Entrepreneurial Regulation

08/30/2013 10:20 AM |

Having just returned from meetings with regulatory authorities in Kenya, Jordan, and Saudi Arabia, I am energized that higher levels of pharmaceutical quality and pharmacovigilance are possible.

But it won’t be easy.

Enhanced levels of excellence will require, if not global harmonization, more aggressive partnerships between agencies around the world.

In other words, it’s time to plan and execute a regulatory Marshall Plan to help build, nation-by-nation, global systems for both quality and safety.  Working together to raise the regulatory performance of all nations will help all nations (even the 20% deemed “capable” by the WHO) to create sound foundations to address a multitude of quality and safety dilemmas such the manufacturing of biosimilars, the control of API and excipient quality, pharmacovigilance and, yes, even counterfeiting.

But drug regulation has to go beyond safety and quality and pharmacovigilance. It’s got to embrace innovation. What we need here at home and around the world is a hunger for entrepreneurial regulation.

Entrepreneurial Regulation is a philosophy that allows agencies such as the FDA to be both regulator of and colleague to industry. Expedited pathways are Entrepreneurial Regulation. Special Medical Use is Entrepreneurial Regulation. REMS are Entrepreneurial Regulation. The exercise of enforcement discretion is Entrepreneurial Regulation. More aggressive use of the Reagan/Udall Foundation is Entrepreneurial Regulation. A more central role for the patient voice is Entrepreneurial Regulation.

Entrepreneurial Regulation makes bodies such as the FDA enablers rather than roadblocks to innovation.

One of the key conundrums of Entrepreneurial Regulation is that there is an inherent tension between predictability and innovation.

The foundational principle of PDUFA is predictability – not speed. And that’s been the focus of the conversation: ambiguity vs. predictability. But, when it comes to innovation, ambiguity is inherent. The pathways to innovation are always ambiguous. Innovation is risky – and not only to investors.

And Entrepreneurial Regulation is likewise risky. But as with all matters regulatory, risk must be viewed alongside benefit – to the public health.

Another level of tension is the relationship between research (R) and development (D). Specifically, the lack of respect between the two. Beyond the disproportionate levels of government funding (when’s the last time you heard anyone talk about “doubling” the FDA budget), nascent relationships between academia (“R”) and industry (“D”) are struggling.

The issue of out-sourcing basic research isn’t new – but it’s mighty contentious. And it’s the new reality of drug development. But, if we are to learn any lesson from the CRO experience, it’s that while we say “partnership,” the danger is that it devolves into a vendor-like relationship. It’s the Golden Rule. He who has the gold makes the rules. Will that be acceptable to high-level, big ego Ivy Hall-ers?

And then there’s the issue of academic priorities, specifically tenure. Does industry funding carry the same weight as NIH grants when it comes to advancing a university career? Not at present. That will have to change.

Need drives change. Just as CROs are finally really partnering with pharma to drive the development of personalized medicine, so too must academe and industry collaborate on the continued evolution of pharmaceutical innovation. It will take discipline and focus. It will be risky. And it will take will. There is a confluence of interest.

When it comes to global safety, quality, and pharmacovigilance standards, there’s a general consensus that a high tide floats all boats. When it comes to Entrepreneurial Regulation demands that we honestly address a tough but fundamental question, how can regulatory agencies around the world (FDA-led by both word and deed) focus on what they can do to facilitate collaboration that results in innovation?

Step One is focus.

In the words of entrepreneur extraordinaire Steve Jobs, “I'm convinced that about half of what seperates the successful entrepreneurs from the non-successful ones is pure perserverance."

And, in the case of Entrepreneurial Regulation, failure is not an option.

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Getting darker at night with increasing light towards morning

08/28/2013 09:44 AM |

And for this she bills how much per hour?

From the pages of the Pittsburgh Business Times,

Developing a regulatory strategy is a key first step in bringing a new medical device to market, Regulatory & Quality Solutions LLC President Maria Fagan said Tuesday.

The plan is needed to align stakeholders, making sure executives, investors and others have a shared vision about the purpose and use of the product, Fagan said in addressing a meeting downtown sponsored by the Pittsburgh Technology Council. Included in the plan should be the device’s intent, benefits and target audience of consumers.

“Think through these things early on,” Fagan said. “From a marketing perspective, where do you want to go?”

And, as a word to the wise, spelling also counts.

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Baucus gets Raucous on Indian Trade Shenanigans

08/25/2013 03:11 PM |

CONGRESSIONAL TRADE LEADERS FIGHT INDIA’S UNFAIR TRADE PRACTICES

In Letter to ITC, Members Seek Investigation of Policies that Discriminate Against U.S. Exports

WASHINGTON –Senate Finance Committee Chairman Max Baucus (D-Mont.), Ranking Member Orrin Hatch (R-Utah), House Ways and Means Committee Chairman Dave Camp (R-Mich.) and Ranking Member Sander Levin (D-Mich.) today requested the U.S. International Trade Commission (ITC) investigate India’s unfair trade practices that discriminate against U.S. exports and investment. 

Noting in a letter that U.S. exports to India are low given the size of its market, the congressional leaders asked the ITC to detail policies India has in place that restrict trade and violate intellectual property rights, as well as the effect they have on U.S. exports, businesses and jobs.

The full text of the letter is available below:

The Honorable Irving A. Williamson
Chairman
U.S. International Trade Commission
500 E Street, S.W.
Washington, DC 20436

Dear Chairman Williamson,

We are writing to request that the U.S. International Trade Commission (Commission) conduct an investigation under section 332(g) of the Tariff Act of 1930 (19 U.S.C. §1332(g)) regarding Indian industrial policies that discriminate against U.S. imports and investment for the sake of supporting Indian domestic industries, and the effect that those barriers have on the U.S. economy and U.S. jobs.

India is an important strategic partner of the United States, yet U.S. exports of goods and services to India remain low.  In 2011, U.S. goods exports to India – the world’s second most populous country – were just $22.3 billion.  Similarly, recent data indicates that U.S. private commercial services exports, sales of services by majority U.S.-owned affiliates, and U.S. foreign direct investment (FDI) in India were also low.

India has risen rapidly and lifted millions out of poverty in the wake of its significant market opening reforms and its efforts to seek foreign investment in certain sectors of its economy over the past two decades.  However, India maintains and continues to put in place measures that appear to contradict its stated domestic growth objectives.  For example, India has a complex, non-transparent tariff and fee system and byzantine and overburdensome customs procedures, and it maintains significant tariff and non-tariff barriers to U.S. goods and service participation in sectors including retail and agriculture.  More recently, India has introduced new localization-forcing measures such as local content and technology transfer requirements in the green technology and information and communications technology sectors.   And India has not yet taken action to fully and effectively protect and enforce copyrights, including in the digital environment, and has applied its patent law in a discriminatory manner, particularly against innovative U.S. pharmaceutical companies, so as to advantage its domestic industries.  

Beyond any particular action India has taken, the government has enunciated a broader policy objective to develop and support Indian domestic industries by forcing foreign firms to use local facilities and suppliers and to transfer their intellectual property to Indian entities.  Government documents indicate that India is likely to adopt additional measures to this end, and expand these sorts of measures to additional sectors, creating significant concern and uncertainty for U.S. exporters and investors.

Finally, we are very concerned about the broader impact that India’s trade policy may be having on the global trading system, both in terms of the model it is setting for other countries and the drag it is exerting on multilateral trade negotiations.

Despite the widespread evidence of these existing and anticipated barriers to U.S. exports and investment in India, the U.S. Government has not conducted a comprehensive economic analysis of the effect of Indian trade policies on the U.S. economy and U.S. jobs.  To assist us in better understanding the effects of these existing and anticipated barriers to U.S. exports and investment in India, we request the Commission to provide a report covering the items described below.

Based on a review and analysis of data and information from available sources, including a survey of U.S. firms, we request the Commission to provide:
 

·         An overview of trends and policies in India affecting trade and foreign direct investment in that country’s agriculture, manufacturing and service sectors, as well as the overall business environment.  The overview should take a historic view, but focus on the period since 2003.  It should include examples of changes in tariff and nontariff measures, including measures related to the protection of intellectual property (IP) rights, and other actions taken by India’s government to facilitate or restrict the inflow of trade and FDI.

·         A description of (1) any significant restrictive trade and FDI policies currently maintained or recently adopted by India as identified by USITC research; (2) the sectors in the U.S. economy most affected by these restrictive policies; and (3) the general competitiveness of sectors in India’s economy that are subject to the identified restrictions.
 

·         Several case studies that examine the effects of particular restrictive measures on U.S. firms that export to or invest in India, or that have not done so because of the measures.  To the extent feasible, the case studies should address the impact of the restrictive measures on both large and small and medium-sized enterprises.
 

·         To the extent feasible, a quantitative analysis of the economic effects of India’s identified restrictive measures on the U.S. economy as a whole, on U.S. trade and investment, and on selected sectors of the U.S. economy.
 

·         Based on the survey and analysis of results, and to the extent feasible, a summary of U.S. firms’ perception of (1) recent changes in India’s trade and investment policies in selected sectors and (2) the effects of these changes on U.S. firms’ strategies towards India (e.g., reducing investment or altering product mix), and analysis of whether the effects of these policy changes differ by firms’ characteristics, such as size, IP-intensiveness, or export status.

We request that the Commission deliver the report to us by November 30, 2014. 

In preparing its report, we do not expect the Commission to make findings regarding the legal merits of any Indian laws or policies. 

As we intend to make the report available to the public, we request that the Commission not include confidential business information in the report.

Sincerely,
 

Max Baucus
Chairman, Senate Committee on Finance

Orrin Hatch
Ranking Member, Senate Committee on Finance

Dave Camp
Chairman, House Committee on Ways and Means

Sander Levin
Ranking Member, House Committee on Ways and Means

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340B Minus

08/22/2013 12:50 PM |

From today’s edition of the South Florida Sun-Sentinel:

Some health providers exploiting drug program

Some major American health care providers are padding their bottom lines by exploiting a federal program meant to help low-income patients. This behavior is netting them billions in ill-gotten gains.

And it could be preventing many vulnerable Americans from accessing the low-cost drugs they need to treat and prevent illness.

This abuse needs to be stopped.

In 1992, Congress created a program — known as "340B" — to help caregivers serving disproportionately large numbers of low-income beneficiaries and uninsured patients. Under 340B, drug manufacturers are required to sell their products at a discount to such institutions.  The discounted prescriptions are dispensed either through the caregiver's in-house pharmacy or through a contractual arrangement with an outside pharmacy.

340B has a noble cause. And many of the medications discounted through 340B do in fact go to clinics, hospitals, and medical facilities providing care almost exclusively to uninsured and poor patients.

However, some 340B participants are exploiting the program.

340B only requires caregivers to meet certain minimal thresholds for the number of medically underserved people they treat.  For many hospitals, these eligibility standards are easily reached, and some are benefiting from the program's deep drug discounts while still serving a relatively affluent clientele.

Moreover, participating caregivers are not actually required to pass drug savings along to their patients. The huge discounts they're getting from pharmaceutical manufacturers don't necessarily translate to lower pill prices for uninsured and low-income patients.

Given what we have recently learned about some hospital administrators inflating charges for a broad variety of basic services, there's good reason to believe many sell those discounted drugs at full price to insured patients and then pocket the difference. Indeed, a report by the Raleigh News Observer last year found hospitals that "routinely mark up prices on cancer drugs two to 10 times or more over cost. In some cases, the mark up is far higher."

Meanwhile, the vulnerable patient populations 340B was intended to help are often still stuck struggling to gain access to affordable pharmaceuticals.

In large part because some healthcare providers are abusing the 340B system, the size and cost of the program are ballooning out of control. The Berkeley Research Group estimates the total the total value of all the medicines sold through the program will more than double from $8 billion in 2010 to $19 in 2016.

Such a surge in expenses might very well be worth if it 340B was largely helping needy patients. But it is not clear that this is actually happening. Although 340B was created to help low-income patients obtain the medicines they need, it has turned into a revenue generator for many hospitals.

Caregivers are now allowed to qualify for the program's deep drug discounts without passing along those savings to patients in need. Administrators are getting rich off a well-intentioned public program.

Too many uninsured and poor patients still don't have access to discounted drugs.

340B needs to be fixed.

Peter J. Pitts, a former FDA Associate Commissioner, is President of the Center for Medicine in the Public Interest.

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It only hurts when I tweet

08/21/2013 08:58 AM |

Docs Need to Get Up to Speed, Social Media Advocate Says

By Kristina Fiore, Staff Writer, MedPage Today

Bertalan Mesko, MD, PhD, is counting on old media to convince more clinicians about the value of new media.

The clinical genomics specialist has just published a handbook on social media in clinical practice -- and he hopes it will bring late adopters up to speed with their social-media-savvy colleagues, and even with some of their electronically empowered patients.

While "expert" patients voraciously pursue credible medical information and communities online, clinicians "usually lag behind," Mesko, who is based in Budapest, said in an email exchange with MedPage Today. Instead of disdaining this kind of behavior, doctors need to see themselves as a gatekeeper of vetted online information and activities, he said.

One of the earliest clinician voices on social media, Mesko has been attempting to make that gatekeeper role easier as the founder of Webicina.com, a clearinghouse of sorts for digital and social media resources. He's also a self-described "medical futurist" who blogs at Scienceroll.com.

Mesko answered some questions about the book, "Social Media in Clinical Practice," and about online engagement via email. An edited version of that conversation follows.

MPT: Why the need for a book on social media adoption for clinicians?

Mesko: Social media has been playing an increasingly important role in medical communication as the Internet now has a crucial place in our lives. While patients relatively easily become empowered or expert patients -- the so-called e-patients, as they are really motivated to use digital solutions to get a better care -- medical professionals usually lag behind.

I've been teaching medical students and physicians about the proper use of social media and other digital technologies for years, and my experience is that even if I created a free e-learning platform for them, they stick to the traditional way of learning new things. Therefore, the need for a practical handbook full of examples and step-by-step instructions about using social media platforms was imminent.

MPT: Many clinicians are apprehensive about social media because of HIPAA and patient privacy. What are the challenges here, and how should clinicians address them?

Mesko: Physicians should know exactly the potential limitations and privacy issues caused by the use of social media. [In the book], I describe bad examples and stories related to these crucial issues.

In one example, a patient added me on Facebook and shared her previous medical records with me even though she was not my patient. I rejected the offer and sent her a private e-mail explaining why (that this is a personal online channel, why our relationship would be professional and I don't want to mix these). She perfectly understood.

I had a colleague who rejected the offer and pretended like nothing happened when they next met. He described the situation as quite awkward.

This happens when you don't know the rules of online communication. If we know the rules, just like in real-life communication, the use of social media becomes safe and efficient.

MPT: Can you give examples of some of the more insightful uses of social media that you've seen among doctors?

Mesko: I'm a member of amazing medical communities -- from Google+ to closed Facebook groups -- in which I can discuss clinical issues with peers worldwide. My Twitter channel with over 32,000 followers lets me crowdsource difficult diagnostic problems, and my blog gives me a chance to connect with tens of thousands of medical professionals without limitations. I published papers in peer-reviewed journals after collaborating with co-authors simultaneously in a Google document.

Social media provides us with a lot of opportunities, but only if we know the potential limitations and security issues. Acquiring such knowledge takes years, and my goal with the handbook was to shorten this time significantly for those medical professionals who would like to become a bit more digital, but at the same time use these online tools in a secure way.

MPT: How did you crowdsource a difficult diagnostic problem?

Mesko: The story happened in the fall of 2009; the New York Times wrote about it in early 2010. When professors at the clinic could not find the solution to a complicated medical issue, I sent a tweet to my tens of thousands of medical followers for useful advice. I tweeted this:

"Strange case today in internal medicine rotation: 16-year-old boy with acute pancreatitis (for the 6th! time). Any ideas?"

I received hundreds of amazing responses from all over the world, and in one day we came up with a potential solution. The final diagnosis was microlithiasis, small stones from the gallbladder causing pancreatitis from time to time without obvious causes. The diagnosis was not so simple because of the size of the stones and as a medical student I wanted to use the power of the medical community I had been building for years, so that I might come up with useful suggestions for my professors. It turned out to be a good idea.

MPT: If there was one use of digital or social media that all clinicians should adopt, what would it be?

Mesko: I think communication methods in real life and in the online world are the same. If medical professionals understand this and create a proper online presence, as well as give their patients a chance to communicate with them through certain online channels, the doctor-patient relationship can become more efficient by saving time for both parties.

Using digital technologies, especially social media, is now an integral part of medical communication, and as more and more patients use these platforms, their physicians must be able to deal with this in an evidence-based manner.

MPT: Are there any definite "don'ts" for clinicians online?

Mesko: I tell my medical students they should definitely not do things online which they would never do offline. That's how simple it is.

MPT: What is the "Participatory Medicine Movement" and what should practicing clinicians know about it?

Mesko: As e-patients are motivated to use digital technologies in their health management, they have additional questions about the Internet, such as where to find reliable medical resources about their conditions. Doctors must be able to respond properly and professionally to these questions as well. The Participatory Medicine Movement leads to some changes in the structure of medicine in which medical professionals will become mediators or guides for their patients online.

In order to reach this long-term goal, doctors should learn new tricks, not just about communicating with patients online, but also about keeping themselves up to date more easily.

MPT: Which smartphone apps should every clinician already have downloaded?

Mesko: It very much depends on certain factors such as specialty, the smartphone or tablet you have, and the way you want to use it in practicing medicine. The key point here is curation. Only curated, validated, and quality smartphone apps should be used by physicians, and they should be able to help their own patients identify similarly good apps themselves.

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Obamacare's Losers

08/20/2013 02:24 AM |

Via USA Today:

Johnny Drake's business is losing 2.3 percent of everything it makes because of the Affordable Care Act.

He's the president of Pathfinder Technologies, a small company in Nashville with fewer than 20 employees, that got hit with an excise tax this year because it makes medical devices
.
Medical device manufacturers are among the federal health law losers, those that will have to pay up to cover the cost of implementing it. Others include high-wage earners, tanning salons and, in some cases, working parents and folks with big medical bills. The law generates revenue through a hodgepodge of new taxes, financial penalties and IRS rule changes.

"Every quarter, we're having to send the federal government a flat 2.3 percent of our revenue," Drake said. "I feel like it's double taxation because at the end of the year, we're sending them our federal income tax as well."

Tanning salon owners started having to pay a bigger tax three years ago. Lyvonn Reese, who owns the Hot Spot Tanning salons in the Nashville area, said the 10 percent tax ate away so much of her profit margin that she had no choice but to pass it along. She itemizes "tanning tax" on customer receipts.

Read the article here.

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App-Sense & Pharma Sensibilities

08/16/2013 10:52 AM |

Congressional Quarterly reports that the Food and Drug Administration has yet to give final guidance on which of the growing number of mobile health apps it will regulate. Draft guidance indicates “enforcement discretion” will be taken toward consumer-oriented mobile apps, especially ones that are more high risk. The draft guidance also notes that clinical apps could “present a potential risk to patients if they do not work as intended.” Many health industry groups would like to see a wider regulatory framework on IT in place before the FDA issues specific mobile app guidance.

That sounds familiar.

For a more detailed look at this issue – with particular emphasis on the issue of enforcement discretion see, “A Regulatory App-ening.”

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Mutual of Pretoria

08/14/2013 06:59 AM |

South Africa seeking mutual recognition with FDA and EMA.

Maybe a better first step is to establish a reference basket of maybe five to six countries. A good model is Singapore. Everyone’s favorite city-state reviews six countries—USA, Canada, Australia, NZ, Japan, Switzerland and the EMA.  If any two of the five have approved, then approval is basically a formality.

Singapore doesn’t have mutual recognition with either the FDA or the EMA, but they unilaterally recognize the benefits of referencing to the “big regulatory dogs”, but did not demand that the FDA reference to their tiny agency.

 New medicine control body a step closer
Tamar Kahn: Business Day

THE establishment of a new regulatory body for medicines is a step closer, after the Cabinet said yesterday that it had referred enabling legislation containing the Medicines and Related Substances Amendment Bill to Parliament.

The bill will replace the Medicines Control Council (MCC) with the South African Health Products Regulatory Agency (Sahpra), an entity with much wider scope.

The Cabinet said the bill sought to establish a strong, efficient and effective medicine regulatory authority. The Department of Health envisages Sahpra as being the solution to the extensive delays besetting the MCC, which takes much longer compared with US or European regulators to approve new medicines and clinical trials.

It is also expected to bring scrutiny to bear on aspects of the market that have largely gone unregulated, such as medical devices and complementary medicines. The new regulatory agency will also be responsible for foodstuffs, cosmetics, disinfectants and diagnostics.

To the frustration of researchers and the pharmaceutical industry, Sahpra has been stuck in the works for years. In 2008, Parliament passed amendments to the Medicines and Related Substances Act, which were not implemented. The bill was subsequently redrafted and published for comment in March. Since then it has taken more than a year to refine the bill and get it through the Cabinet.

Department of Health director-general Precious Matsoso said in June that one of the key changes made to the draft legislation was the inclusion of provisions for Sahpra to be a public entity with an independent board chaired by a CEO. It would also have a stronger governance structure than the previous draft, which had a CEO appointed by the Health Minister and gave final authority for the approval of new products to the Minister. The draft bill also included measures to shorten the registration time for medicines and medical devices by allowing mutual recognition agreements between Sahpra and other regulatory authorities such as the US Food and Drug Administration.

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Interface Race

08/13/2013 08:41 AM |

From the pages of Health Affairs:

Electronic Communication Improves Access, But Barriers To Its Widespread Adoption Remain

Abstract

Because electronic communication is quick, convenient, and inexpensive for most patients, care that is truly patient centered should promote the use of such communication between patients and providers, even using it as a substitute for office visits when clinically appropriate. Despite the potential benefits of electronic communication, fewer than 7 percent of providers used it in 2008. To learn from the experiences of providers that have widely incorporated electronic communication into patient care, we interviewed leaders of twenty-one medical groups that use it extensively with patients. We also interviewed staff in six of those groups. Electronic communication was widely perceived to be a safe, effective, and efficient means of communication that improves patient satisfaction and saves patients time but that increases the volume of physician work unless office visits are reduced. Practice redesign and new payment methods are likely necessary for electronic communication to be more widely used in patient care.

1. Tara F. Bishop1,*,
2. Matthew J. Press2,
3. Jayme L. Mendelsohn3 and
4. Lawrence P. Casalino4

1. ¹Tara F. Bishop (tlfernan@med.cornell.edu) is an assistant professor in the Departments of Public Health and Medicine at Weill Cornell Medical College, in New York City.
2. ²Matthew J. Press is an assistant professor in the Departments of Public Health and Medicine at Weill Cornell Medical College.
3. ³Jayme L. Mendelsohn is a research coordinator in the Department of Public Health at Weill Cornell Medical College.
4. ⁴Lawrence P. Casalino is the Livingston Farrand Associate Professor in the Department of Public Health at Weill Cornell Medical College.

1. ↵*Corresponding author

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"D" is for Dual Eligibility

08/12/2013 08:15 AM |

From the pages of the South Florida Sun Sentinel …

Competition from insurers is benefit for consumers

How do you know a federal government program is working? When states start using it as a model for their own initiatives. That's what's happening with Medicare Part D, the prescription drug program for seniors. States are incorporating its unique market-based structure into their own healthcare programs to expand access and manage costs.

Under Medicare Part D, seniors select their prescription coverage from a host of private insurers. States are now applying that principle of free market competition to their Medicaid programs, and the results have been excellent.

Kansas, Louisiana, and Florida have received federal waivers that allow them to experiment with providing Medicaid services through private insurers. The states pay a fixed amount to insure each Medicaid enrollee, and then set minimum benefits that plans must provide.

But it's private insurers that actually supply the plans, and it's the enrollees themselves who decide which plan they would like. As a result, insurers can't provide the bare minimum — Medicaid enrollees can easily select a more appealing plan based on their own needs.

The results have been impressive. When Florida recently ran a pilot program, participating counties outperformed others 64 percent of the time on measured health outcomes. In Louisiana, where those on private insurance have the option of returning to the state's traditional Medicaid program, only one-third of 1 percent have chosen to do so. And because these states expect to save money, other states, including North Carolina, Texas, and Utah, are now considering similar policies.

And these programs don't just promise to save money, they feature various innovations to ensure quality. For example, in Florida, insurers are required to conduct customer-satisfaction surveys. While some states have increased the amount they will pay for coverage of high-risk patients, giving insurance companies an incentive to cover these patients and keep them healthy. This type of risk-based pricing helps patients receive more preventive care, according to a report last year by the Urban Institute.

Nationwide, 36 states and the District of Columbia provide at least some of their Medicaid services through private insurers — and these programs are expected to grow as the new health-care law expands access to Medicaid. States already spend up to one-third of their budgets on Medicaid, so an opportunity to save money while providing better service is an obvious win.

All of this is exciting — but it's not surprising, at least not to those who are familiar with the success of Medicare Part D. By letting seniors choose between private drug plans, Part D has cost the government about 45 percent less than initially projected when Congress enacted the program in 2003.

Out-of-pocket expenses for seniors are also lower than expected. And in a recent survey, 90 percent of Part D beneficiaries said they were satisfied with the program.

Given Medicare Part D's success in its own right and as a model for other healthcare programs, it's bizarre that the president and some in Congress would like to undermine the competition that makes Part D work.

The president's most recent budget, as well as bills introduced in Congress by Sen. Jay Rockefeller and Rep. Henry Waxman, would require drug companies to give "rebates" to the government for the drugs purchased for low-income seniors — known in technical parlance as "dual eligibles" because they qualify for both Medicare and Medicaid.

To put it simply, these politicians would substitute government price controls for the competitive marketplace that has been so effective in keeping costs down. As the Congressional Budget Office has reported, the private plans provided through Part D are already negotiating low prices for drugs. Requiring drug makers to, instead, sell their products for below-market prices will force manufacturers to raise prices on other consumers, including most seniors. Such rebates could increase seniors' premiums by 40 percent, according to a study by former CBO director Douglas Holtz-Eakin.

States may be the laboratories for innovation, but the big lesson from Part D is clear: Competition between private insurers reduces costs and encourages better health care. Some states are learning this lesson and applying it to their Medicaid programs. The federal government should be encouraging this market-based reform, not trying to undermine it.

Peter J. Pitts, a former FDA Associate Commissioner, is President of the Center for Medicine in the Public Interest.

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Oregon Trail?

08/09/2013 07:11 AM |

From the pages of the Wall Street Journal.  Frightening.

Rationing Health Care in Oregon

BY: HOPE LANDSEM

Liberal states often preview health-care central planning before the same regulations go national, which ought to make an Oregon cost-control commission especially scary. On Thursday a state board could change Oregon's Medicaid program to deny costly care to poor patients who need it most.

Like most such panels, including the Affordable Care Act's Independent Payment Advisory Board, the Oregon Health Evidence Review Commission, or HERC, claims to be merely concerned with what supposedly works and what doesn't. Their real targets are usually advanced, costly treatments. That's why HERC, for example, proposed in May that Medicaid should not cover "treatment with intent to prolong survival" for cancer patients who likely have fewer than two years left to live. HERC presents an example to show their reasoning for such a decision: "In no instance can it be justified to spend $100,000 in public resources to increase an individual's expected survival by three months when hundreds of thousands of Oregonians are without any form of health insurance."

Amazingly enough, the Affordable Care Act quashed that one. The law says coverage decisions cannot discriminate against people because of their diagnoses and life expectancies.

So HERC changed a few words of its proposal. Before, the plan would have limited treatment based on life expectancy. Now, the plan will determine whether an individual continues to receive potentially life-saving treatment based on the severity of the illness, using ambiguous performance statuses, a scale that tries to quantify a cancer patient's well-being. Some difference.

This is a recurring theme in HERC's Medicaid overhaul. The commission also suggested guidelines that would limit to once a week the number of times some diabetics could check their blood sugar, down from the three tests a day the American Diabetes Association recommends now. Outrage from diabetics, not to mention medical experts, forced the commission to postpone that vote.

The public isn't receiving the cancer proposal any better. In a letter to HERC, a Willamette Valley Cancer Institute and Research Center patient navigator, a trained health-care worker responsible for educating cancer patients and guiding them through various treatment options, writes that "patients deserve treatment that is available based on the best evidence, not on a timeline." We'll learn today if HERC is willing to restrict potentially life-saving treatment in favor of the left's one-size-fits-all health approach.

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CMPI Event: “Personalized Medicine and Responsible Access to Pain Medication.”

08/07/2013 02:56 AM |

Please join the Center for Medicine in the Public Interest (www.cmpi.org) and some of the nation’s top experts for an interactive panel discussion of “Personalized Medicine and Responsible Access to Pain Medication.”

A Capitol Hill Briefing

When: September 10, 2013 from 9:30AM – 2:15PM

Where: Rayburn House Office Building B338, Washington DC

RSVP: Mario Coluccio at mcoluccio@cmpi.org

BREAKFAST & LUNCH TO BE SERVED

AGENDA

9:30- 10AM:  Breakfast and Registration

10AM: Welcome
            Peter Pitts & Robert Goldberg, Center for Medicine in the Public Interest

10:15: Keynote: FDA Regulation and Responsible Access to Pain Medication
            Douglas Throckmorton, Deputy Director, Regulatory Programs, CDER, FDA

11:00: Access to Pain Medications: The Role of Patients and Manufacturers
            Moderator: Steve Usdin, BioCentury
            Cindy Steinberg, US Pain Foundation
            Bob Twillman, American Academy of Pain Management
            Stuart Kim, Mallinckrodt Pharmaceuticals

11:45: Break and Lunch

12:00: Issue:  Pain Medications:  Two Reporters Views
            Moderator: Peter Pitts, CMPI
            Barry Meier, New York Times
            Judy Foreman, Syndicated Columnist

1:30:  Closing Keynote: Pain Medications and the Future of Personalized Medicine
            Introduction: Bob Goldberg, CMPI
            Charles Inturrisi, Weill Cornell Medical Center
 
2:15: Closing Remarks
 
Peter Pitts & Robert Goldberg, Center for Medicine in the Public Interest
 
RSVP: Mario Coluccio at mcoluccio@cmpi.org

BREAKFAST & LUNCH TO BE SERVED
 

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B Cause

08/06/2013 07:36 AM |

From the pages of Forbes.com.

Medicare Budget Cuts Could Threaten Cancer Patients' Access To Drug Treatments

By Peter J. Pitts

Earlier this month, Forbes guest commentator John Wilson celebrated cuts planned for Medicare Part B as a result of sequestration and called for additional “savings” to be wrung out of the program.

Across-the-board federal budget trimming has forced the Centers for Medicare and Medicaid Services (CMS) to significantly reduce the reimbursement rate for healthcare providers that participate in Part B, which covers drugs that have to be administered under professional supervision.

Mr. Wilson thinks these rate cuts are a good start — and he wants more. He claims that “Part B drugs have a history of CMS overpayments” and suggests additional reimbursement reductions won’t have any ill effects.

He’s mistaken.

The model for Part B provider payments is working well to bring down long-term healthcare costs and ensure enrollees have access to needed medication. Additional reimbursement reductions will compromise care in communities throughout the country while doing little to curtail Medicare expenses.

Under Part B, doctors pay for medications on their own and are then reimbursed according to a formula: the average market price for that drug plus an add-on to cover administrative expenses. That additional compensation above the prevailing price is crucial. It helps participating healthcare providers finance other crucial, resource-intensive aspects of treatment, like drug acquisition and storage.

Under this unique, market-like setup, caregivers have an incentive to find the best treatment for the lowest price. Indeed, the Congressional Budget Office projected that this reimbursement system would generate $16 billion in savings over its first decade of operation. And yearly cost growth for the program has been below overall medical inflation.

One major study found that Part B’s reimbursement formula reduced drug spending by over seven percent during the program’s first year of operations. And it limited Part B’s average annual expense growth rate to just 2.4 percent, compared to nearly 11 percent for all of Medicare.

Community health clinics are major participants in Part B. These locally oriented operations tend to be significantly more cost-efficient than larger hospitals. By properly compensating clinics and encouraging them to treat Medicare enrollees, Part B saves the government money over the long-run. For instance, research from the consulting group Milliman has found that Medicare saves an average of $6,500 per year when a patient receives chemotherapy treatment at a clinic rather than a hospital.

All in all, Part B’s reimbursement formula has been working terrifically well. But as a result of sequestration, CMS will be cutting that administrative add-on by about two percentage points — from six to four percent. And some in Washington are looking to ratchet back this reimbursement even further, to closer to three percent.

Further cutting this rate would seriously threatens the financial viability of many of the community health clinics currently participating in Part B. These operations already run on exceedingly thin profit margins. They depend on proper compensation from Part B and other public programs to stay afloat. Reducing reimbursements would force many clinics to close and physicians to turn away enrollees.

New cuts would make a bad situation even worse, particularly in the realm of cancer treatment. According to the Community Oncology Alliance, over the last six years, 288 cancer clinics have closed. Another 469 have entered into a contractual relationship with a hospital or been acquired by a hospital. And 407 report they’re struggling financially.

The American Society of Clinical Oncology predicts that Part B cuts could force up to three-quarters of the remaining cancer clinics in the country to start redirecting Medicare patients to other caregivers.

Mr. Wilson also argues for scaling back the intellectual property protections currently afforded an advanced class of pharmaceutical drugs called biologics. These are highly complex treatments derived from living organisms. In addition to standard patent controls, biologics are also provided 12 years worth of data protection preventing generic competitors from accessing the original innovators research information.

The provision establishing these 12 years of data protection was included in the President’s 2010 health care reform law — and it was one of the few provisions that enjoyed broad bipartisan support from both chambers of Congress.

After noting that 12 years is “far longer than for most other drugs,” Mr. Wilson joins the chorus of misinformed critics calling for biologic data exclusivity to be scaled back to seven years. He thinks such a move would save the public health system billions in drug expenses by expanding the pool of low-cost generic alternatives.

But that 12 year set point isn’t arbitrary. Virtually all the research on this subject shows that that’s about as long as it takes for the average biologic to break even in sales. The average new drug costs on average $1.2 billion to research, develop and bring to market. And just two out of every ten new drugs ever turns a profit.

Cutting down the period of data protection to just seven years would flood the biologic market with generic competition well before most innovators have had time to get out of the red. Drug developers would be much less likely to invest in new products in the future and patients would be deprived of new breakthrough treatments.

Mr. Wilson and I are in agreement that public officials need to find effective ways of controlling Medicare costs without compromising enrollee care. But further cutting Part B reimbursements and reducing the protection of intellectual property for innovative drug companies doesn’t fit the bill — it will undermine community caregivers and choke off patient access to needed medicines.

Peter J. Pitts, a former FDA Associate Commissioner for External Affairs, is President of the Center for Medicine in the Public Interest.

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How We Can Cure Cancer Sooner

08/05/2013 01:04 PM |

Tailoring cancer studies to fit people who need to get better 

Imagine if our immune systems could vanquish cancer in much the same way they take on the common cold. New research could turn such science fiction into fact.

Currently in the works are several new drugs that can order certain white blood cells - the immune system's warriors - to attack cancer cells. If they prove successful, a world free from cancer could be one step closer. Researchers are increasingly exploring how we can personalize the fight against cancer - harnessing the unique characteristics of our own bodies to beat the disease. Such personalized approaches offer our best shot at eradicating cancer - and should be at the heart of our battle plan against it. Medical science has already made progress.

Since 1990, new medicines have doubled the number of cancer survivors - from six million to 13 million. They have given patients collectively about 43 million years of additional life. These aren't years of pain and desperation. Cancer survivors add about $4.7 trillion in value to the economy just by living and working longer. Today, every dollar spent on new cancer medicines reduces spending on hospitals and doctors by $7. Yet the actual amount we spend on such treatments is small - about 1 percent of total health-care spending. Indeed, spending on innovative cancer research and therapies has already delivered a hefty return on investment. We should double down on that approach.

Step one is to put patients in charge of cancer research. How? Patients can use online communities to test treatments, design studies, and determine better ways to tackle their illnesses. They're already keeping tabs on their health with fitness monitors and tablets. Research should be shaped by these real-time, real-world experiences in combination with information about the particular genetic mechanisms that make their tumors tick. As genomics professor Eric Topol argues: "It is time for a jailbreak; it is time for the rise of the consumers to drive the future of medicine. It is their DNA, their medical data, their cellphones, and their own health at stake."

That jailbreak should include replacing one-size-fits-all research with personalized cancer studies. The Human Genome Project empowers researchers to do so. Personal genomes can now be sequenced in a few hours for under $500. Such sequencing can yield medicines that are truly personalized. Several cancer organizations, including the International Myeloma Foundation, StandUp2Cancer, and the Sarcoma Foundation of America, require researchers to look for genetic cues that could lead to cures. This should be the rule, not the exception.

These personalized approaches could also allow regulators to get new cancer medicines to market faster. Developing a new cancer medicine takes 8.8 years, on average - much longer than for other drugs. Most of this time and effort is spent testing medicines in people who researchers know won't benefit. But by focusing solely on patients and their specific cancer-causing genetic mutations, researchers could identify what therapies work early on. That could mean approving cancer therapies as fast as HIV medicines - in two to three years.

Government officials can also get personalized treatments into the hands of patients more quickly by requiring health plans to pay for them. Advances in cancer treatment are saving lives and cutting health-care costs. But many health-insurance plans haven't caught up with the times. Many cancer patients are forced to choose between a treatment that could save their lives - or one that's paid for. Insurers should instead pay for the right treatment for the right patient.

Under the health-care status quo, cancer treatment is divided up according to who gets paid. Innovations that save money are pitted against services that lose money. New "Charter Cancer Communities" can solve that problem by focusing specifically on the value of care. Like public charter schools, these communities would have greater flexibility to use and pay for the combination of treatments that deliver real value. And they'd be accountable to the member organizations and to the patients they serve. Thanks to recent advances in medical science, we're closer to a world free from cancer. If we ratchet up our investments in personalized medicine, that world can become a reality.


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A Limited Use Consensus -- in more ways than one

08/05/2013 07:25 AM |

On Thursday I joined a select group of FDA and healthcare policy experts at the joint FDA/Engelberg Center for Health Care Reform (Brookings Institution) to discuss, debate, and digest the many issues surrounding the thorny opportunity known as Special Medical Use (SMU).

Expertly chaired by Mark McClellan (my former boss at the FDA and the hardest working man in healthcare policy), the day was filled with honesty, ideas, identification of roadblocks – and frustration.

But all to the good.

The meeting was also filled with many senior thinkers and heavy hitters from the agency (Rachel Sherman, John Jenkins/OND, Janet Woodcock, among others) as well as senior Hill staffers, officials from the biopharmaceutical industry, BIO, PhRMA, payers, patient organizations, and academia. The list of attendees can be found here.

First of all, permit me to recommend the short briefing paper that McClellan’s team developed in advance of the meeting, “Special Medical Use: Limited Use for Drugs Developed in an Expedited Manner to Meet an Unmet Medical Need.” Worthwhile reading both as a primer and a guide to many of the regulatory quandaries surrounding SMU.

The meeting began with McClellan commenting that we needed to “channel PCAST” and making the key distinction (regularly confused) that the difference between special medical use (from a labeling perspective) and REMS – is that REMS is for known safety risks.

And then came Janet.

Her remarks focused understanding SMU along the “safety and certainty” continuum.  SMU medicines are for sub-segments of a patient population (with a serious and life-threatening disease) that are not effectively served by current therapies. “There’s a difference between headaches and HIV.” And that wasn’t a throwaway line. In fact, there was zero mention or acknowledgement that SMU should be a pathway exclusively for anti-infectives. Janet strongly affirmed that the FDA has no interest in interfering in the practice of medicine. To the contrary, she believes the best way to ensure that SMU products are used appropriately is through clear and distinct labeling – special logo, etc.

This makes perfect sense and is a logical extension of the agency’s Safe Use of Drugs initiative.

Janet also raised the issues of SMU products and industry promotional practices. Her feeling is that (as with other Breakthrough Designation programs) that pre-view of all marketing materials would be an SMU prerequisite. She also raised the interesting question of seeking to limit on-label communications in order to drive “appropriate messaging from launch because appropriate messaging leads to appropriate use.”

Tom Abrams, call your office.

Janet also noted the fear of many in industry that the FDA would “deem” an SMU designation. She made it clear that would not be the case.

After Dr. Woodcock’s opening keynote, the first panel of the event focused on “The Special Medical Use Pathway Proposal.” The panelists were Jim Greenwood (BIO), John Castellani (PhRMA), Jack Lasersohn (The Vertical Group), Margaret Anderson (FasterCures), and Jeff Allen (Friends of Cancer Research).

And, while there were no fireworks – there was a degree of discomfort – specifically on SMU and legislation, regulatory authority, and therapeutic areas of use.

Here’s how Steve Usdin at BioCentury saw it:

BIO, PhRMA split on special use pathway

The leaders of the Biotechnology Industry Organization and the Pharmaceutical Research and Manufacturers of America staked out separate positions on the creation of a Special Medical Use pathway proposed by the President's Council of Advisors on Science and Technology and senior FDA officials …

BIO President and CEO James Greenwood said the group's board "strongly approves" of SMU, endorses congressional action to create the pathway and feels it should be "broadly applied" to drugs for a wide range of diseases. John Castellani, PhRMA's president and CEO, declined to endorse creation of an SMU pathway and said FDA has sufficient authority to approve drugs for special populations, though he stressed the urgency of facilitating development of new anti-infective drugs. Members of Congress have indicated that enactment of legislation creating an SMU pathway is contingent on unanimous support from industry, so PhRMA's skepticism about the need for an SMU pathway could scuttle the concept.  

While the general sense of Usdin’s comments is correct, it may not be quite so black and white.

Both Greenwood and Castellani support the concept of SMU. And that’s an important point of departure. Greenwood, while supporting the concept of legislation, believes that the FDA already has the regulatory authority to create a Special Medical Use pathway and the ensuing labeling for such products. He said that, rather than pushing for actual legislation, that a “sense of Congress” might be a better idea.

Dr. Woodcock, call your lawyers.

Castellani agreed that the agency already as the authority to move forward. So there’s important convergence there. But he was less clear on the issue of an SMU pathway that went beyond anti-infectives. I specifically asked him if he felt the SMU pathway should be limited to anti-infectives. His answer was to reiterate PhRMA’s concerns about “direct or indirect” impact on the practice of medicine and possible agency hindrance of appropriate promotional communication.

Both Margaret Anderson and Jeff Allen feel that the SMU pathway should be open to any appropriate therapeutic area. Jeff Allen made a key point, that the SMU pathway is possible because of advances in science. It sounds like a simple point – and it is – but it’s important to recognize that SMU is the next logical step towards personalized medicine.

Margaret Anderson also got real by pointing out that, minus additional budget, the FDA will only be able to go so far. “How much innovation,” she asked, “can the agency be expected to handle?”

Indeed.

The general consensus was that SMU should be piloted with anti-infectives – but proceed rapidly to where the need is the greatest. Bravo – but highly subjective.

John Castellani, call your office.

Panel Two operated under the moniker, “Implementation and Impact of Special Medical use Products in Clinical Practice” – but this was really the payer panel.

Sam Nussbaum (WellPoint) brought the urgent issue of patient outcomes into the conversation. His point was (per reimbursement) that if the SMU process works as designed (limited use with an enhanced risk/benefit profile in a defined sub-population) that they would be swiftly tiered for reimbursement.

Pursing the topic of the role payers have in outcomes, Ed Septimus (HCA Healthcare) stated that, “Stewardship is resource intensive.” True – but lack of stewardship is even more costly in terms of scarce dollars and clinical sense – and patient lives.  Septimus also introduced the concept of mandatory patient registries.

I asked the panel if, as part of the regulatory process, the FDA should mandate patient registries for SMU medicines. There was an uncomfortable silence.

Gerald del Pan, call your office.

Panel III: “Postmarket Considerations to Promote Safe Use and Continued Evidence Development of Special Medical Use Products” opened with Preeti Pinto (former AZ compliance chief and now consultant extraordinaire to the regulatory stars) stating that, “Marketers will promote the product to the full extent of the label.” No surprise there, but a real issue when it comes to promoting safe use of SMU medicines. Can marketers self-regulate? Is that even feasible? Short of an OPDP SWAT team for SMU medicines (with the authority to apply harsh penalties) how can marketers (per Preeti’s prescience) be made to “do the right thing?” What are the appropriate tools for post-marketing surveillance and how can they be validated?

Rob Califf (Duke Translational Medical Institute and Duke University Medical Center) pointed out that the label has become nothing more than a tool for legal protection – dismissing any future utility. But, in a post Wyeth v. Levine world, with preemption off the table, perhaps now is precisely the right time to revisit the public health utility of the PI.

Perhaps, as part of the SMU pathway, the FDA should adopt the EPARS (European Public Assessment Reports) system. The European Medicines Agency (EMA) publishes an EPAR for every medicine granted a central marketing authorization by the European Commission. EPARs are full scientific assessment reports of medicines authorized at a European Union level. 

Rachel Sherman, call your office.

An “almost” consensus of the meeting was the SMU pathway should proceed apace with anti-infectives as the pilot program and under existing regulatory authority. Almost. Maybe.

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Special Medical Use: Take One

08/02/2013 07:23 AM |

Yesterday I participated in the Brookings Institution/FDA meeting on Special Medical Use. I am gathering my notes and will have a more complete report ready for Monday.

In the meantime, here is an interesting take on the issue from the pages of Specialty Pharmacy Times.

FDA’s New Expedited Approval Program Provides Major Communication Breakthrough for Industry

Drugs that show a clear or substantial benefit to patients in the earliest stages of clinical trials can now reach the market more quickly, thanks to the FDA’s new Breakthrough Therapy designation. The expedited pathway, introduced by the Food and Drug Administration Safety and Innovation Act of 2012, has the potential to shave off nearly 75% (or by some accounts, nearly 2 years) of the time it typically takes the FDA to review a new drug application.

One of the major innovations of the expedited drug development process appears to be that drug companies now have direct access to something that previously eluded them: FDA officials. According to Reuters, efforts to bring promising therapies to market have been well-coordinated through the use of these pathways, and communications “that might typically take weeks and months” have been occurring more quickly, with fewer gaps between interactions.

The FDA began granting breakthrough designations in January 2013. To date, the agency has received 77 requests. Of these, the regulatory body has granted 25 designations and denied 24. Kalydeco (ivacaftor), Vertex’s cystic fibrosis drug, was approved under the breakthrough designation pathway earlier this year. Anticipated blockbusters such as Genentech’s obinutuzumab and Janssen/Pharmacyclics’ ibrutinib have snagged breakthrough designations as well. Obinutuzumab was granted the designation for the treatment of chronic lymphocytic leukemia (CLL), and ibrutinib was granted 2 breakthrough designations: one to treat patients with CLL and the other for patients with mantle cell lymphoma.

Although getting treatments to patients more quickly is definitely a good thing, a recent blog from Context Matters points out that, based on its calculations, the FDA’s previously established programs to expedite therapies (including Fast Track, Accelerated Approval, and Priority Review) have not produced lasting improvements in approval time. “Based on our preliminary analysis, in 2008 the average cycle time for ‘Priority’ was 10.1 months, versus the ‘Standard’ which was 21.2 months. In 2011, the average cycle time for ‘Priority’ was 19.5 months, versus ‘Standard’ which was 17.5 months,” the blog authors wrote. “What’s interesting here is that over time the fast lane has apparently become just another standard lane; in 2011 it was actually even slower.”

The group at Context Matters compared the process in the United States to that of the one used by the European Medicines Agency (EMA) and concluded that despite not having “fast” or “standard” designations, the EMA approval process took an average of just 3.5 months during 2012.

The FDA needs to have the proper infrastructure and support to successfully reduce approval times, says Peter Pitts, a former head of communications for the FDA who is a board member of Context Matters and co-founder of the Center for Medicine in the Public Interest. “Clearly, when you are looking at products that have less data behind them, you need more senior people who can devote greater resources to studying them, and that also takes time away from other programs that the agent needs to review,” Pitts told Specialty Pharmacy Times. “It’s one thing to give the FDA more authority, it’s another thing to give the FDA greater responsibility, but if you don’t give them the resources to get it done, to a large degree it is just rhetoric.”
 
Despite the improvements in communication and collaboration between drug developers and the FDA, manufacturers still must overcome many hurdles on the way to getting therapies approved by the agency. Sponsors must have more resources available in less time in order to push their therapies through the abbreviated regulatory process, and because many of the drugs being submitted for review under the new designation are targeted therapies, they frequently must be developed in concert with a companion diagnostic test. Such diagnostics would have to be approved separately by the FDA’s Center for Devices and Radiological Health, and guidance governing their development has been shaky so far.

Once therapies gain approval through the breakthrough therapy program, they still may face challenges in getting to patients as health plans may be reluctant to provide reimbursement for therapies with a limited amount of evidence regarding patient outcomes. Other issues that may thwart successful market penetration of drugs traveling through this pathway include timing of facility inspections and obtaining drug approval in other countries (where the breakthrough designation is not recognized or accepted). A lack of FDA resources may also affect the success of the breakthrough pathway in getting therapies to patients with few or no treatment options.

But, speed to market isn't the only metric that matters, Pitts points out. "Breakthrough designation doesn’t always mean the product gets approved...sometimes it means that because you fail faster, you don’t waste money on programs that don’t pan out."

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High and Mighty

07/31/2013 07:16 AM |

The Washington Post reports Capital City Care dispensary sold Washington DC’s first legal marijuana “in at least 75 years” on Monday. The sale is a result of a “15-year struggle to legalize medical marijuana in the district,” the culmination of a battle that “dates to the mid-1990s, when HIV/AIDS activists first fought to put medical marijuana on the citywide ballot.” About 70% of DC voters approved a 1998 legalization initiative, only to have it squashed by Congress for over a decade. But after Congress lifted the restriction in 2009, it still took several years for the city government to establish a “strict regulatory and licensing regime limited to city residents with specific chronic illnesses,” while also minimizing the “risk of future federal intervention.”

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NEHI and a mile wide

07/30/2013 06:13 AM |

The New England Health Institute has released a new report calling on public and private policymakers to adopt six “priorities for action” to improve the way patients take their prescription medicines across the US.

NEHI has created six “priorities for action” to improve the way patients take their prescription medicines across the US. They are: 

1) Promote sharing of best practices and lessons learned from pilots of new medication management techniques

2) Support large-scale implementation of promising, evidence-based “tactics” for improved medication management

3) Continue development of metrics of medication use that will spur adoption of proven medication management strategies

4) Support continued rapid adoption of electronic prescribing and electronic medical records with capabilities that support evidence-based interventions for improved adherence

5) Continue to improve Medication Therapy Management services in Medicare Part D including improvements in program services and targeting; consider wider adoption of medication management by other health care payers

6) Integrate medication adherence research, policy development and advocacy with broader efforts that aim to improve use of medicines, including those focused on patient safety

The report concludes:

Proponents of better patient adherence should rally behind a comprehensive vision of good medication use that encompasses interventions to promote adherence, and promote a vigorous agenda for policy change that will create incentives for proven adherence interventions.

Words to the wise.

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Howard Dean sees the light?

07/30/2013 05:02 AM |

Howard Dean on IPAB:

One major problem is the so-called Independent Payment Advisory Board. The IPAB is essentially a health-care rationing body. By setting doctor reimbursement rates for Medicare and determining which procedures and drugs will be covered and at what price, the IPAB will be able to stop certain treatments its members do not favor by simply setting rates to levels where no doctor or hospital will perform them.

There does have to be control of costs in our health-care system. However, rate setting—the essential mechanism of the IPAB—has a 40-year track record of failure. What ends up happening in these schemes (which many states including my home state of Vermont have implemented with virtually no long-term effect on costs) is that patients and physicians get aggravated because bureaucrats in either the private or public sector are making medical decisions without knowing the patients. Most important, once again, these kinds of schemes do not control costs. The medical system simply becomes more bureaucratic.

The nonpartisan Congressional Budget Office has indicated that the IPAB, in its current form, won't save a single dime before 2021. As everyone in Washington knows, but less frequently admits, CBO projections of any kind—past five years or so—are really just speculation. I believe the IPAB will never control costs based on the long record of previous attempts in many of the states, including my own state of Vermont.

Read the full piece in the Wall Street Journal.

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Arrant Transparent

07/29/2013 09:07 AM |

Speaking of clinical trial data transparency – here’s an excellent analysis from the August edition of the Burrill Report ...

Fight for Transparency Heats Up: Industry turns to courts to stop policy in EU, offers own approach

Daniel S. Levine

Tom Jefferson had nagging concerns about his review for the Cochrane Collaboration of clinical studies of the flu drug Tamiflu, but they crystallized in 2009 when a Japanese pediatri­cian raised a question about the work via email. The doctor want­ed to know why the review in­cluded eight unpublished trials that Jefferson and his colleagues hadn’t seen, and had only been included as summaries in an­other study funded by the drug’s producer Roche?

The solution seemed simple enough. Jefferson, an epidemi­ologist based in Rome who was conducting the updated analy­sis for the Cochrane Collabora­tion, an independent group that provides guidance to health­care professionals on the use of drugs, would reach out to the authors of the studies and get them. But the authors either told him they didn’t have the data, said they had never seen the data, or ignored him completely. When he asked Roche for the studies, it said it would provide them, but insisted at first that he sign a confidentiality agree­ment, a condition that he found unacceptable.

The controversy came against a backdrop of worldwide fear of a global pandemic of bird flu that sent the World Health Or­ganization, the Centers for Dis­ease Control and Prevention, and governments around the world spending billions of dollars to stock­pile Tamiflu. Despite the spending, Jefferson says there were questions about whether the drug did anything to prevent transmission of flu, mini­mize complications, or whether the health benefits the drug provided jus­tified the risks of using it. Roche, in a statement posted on its web site and updated at the end of February 2013, said that it had disagreed with the analysis plan Cochrane Collaboration shared with it because it was at odds with how Tamiflu has been reviewed and approved by regulatory authori­ties in more than 80 countries. The company said it stood by the “robust­ness and integrity” of its data support­ing the safety and efficacy of the drug.

“The fight for the last four years hasn’t been so much for clinical study reports,” Jefferson says, “but to have clinical study reports without having to sign confidentiality agreements, funny handshakes, rolling up our trouser leg, and so on.”

The four-year battle to obtain clini­cal trials data—he believes that there have been 123 studies (74 of which Roche sponsored)—has made Jeffer­son a flag bearer in an international fight over clinical trials data transpar­ency. The controversy, which is by no means limited to Tamiflu and Roche, is now reaching a critical point. A le­gal battle is underway in Europe over a European Medicines Agency trans­parency policy expected to take effect at the start of 2014 that would make public clinical trial data once a drug is approved. At the same time, in the United States, the Institute of Medi­cine is at work on a consensus study on the issue and the U.S. Food and Drug Administration is seeking com­ment on a clinical data transparency policy of its own.

Much is at stake as the policymak­ers embrace comparative effectiveness and the advent of Big Data provides a new means to ferret out untapped information hidden within clinical study reports—the detailed narrative reports at the individual patient level that can run to tens of thousands of pages in a single trial.

Proponents of clinical data transpar­ency argue that it will provide doctors with greater insight into the safety and efficacy of the drugs they prescribe, improve care, cut waste, and minimize scientific misconduct and fraud. Peter Doshi, a post doctoral comparative ef­fectiveness researcher at Johns Hop­kins University, who is also working on the Tamiflu review for the Cochrane Collaboration, points to a list of block­buster drugs that were marketed only to have significant health risks come to light through academicians analyzing clinical trials data. “The whole system has depended on trust in medicine, trust in people to report properly, trust in people to have enough information to credibly analyze trials,” he says. “We know enough not to be so trusting anymore.”

Industry, while not of one mind, says that in principle it doesn’t oppose transparency, but expresses concerns about how it is done. These concerns include the threat to confidential com­mercial information, the challenge to the authority of regulators, the risk to patient privacy, and the opening of the data to inappropriate uses that could lead to the publication of misleading results and public health scares.

Lawsuits in Europe

In May, the General Court of the European Union prohibited the Euro­pean Medicines Agency from releas­ing data from two AbbVie and Inter­mune trials in an interim ruling, part of a challenge by the two drugmakers to the agency’s decision to grant access to information the companies provid­ed as part of their market approval ap­plications. The challenge is the first to be made to the EMA’s three-year old access-to-documents policy.

Since November 2010, the Agency has released more than 1.9 million pages in response to such requests. The legal battle involves documents relat­ing to AbbVie’s rheumatoid arthritis drug Humira and InterMune’s Esbriet, a treatment for idiopathic pulmonary fibrosis, an unexplained chronic and progressive scarring of the lungs. In both cases, competitors of the compa­nies were seeking the data.

“Biopharmaceutical companies support responsible data sharing that protects patient privacy, maintains the integrity of the regulatory review pro­cess, and preserves incentives for bio­medical research,” said Matt Bennett, senior vice president for the Pharma­ceutical Research and Manufacturers of America in a statement at the time. “Unfortunately, the EMA’s current and proposed policies fail to respect these principles.”

The ruling comes amid increasing pressure from medical journals and patient advocates on pharmaceutical companies to provide complete trans­parency and make public all of their clinical trials data. It also comes as the EMA readies implementation of a new policy to proactively publish data from clinical trials supporting the approval of new drugs once a decision has been made. The EMA said it would continue the process of drafting its policy on publication of clinical trials data.

Ben Goldacre, author of “Bad Phar­ma” and co-founder of the AllTrials campaign, which seeks publication of all results from all clinical trials, called the ruling “a disgrace.” “There is no justification for withholding informa­tion about the methods and results of clinical trials from doctors, research­ers, payers and patients, who need all the information on a medicine to make truly informed decisions,” he says.

The EMA says it welcomes the op­portunity for legal clarification of the concept of commercially confidential information, but expressed “regret” over the decision to grant interim re­lief to AbbVie and InterMune.

FDA, IOM, weigh transparency

In the United States, although ef­forts to bring about transparency have moved slower, they are advancing. In October 2012, the Institute of Medicine held a two-day workshop to explore the benefits of sharing clinical research data, the issues surrounding it, and how best to do so. The IOM is now working on a consensus study that some hope could provide a roadmap for the devel­opment of a clinical trials data transpar­ency policy in the United States.

A group of leading pharmaceutical and biotechnology companies has been involved in the IOM process and issued a press release hailing the effort. “This industry group is strongly in support of enhanced access by third party re­search to clinical trial data generated by industry and academia in a manner that ensures that patient confidential­ity is preserved, scientific integrity is maintained, and intellectual property rights and confidential company infor­mation are protected,” they said in a joint statement.

The Food and Drug Administration is seeking public comments through August 5 on a policy to make available de-identified and masked data derived from medical product applications. But advocates of transparency say such data will be of limited utility because the policy under consideration would de-identify it and remove the data’s link to a specific product, study, or application.

Peter Pitts, president of the Cen­ter for Medicine in the Public Interest and chairman of a conference on clini­cal trials data transparency held by FDANews July 23-24, says it’s “a non-arguable fact that FDA is at the nexus of vast amounts of clinical data that if it was appropriately shared it would be of tremendous value.” He says that sharing information, among other things, could help companies fail faster, which would save a lot of money and allow for reinvestment of diminishing resources. But such an effort, he says would require information technol­ogy resources the agency is without, decisions on what is and isn’t redacted, discussions about intellectual property rights and commercial confidentiality, and funding for it all.

“We’ve come to the point now, rela­tive to clinical trials data, that transpar­ency is a good thing. The question now becomes, ‘How do you accomplish that and what exactly does transparency mean? And how quickly does it be­come transparent? And how much of it becomes transparent and how much of it remains confidential? Is it transpar­ent through corporate entities, or is it transparent through a consortium of corporations? Is it transparent through a government organization? And who ultimately decides? Can people opt out? And if people opt out, can you really have transparency?’” says Pitts. “They are all very tough questions.”

(Note: For more on the FDA News conference, see Cry “havoc,” and let slip the dogs of data transparency.

Progress, but no victory

Four years after Jefferson began his efforts to access clinical study reports from Roche and GlaxoSmithKline, which produces the Tamiflu competi­tor Relenza, both of which are in the class of drugs known as neuramini­dase inhibitors, it is Roche and GSK that arguably have the clearest defined and most advanced efforts to provide clinical trials data to outsiders.

Roche has complied with the origi­nal request the Cochrane reviewers made in 2009, although the researchers continue to pursue access to additional trials data of which they subsequently learned. The Cochrane Collaboration expects at the start of 2014 to publish a new review of neuraminidase inhibi­tors based on 23 clinical studies on Re­lenza and up to 20 studies on Tamiflu.

In February, GSK announced it had signed on in support of the AllTri­als campaign and has vowed to pub­lish clinical trials data of all approved drugs dating back to 2000, the date the company was formed through the merger of Glaxo Wellcome and Smith­Kline Beecham.

“We are committed to being trans­parent with our clinical trial data to help advance scientific understanding and inform medical judgment,” Patrick Vallance, President, Pharmaceuticals R&D, GlaxoSmithKline, said in an­nouncing support for the campaign. “Our commitment also acknowledges the very great contribution made by the individuals who participate in clin­ical research. All those involved in the conduct and publication of clinical re­search, whether healthcare companies like GSK, academia or research organi­zations, have a role to play in ensuring that the data they generate are made publicly available to help bring patient benefit.”

In response to questions from The Burrill Report, GSK said it believes a broader solution for providing access to trial data from across the research com­munity needs to be developed and it has been in discussion with trials sponsors from industry, academia, and research charities working in partnership to cre­ate one. GSK says one solution would be the establishment of an independent data custodian to which research spon­sors would deposit anonimized data after a project has been completed and clinical studies are published. Research­ers could then submit scientific propos­als and analysis plans to independent custodian to request access.

The Pharmaceutical Research and Manufacturers of America and the Eu­ropean Federation of Pharmaceutical In­dustries and Associations at the end of July jointly issued a list of principles for clinical trial data sharing. They call for a voluntary plan that would make data available to only to “qualified” research­ers who sign non-disclosure agree­ments. They say they would only share data for which they have the informed consent of study participants to do so.

(Note: The full principles can be found on the PhRMA website. PhRMA said implementation of the commitments in the principles will begin on January 1, 2014.)

Companies will start reviewing requests for data from researchers at that point, but Castellani said companies will need to take into account patients’ informed consent. He said it will not be possible to share data from many earlier trials because patients participating in them did not consent to such release.)

But advocates worry that the vic­tories in studies extracted from phar­maceutical companies to date may be short lived, in large part due to the legal battle in Europe over the EMA’s transparency policy. “Societal expec­tations have dramatically moved in a direction of greater transparency, but there remain serious risks to the pro­cess that could potentially derail a lot of the progress that has been made,” says Johns Hopkin’s Doshi. “The prog­ress we’ve seen with Tamiflu may have been a one-off victory for what other­wise may be a return of data secrecy. Many people think the goals of trans­parency have been achieved, but they have not.”

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