Amazing Pace

05/07/2012 01:06 AM |

According to FDA Commish Peggy Hamburg, since President Obama gave the agency new authorities to address drug shortages new shortages have fallen (year-to-year) by more than half.  There have been 42 new shortages in 2012 compared to 90 at the same time last year.

But is this decline really due to the FDA’s ability to demand earlier information about potential shortages from manufacturers? 

“I am both amazed and delighted to see the progress that’s been made, said Hamburg.  Key word:  “Amazed.”

While the FDA’s new authorities are both timely and important, there are many pieces to the drug shortages problem – not the least of which is that (when it comes to hospital injectables) 30% of manufacturing capacity is off-line due to FDA inspection issues. That’s a lot of capacity. In fact, according to the agency, 43% of reported potential shortages were due to manufacturing issues.

Greater cooperation between agency and manufacturers is required so that this gap can be corrected before problems arise.  A 30% hole in manufacturing is more than a hint that something’s amiss on both sides.

Is it a victory when you are solving the problems you create?


  Read More & Comment...

The Calendar Days of our Lives

05/04/2012 07:18 AM |

Peggy Hamburg:  Calendar Girl

Big hat tip to http://www.fdalawblog.net for the excellent reporting.

GAO Report Says That FDA Has Met Most PDUFA Performance Goals; Agency Plans to Take Steps to Address Lingering Stakeholder Concerns

A report released earlier this week by the Government Accountability Office (“GAO”) says that FDA has met most of the Agency’s PDUFA performance goals for priority and standard original NDA and BLA submissions and for priority and standard original efficacy supplements to approved NDAs and BLAs, although in each case FDA review times have increased slightly.  The GAO’s analysis covers applications in the Fiscal Year 2000 to 2010 cohorts, as well as preliminary information for applications submitted in Fiscal Year 2011. 

The GAO report was sent to Senators Richard Burr (R-NC) and Tom Coburn (R-OK).  Both Senators have long criticized FDA, saying that the Agency‘s “regulatory malaise” harms patients and manufacturers.  Senator Burr has also threatened to delay the passage of PDUFA and other user fee legislation unless FDA speeds up application approval times.  In addition, Senator Burr was successful in getting an amendment added to the FDA appropriations bill passed last year that seeks to “improve the transparency and accountability of the FDA in order to encourage regulatory certainty and innovation on behalf of America’s patients.”  That amendment requires the submission of information to Congress on, among other things,

• “the average number of calendar days that elapsed from the date that drug applications (including any supplements) were submitted to such Secretary under [FDC Act § 505] until the date that the drugs were approved under such section 505;” and
• “the average number of calendar days that elapsed from the date that [BLAs] (including any supplements) were submitted to such Secretary under [PHS Act § 351] until the date that the biological products were licensed under such section 351.” 

The GAO’s analysis shows that except for Fiscal Year 2008, FDA met PDUFA goals in all of the Fiscal Year 2000 to 2010 cohorts.  (FDA recently provided similar statistics in testimony before Congress.)  Moreover, the GAO found that an average of 44% of all original NDAs and BLAs submitted to FDA in Fiscal Years 2000 to 2010 were approved during the first review cycle and 75% were ultimately approved.  FDA and industry stakeholders the GAO interviewed suggested that FDA failed to meet Fiscal Year 2008 goals as a result of implementation of the Risk Evaluation and Mitigation Strategy (“REMS”) requirements added to the FDC Act by the 2007 FDA Amendments Act.

Although FDA met most PDUFA goals for the Fiscal Year 2000 to 2010 application cohorts (and is on track to meet Fiscal Year 2011 goals for the applications submitted in that cohort), the GAO’s analysis (reflected in the tables below) shows that average FDA review times (i.e., the time elapsed from when FDA received a submission until it issued an action letter) hve increased slightly from Fiscal Year 2000 through Fiscal Yeat 2010 for both priority and standard NDAs and BLAs and priority and standard original efficacy supplements to approved NDAs and BLAs.

With respect to Senator Burr’s request for the average number of calendar days that elapsed from the date of NDA or BLA (including supplement) submission to final FDA action, the GAO says that it was unable to calculate average FDA review times in any meaningful way because most cohorts were still open; that is, “fewer than 90 percent of submissions had received a final action such as approval, denial, or withdrawal.”  Specifically, for priority original NDAs and BLAs, only four cohorts had at least 90% of submissions closed (Fiscal Years 2001, 2002, 2005, and 2006), and for standard original NDAs and BLAs, only one cohort had at least 90% of submissions closed (Fiscal Year 2002).  For priority efficacy supplements, only four cohorts had at least 90% of submissions closed (Fiscal Years 2000, 2001, 2004, and 2007), and for standard efficacy supplements, only one cohort had at least 90% of submissions closed (Fiscal Year 2005).

Stakeholders the GAO interviewed identified some issues that they believe hamper the NDA and BLA approval process, including REMS implementation, the use of outside expertise for reviewing applications, insufficient communication between FDA and stakeholders, and a lack of predictability and consistency in FDA reviews.   FDA commented in the Agency’s response to the GAO report that it is taking or has agreed to take steps (as part of PDUFA V) that may address these issues, including issuing new guidance, establishing new communication-related performance goals, training staff, and enhancing scientific decision making.

  Read More & Comment...

Heavy Meddle

05/03/2012 09:12 AM |

The American Recovery and Reinvestment Act (aka, “the stimulus package”) provided AHRQ with $29.5 million for a program on academic detailing and the “communication of CER results to physicians.”

One contract, for $11.7 million, went to Total Therapeutic Management (TTM) and is specifically intended for physician outreach and education.

(TTM is a company that focuses on chart abstraction, data mining, and physician and patient education for a predominantly commercial client base -- health plans, pharmacy benefit managers, employers, and pharmaceutical companies, etc.)

The goal of this contract is to integrate AHRQ’s comparative effectiveness research, products, and tools into clinical practice through 9,000 on-site, face-to-face visits with clinicians, nurses, health plan formularies, benefit managers, and other healthcare professionals.

I recently interviewed Barry Patel, the president of TTM.  Here are some snippets from our conversation:

How will the government decide which doctors are to be visited? Will ‘‘high prescribers’’ of on-patent medicines be on a priority list?

TTM’s top priority is ‘‘high volume’’ practices across 150 Metropolitan Statistical Areas (MSAs). So, rather than focusing on offices with disproportionately high negative patient outcomes, the government is directing its efforts against those doctors who are high prescribers—which is a pretty good indicator about what government detailing is all about—decreasing cost rather than improving care.

When it comes to government detailing (at the taxpayers’ expense), what are the metrics for success?

According to Mr. Patel, the only metrics are whether or not a physician says the sessions have been useful and asks the detailer to come back to discuss other topics. In other words, the metrics are subjective and anecdotal -- not clinical.

Interestingly, Mr. Patel doesn’t even agree with either the term academic detailing or counter detailing. ‘‘We aren’t counter anything. We’re not there to undo anything. It’s not good versus bad. Our visits aren’t details, they’re the beginning of a process.’’ And, as far as ‘‘academic’’ goes, Mr. Patel uses that term because “that’s the phrase AHRQ uses and placed in the contract. Our people are patient-centered outcomes consultants, PCOCs.” And “his people” are largely pharmacists and nurses.

A former Merck employee, Patel likens his PCOCs more to pharmaceutical company Medical/Science Liaisons (MSLs) than field representatives. ‘‘They’re not discussing product-specific information, but the findings of comparative effectiveness studies.”

How does TTM schedule their appointments with targeted physicians?

According to Mr. Patel, when his ‘‘outreach experts’’ phone physicians to request appointments, the fact that the meeting will result in CME credits is always mentioned. Would a pharmaceutical company be permitted to offer such an enticement? Would such an offer be ‘‘sunshine-able’’ under state and federal guidelines? And, if so, why don’t government detailers have to share the details of their valued benefactions?

Interestingly, according to the Accreditation Council for Continuing Medical Education (ACCME), government is exonerated from having a commercial interest. (A commercial interest is any entity producing, marketing, re-selling, or distributing healthcare goods or services consumed by, or used on, patients.)

Our nation’s single largest payer, Uncle Sam, is not deemed to have a conflict of interest when it comes to designing and providing physician CME.

What’s wrong with this picture?

  Read More & Comment...

The Rise of the Administrative State

05/02/2012 12:01 PM |

Bob Moffitt,  The Heritage Foundation’s senior fellow in domestic and economic policy studies gave a lecture to members of Congress recently.  It addresses the historical and cultural impulse that has turned government into a mechanism for producing equality of results instead of a system the that increases opportunity by advancing liberty.   

At a time when more government agencies have been created to regulate and redistribute economy activity, with a focus on finance,  energy production and of course health care, it is useful to reflect upon how this fundamental shift in power and direction will shape our future.   Most important in my mind is the way in which the administrative state is being used to create ideological no-fly zones for those who disagree or challenge the guiding principles of the agency and the professional experts who feed off it.  

Bob Moffitt's lecture is an important contribution to this discourse.    Harry Truman noted" Once a government is committed to the principle of silencing the voice of opposition, it has only one way to go, and that is down the path of increasingly repressive measures, until it becomes a source of terror to all its citizens and creates a country where everyone lives in fear.”   Keep Truman's warning in mind as you read Moffitt's lecture. 

http://www.heritage.org/research/reports/2012/03/why-congress-must-confront-the-administrative-state?query=Why+Congress+Must+Confront+the+Administrative+State  Read More & Comment...

And speaking of benefit/risk ...

05/02/2012 06:57 AM |

BioCentury reports:

IOM: FDA should consolidate benefit-risk data for each drug

The Institute of Medicine said FDA should consolidate benefit-risk information for approved drugs into publicly available repositories created for each product. In a report released Tuesday, IOM said the agency already collects much of the information needed but that it is spread across multiple records. The report recommended the creation of a publicly available document containing information for each product from its approval throughout its entire time on market, including safety issues, regulatory actions and any restrictions. FDA said in a statement it supports the general concept, but added that it would be "very challenging to implement this recommendation within our current resources without seriously compromising other critical regulatory activities."

IOM also noted in the report that there is no universal set of criteria to determine when FDA should require a postmarketing study to evaluate a drug's safety. However, the report noted that the agency should require additional post-marketing safety research when a drug's benefits or risks are particularly uncertain, including first-in-class drugs approved based on surrogate endpoints, drugs for which endpoints provide conflicting evidence about risk or drugs with a strong biological rationale for a particular side effect. IOM, which conducted the review at FDA's request, issued a preliminary report in 2010.

  Read More & Comment...

RIP RCTs

05/02/2012 01:19 AM |

An article in The Scientist makes much about how using raw data from randomized clinical trials can change results. But while the article trumpets the importance of transparency and equates more clinical trial data with better science, in fact the massive amount data being generated from randomized clinical trials is nearly beside the point because data from real world use and the collection of biological and genomic information about treatment response can be collected more quickly and at a lower cost. So why are academics and interest groups obsessing about more trial data? I think data diving gives them a reason to apply for grants and an endless supply of cherries to pick to score policy and political points. Dumping more data based on evidence generation methods of the 19th century is no way to take patients and their needs seriously. http://the-scientist.com/2012/05/01/data-diving/   Read More & Comment...

Avandia and Children With Type 2 Diabetes 1, Steve Nissen 0.0

04/30/2012 10:14 AM |

A recent article in the New England Journal of Medicine evaluating the results of an experiment on how to treat type 2 diabetes in children raises some interesting questions:

http://www.nejm.org/doi/full/10.1056/NEJMoa1109333?query=featured_home#t=articleBackground

The Treatment Options for Type 2 Diabetes in Adolescents and Youth (TODAY) study found that metformin and Avandia were superior to other treatments in controlling the disease in kids.

I have two questions:  Will Steve Nissen admit he was wrong, again?  Also,  will doctors start to use existing diagnostics that identify those who do not respond to metformin, at least in kids.  Isn't that choosing wisely?  



  Read More & Comment...

The Learned Disintermediary

04/30/2012 08:18 AM |

Patient, diagnose thyself -- and then reach a little deeper into your wallet.

Physician, step aside.

Pharmacist, step up to the plate.

Maybe.

The FDA may soon permit Americans to obtain some drugs used to treat conditions such as high blood pressure and diabetes without obtaining a prescription.

The FDA says over-the-counter distribution would let patients get drugs for many common conditions without the time and expense of visiting a doctor, but medical providers call the change medically unsound and note that it also may mean that

Under the changes that the agency is considering, patients could diagnose their ailments by answering questions online or at a pharmacy kiosk in order to buy current prescription-only drugs for conditions such as high cholesterol, certain infections, migraine headaches, asthma or allergies.

By removing the prescription requirement from popular drugs, the Obama administration could ease financial pressures on the overburdened Medicare system by paying for fewer doctor visits and possibly opening the door to make seniors pay a larger share of the cost of their medications.

The change could have mixed results for non-Medicare patients. Although they may not have to visit a doctor as often, they could have to pay out more money for medications because most insurance companies don't cover over-the-counter drugs.

How will this impact patient compliance?  How will this effect one condition masking another, more serious one?

How will this change the role of the pharmacist?

Comments on the FDA proposal are due by May 7.

  Read More & Comment...

PDUFA -- If and When and How

04/30/2012 07:12 AM |

“Cozy Deal” (New York Times, April, 29, 2012) argues,

“The best approach would be for the government to fully finance the F.D.A. That is unlikely to happen. So before it ratifies any new deal on user fees, Congress must ensure that patient safety is the first priority.”

Sure, but unlike the Gray Lady we must all live in the real world. User fees are the cost of predictability -- not just for innovators who wants to know if and when they can commence marking new products but also for patients and physicians who want to know if and when these new treatments will be available.  Predictability is also an issue of how. And this rests on the ability of the FDA to create and communicate a thoughtful and patient-centered model of risk/benefit analysis. Thankfully this is a central part of the current reauthorization debate.

  Read More & Comment...

A Methodology to PCORI's Madness

04/27/2012 07:49 AM |

Talk about positive outcomes!

It pays to have friends on PCORI’s Methodology Committee.  Five of the seven grantees selected for PCORI funding are directly affiliated with members of that panel. In dollar terms its even more lopsided. Of the $799,524 awarded, $721,940 went to institutions with direct representation on the conclave.

If those committee seats are pro bono somebody should ask -- Cui bono?

  Read More & Comment...

Celebrating Israel's Independence and Existence

04/26/2012 03:18 PM |

Zach On Patrol Near Gaza 2012




Today is Yom Haatzmaut  (Israel Independence Day) and Peter Beinart will be celebrating it by giving a lecture at a synagogue about his anti-Israel book, “The Crisis of Zionism.”  In doing so, Beinart joins Gunter Grass and the producers of the 60 Minutes segment (blaming Israel for the withering away of the Palestinian Christian community) in proposing that Israel can only be purified if it ceases to exist as a Jewish state.

Victor Davis Hanson points out that the new anti-Semitism regards the creation of the Jewish state as a manifestation of the Western hegemonic impulse.  It  barely concedes that  Israel is democratic and protects human rights in a way unlike its autocratic neighbors.   It’s proponents ignore the systematic persecution of Christians in Arab countries, the torture and extortion of Hamas, the ongoing slaughter in Syria, the resurgence of the Islamic Brotherhood.

Rather, they are focused on forcing Israel to give up what the Economist calls the  “Auschwitz Complex.” accrued from the Holocaust, a pathology which they say its unwillingness to  “to give up its empire” (by crawling back into the 1949 borders of the state and absorbing millions of Palestinians).

The remembrance of the Holocaust is a searing reminder of the necessity of Israel.   Yom Hashoah is recalled by a two minute siren -- invoking the shofar – that turns all moving things and people into standing monuments.. The moment stretches on, the wail growing tighter and thinner, like a taut thread, binding all together, until it unwinds.

But Yom Ha-Shoah is only the tragic beginning of the story, not the end.  Seven days after,  Yom Ha’atzmuat (Israel Independence Day) is celebrated.  The day preceding that holiday, Israel observes Yom Ha-Zikaron ( Memorial Day)  to reaffirm (as written in the Israel Defense Force manual) “that establishing the State of Israel was intended to provide an answer to the problem of the existence of the Jewish people, in view of the fact that all other solutions had failed.  The Holocaust proved, in all its horror, that in the twentieth century, the survival of Jews is not assured as long as they are not masters of their fate and as long as they do not have the power to defend their survival.”

The movement from Yom Hashoah, to Yom Ha-Zikaron to Yom Ha'atzmaut reminds people, as Rabbi Irving Greenberg notes, that the “ State of Israel is not a reward or a product or an exchange for the Holocaust; it is a response. The Jewish people responded to the total assault of death by an incredible outpouring of life.”

And with an enduring sense of purpose.  When my son immigrated to Israel in 2007 and joined the IDF he was repeatedly asked why he did so.  He could never fully explain to others or to himself until his unit was called into battle during 2008 Operation Cast Lead against Hamas. 

He was in an APC with his brothers in arms.  He looked around at his comrades with who he shared the cramped space. 

Zach writes:

“To my right was the sharpshooter, an Ethiopian by the name of Redit Mullah. His parents were thrown onto Israeli chartered planes and saved from political instability and war in the 1991 Solomon Operation, when Israel rescued and integrated thousands of Ethiopian Jews into its growing populace. 

To my left was my friend Avichai Galilee, of Yemenite descent, whose grandparents were similarly rescued from persecution and vehement Arab violence by a massive and unprecedented Air-Evac operation known as ‘Operation Magic Carpet’ in 1950.
In back of me were two soldiers, of Russian and Iraqi descent.  They both shared the same legacy of being rescued and welcomed by the Jewish state when they had no place else to live as Jews.   It did not matter that the Israeli economy was anemic at the time and that we had few resources to spare. We did it anyways.

It then finally made sense to me: The state of Israel is there for us, and so should we be there for the state of Israel. Riding away in our APC’s it was us who was thanking Israel and paying a debt of gratitude for all it had done for us. “

Tomorrow Israel celebrates this spirit.  So do most Americans, who know that a democracy can achieve great things only if the citizens believe it is worth defending.   Only people like Beinart want to strip the Jewish state of this eternal essence.
  Read More & Comment...

10 More Reasons Why PCORI and CER Are Obsolete

04/26/2012 03:01 PM |

Breast cancer is at least 10 different diseases, each with its own genetic signature and pattern of weak spots, according to a new landmark study that promises to revolutionize diagnosis and prognosis, and pave the way for individualized, tailored treatment.

The study group, METABRIC (Molecular Taxonomy of Breast Cancer International Consortium), reports its findings in the 18 April online issue of Nature.  The Cancer Research UK-funded study is the largest global gene study of breast cancer tissue ever conducted, involving a large team of researchers, primarily in the UK and Canada.

Led by Professor Carlos Caldas from Cancer Research UK's Cambridge Research Institute and Professor Sam Aparicio from the British Columbia Cancer Centre in Canada, the team uncovered crucial new information about breast cancer. The researchers analyzed the DNA and RNA of breast tumor samples from nearly 2,000 women who had been diagnosed between five and 10 years ago, and for whom information about the tumor characteristics had been meticulously recorded. They compared this with the women's survival, and other information, like their age at diagnosis. Because the study was able to look at many tumors with rich data on each, it identified new patterns and "clusters" in the data not spotted before. In the study, the team classified breast cancer into at least 10 different subtypes: each characterized by common genetic features that link to survival. This suggests we need to rethink what we call breast cancer and start looking at it as at least 10 different diseases, each with its own molecular fingerprint and pattern of weak spots.

Dr Harpal Kumar, chief executive of Cancer Research UK, told the press the study will completely change the way we look at breast cancer.  Caldas, who is also Professor of Cancer Medicine at Cambridge's Department of Oncology, said "breast cancer" should be regarded as an umbrella term for a range of diseases. The findings could change the way drugs are tailored to treat individual women with breast cancer. The team also discovered several previously unknown genes that drive breast cancer. Each of these is a potential target for new drugs, and should boost worldwide efforts to discover and develop new treatments. The study also reveals the relationship between these breast cancer genes and known signalling pathways, the networks that control cell growth and division. This invaluable knowledge will help identify how variants of these genes cause cancer by interfering with cell processes.

Over decades, the METABRIC project has produced a "goldmine" of data, says Caldas. The process is not unlike that of cartography. At first, intrepid explorers discover new continents, defined by outlines and some rough impressions of terrains and landscapes. Then gradually, as mapping techniques improve, the data becomes more detailed and more precise. The METABRIC team now has a detailed "map" of thousands of individual tumors that have been analyzed and re-analyzed in many different ways and linked to detailed information about the fate of the women they came from.

Not only has the team performed all kinds of analysis on the DNA of the tumors (for instance the map is now annotated with copy number changes and single letter variations or SNPs, for each tumor), it has also conducted a detailed analysis of their RNA so they can tell which genes were active in each sample. Altogether they did this for more than 30,000 types of RNA, each corresponding to the activity of a single gene. "We've drilled down into the fundamental detail of the biological causes of breast cancer," said Caldas, "we've moved from knowing what a breast tumour looks like under a microscope to pinpointing its molecular anatomy". "Our results will pave the way for doctors in the future to diagnose the type of breast cancer a woman has, the types of drugs that will work, and those that won't, in a much more precise way than is currently possible," he added.

Aparicio said: "The new molecular map of breast cancer points us to new drug targets for treating breast cancer and also defines the groups of patients who would benefit most." Caldas said these results will not affect women diagnosed with breast cancer today, but he envisages future breast cancer patients will receive treatments tailored specifically to the genetic fingerprints of their tumors. From this huge leap forward, the next step is to find out how the tumors in each of subgroup behave.

Caldas had this to say to the patients behind the study: "I want to stress, this study wouldn't have been possible without the breast cancer patients who donated their samples and agreed to take part in the study. None of this would have happened without them, and I'm so grateful for their participation." For an excellent account of how we have increased our understanding of breast cancer, plus a table showing the 10 disease subtypes, see Henry Scowcroft's post in the Cancer Research UK Science Update blog.  Read More & Comment...

P2P Healthcare & the Portals of Power

04/26/2012 07:54 AM |

Transparency (via social media) is leading to erosion in trust of once sacrosanct gurus such as physicians, corporations, their avatars and other “experts” (not the least of which is the mainstream media).

It’s been a painful and swift denuding of influence. Rather than being slowly disrobed, yesterday’s unquestioned experts have been roughly stripped of their gravitas and authority. Some have behaved badly, the majority has ignored it. Too few have gotten the message – adapt or die. You can’t airbrush social media.

Perhaps (and hopefully) this isn’t so much a downward spiral as it is (in the words of Schumpeter) “creative destruction.”

As Schumpeter writes in Capitalism, Socialism and Democracy, “The fundamental impulse that sets and keeps the capitalist engine in motion comes from the new consumers, goods, the new methods of production or transportation, the new markets, the new forms of industrial organization that capitalist enterprise creates.”

He would have loved social media.

While various “emperors” are being exposed as having no clothes, the void is being filled with robust and real-time peer-to-peer communications. Alas, there are also many ascendant false prophets. The Internet is full of them. Some are well-meaning (but still dangerous) idiots (such as the anti-vaccine crowd), others pure charlatans ("Cure your cancer in Mexico!").

As Don Draper once said, “I'm enjoying the story so far, but I have a feeling it’s not going to end well.”

Social media is a wonderful “green field of opportunity.”  But to maximize the opportunity, we must accommodate the reality of a messier world.  Social media, almost by definition, is messy – and the regulatory framework (or lack thereof) is equally so. And it’s not likely to get much better. Get used to it.

All this doesn’t mean that social media is a bad thing. Nobody said it was going to be easy. If we want to change the healthcare paradigm (and for some that’s a big “if”), then changing the way people learn, discuss and address healthcare issues is a crucial element. And, unlike other aspects of healthcare change – it is happening with great rapidity.

Impact and influence happen when what you have to share is to the benefit of the seeker — not to you.  And that requires a level of focus, acumen and honesty that is always hard and often lacking – especially when it comes to healthcare marketing. As the saying goes in our nation’s capital, “if you’re not at the table, you’re on the menu.”

In the words of Winston Churchill, “Ease is relative to the experience of the doer.”

We’re still looking to healthcare professionals for technical solutions (physicians are no longer the first and last word, but the first among equals). When it comes to practical advice, it’s an increasingly peer-to-peer proposition. Today (for better or worse) we are all “learned intermediaries.” (But some as more learned than others – a fact we need to recognize and advocate.)

Welcome to the new world of P2P Healthcare where social media holds the keys to the portals of power.  And as Dr. James Fowler of the University of California at San Diego, opined, “Pharma must realize their own network power.”

Social media is communications at the speed of life. As Marshall McLuhan wrote, “At electric speed, all forms are pushed to the limits of their potential." 

(Still think you can wait for more precise and directive FDA regulations?)

  Read More & Comment...

PDUFA Update

04/25/2012 11:07 AM |

The Energy and Commerce Health Subcommittee PDUFA Reauthorization Markup scheduled for tomorrow has been postponed.  The Health Subcommittee Markup will occur on Tuesday, May 8.  The Full Committee may have opening statements on the user fee bills Wednesday, May 9 and possible Full committee mark-up Thursday, May 10.  The Chairman cited continued staff negotiations for the reason for the postponement  Read More & Comment...

Endgame for PDUFA Primaries

04/25/2012 06:55 AM |

The PDUFA "primary season" is almost over

Today, BioCentury reports, the Senate HELP Committee will deliberate on a PDUFA reauthorization bill that would relax conflict-of-interest restrictions on advisory committee members, and enact new provisions intended to improve risk-benefit decision-making, facilitate global harmonization of clinical trials and promote regulatory science.

The PDUFA reauthorization manager's amendment would eliminate limits on the numbers of conflict-of-interest waivers FDA can issue for advisory committee members. It would require FDA to "implement a structured risk-benefit assessment framework in the new drug approval process to facilitate the balanced consideration of benefits and risks, a consistent and systematic approach to the discussion and regulatory decision-making, and the communication of the benefits and risks of new drugs." It also would instruct FDA to work with international regulators and industry to "encourage uniform, scientifically-driven clinical trial standards" that would facilitate simultaneous global development of new medical products. Another new provision would require FDA to identify regulatory and scientific gaps that impede product reviews and approvals and draft plans with specific milestones for addressing the gaps.

The House Energy and Commerce Subcommittee on Health will mark up its version of the FDA user fee reauthorization legislation on Thursday.

Is the final vote a sure thing?  Probably.

But is any election ever a sure thing?

  Read More & Comment...

Creating a Patient Centered Path To Innovation

04/24/2012 06:08 PM |

I attended the Value Driven Engineering conference sponsored by The Austen Bioinnovation Institute of Akron ( http://www.abiakron.org/vde-home) and ABIA's CEO, Dr. Frank Douglas. The meeting brought together a combination of engineers, entrepreneurs, doctors, patients, government officials to discuss ways to make innovation sustainable by making it patient centered. The shift in perspective brings about a shift in how both value and cost are defined and measured as well as how new medical products are developed. Peter Drucker once observed: “Quality in a product or service is not what the supplier puts in. It is what the customer gets out and is willing to pay for. A product is not quality because it is hard to make and costs a lot of money, as manufacturers typically believe. This is incompetence. Customers pay only for what is of use to them and gives them value. Nothing else constitutes quality.” Value (from the patient perspective) is defined as what benefits end users look for and get from a new technology. This is not a new concept, but it's application in the design and purchase of medical innovations is indeed, innovative and in many ways radical. It means that elegant technology has to enhance experience and solve problems. It means that consumers of products will have to be involved in the design of products in some meaningful manner. And it means that product design must be refined by real time, real world experience. Does it mean that new products have to be cheaper or reduce the cost of treating patients. Sometimes, and likely more often than not. But that shift does not mean giving up quality. Indeed, a Johns Hopkins University engineering students are developing a fetal heart monitor that is ten times cheaper than the state of the art machine used in America and is less invasive and more precise. It's also a product anyone can use. Often they are products that have a higher price but reduces the complexity of care, makes it more convenient and frees people up to work more and be more independent, not less. And they also fill an unmet clinical need. A growth factor in heart stem cells can be injected in minutes to help repair left ventricles and save lives. The biggest hurdle to VDE is, the conferees agreed, was reimbursement requirements, especially those that require CER as another step to adoption. VDE uses and depends upon real work use and reworking of products to improve performance and reduce cost. Companies can complete this virtuous cycle more frequently and with less interference offshore. Which is why innovation is leaving America. The choice is between VDE, a prospective partnership that designs products based on reducing cost and consumer value and CER, an after fact hurdle that patients must scale to access innovation.   Read More & Comment...

Gasp -- ASP!

04/23/2012 07:45 AM |

When it comes to drug shortages, pounding Big Pharma isn’t the answer. Finally someone is focusing on the perverse economic incentives of Average Sales Price (ASP) as a key factor behind the problem.  And that someone is Senator Orrin Hatch (R, UT.)

His Patient Access to Drugs in Shortage Act is out for comment through April 25, 2012. This proposed legislation is the only bill dealing with the economic causes of the shortages.

Some important codicils include:

* Price Stability — The draft would change the Medicare reimbursement rate for generic injectable products with 4 or fewer active manufacturers from Average Sales Price (ASP) + 6% to Wholesale Acquisition Cost (WAC) in order to achieve market price stability.

* Medicaid/340B Rebate Exemption — The draft exempts generic injectable products with 4 or fewer active manufacturers from Medicaid rebates and 340B discounts in order to achieve market price stability.

* Extended Exclusivity — Manufacturers who hold an approved application for a drug that would mitigate a shortage can extend by 5 years any period of exclusivity, even if the drug is eventually moved from drug shortage designation.

* Drug Shortage Database — The Secretary would establish a mechanism by which health care providers and other third-party organizations may report evidence of a drug shortage.

This last point may sound innocuous – but it’s crucial. There are many players in the drug shortage game – not just innovator pharmaceutical companies and the FDA. There are generics manufacturers, hospitals, Group Purchasing Organizations (GPOs), and physicians.  (For more on the GPO issue, see this new op-ed in today’s Washington Examiner).

As the Japanese say – don’t fix the blame; fix the problem.

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Depth of a Salesman

04/20/2012 07:38 AM |

Earlier this week the Supreme Court heard arguments as to whether representatives of pharmaceutical companies are entitled to overtime. The answer, it seems, turns on whether those visits are actually sales calls.

According to Paul D. Clement, lawyer for the defendant, the pharmaceutical sales representatives plaintiffs, “ … were hired for a sales job. They were given sales training. They attend sales conferences. They are assigned to sales territory, and they are evaluated and compensated as salespeople.”

But, commented Chief Justice John G. Roberts Jr, “They don’t do sales. Your long list sort of stopped one step short. They don’t make sales.”

As the New York Times accurately reports, industry reps “do not sell products in the strict sense of the term. Rather, they encourage doctors to write prescriptions for patients to buy drugs from pharmacies.”

Interesting semantical and practical questions and it’ll be interesting to see how the Supremes rule.  In the meantime here’s a related question – depending on the final judgment – will government detailers (aka “academic detailers”) qualify overtime pay? They’re not supposed to be “selling” anything either.  

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My Pick For FDA Chief Innovation Officer

04/19/2012 11:53 AM |

http://www.bizjournals.com/boston/news/2012/04/18/bio-ceo-jim-greenwood-on-innovation.html

I like BIO CEO Jim Greenwood's proposal to give FDA a chief innovation officer who who would examine whether the number of drug rejections is reasonable, or whether it is stunting innovation and causing unwarranted delays.

“People who die because a product is not approved quickly enough are just as dead as those who die because a product is not safe,” Greenwood said. “It’s not just the job of the FDA to protect us, it’s also to promote innovation.”

My pick for the position:  Janet Woodcock.   No one (except for Bob Temple) could and has balanced these two missions better.  And no one has done more to promote personalized medicine, which allows products to more efficiently embody safety and innovation.

There are a lot of important bi-partisan ideas to accelerate the innovation process.  A chief innovation officer should not be a ceremonial add-on or be limited to reviewing product development after the fact.  That official could report to the Commissioner, search out and encourage companies with innovation products for fast track approval, be in on the development of products from the beginning.   That's something Dr. Woodcock would and could do effectively.



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Empower FDA By Eliminating CER

04/19/2012 11:39 AM |

My heart is with Mike Rogers and other members of Congress who want to expand FDA's mission to include  job creation and economic growth.   And I get the message they are trying to send to the public and the FDA about what's important.  CMPI has consistently made the case that medical innovation is the primary source of longevity and prosperity.  

But the way to change FDA to promote those broader objectives is to hold it accountable to new standards for product approval and oversight reflecting dramatic changes in the science sustaining innovation.   Congressman Rogers and others can help the FDA by getting them out of the comparative effectiveness business. Reauthorization of PDUFA will give the agency more resources and more direction with regard to increasing the predictability in the development process for drugs, devices, diagnostics and combination products.  However, the FDA would get a boost if legislation stipulated that any products that use biomarkers or other tools to target treatments and improve the ability to monitor disease progression or response would be automatically approved for use in Medicare, Medicaid, etc. 

The more we can defang what is an increasingly worthless CER enterprise the more FDA can achieve the mission the Congressman Rogers wants the agency to carry out.   CMPI will be releasing a study, supported by the Kauffman foundation, that estimates how much innovation and health value our nation will forfeit if CER becomes the focus of commercialization instead personalized medicine.   The estimates, even with conservative assumptions built in, are staggering.  

Every time someone at FDA has to pay lip service to CER it is a signal to companies that all the work put in on diagnostics, combination products and targeted treatments will go to naught because they have to wait for a bunch of underachieving health economists and other professionals to conduct systematic reviews or horizon scams, I mean scans, or clinical trials.   Then again, as I noted in my last post, it looks at though PCORI will be handing out dough to to figure out how to conduct patient-centered research and how to engage patients, though the last time I checked no one -- except the stakeholders who will also get the PCORI cash --  was asking for such government help. 

You want to make medicine patient-centered?  Produce patient-centered medicine.   Tools and treatments that reduce hassles, improve life, increase health. 

That's something FDA can, should and is trying to do in collaboration with companies.  If you asked people should we spend $3 billion on medical decision making or the same amount making better medicines more quickly available to people who need them, I don't have any doubt that faster approvals would win.

The FDA could do a lot with $3 billion over the next decade and do much more for our health and nation than PCORI ever will.  Just a suggestion if Congressman Rogers wants to amend his bill....



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