Peggy Hamburg: FDA's 21st Century Commissioner

02/05/2015 07:43 AM |

Come steppin' down the stairs pretty Peggy-O.

News today that FDA Commissioner Hamburg will resign tomorrow.

More on this to be sure – but for now let’s just say that she’s leaving the agency in a better place than she found it. She set the tone for the 21^st Century FDA -- an impressive and gutsy millennial course.

Here’s the New York Times report.

Much on the horizon for her successor, beginning with working to shape PDUFA VI.

And who better to do this than … Rob Califf.

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A Wish For World Cancer Day: Higher Drug Prices

02/04/2015 06:09 PM |

Today is World Cancer Day.   The goal of this annual event is to generate support and awareness to ensure "all people have the right to access quality, effective cancer treatment and services on equal terms,regardless of geography and without suffering economic hardship as a consequence."

The way to do so: high prices for medicines. 

As Tomas Philipson points out in his op-ed today in Forbes,  the doctors who are only able to pass themselves off as economists because they refuse to debate real economists with real evidence, claim a 'just price' is a price that is... well, less than what they think is 'fair.'   Or a price that is set by government.  Which is one and the same to these faux financial experts.   

But a just price is, as Philipson notes, ithe price that brings better medicines to market that leading to lower prices for health and staying aive.  Which includes eliminating the use of the high priced and much more costly surgical and  hospital based services that enrich the institutions of the would-be economists:

"The current pricing debate is misguided because it is the price of health, and not healthcare, that is the key to patient health. Before a new innovation, the price of better health is effectively infinite for some patients who do not respond to existing treatments because they cannot buy better health. Thus, regardless of price charged for a new treatment it always lowers the marginal price of health for some patient populations. For example, before the breakthrough therapies for HIV in 1996, infected patients could not buy a longer life at any price.  Regardless of the price of the new HIV drugs that came on the market, the price of buying a longer life thus fell. When generics come in, the price of health falls even further but is not captured as returns to the innovators ultimately responsible for it.

What these misperceptions make clear is drug pricing is more complicated than self-interested buyers (Memorial Sloak Kettering, MD Anderson) will have you believe. Although their misguided policy proposals are of course well-intended, if taken seriously they could have very harmful impacts on patients who have no existing options to limit the impact of  their diseases."

These misperceptions persist in large part because their purveyors duck and dodge when asked to debate or discuss in pubiic, one on one.   Rather, they retreat to the security of publications, forums and programs that allow them to manufacturer serious sounding statements that are factually inaccurate and misleading. 

If I didn't know better, I'd say that these pretend economists are afraid to engage directly with Philipson, Frank Lichtenberg,  Tufts's Josh Cohen or anyone else that would challenge their version of the truth.

The danger is that if these opinions become the foundation for pricing policy here, it will reduce investment and ensure that onlly the very rich get access to life saving drugs.   


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The Urban Myths of Expanded Access

02/03/2015 06:05 PM |

Today, senior FDA officials and representatives from leading patient and health policy organizations are meeting in Washington, DC to discuss the urgent and contentious issues surrounding expanded access to investigational drugs.  Yesterday I keynoted the “Expanded Access Programs: New Models for Stakeholder Collaboration, Program Design, Supply Equity and Access for Investigation Drugs” conference. I was pleased to share the day with colleagues such as Richard Klein (Patient Liaison Program Director, FDA’s Office of Health and Constituent Affairs) and Diane Dorman (Vice President for Public Policy at NORD).

The full program can be found at www.cbinet.com/eap.

Some points from the conference worth mentioning:

There is “therapeutic misinterpretation” of early scientific data resulting in some patients asking for access to investigational products based on mouse data! Patients see reporting of promising early data in places like USA Today and immediately call the FDA.

Better “expanded access IQ” isn’t just reserved for patients – but is also needed for physicians. Many doctors are unsure of just what there roles and responsibilities are relative to expanded access protocols. Is this even taught in medical school? And then there's the issue of physicans being concerned (and rightfully so given our litigious society)) about liability issues.

The urban myth that adverse events reported through expanded access programs derail the success of FDA approval is not supported by facts. Richard Klein repeatedly told the conference that (to his knowledge) there were no examples of this – and he’s contacted his FDA colleagues in search of such circumstances. Perhaps it’s time for industry to gather examples – if they, in fact, exist – and share them with the FDA as a tool to correct the problem.

Is “expanded access” even the right term to use? It means one thing in the US, and completely unrelated things in other countries. In the UK, for example, “expanded access” is a reimbursement strategy for approved products.

How can IRBs be restructured to expedite review rather than being a bottleneck?

In the words of the great health policy guru, Buffalo Springfield, There's something happening here / What it is ain't exactly clear.

Perhaps, as part of the FDA’s current initiatives to enhance both the timeliness and weight of the patient voice, expanded access plan development and execution should involve patient organizations. Maybe it’s time to harness that power to make the process both more-inclusive and better.

Another issue that remains at-large is who pays for access to these unapproved drugs? What a company can charge is regulated (via draft guidance), but sometimes the drug company will bare all costs, other times some costs, and just as often it’s the patient who writes the check. And IRB and other related costs are often borne by the patient. Perhaps there’s a role for the Federal government.

How about a fund that pays for access for any approved FDA expanded access IND or protocol? I propose that this issue should be a key part of the pending 21^st Century Cures legislation being drafted by Representative Fred Upton, the Chair of the House Energy & Commerce Committee and by Senator Lamar Alexander in the Senate.

All sides want the same thing -- expedited expanded access programs. But name-calling and bridge burning doesn't bring anyone closer together or experimental drugs to dying patients any faster.

Let’s expedite access by enlarging the Expanded Access Ecosystem.

My complete keynote remarks (including commentary on Abigail Alliance v. von Eschenbach and the Right-to-Try debate can be found here.

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Warren Down the Rabbit Hole

02/03/2015 04:26 PM |

When it comes to myths about healthcare, one of the great shibboleths is that Big Pharma profits from innovation “that comes primarily from the NIH.” It’s never been true – and now a new study confirms it.

A study in Health Affairs by Bhaven N. Sampat and Frank R. Lichtenberg (What Are The Respective Roles Of The Public And Private Sectors In Pharmaceutical Innovation?) http://content.healthaffairs.org/content/30/2/332.full.html puts the issue in a data-driven perspective that gives the NIH its due – but in the proper frame of reference.

For example, according to Sampat and Lichtenberg, fewer than 10 percent of drugs had a public sector patent, and drugs with public-sector patents accounted for 2.5 percent of sales, but that the indirect impact was higher for drugs granted priority review by the FDA. (Priority review is “given to drugs that offer major advances in treatment, or provide a treatment where no adequate therapy exists.)

478 drugs in our sample were associated with $132.7 billion in prescription drug sales in 2006. Drugs with public-sector patents accounted for 2.5 percent of these sales, while drugs whose applications cited federally funded research and development or government publications accounted for 27 percent.

This is an important article that contains much data analysis worthy of careful consideration by the healthcare policy community.

Specific consideration should be given by Senator Elizabeth Warren, whose new draft legislation, the so-called “Medical Innovation Act,” would result in less R&D investment by the true drivers of progress – the private sector – resulting in less innovation. Senator Warren’s suggestion that a percentage of private sector profits be used as a “fee” would have a chilling effect on future investments in R&D by biopharmaceutical companies subject to the legislation. 

Senator Warren’s proposal inaccurately argues that because the proposed fee would only be coming from net profits – after business and R&D expenses have been accounted for – it will not impact investment in drug development. Given the lengthy R&D process to develop new medicines – an average of at least 10 years for one new drug – biopharmaceutical R&D is characterized by long-term R&D investment strategies across R&D portfolios. Thus, a portion of profits earned by innovative biopharmaceutical companies today are used to fund future research. If a percentage of those profits are used for the proposed fee, then there may be a chilling effect on future investments in R&D by biopharmaceutical companies subject to the rule.  Facts are pesky things.

The National Institutes of Health plays a vital role in basic research and early discovery, but is robbing Productive Peter to pay Government Paul the best bang for the buck when it comes to advancing public health?

As Sampat and Lichtenberg show, the engine of innovation is the biopharmaceutical industry, which spends in excess of $50 billion annually on research and development. It's not a competition; the NIH and industry complement each other's efforts. But context matters.

The NIH focuses on basic research, the study of fundamental aspects of phenomena without specific applications. The biopharmaceutical industry addresses most of its R&D toward clinical research, science focused on the actual development of new medicines. The NIH provides grants to academic institutions. Industry employs the scientists who do the work and, increasingly, funds academic research.

Alas, headlines for hyped and misleading "NIH-funded cures" are far sexier than those for "more money for drug regulation." Pursuing misguided policies that siphon funding from the groundbreaking medical research happening in the biopharmaceutical industry will have devastating consequences for patients and society. The proposed legislation would result in fewer medicines for patients and lost jobs at a time when our economy can least afford it. Senator Warren and others should pay heed to the facts and avoid the fiction. They are inversely important to advancing 21st-century healthcare.

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Is Chris Christie An Anti-Vaccine Wingnut?

02/03/2015 11:08 AM |

I think everyone who is running to paint any Republican candidate as a climate denying, Creationist, anti-vaccine Neanderthal would agree that records matter.  That is, what you do is more important than a curveball question during a press mashup thrown by a reporter seeking a ‘gotcha.’

As a proud citizen of the Garden State, I am obligated to respond to the implication that Chris Christie is a anti-science wingnut who thinks parents can choose to have their kids infect others with measles and other communicable diseases.

Turns out New Jersey’s childhood immunization rates are some of the highest in the United States.

It is number 1 compared to all other states for measles-mumps-rubella (MMR) vaccination rates.

New Jersey’s immunization rates are above the national average for major childhood vaccines.

It’s immunization rates are higher than California.  And Kentucky.


Source: CDC
http://www.cdc.gov/vaccines/imz-managers/coverage/nis/child/data/tables-2013.html


With regard to Governor Christie’s alleged support of vaccine choice, his statement to “take a hard look” at legislation introduced by a Democrat in the NJ legislature to weaken vaccine requirements stands in valiant comparison to the pandering of then candidates Obama and McCain on vaccines causing autism, the do as you please stance of Rand Paul and the outright lunacy of Michelle Bachman.

Do people forget that Christie was attacked for requiring a hospital worker returning from Africa to be quarantined?   The idea that the governor of a state that has aggressively and successfully pushed to raise immunization rates – along with other public health metrics  – is absurd.  






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Optimizing Patient Safety -- It's all about the base

02/02/2015 07:24 AM |

A joke inside the FDA is that the Brief Summary is like the Holy Roman Empire: neither brief nor a summary. But it's an important jump start for a serious question—namely, how can safety information be made more user-friendly? For an answer, I turn to one of my favorite doctors, Dr. Seuss: “Sometimes the questions are complicated and the answers are simple.”

The theory behind safe use is that the most effective way to make a drug safer is to be sure it's used as directed. That means putting safety information in places and formats most accessible to patients. In 2015, that means mobile apps. According to a study from Adherent Health, nearly 75% of prescription-takers use mobile apps, including most older adults and seniors. Indeed, mobile-app adoption rates are high across all medication-taking adult age groups. Plus, new-user data from app-based safe use and outcomes support platform Mobile Health Library (MHL) confirms that, when Important Safety Information is mobile-friendly, it's regularly accessed by on-treatment patients.

Safety information is knowledge. And, in pursuit of public health, knowledge is power. 

Consider one year's worth of data for MHL's patient-support app for generic Exemestane: Of all engagement activities available to users, those concerning “side effects and safety” are most popular (at 28%), followed by “savings and refills” (16%), “about condition” (15%), “about Exemestane” (14%) and “dosing reminder” (13%). The safe-use numbers for branded medicines are even higher. For one medicine (a branded epilepsy treatment), patients are accessing the MedGuide at 90%+ rates.

When you match this with data showing the benefits that prescribing physicians (and nurse prescribers) want from patient-support apps, it's an almost-perfect fit. Ninety-four percent of primary care providers list “take Rx as directed” as their number one app priority, with “Rx and disease understanding” a close second (91%) and “conversation reinforcement” third (83%). Again, it's all about the base. 

So if the business is truly serious about giving patients what they want, then it needs to convey safety information in the ways they want it. When it comes to safety, it's time for both the FDA and Big Pharma to start saying “App-y New Year.”

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21st Century Cures: The Future is Now

01/30/2015 07:46 AM |

In mid-January Representatives Fred Upton (R/MI) and Diana DeGette (D/CO) previewed their goals for the 21st Century Cures initiative. 


The bipartisan duo wanted to “modernize clinical trials to streamline the approval of drugs and devices,” in part by reducing paperwork and promoting adaptive trials, help FDA “better integrate the patient perspective into the regulatory process,” including using public-private partnerships to strengthen science around biomarkers and patient-reported outcomes, promote “better access to and sharing of information such as genomic and other clinical data to foster more collaboration among researchers," and to invest in programs for young scientists.


Upton -- the chair of the U.S. House's Energy & Commerce Committee -- and DeGette also planned to “incentivize new drugs and devices for unmet medical needs” by “streamlining the premarket process while establishing mechanisms to better capture real world evidence post-market." They also said they will examine incentives, including “exclusivity or simplifying the reimbursement process,” to stimulate the development of new drugs and devices for unmet medical needs.

Two weeks later the committee released a discussion draft and a Discussion Document without support from Democrats. According to Rep. Diana DeGette, “While I don’t endorse the draft document, I know that with continued engagement, we can reach a bipartisan consensus to help advance biomedical research and cures." Inside the Beltway that’s known as “playing nice.”

Not so nice was Representative Frank Pallone, Jr. (D/NJ), the ranking Democrat on the Energy & Commerce Committee. Mr. Pallone is “disappointed that the discussion document released today by Chairman Upton does not reflect true bipartisan collaboration.” Pallone added that the “nearly 400 page draft could create more problems for our health care system than it solves.” While he did not cite any specific concerns, (did he even read the report?) he did note that the draft “does not include any real dollars to fund additional basic research at the National Institutes of Health.” Real dollars -- as opposed to what? Was Senator Warren whispering in his ear? 


Per a report in BioCentury, Some provisions in the discussion draft are likely to be sticking points for Democrats, including proposals to exert more centralized control over NIH funding decisions by setting fixed, renewable four-year terms for institute directors, and making directors personally responsible for ensuring that the goals of every grant award are consistent with “a national priority and have public support.” 
FDA is likely to oppose scores of provisions in the discussion draft that would impose new mandates but do not provide additional funding. For example, the draft would require FDA and HHS to produce about 35 draft or final guidance documents, most of them on tight deadlines. 
The committee left placeholders in the draft for several topics that are potential political flash points, such as communication about off-label uses of approved products, and regulation of diagnostics.

The draft includes a provision allowing FDA to approve a drug with breakthrough designation on the basis of a single Phase II study, contingent on postmarket clinical trials and/or data from observational studies and registries. FDA could withdraw breakthrough approvals if sponsors failed to provide the required data, if evidence emerged showing the drug was not safe or effective, or if the sponsor disseminated false or misleading promotional material. 
The committee included provisions creating tight deadlines for FDA to assess applications for qualification of biomarkers and other surrogate endpoints.

The draft also attempts to extend and formalize FDA’s efforts to incorporate patient experiences and preferences into regulatory decisions, including using patient data in structured benefit-risk assessments. In other words, it’s time to move from a patient-centered drug development initiative to a patient-driven drug development program.

The draft also incorporates legislation on compassionate access introduced by Michael McCaul (R-Texas), as well as the Modernizing Our Drug & Diagnostics Evaluation and Regulatory Network (MODDERN) Cures. 
The discussion draft has several provisions intended to make CMS decision-making more transparent and consistent, including a process for device and drug sponsors to appeal coverage decisions

Title IV of the draft legislation calls for “Accelerating the Discovery, Development, and Delivery Cycle and Continuing 21^st Century Innovation at NIH, FDA, CDC, and CMS.” Bravo. Mr. Upton and his team at E&C understand that innovation is an eco-system. And that's precisely what 21st Century Cures is all about, creating a sustainable innovation eco-system with government players as an integral part.

One way FDA can help is to assist innovators to fail faster. Killing clinical programs earlier in the development process can save billions that can then be more fruitfully reinvested. When Thomas Edison was asked why he was so successful, he responded, “Because I fail so much faster than everyone else.” Consider the implications if FDA could help companies to fail faster. The following figures are illuminating:

• A 10% improvement in predicting failure before clinical trials could save $100 million in development costs.

• Shifting 5% of clinical failures from phase 3 to phase 1 reduces out-of-pocket costs by $15–20 million.

• Shifting failures from phase 2 to phase 1 would reduce out-of-pocket costs by $12–21 million.

One way to achieve this is for the FDA to finalize and formalize a risk/benefit grid, so that the factors behind regulatory decisions can be better understood – and future ones become more predictable. The EMA does this very successfully. Not having such a regulatory tool gives the FDA the Power of Ambiguity. But predictability is power in pursuit of the public health. Ambiguity also weighs heavily on the soul of those making investment decisions in riskier R&D propositions.

Since FDA, NIH, CDC, and CMS all fall under the purview of the Department of Health & Human Services, perhaps the 21^st Century Cures legislation should be more directive relative to the co-ordination of government efforts.

How about a 21^st Century Cures Czar?   Read More & Comment...

Gaming the System. Denying Care.

01/29/2015 02:08 PM |

I’m shocked to discover that there is gambling going on in this establishment.

From today’s New York Times:

Study Finds H.I.V. Drugs Priced Out of Reach

Drugs to treat H.I.V. and AIDS are being priced out of reach for many patients enrolled in insurance plans through the new health care exchanges, despite warnings that such practices are illegal under the Obama administration’s health care law, according to a new analysis by Harvard researchers.

The study, to be published on Wednesday in an article in The New England Journal of Medicine, looked at 48 health plans in 12 states and found that a quarter of the plans showed evidence of what researchers called “adverse tiering,” or placing all of the drugs used to treat H.I.V. in a specialty tier where consumers are required to pay at least 30 percent of the cost of the drug.

The financial impact can be drastic, the researchers found: A patient taking a common H.I.V. treatment, Atripla, would pay about $3,000 more a year in a restrictive plan compared with someone enrolled in a more generous plan, even after accounting for the fact that the more restrictive plans tended to charge lower monthly premiums.

“That’s really a large cost difference, and really is a very significant financial constraint for those with chronic conditions, particularly H.I.V.,” said Douglas B. Jacobs, the lead author of the study, who is pursuing degrees in public health at the Harvard T. H. Chan School of Public Health and medicine at the University of California, San Francisco.

The study, which did not name the insurers whose plans it analyzed, looked only at the midlevel, or silver, plans offered in the marketplaces because they are the most popular plans among consumers. More comprehensive plans, known as gold and platinum plans, are often more generous in coverage but carry higher premiums. Insurers have said that these may be a better choice for people who have serious medical conditions.

Clare Krusing, a spokeswoman for America’s Health Insurance Plans, an industry trade group, said that a crucial component of the marketplace was consumer choice, and that the study should have included an analysis of the gold and platinum plans. “Individuals have diverse health and financial needs, and health plans have designed a wide range of coverage options, including those with lower cost-sharing, so individuals can pick the policy that is best for them,” she said.

Health insurers are prohibited from discriminating against people with specific medical conditions under the new federal health care law, and the law contains some provisions that help prevent such practices. Patient advocates and others, however, have said that some companies appear to be skirting the law by restricting access to all drugs that treat certain conditions.

In May, two consumer groups filed a federal complaint asserting that four insurers in Florida had discriminated against people with H.I.V. by making their drugs more costly and difficult to obtain. All of the companies have since agreed to make changes that would lower the cost of the drugs in 2015, although the federal complaint is still pending.

The latest study is “more confirmation that this is happening, not only in Florida, but in other states as well,” said Carl Schmid, deputy executive director of the AIDS Institute, one of the groups that filed the federal complaint.

In December, the Obama administration said that it would investigate insurers’ prescription drug coverage, and told insurers that companies that place most or all drugs that treat a condition on the highest-cost tiers are effectively discriminating against people with those conditions.

In an effort to contain the rising cost of prescription drugs, many insurers are requiring extra steps for people who need the drugs or raising out-of-pocket amounts that patients must pay. The insurers say such practices are necessary to keep premiums low and to encourage patients and their doctors to make cost-effective decisions, like choosing a cheaper generic over an expensive brand-name drug.

But what makes these cases different, the researchers said, is that the insurers restricted access to all drugs that treat H.I.V., even less costly generics, leaving patients who have the virus with few options.

Limiting access to H.I.V. drugs could benefit insurers because it might discourage people with such conditions from signing up for coverage. But the study’s authors warned that if left unchecked, a small number of insurers — those offering the most generous coverage — could end up shouldering the burden of caring for the sickest patients.

If the more generous plans end up with a disproportionate share of sicker members, they could, in turn, adopt more restrictive practices. “It could, in essence, be a race to the bottom on drug benefit designs,” Mr. Jacobs said.

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Biosimilar Interchangability: When Irish Eyes Aren't Smiling

01/28/2015 10:02 AM |

A new poster presentation at the European Crohn’s and Colitis Organisation, titled, “Biosimilar but not the same,” offers some timely and important real-world data on the differences between originator biologics and their biosimilar cousins.

The study, from Mercy University Hospital, University College Cork, Centre for Gastroenterology, Mercy University Hospital, Cork, Ireland, studied the clinical impact of both the innovator product (Remicade) and it’s EMA-approved biosimilar (Inflectra). The findings are important. Specifically, the rates of surgery in Infliximab and Inflectra groups were significantly different.

80% of the Inflectra group required hospital readmission versus 5% of the infliximab (Remicade) group. (p=0.00004). 60% of patients in the Inflectra group needed steroid augmentation of standard steroid tapering protocol with 50% requiring multiple increases in steroid dose versus 8% of patients in the Infliximab (p-value = 0.0007). Over the course of 8 weeks, 93% of patients in the Inflectra group had an increase in CRP with 7% remaining unchanged whereas 100% of patients in the infliximab group had a decrease in CRP (p=<0.001).

The conclusion is not ambiguous, “Our results suggest that biosimilars may not be as efficacious as the reference medicine. The results found reflect the ECCO statement position that the use of most biosimilars in IBD will require testing in this particular patient population and cannot be extrapolated from other disease populations."

The complete poster can be found here.

These First World data points about a product from a respected manufacturer (Hospira) cannot be ignored and must be used to inform the policy debate over nomenclature, interchangeability, label extrapolations, and overall pharmacovigilance practices.

Attention must be paid.
 

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PCORI's Survival Depends on Shift to Precision Medicine

01/27/2015 04:53 PM |

The Patient Centered Outcomes Research Institute (PCORI) has given nearly $300 million away to groups that are conducting research that is less then, shall we say, transformational..   Most of the money is going to comparaative effectiveness research that pays lip service to differences in individuals when in fact it is just coming up with one size fits all comparisions with a bit of control for age, gender, ethnicity, disease stage.  For instance, PCORI is funding a study http://Which Medicine Is Best for Children with Epilepsy?   Really?  Drug A over Drug B or Drug C?   

PCORI is funding mainframe medicine when cloud=based, crowd sourced technologies that integrate genomics, systems biology and patient provided data to match people to treatments.  It is investing heavily in PROMIS and it's own CER research network.. both are based on technologies and natural language processing algorithms that are old, clunky and barely accessible to patients.   

The new Congress has just launched a 21st century cures initiative.  A close look at the legislation and it's goals suggest that PCORI is ill-suited to the goal of using 21st century bioinformatics to increase the role and power patients have over access to new treatments.  PCORI is funding yesterday's research methods.

Congress should hold oversight hearing on PCORI and ask some hard questions about it's research focus.   It doesn't have to exist at all or in it's current form.  Indeed,  the money from PCORI and other stand alone pots of money could be integrated into a public-private venture to support biomarker development and precisiono medicine that the legislation calls for.  

PCORI had better show it's ready to shift it's gears and mission if it wants to survive.  Read More & Comment...

Will Congress Produce Cures?

01/27/2015 04:19 PM |

The Energy and Commerce committee 21st Centure Cures Initiative has produced an important piece of legislation designed to accelerate the adoption of precision medicine.  The way to tell if it will pass is how much time and effort AHIP and the PBMs spend trying to gut the recommendations to advance personalized or precision medicine.  If they cared about the cost of medicines, they would propose ways to reduce the time and money needed to develop and adopt precision medicine.   The same goes for critics who say nothing about the value of medicines and are not an advocates for 21st century cures.

Here is the committee's press release and the legislation itself.  http://1.usa.gov/1BvrGio



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Patient-Driven Care with a Capital PFF

01/27/2015 09:36 AM |

A real-world step towards both helping patients now and gathering useful data to advance better care.

PULMONARY FIBROSIS FOUNDATION ANNOUNCES EXPANSION
OF FIRST-OF–ITS-KIND CARE CENTER NETWORK
PFF Adds 12 Sites, Bringing Total to 21 Centers Across the Country

CHICAGO, January 27, 2015 – The Pulmonary Fibrosis Foundation (PFF) today announced the expansion of its PFF Care Center Network with the selection of 12 additional sites, bringing the total number of medical centers to 21 in 20 states. Launched in 2013, the PFF Care Center Network is comprised of leading medical centers with specific expertise in treating pulmonary fibrosis (PF), a group of lung disorders including idiopathic pulmonary fibrosis (IPF) that are often difficult to diagnose and manage and that are associated with survival rates of less than five years following diagnosis in certain diseases.  

“As the leading advocate for the pulmonary fibrosis community, we are dedicated to advancing the care of people living with this deadly disease, and this starts with providing greater access to experienced care teams,” said Gregory P. Cosgrove, M.D., chief medical officer of the PFF. “Working together, institutions within the Network will identify and share best practices, which foster better care and ultimately enable more institutions that embrace these practices to be certified as a PFF Care Center site.”

As part of the selection process, a panel of peer reviewers, comprised of current members of the PFF Board of Directors, Medical Advisory Board, PFF Care Center Network and PFF Patient Registry Steering Committee, reviewed and scored applications.

“When selecting sites to add to the PFF Care Center Network, we consider a center’s specific programs and its geographic location in order to best serve the needs of the broader pulmonary fibrosis community,” said Kevin Flaherty, M.D., M.S., chairman of the Steering Committee of the PFF Care Center Network. “The new centers selected provide the highest quality patient care and an individualized approach to treatment in accordance with best evidence-based recommendations. We welcome these new centers and look forward to continuing to expand the Network in the coming year.”

The Pulmonary Fibrosis Foundation Care Center Network

The PFF Care Center Network uses a multidisciplinary approach to deliver comprehensive patient care, forming specialized care teams comprised of experts in interstitial lung disease in pulmonary medicine, rheumatology, radiology and pathology. This multidisciplinary approach is critical to managing a complex disease like PF and ensuring people with PF receive an accurate diagnosis, obtain quality clinical care, and acquire important support services.

The institutions newly certified as PFF Care Center Network sites include:

• Inova Fairfax Medical Campus
• Mayo Clinic in Rochester, Minn.
• Medical University of South Carolina
• New York-Presbyterian/Columbia University Medical Center and New York-Presbyterian/Weill Cornell Medical Center
• Piedmont Healthcare
• The University of Arizona Interstitial Lung Disease Program at the University of Arizona Medical Center-University Campus in Tucson
• The University of Kansas Hospital
• Tulane University School of Medicine
• University of Alabama at Birmingham
• University of Miami Miller School of Medicine
• University of Pennsylvania

Washington University School of Medicine in St. Louis   Read More & Comment...

Rob Califf at FDA -- Finally!

01/26/2015 02:33 PM |

Dr. Robert Califf named FDA Deputy Commissioner for Medical Products and Tobacco

U.S. Food and Drug Administration Commissioner Margaret A. Hamburg, M.D., today appointed Robert Califf, M.D., a recognized global leader in cardiology, clinical research, and medical economics, as FDA Deputy Commissioner for Medical Products and Tobacco.  

In this position, Dr. Califf will provide executive leadership to the Center for Drug Evaluation and Research, the Center for Biologics Evaluation and Research, the Center for Devices and Radiological Healthand the Center for Tobacco Products. He will also oversee the Office of Special Medical Programs in the Office of the Commissioner. Dr. Califf will play a critical role in providing high-level advice and policy direction on the agency’s medical product and tobacco priorities and will manage cross-cutting clinical, scientific and regulatory initiatives in several key areas for the agency, including personalized medicine, orphan drugs, pediatric science, and the advisory committee system.

“I am delighted to announce this important addition to FDA’s senior leadership team,” said FDA Commissioner Margaret A. Hamburg, M.D. “Dr. Califf’s deep knowledge and experience in the areas of medicine and clinical research will enable the agency to capitalize on, and improve upon, the significant advances we’ve made in medical product development and regulation over the last few years.”

Dr. Califf is currently serving as vice chancellor of clinical and translational research at Duke University. Other prominent roles during his tenure at Duke include director of the Duke Translational Medicine Institute (DTMI), and professor of medicine in the Division of Cardiology at the Duke University Medical Center in Durham, North Carolina. Before serving as director of DTMI, he was the founding director of the Duke Clinical Research Institute, the world’s largest academic research organization. Dr. Califf is recognized by the Institute for Scientific Information as one of the top 10 most cited medical authors, with more than 1,200 peer-reviewed publications.

During his career, Dr. Califf has led many landmark clinical studies, and is a nationally and internationally recognized expert in cardiovascular medicine, health outcomes research, health care quality, and clinical research. He is one of our nation’s leaders in the growing field of translational research, which is key to ensuring that advances in science translate into medical care. He was a member of the Institute of Medicine (IOM) committees that recommended Medicare coverage of clinical trials and the removal of ephedra from the market and of the IOM’s Committee on Identifying and Preventing Medication Errors. In addition, he served as a member of the FDA Cardiorenal Advisory Panel and FDA Science Board’s Subcommittee on Science and Technology. Currently, he is a member of the IOM Policy Committee and liaison to the Forum in Drug Discovery, Development, and Translation.

Dr. Califf will join the FDA in late February.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human drugs, including vaccines and other biological products for human use, veterinary drugs, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

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Opioid's Atlantic Charter

01/26/2015 12:02 PM |

For a balanced look at the current debate over opioids, check out A Delicate Balance: The challenge of treating pain—and stopping prescription drug abuse—in America. It’s timely, comprehensive, science-based sponsored content in The Atlantic and well worth a read.

ABOUT THIS SERIES

Pain exists. It’s pervasive. And, yet, the drugs prescribed for its treatment are often abused. A Delicate Balance explores several approaches to helping achieve the public health goal of stopping prescription opioid abuse while effectively treating people who live with chronic pain.

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Obama's Precision Medicine Proposal: Guess Who Hates It?

01/23/2015 01:56 PM |

In his State of the Union address, President Obama proposed a federal initiative to accelerate the development of precision medicine and ensure that everyone would have “access to treatments that deliver the right treatment at the right time.” 

To illustrate his point, the President invited Bill Elder, a 27-year-old medical student with a rare form of cystic fibrosis to sit with First Lady Michelle Obama during his speech.    Mr. Elder has been using Kalydeco, which, as the president pointed out “has reversed a disease once thought unstoppable” by turning off the genetic mutation causing his disease.

But Kalydeco, is NOT easily available under most health plans.  Ask Chloe Jones, a 14-year-old Arkansan with the same type of cystic fibrosis mutation.   Arkansas’ Medicaid agency has refused to give her Kalydeco.  Instead she has to first fail to respond to older, less-expensive therapies that treat the symptoms but not the underlying cause.   (Kalydeco costs about $200,000 a year.) In other words, she has to get sicker and a step closer to death.

Sadly, Chloe is not alone.  She and hundreds of thousands of other patients are denied the precision medicines the president celebrates. Instead of ensuring access to precision medicines for the right patient at the right time, pharmacy benefit management companies  (PBMs) that put together drug formularies for health plans are only offering drugs from biotech firms that give them biggest cash discount. And then insurers are requiring kids like Chloe to get sicker or pay thousands of dollars before getting targeted treatments.

After the rebates are harvested by PBMs by excluding precision meds, health plans require patients to pay thousands of dollars out of pocket to get the one’s that are left.  Managed Care magazine points to a study by Avalere Health that revealed “many insurers require copayments of 10% to 40% for 19 classes of medications for patients with chronic conditions. More than 60% of silver plans put all covered medications for patients with multiple sclerosis, rheumatoid arthritis, Crohn’s disease, and certain cancers in the highest formulary tier.”

While most plans cap out of pocket costs, most consumers can’t afford thousands of dollars upfront.   As a result, patients don’t take the medicines that could save their lives.  Health insurers and PBMs know it and profit from it.

On top of all that, health plans and PBMs don't pay for the genetic tests that match people to the right medicines.  They want randomized clinical trials to demonstrate accuracy know it will take years to set up studies and get results. 

The orchestrated outrage over drug prices, led by John Rother and his AHIP funded National Coaltion on Health is nothing but part of cynical plan to extort rebates and limit access to precision medicines.  It is noteworthy that AHIP, and the PBMs have contributed nothing to the discussion around the bi-partisan 21st Century Cures Initiative.  If they cared about the cost of medicines, they would propose ways to reduce the time and money needed to develop and adopt precision medicine.   The same goes for critics who say nothing about the value of medicines and is not an advocate for 21st century cures.  

In fact, the pay for play schemes indicate that PBMs and insurers can only profit from precision medicine by rebates.   As Eric Topol points out in his new book "The Patient Will See You Now", the health data now controlled by payors and physicians will be generated and controlled by patients themselves.  Using smartphones patients will be able to find the right treatment for them based on their genetic and clinical data.   Where's the  need for middlemen at that point?  PBMs are the Blockbuster Video and Tower Record Stores of the healthcare system.  NO ONE will miss them when they are gone. 

We need to hasten the creative destruction of PBMs.    We can start by replacing fail first or step therapy approaches with value-based precision therapy.   And we should elimiinate the discriminatory practice to placing precision medicines in the most expensive formlary tiers and the coo-insurance costing thousands.    Finally, we need to expand the use of new medicines in the real world.  

Ultimately, the rebate and co-pay schemes plump up profits to prop up an outdated business model. PBMs and insurers are skimming off billions of dollars with drug benefit designs that target the most chronically ill and vulnerable patients. No wonder, patient groups have sued health plans for discrimination.  Change can't come fast enough.    Read More & Comment...

Cognitive Dissonance Over Opioids

01/23/2015 09:37 AM |

Are opioids “bad?” Certainly they can be addictive and that fact can’t be understated. That’s precisely why they are controlled substances. And still nascent abuse deterrent technologies are helping to further decrease the opportunities for improper use. But the value of opioids is, when used as directed, they are highly effective in combatting the scourge of pain.

The truth, of course, is that opioids aren’t bad. The problem is that they’re not perfect nor are they perfectly safe – just like 100% of prescription drugs on the market. “Safe” is a relative term. Opioids are safe when they are used as directed. Are chemotherapy drugs “bad” because of their horrible side effects? Of course not. Why – because the alternative is far worse. So too the case with opioids. For both physicians and the tens of millions of Americans with chronic pain, the absence of opioids would be disaster.

That’s why the FDA and other pertinent constituencies view the future of pain medication through the lens of “safe use.” How can we enhance appropriate prescribing, dispensing, and patient behavior? Abuse deterrent technologies are part of the answer, but better physician, pharmacist, and patient education must be another pillar. The FDA has made this a policy priority. FDA Commissioner Hamburg has called for “Improving appropriate prescribing by physicians and use by patients through educational materials required as a part of a risk mitigation strategy for extended-release and long-acting opioids.”  Now it's also time for the DEA to work with the FDA to develop smart policies for specific education as a must-have for prescribing rights.

Alas, there are too many pundits, politicians, and self-anointed citizen advocates who are keen to focus on placing blame. It’s a savvy strategy for media attention but does little to advance the public health. There’s no value in fixing the blame for medicines that aren’t 100% safe. No medicine is 100% safe. That cynical approach just leverages ignorance to produce anger. There’s tremendous value in fixing the problem – through advancing the safe use of opioids – a crucial weapon in the armamentarium against pain.    Read More & Comment...

When Lawyers Play Doctor

01/21/2015 09:39 AM |

When it comes to medicine, who should make decisions about safety and efficacy – and on what evidence should those choices be made? At present, the Food & Drug Administration has the responsibility to determine approvals and labeling language based on a scientific review of the evidence. Should this authority be ceded to the tort bar?

Consider the recent spate of litigation against the manufacturers of opioid pain medications. One example is the City of Chicago’s lawsuit against multiple manufacturers of opioid pain treatments. In the United States District Court for the Northern District of Illinois (Eastern Division), the City of Chicago’s First Amended Complaint (“FAC”) seeks to limit the ability of Chicago doctors to treat the chronic, non-cancer pain of patients in the manner doctors deem most appropriate. Although the Food and Drug Administration has approved certain opioid pain medications for the treatment of chronic non-cancer pain, the FAC seeks to deprive patients and doctors of that treatment choice by having six lay jurors determine that “the use of opioids to treat chronic pain is not ‘medically necessary’ or ‘reasonably required’ in that their risks do not exceed their benefits.”

The FDA has determined that opioids serve an important public health role: “When prescribed and used properly, opioids can effectively manage pain and alleviate suffering—clearly a public health priority. Chronic pain is a serious and growing health problem: it affects millions of Americans; contributes greatly to national rates of morbidity, mortality, and disability; and is rising in prevalence. At the same time, there is no dispute that opioids pose significant public health risks: Opioids also have grave risks, the most well-known of which include addiction, overdose, even death. The labeling for these products contains prominent warnings about these risks. Moreover, the boxed warning states that all patients should be routinely monitored for signs of misuse, abuse, and addiction.

In September 2013, the FDA ruled on a citizen’s petition filed by a group of clinicians, researchers, and health officials called Physicians for Responsible Opioid Prescribing (“PROP”). Like the Chicago FAC, the Petition directly challenged the use of opioids for “chronic non-cancer pain.” PROP contended that the “long-term safety and effectiveness of managing [chronic non-cancer pain] with opioids has not been established,” and requested that the FDA, inter alia, impose a “maximum duration of 90-days for continuous (daily) use for non-cancer pain.” The FDA carefully reviewed the Petition and more than 1900 related comments. The agency assessed the relevant literature. It held a two-day public hearing at which it received “over 600 comments” and dozens of experts and concerned citizens testified. The FDA noted that “the majority of comments” “opposed PROP’s requests” and that “many professional societies,” including the American Medical Association, “did not support the Petition and stated that the data cited by PROP did not support PROP’s requests.” After completing a 14-month review, the FDA determined that opioids should continue to be available for the treatment of chronic pain, while also directing further study and certain labeling changes for some opioid drugs. Significantly, after being presented with the same assertions as those now alleged in the Amended Complaint, the FDA made two findings directly at odds with the underlying premises that form the cornerstones of the FAC.

But the lawsuits keep coming. The FDA has the authority, the ability, the means, the mission, and the mandate to manage the health care risks and benefits inherent in the products it regulates on behalf of the American public.

A more balanced legal system will occur only when elected officials determine the time has come for real tort reform, as it affects pharmaceutical companies.  But that day is likely very far off. Healthcare leaders must devote their most aggressive efforts toward reform.

Maybe when our elected officials understand that it's the health of their constituents versus the pocketbooks of lawyers, our public servants will finally get serious on tort reform.  Read More & Comment...

Bach to the Future

01/20/2015 09:42 AM |

Time and again, the dangerous idiots of healthcare reform claim that there's no difference between so-called "me-too" drugs, and that they're simply a way for drug companies to turn a quick profit. The New York Times,in an editorial, claimed that "the nation is wasting billions of dollars on heavily marketed drugs that have never proved themselves in head-to-head competition against cheaper competitors." Wrong.

A recent op-ed in the New York Times, (Why Drugs Cost So Much, NYT, January 14, 2015) by Peter Bach is only the latest in a series of such bandwagon drivel – and he should know better.

The truth is that despite the assertions in and by the New York Times, different drugs are indeed different, even if you describe them as "me-too" medications. Likewise, even though every brand of store-bought peanut butter contains peanuts, oil, sugar, and salt, no one would argue that Skippy and JIF taste exactly the same.

Time and again, different medicines have proved themselves where it counts - in the bodies and biochemistries of patients. No two patients are alike, and the more options available, the more likely doctors are to find what works best for each patient. A few points to consider:

* When patients have access to more effective medications, their overall health improves, even as their overall medical expenses go down. That, in turn, reduces national health-care spending and boosts the economy. Value must be measured in patient outcomes.

* Healthcare innovation saves lives, saves money, promotes economic growth, and provides hope for hundreds of millions of people (both patients and care-givers) in the United States and around the world.

* If we do not support the development of new medicines through timely licensing and fair pricing, innovation will be stopped in its tracks – and that is not an acceptable public health outcome.

* Regulators can be partners in innovation three ways: Through robust oversight, through active collaboration, and, most importantly, by being an innovation enabler.

 The deviation between value and pricing in a distinction with a difference – and while both are important, it is value that’s the higher priority since driving patient outcomes is the higher calling (and better long-term economic investment).

After all, as Yale economist William Nordhaus has written, "The social productivity of health care spending might be many times that of other spending.”

Luckily, America's doctors, drug researchers and patients are smarter than that. Just like there aren't any "me-too" patients, there aren't any "me-too" drugs. And if cars and peanut butter are going to be personalized, then medicine is too important not to be.

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Does Cutting Drug Prices Kill People?

01/15/2015 01:34 PM |

Peter Bach yet again tries to make the case that new drugs are too expensive.

He writes: "The price of drugs is so unfair and mystifying that publlic often pays a high price when the identical drug is available at a fraction of the cost."

Actually, he didn't write that.  It's from another New York Times article: " Drug Prices Here Held Inequitable. "   It was written 40 years ago.  Burks, E. (1965) Drug Prices Here Held Inequitable. The New York Times Retrieved from https://secure.pqarchiver.com/

What he did write is another variation on this very old theme: "We can free insurers and government programs from the requirement to include all expensive drugs in their plans as we explain to the public that some drugs are not effective enough to justify their price. If we do this, we can be confident that manufacturers will lower their prices to ensure their ability to sell their products. Or we can piggyback on the gumption of bolder countries, and demand that policy makers set drug prices in the United States equal to those of Western Europe. Either approach would be vastly superior to the situation we have today."

First the facts.  

1. Nearly 90 percent of all brand drugs are discounted.  Companies pay rebates to Medicare, Medicaid, and the Veterans health system.    In the private sector (including Obamacare plans) drug firms pay rebates to pharmacy benefit management companies to get preferred listing.  

2. Bach is incorrect in claiming insurers have to cover every ‘expensive’ drug.  Express Scripts has 66 products on its 2015 formulary exclusion list, compared with 48 in 2014. CVS Caremark’s 2015 list has 95 products, including 72 carryovers from the 2014 edition.  

He loves this idea.  But what about patients?

From Managed Care Magazine:  

"The problem with this approach is that by adding more tiers health plans have confused employees and made some medications more expensive — the antithesis of a value-based strategy. They also have retained cost-sharing mechanisms that make high-priced drugs unaffordable for many patients, Vogenberg says. A 25% copayment on a drug priced at $1,000 a day costs a patient $1,750 each week or more than $7,000 per month. Many patients simply cannot afford their medications, even though commercial plan members often use copayment assistance programs from pharmaceutical manufacturers and can avoid such high cost-sharing amounts, says Brenda Motheral, RPh, MBA, PhD, president of Artemetrx, a specialty drug management consultant.

Bach is silent as PBMs pocket rebates while at the same time sticking patients with the higher costs of meds.


3.  Compared to Europe and Canada, on average Americans then pay slightly more for new medicines  in exchange for faster and broader access.   On average we get twice the new medicines about a year and a half earlier with fewer restrictions.  When all is said and done, using Euro-prices would save a grant total of 8.6 billion.. less than one third of one percent of total health care spending."

4.  Contrary to Bach, Americans also pay less out of pocket for medicines than in Europe. On average, out of pocket costs are about $800.  

4.   European speand more on cancer care as a percentage of health care and less on cancer drugs.  

Nowhere in Bach's article does he mention the value of broader, faster access to new medicines.   Let's stick with the European comparision.

Cancer mortality rates in the US are lower than most anywhere in Europe for every major cancer.  And five year survival rates (adjusted for lag time and incidence) are much higher and increase faster.  That doesn't include the life years lost when people with advanced forms of cancer die waiting a year and half for a new medicine or have strict limits place on access.    

American get broader access to better drugs that save money at about the same price as medicines elsewhere.  Price controls and PBM restrictions on access to new drugs actually increases cost and makes people sicker.  And probably let a lot of people die waiting for new medcines.  If a new medicine adds 3 months on average to the life of a cancer patient it would mean that each year (based on 590,000 cancer deaths a year) someone is denied access robs people with cancer of about 145000 life years annually. 


Back claims other systems are vastly superior.  The evidence suggests otherwise.  Cutting drug prices does kill people. 

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Deterring Opioid Abuse: A Success Story

01/15/2015 09:06 AM |

The gain in pain is plainly in the main.

Abuse of opiate-based prescription painkillers such as oxycodone and morphine peaked around 2010-2011 and now may be on the decline in the United States, according to an analysis of databases designed to track illicit use of the drugs.

New laws, programs and policies, such as prescription tracking systems and the reformulation of oxycodone to make it harder to abuse, may be combining to reverse the once-growing trend, researchers said.

"I think we're at an inflection point and we're starting to turn this steamship around," said Dr. G. Caleb Alexander, co-director of the Johns Hopkins’ Center for Drug Safety and Effectiveness, who was not involved in the research.

The new study, published in the New England Journal of Medicine, looked at data collected between 2002 and 2013 from substance-abuse treatment centers, poison centers, college students and drug-diversion investigators.

There were "large increases in the rates of opioid diversion and abuse from 2002 to 2010, but then the rates flattened or decreased from 2011 through 2013. The rate of opioid-related deaths rose and fell in a similar pattern," write the study authors, led by Dr. Richard Dart of the Rocky Mountain Poison and Drug Center at the Denver Health and Hospital Authority in Colorado.

The full NEJM article can be found here.

Now we need to continue to move (and act) beyond the rhetoric and pursue additional solutions. And at the top of the list is the foundation of the Hamburg Manifesto -- prescriber education.  Read More & Comment...