Latest Drugwonks' Blog

Without commercial interruption please read this important blog from Drug Channels about how insurers and PBMs are rigging drug prices to maximize profits and discourage access.  

Insurance is -- to paraphrase the song Me and Bobby McGee -- has become another word for nothing left to lose, especially if you are one of the 4-5 percent of patients fighting cancer,  automimmune diseases and orphan conditions.   As Adam points out, the system is rigged against a minority of people.  That's discrimination.  

There is plenty of revenue from PBMSs, insurers and pharma that could be used to eliminate out of pocket spending on drugs.   that mean drug companies can charge whatever they want, as critics claim?  Hardly.   Rather it means that drug spending will have to be part of a solution that are offered to consumers based on what they value.   New medicines do not drive up health care costs long term, rather they make staying healthy more affordable and convenient.    Every economic analysis shows that.  

Instead of finger pointing about who is to blame, it's time to ramp up the shift to figuring out what value-based purchasing is and making it work.  And it's time to find more ways to reduce the cost, time and uncertainty of drug development.   Both challenges can be overcome by applying precision health tools to improve population well-being and leveraging new medicines to deliver even more value directly to people.    


Written by Rafael Fonseca MD and Robert Goldberg PhD

Two recent articles in the NEJM (1) e) and Annals of Internal Medicine (2) propose that restrictions or eliminations of co-pay assistance programs by pharmaceutical companies are needed since they distort the market and therefore, while few benefit, many suffer in the form of higher healthcare expenditures. They are factually correct but the conclusions point to a larger problem; the third party payer system in health care. When consumers (in this case patients) are detached from the price consequence of transactions they will be less careful in their selections. However, the proposal presented in these articles are, at least in the short-term, patient unfriendly and immediate implementation of their recommendations would limit, severely, access; particularly for patients with serious or life-threatening conditions. The Annals article is notorious in that it fails to mention the increased cost-sharing pressure that patients are facing from the payers.

So what are the solutions?  Ideally, healthcare should be divided between the routine care and the catastrophic care.  Routine care should involve much more “skin in the game.”  In my view, regular care should not be covered under traditional heath insurance but be bought directly, with compassionate support tools for those in need.  In this type of care I could envision more direct pressure in the selection of older medications for which many alternative exist. There are many options for anti-hypertensive medications, statins, etc.  In the absence of a third party payer system the Epipen would never cost $600.  This is where consumer pressure, with co-pay requirement as a discriminator, should be employed; vigorously, if you may.  I would not disagree that if an equivalent generic exists, a true equivalent, then having the patient paying more for a branded product is fine.  This is not to say that branded products and generics are always the same.  Sometimes production quality may suffer or sometimes an alternative may not be the same as the original, similar and yet not the same product.  But if you want the branded statin maybe you should pay a bit more.  Who could argue with that?

However, for more expensive medications, medications used for serious, chronic severe or catastrophic illnesses the pressure seems misguided.  For instance preventing co-pay assistance for patients with cancer seems inhumane.  Dusetzina and colleagues have documented that lack of supplemental insurance delays initiation of treatment for CML (3). Should we disincentivize the use of medications that can be lifesaving for a cancer patient so they consider inferior treatments?  Should we place additional burdens in someone who is at high risk for bankruptcy? Someone who cannot work or whose caregiver cannot work?  That seems inappropriate.  Furthermore, for many of these newer and expensive medications there are no suitable alternatives (4).  Dr. Bach and colleagues were able to reduce the price of a competing monoclonal antibody, but that is a rarity in oncology and more of an exception. Should myeloma patients forgo lenalidomide in favor or thalidomide and face certain peripheral neuropathy?  Or should a diabetic myeloma patient be forced to be treated with bortezomib instead of carfilzomib and again face neuropathy? To create a disincentive as this for people facing serious medical illness appears to me as a fundamental violation of medicine’s stance of benevolence. Rather this has the appeal of altruism, sacrifice the few for the benefit of the many.

Lastly, government regulations that eliminate this co-pay assistance are demonstrably patient unfriendly. Commercially insured patients can receive this directly and in another study by Dusetzina she shows that the average co-pay for specialty medications is only $35 (5). In another study it was shown that 90% of myeloma patients pay less than $100 per month for lenalidomide, a backbone for the treatment of this disease. In the meantime, Medicare beneficiaries struggle to find indirect support with funds provided by the pharmaceutical companies to third parties. I’ve written about this topic before, but again is a built-in disincentive to lessen the use of medications (6).  What if these medications save dollars by preventing hospitalizations and decreasing expenses elsewhere?  Once again it is pound foolish to only concentrate on the price of drugs.

1.    Dafny LS, Ody CJ, Schmitt MA. Undermining Value-Based Purchasing - Lessons from the Pharmaceutical Industry. The New England journal of medicine. 2016. Epub 2016/10/13. doi: 10.1056/NEJMp1607378. PubMed PMID: 27732125.
2.    Ubel PA, Bach PB. Copay Assistance for Expensive Drugs: A Helping Hand That Raises Costs. Annals of internal medicine. 2016. Epub 2016/10/11. doi: 10.7326/M16-1334. PubMed PMID: 27723893.
3.    Winn A, Keating N, Dusetzina S. Factors Associated With Tyrosine Kinase Inhibitor Initiation and Adherence Among Medicare Beneficiaries With Chronic Myeloid Leukemia. Journal of Clinical Oncology. 2016;10.1200/JCO.2016.67.4184.
4.    Fonseca R. 2016. Available from:
5.    Dusetzina SB. Share Of Specialty Drugs In Commercial Plans Nearly Quadrupled, 2003-14. Health Aff (Millwood). 2016;35(7):1241-6. Epub 2016/07/08. doi: 10.1377/hlthaff.2015.1657. PubMed PMID: 27385240.
6.    Fonseca R. 2016. Available from:
From the pages of today's edition of the Boston Herald:

Insurers not covering new cancer treatments

Poor insurance coverage is causing cancer patients to miss out on cutting-edge technologies that use gene analysis to determine the best treatments — a fact a former commissioner of the Food and Drug Administration is calling another example of Obamacare’s failure to provide Americans with high-quality health care.

“There’s a degree of dishonesty about what the Affordable Care Act provides, and that is starkly clear when a patient has a serious type of cancer,” said former FDA Associate Commissioner Peter J. Pitts, who now serves as president of the Center for Medicine in the Public Interest. “The soundbite of the ACA is, many more Americans now have health insurance. But the health insurance isn’t worth the paper it’s written on.”

Pitts said companies are covering the old-fashioned, less-effective chemotherapy regimens rather than more sophisticated approaches and prescription benefit managers are opting not to reimburse the more innovative, expensive treatments. Instead, they are negotiating rebates with pharmaceutical companies that they pocket, rather than passing the savings along to the patient.

“It’s time for insurance companies and prescription benefit managers to step up and do the right thing by putting patients first,” Pitts said. “When they choose not to reimburse for a product because it doesn’t earn them enough money, even though it could save a patient’s life, I think it’s despicable.”

And coverage for the treatments tends to vary by tens of thousands of dollars nationwide, depending on the company and geographic region, according to a University of Texas MD Anderson Cancer Center study published Monday in the journal Cancer.

The study found that insurance costs varied by as much as $47,000 for women on a chemotherapy plan that included the drug Herceptin, which is used to treat breast and stomach cancers by keeping cell growth at bay — and out-of-pocket costs range from $2,700 to $3,400.

The extra costs caused by these variations leads to an additional $1 billion spent to treat breast cancer in the U.S. each year, the study found. But cancer patients will often be covered for a treatment if it has been proven effective by the FDA, according to Dr. Harold Burstein, a breast cancer specialist at Dana-Farber Cancer Institute, who said: “The business of cures not being covered is extraordinarily uncommon.”

Many of the additional costs for the patient, he said, come in the form of lost income and child care.

“Those things are harder to measure than direct hospital bills,” he said, “and they generally have more of an impact.”
Anthem Health has issued a decision deny coverage of Exondys 51, the first drug to treat a form of Duchenne’s muscular dystrophy.  As STAT’s Ed Silverman reports : Anthem “claims that the drug is “not medically necessary” and that “the clinical benefit … has not been demonstrated.” Duchenne is a rare disease that confines boys to wheelchairs and condemns them to an early death.

The decision was followed by this: "Exondys 51 failed to show it improves health outcomes, and therefore it is not a covered benefit for our members," Anthem spokeswoman Leslie Porras said in an emailed statement on Friday.

Not exactly.   Anthem conveniently ignored the fact that the FDA does not approve drugs that have no medical benefit or more precisely,  the FDA approves drugs that are likely to promote treatment of specific conditions.  Anthem claims it is denying coverage because a multitude of limitations cast further doubt on the reliability of dystrophin levels as a surrogate endpoint for clinical efficacy in DMD (FDA, 2016).   But these limitations were considered and approval was given.  In fact, in ignoring the FDA’s decision Anthem is taking refuge in the statements of FDA reviewers who raised concerns about clinical trials but were overruled by Janet Woodcock and Robert Califf, the FDA commissioner.  

I wonder if Anthem would have a problem with doctors and its utilization review munchkins ignoring the company’s medical policy on any treatment based on evidence ultimately rejected by Anthem.   And I wonder what a disability rights attorney will do with the fact that Anthem is willfully misapplying the term medically necessary.  

In fact, Anthem has no problem covering acupuncture, massage therapy, spine manipulations and naturopathy.   

Compare Anthem’s demand for more evidence before covering Exondys with the statement accompanying it’s decision to cover acupuncture:

"Acupuncture as a therapeutic intervention is widely practiced in the United States. There have been many studies of its potential usefulness. However, many of these studies provide equivocal results because of design, sample size, and other factors. The issue is further complicated by inherent difficulties in the use of appropriate controls, such as placebo and sham acupuncture groups…Further research is likely to uncover additional areas where acupuncture interventions will be useful.”

Anthem covers treatments that have less medical evidence of benefit for less severe ailments than DMD.  And it covers treatments based on the it’s belief that research will find acupuncture useful” (though not clinically effective.)   That’s because they attract millions of fairly healthy consumers.

There are about 17000 boys with DMD.    Providing the drug also means paying for all the other supportive services, emergency care, hospitalizations that such kids may require.  To Anthem, spending money to help boys with DMD stay independent and alive is a money loser. 

That’s the only evidence Anthem cares about.  
From today's edition of Morning Consult ...

21st Century Pharmacovigilance and the Role of Artificial Intelligence

Artificial intelligence has an unimaginable potential. Within the next couple of years, it will revolutionize every area of our life, including medicine — and pharmacovigilance. Usually, we make sense of the world around us with the help of rules and processes that build up a system. The world of Big Data is so huge that we will need artificial intelligence to be able to keep track of it.

With the evolution of digital capacity, more and more data is produced and stored in the digital space. The amount of available digital data is growing by a mind-blowing speed, doubling every two years. In 2013, it encompassed 4.4 zettabytes, however by 2020 the digital universe — the data we create and copy annually — will reach 44 zettabytes, or 44 trillion gigabytes.

In a world increasingly driven by outcomes reporting and Big Data, more patient-level information from individual consumers is not always synonymous with better information. The good news is that artificial intelligence will facilitate what the pharmacovigilance ecosystem lacks today — a coordinated and efficient systems for developing actionable evidence on safety and effectiveness.

Today, the absence of these capabilities significantly affects the public health by creating obstacles for patients and clinicians to receive the meaningful information they need to make informed decisions, perpetuating unnecessarily long delays and gaps in effective and timely safety communications and recall management, hindering the timely development of new and innovative treatment options, and increasing the overall costs and inefficiency of the healthcare system.

In considering the role artificial intelligence can play in the both the near and long-term future of outcomes centricity, we need to discuss the concept of Design Thinking which requires cross-examination of the filters used in defining a problem and to revise the potential opportunities before developing strategies and tactics. Design Thinking requires cross-functional insights into a problem by varied perspectives as well as constant and relentless questioning. In Design Thinking observation takes center stage. In the “Sciences of the Artificial,” Herbert Simon has defined “design” as the “transformation of existing conditions into preferred ones.”

Unlike Critical Thinking, which is a process of analysis, Design Thinking is a creative process based on the creation of action-oriented ideas. AI can be a revolutionary tool to develop those action-oriented ideas. And, just for the record, “action-oriented” and “pharmacovigilance” are not mutually exclusive terms.

There is so much data to utilize: patient medical history records, treatment data — and lately information coming from wearable health trackers and sensors. This huge amount of data must be analyzed not only to provide patients who want to be proactive with better suggestions about lifestyle, but also to serve providers with instructive pieces of information about how to design healthcare based on the needs and habits of patients, and provide regulators not just with more data, but better date in context. Can AI usage for adverse event reporting and prediction be far behind?

Artificial intelligence is already found in several areas in health care, from data mining electronic health records to helping design treatment plans, from health assistance to medication management.

Artificial intelligence will have a huge impact on genetics and genomics, helping to identify patterns in huge data sets of genetic information and medical records, looking for mutations and linkages to disease. There are companies out there today inventing a new generation of computational technologies that can tell doctors what will happen within a cell when DNA is altered by genetic variation, whether natural or therapeutic. Imagine the predictive capabilities for pharmacovigilance.

But making knowledge actionable requires the application of proven analytical methods and techniques to biomedical data in order to produce reliable conclusions. Until recently, such analysis was done by experts operating in centers that typically restricted access to data. This “walled garden” approach evolved for several reasons: the imperative to protect the privacy and confidentiality of sensitive medical data; concern about the negative consequences that could arise from inappropriate, biased, or incompetent analysis; and the tendency to see data as a competitive asset. Regardless of the specific reason, the result has been the same: widespread and systemic barriers to data sharing.

If we are to reverse these tendencies and foster a new approach to creating evidence, we must bear in mind that there must be a common approach to how data is presented, reported and analyzed and strict methods for ensuring patient privacy and data security.

Rules of engagement must be transparent and developed through a process that builds consensus across the relevant ecosystem and its stakeholders. To ensure support across a diverse ecosystem that often includes competing priorities and incentives, the system’s output must be intended for the public good and be readily accessible to all stakeholders.

For any of this to work — and especially in the world of pharmacovigilace, we must view artificial intelligence through the lens of 21st century interoperability: the idea that different systems used by different groups of people can be used for a common purpose because those systems share standards and approaches.

And as Philip K. Dick wrote, “Reality is that which, when you stop believing in it, doesn’t go away.”

Will our socio-economic “technology gap” lead to a more pronounced “adherence/compliance gap?” It’s an important question. That’s why it’s crucial we remember there is no one-size-fits all solution. But that mustn’t mean we disregard the reality of the growth and pervasiveness of apps, mobile apps. Let’s face it, when it comes to mobile phones, any gap is rather narrow.

As the American industrialist Walter O’Malley once opined, “The future is just one damned thing after another.” Much depends not just on infrastructure, but also on capabilities, and trust.

The end goal is the same for all stakeholders — ensuring optimal use of resources for health care systems; improving access to value-adding medicines for patients; and appropriate reward for innovation.

As management guru W. Edwards Deming once quipped, “Change is not required. Survival is not necessary.”

Artificial Intelligence is here. Fasten your seat belts.
The FDA has announcing a public meeting to discuss scientific and technical issues relating to formulation development and pre-market evaluation of opioid drug products with abuse-deterrent properties. The meeting is intended to give FDA the opportunity to discuss, and seek public input from stakeholders on, the approach to testing FDA recommended in its draft guidance “General Principles for Evaluating the Abuse Deterrence of Generic Solid Oral Opioid Drug Products.” The meeting will also provide an opportunity to discuss FDA’s efforts to develop standardized in vitro testing methodologies for evaluating the abuse deterrence of opioid drug products. FDA is seeking input from all stakeholders, including patients, health care providers, health care payers, the pharmaceutical industry, patient advocates, academics, researchers, and other government entities.

The FDA may hold one or more additional meetings in the future to discuss the risk-benefit paradigm for opioid drug products to ensure that FDA is appropriately considering the full public health impact of prescription opioid drug products and the post-market impact (“real world effects”) of abuse-deterrent opioid drug products.

The public meeting will be held on October 31, 2016, from 8:30 a.m. to 4:30 p.m. and November 1, 2016, from 8:30 a.m. to 4 p.m.

Full details can be found here.

Amy's Army

  • 09.30.2016
  • Robert Goldberg

Amy Stein Harvey  
My cousin Amy Stein Harvey passed away after living with ovarian cancer for 7 years. She was diagnosed at Stage III C and was told she would likely die within 3 years.   Not only did she "beat the odds" in addition to holding down a job and raising kids, Amy become a powerful advocate for cancer research and for legislation requiring new oral cancer drugs to have the same cost sharing as injectible.   She lived in Michigan, still one of only 8 states that have refused to enact oral parity.  Three days before she died, her friends and supporters participated in a walk in support of oral parity and research.  Amy's Army, as they call themselves will continue her fight.

 Her last post on Facebook (see below) is beyond inspirational.  She teaches us that the source of our urge to live longer is the desire to love and deepen our capacity to love.   

Amy, we will complete the mission you started.  We miss you and will always love you.  Give my mom a kiss for me... 

"Today marks another milestone for me. Today is my 7 year cancerversary! I have been fighting this awful disease for 7 long years. Some days are better than others, but I know that the next day things will turn around. They say that women diagnosed with stage IIIC ovarian cancer don't have a good survival rate. Well, take that stupid cancer. I have beaten the odds!! This past year has had more downs than ups, but I am hopefully on the right track now. I have gone to Washington DC and Lansing to let my voice be heard and put a face to a stastic, I have taken a fabulous cruise to Alaska, and I was able to see Benjamin finish elementary school. Cancer, you listen to me... you're never going to get me down! I am a fighter from the word go and I am going to win this war!!! So, I want to thank everyone for all they have done for our family and most importantly standing next to me in the fight of my life!!!"

BMJ slings mud at FDA -- for shame!

  • 09.28.2016
  • Peter Pitts
A new report in the British Medical Journal “reveals” that many U.S. government regulators who review the safety of prescription drugs later go on to work for the pharmaceutical industry.

As opposed to – the automotive industry?

The authors identified individuals working at the FDA who were charged with reviewing hematology-oncology drugs from 2001 to 2010. They identified 55 reviewers. They found that 49 per cent were still employed with the FDA. Of the half that left, 58 per cent had moved on to jobs in the biopharmaceutical industry or as consultants to the industry. They could not find employment information for about 31 per cent of the reviewers who no longer work for the FDA. According to authors Jeffrey Bien and Vinay Prasad, “The transition from regulator to advising companies seems logical, but it raises concern as to whether regulators indefatigably act in the public interest.”


Here’s what the FDA has to say:

"Federal laws and FDA ethics rules cover issues like outside employment, avoiding real and apparent conflicts of interest, recusals, disclosure requirements, protecting confidentiality, a ban on gifts from regulated industry, and avoiding conflicts should a federal employee choose to seek or negotiate outside employment," the spokesperson said. "Furthermore, past federal employees are bound by additional rules protecting the confidentiality of information they worked on while in federal service, a cooling-off requirement for senior employees, and other important rules against switching sides, contacting former employees, and contacting agency leaders."

And as the Pink Sheet reports:

“Peter Pitts, President of the Center for Medicine in the Public Interest and a former FDA associate commissioner, also slammed the findings of the report. He echoed FDA's comments, while also describing its authors as "ignorant."

"Former FDA employees understand the difference between sharing their expertise with industry and trying to unfairly influence their former colleagues," Pitts told the Pink Sheet. "They are the first to point out their jobs aren't to influence the agency but rather to share how best to properly communicate relevant information, and completely within appropriate limits. Former FDA employees understand the difference between sharing their expertise with industry and trying to unfairly influence their former colleagues. They are the first to point out their jobs aren't to influence the agency but rather to share how best to properly communicate relevant information, and completely within appropriate limits." Pitts added that he has never met a former FDAer "who ever asked for a short cut or an inappropriate edge. Shame on those who would allude otherwise." He further said that people should be applauding the fact that industry, like FDA, is looking to attract "the best and the brightest" to spur innovation.

Any former FDAer who tried to inappropriately influence an agency decision on behalf of a client would become persona non grata – and rightly so. What the authors seem not to understand is that there’s a world of difference between expert navigation and conflicts of interest.

Today Mylan CEO Heather Bresch testifies before a Congressional committee about EpiPen costs.   

I hope she is brutally honest and acknowledges that drug pricing is rigged by health plans, government programs and – above all – PBMs to extort as much money from drug companies and consumers as possible.  

She should explain how and why the price people pay at the drug store has little to do with drug companies raising prices and more about how much insurers and the companies that administer the drug benefit – pharmacy benefit management companies (PBMs) such as Express Scripts and CVS  -- want to charge and how much money they can make off of consumers. 

 The price charged to consumers is set by payors.  And that price is set to increase the spread between net drug prices and retail prices as the chart below shows.

More precisely, the behavior of PBMs – often portrayed as a consumer friendly efforts to keep drugs affordable – fits the classic definition of a cartel and price fixing.

A cartel is an organization of sellers or buyers that agree to fix selling prices, purchase prices, or reduce production using a variety of tactics.  The legal constructs of a cartel vary.  However, the acid test of any cartel is whether all consumers are harmed or helped by the practices. 

The PBM approach to pricing is more akin to a cartel than to a buying group that reduces the cost of the product or the production of the good in question. 

Epipen is a great example. 

Step One:  Create a monopoly or limit competition

Last November Sanofi ($SNY) pulled the main competitor for EpiPen--Auvi-Q--from the market, a turn of events that at time looked as if it “should keep Mylan dominating the epinephrine injection field.”
Mylan already had 85 percent market share.  

Then CVS and Express Scripts removed another competitor, Andrenaclick, from it’s formularies.   Andrenclick retails at $141 while EpiPen retails at around $600. 
Most people pay about $50 per Epipen pack.  But increasingly other plans have decided to make consumers pay thousands of dollars out of pocket before covering them.  Still others require consumers to pay up to half of the retail price of cost the product (About $600).  Some pay less than $3 because they are on Medicaid. 

Step 2.   After eliminating competition, fix both the acquisition and retail price of the drug to maximize profit. 

The PBMs then demanded higher rebates for giving Mylan privilege of being only covering EpiPens.  They were current getting about $400 rebates per EpiPen pack. 

When Mylan balked, CVS and Express Scripts moved EpiPen from the lowest cost sharing tier to the highest cost sharing tier most likely to pressure Mylan and to capitalize on that fact that people need the product to lead normal lives. 

Step 3. Require patients to pay part of or all of the retail price in order and NOT not the rebated price of a drug to generate even more profit. 

Indeed, here’s something Ms. Bresch should make clear: Consumer cost sharing for every other health care good or service is based on the discounted price insurers negotiate.  Except for prescriptions drugs.  On the contrary, we pay a share not of the rebated price of a drug, but the retail price.    

Step 4.  Force companies to cover the huge out of pocket costs of consumers based on the retail price. 

On top of rebates Mylan, under pressure for the increase in out of pocket cost is increasing the amount of money going to patients directly to reduce out of pocket costs.  Which means that Mylan is now forced to fork over rebates and cover the out of pocket cost of the drug, which also goes to the PBMs and insurers. 

The Mylan episode is repeated millions of times every day.  PBMs exclude drugs to create monopolies or cartels in exchange for more rebates.  Indeed, 77 percent of the retail price increase in drugs goes to rebates. This means much of the price increase imposed on patients reflects the cost of rebates that PBMs and other claim make medicines ‘affordable’.  

The Mylan sticker shock was due in part to the ability of PBMs to create monopolies, fix prices to maximize profit.  Indeed, the price fixing takes place at both the wholesale and retail level. Such practices are designed to increase the amount of cash they can collect by drug companies that – after paying rebates – provide to consumers to reduce out of pocket costs. 

Rebates are part of a rigged system that provide cash for big insurers, PBMs and hospital systems while patients wind up paying more, not less.  Manipulation of the quantity, acquisition cost and fixing the sale and resale price of products are classic aspects of a cartel.  Or at the very least, the rigged system is a series of “vertical price-fixing agreements, whereby a seller and a buyer agree with respect to the price at which the buyer will resell, have long been illegal per se. As is true with horizontal agreements, it does not matter how reasonable the agreed-upon price may be or how many good and sound reasons there are for the agreement. Nor does it matter whether the fixed price is a maximum rather than a minimum (although the per se rule against fixing maximum resale prices has encountered substantial criticism recently). 

It’s time for a change.  It’s time to end the rigged system which, in addition to the rebate ripoff includes the profits hospitals pocket when marking up and re-selling drugs that companies sell at a discount to help the poor.   

The FDA's press release announcing the accelerated approval of Exondys -- the first drug for Duchenne muscular dystrophy -- sends a very important message.

The prose is PR-speak 101.  The meaning is in the messenger:

"The U.S. Food and Drug Administration today approved Exondys 51 (eteplirsen) injection, the first drug approved to treat patients with Duchenne muscular dystrophy (DMD). Exondys 51 is specifically indicated for patients who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping, which affects about 13 percent of the population with DMD.
“Patients with a particular type of Duchenne muscular dystrophy will now have access to an approved treatment for this rare and devastating disease,” said Janet Woodcock, M.D., director of the FDA’s Center for Drug Evaluation and Research. “In rare diseases, new drug development is especially challenging due to the small numbers of people affected by each disease and the lack of medical understanding of many disorders. Accelerated approval makes this drug available to patients based on initial data, but we eagerly await learning more about the efficacy of this drug through a confirmatory clinical trial that the company must conduct after approval.”

Why is this release different from all other releases?

In most FDA releases about new drug approvals, the director of the division that reviewed the drug is quoted.  

In this release, Janet Woodcock, who runs the whole show is quoted.  

Woodcock has been publically involved in taking the FDA's good intentions about involving patients in the design of clinical trials and in defining study endpoints as well as encouraging scientifically rigorous alternatives to randomized clinical trials that can bring medicines to market faster as well as allow more patients to participate in a study without randomization.  

After the FDA committee initially rejected Sarepta's application for accelerated approval,  virtually attacking the patient community for not supporting a randomized trial.  

So of course so-called patient advocacy organizations that attack and question new medicines at every turn hailed the first denial.   The expertly and eternally ignorant Diane Zuckerman, president of the National Center for Health Research sums up the anti-innovation tunnel vision: "If this drug is approved — on the skimpiest, most pathetic data I’ve ever seen — then it is going to set a precedent that is very uncomfortable." 

Woodcock stepped in at that meeting to note that too often the FDA focuses on not approving drugs because they are relatively safe and effective but that it often engages in "little consideration of another error, which is failing to approve a drug that actually works”. But most of this consequence is borne by patients who have little say."

Patients had a lot to say about the study design and basis for Exondys 51's  approval.  The FDA's bureaucrats and it's supporters among the handful of elites used the initial denial to derail such involvement.  How ironic that these individuals, who pontificated about using data, not emotion, to determine drug approvals, made extreme and factually false statements (See, Zuckerman, Diane) before and after the denial.

 Exondys 51 is now approved.   So too is a pathway that allows patients who bear the risk of clinical trials and the burden of disease to have a say in how medicines should be measured and used.  DMD advocate Christine McSherry, Executive Director of the Jett Foundation, stated that the DMD community’s involvement in drug approvals “will change the way people confront barriers to justice in society. This will be historic not just in Duchenne, not just in rare disease, but for every ordinary individual who has felt the need to change a broken system but never knew how to do it.”

Christine McSherry and her son Jett

Increasingly medical advances determine not only whether we live or die, but how we will live and die.  Ordinary citizens, like Christine McSherry who are potential patients and the friends and relatives of such patients have as much to say on these innovations as any so-called expert. 

That's what Janet Woodcock's announcement means.   

Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.

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