Latest Drugwonks' Blog

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.”

Hogwash.

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.   
Here’s a new op-ed from the Cleveland Plain Dealer. It speaks directly to today’s FDA approval of Sarepta.

Consider what the editor chose to add to my attribution at the bottom of the article:

“Peter J. Pitts, a former FDA associate commissioner, is the president and co-founder of the Center for Medicine in the Public Interest. CMPI receives some funding from biopharmaceutical firms, which could benefit from adaptive trials.”

I suggested that he also add that adaptive trials benefit patients. He declined.  I wonder what the parents of children with Duchenne muscular dystrophy would say?

Build better drugs faster with nontraditional, adaptive clinical trials

Breast cancer surgeon Dr. Laura Esserman of San Francisco sings to her patients as they go under anesthesia. She tackles any song request, whether it's a top 40 hit or a Broadway ballad. This same patient-centric attitude drove Dr. Esserman to participate in adaptive clinical trials, a game-changing way to test new medications.   

It's past time that other researchers think beyond traditional clinical trials.

Adaptive trials can make drug testing both more efficient and accurate. If regulators were to encourage greater use of adaptive trials and facilitate sharing of electronic health record data, researchers could deliver better drugs to patients, faster. 

In a traditional clinical trial, researchers plan out every element of the trial -- from the number of participants to the type of data to be collected -- before they begin testing. They stick to this rigid master plan until the trial is complete. 

But in an adaptive trial, researchers preplan certain modifications that they can make partway through the trial, based on the results they've uncovered so far.  

Think of it in terms of mapping a run. A runner might look at a map and realize that at one point, the road will fork. Instead of deciding whether to go left or right before he ever begins jogging, he might choose to postpone the choice until he actually reaches the fork. Perhaps he'd like to observe which path is muddier or which path has less traffic -- and he'll only know when he gets there.

Similarly, researchers administering adaptive clinical trials can modify their tests as they make observations. Like the runner, they have to preplan what choices they'll make and when. But the more flexible trial formula enables them to alter the trial in response to real-world results. 

The staff at the Case Western Reserve University/University Hospitals AIDS Clinical Trials Unit is using a new video, brochures and other community outreach efforts to invite women without HIV to participate in HIV-related studies.

Dr. Esserman's trial, for example, personalized testing by splitting breast cancer patients into different groups depending on various measurements of their health. She tested a combination of therapies on these patients -- and only continued testing those that were found initially effective. By not having to run a dud treatment through the course of a standard clinical trial, the adaptive design reduced the cost, time, and number of patients needed for the trial. 

Similarly, a group of Florida physicians used an adaptive trial to test and quickly identify the most effective dosage for a drug that relieves post-operation pain. 

By not having to run a dud treatment, adaptive design reduced the cost, time, and number of patients needed.

The U.S. Food and Drug Administration (FDA) currently permits adaptive trials in limited instances. Letting researchers use this model more often would improve the drug development process. 

Another way to improve the process is to enable doctors, insurers, and drug companies to share data on patients' health outcomes after they take new FDA-approved medicines. By analyzing this data, they could uncover patterns that can't be detected even in the largest clinical trials.

Companies like Explorys are pioneering Big Data in the health care realm, raising prospects of a Cleveland niche in an emerging industry. For instance, a clinical trial for a diabetes drug might include 1,000 participants, ten of whom are Native American. Perhaps 60 percent of all patients respond well to the drug, but all ten Native Americans get their blood sugar under control thanks to the medicine. 

It's impossible to tell from such a small sample size whether the medicine really is vastly more effective for Native Americans, or whether those ten patients just got lucky.

The real world offers much larger sample sizes. Assume a million patients take the drug after the FDA approves it, and 10,000 are Native American. If doctors, insurers, and research firms had access to those patients' electronic health records -- with names and other identifying information stripped away, of course -- they'd be able to determine if the drug should be the go-to prescription for Native Americans with diabetes, or if the promising results from the clinical trial were a fluke.

Changing regulations to permit greater sharing of such data would help researchers unveil rare side effects, complications, or "miracle" results that crop up. That would make drugs safer and ensure that the right patients receive the best treatments possible.

Adaptive trials and increased data sharing would deliver better medicines to patients faster and with greater safety. That's something to sing about. 

Peter J. Pitts, a former FDA associate commissioner, is the president and co-founder of the Center for Medicine in the Public Interest. CMPI receives some funding from biopharmaceutical firms, which could benefit from adaptive trials.

When is access not access?

  • 09.17.2016
  • Peter Pitts
From the current edition of Newsweek

There Is a Sure Cure, but It’s Not Covered by Your Health Insurance Plan

When 41-year-old New Jersey resident Amy Speicher was diagnosed with advanced skin cancer, she feared the disease as well as the hair loss, vomiting and crippling fatigue that usually come with cancer treatment.

None of that came to pass. Speicher sailed through her treatment, balancing parenting and a full-time job. How? Her insurance paid for a groundbreaking immunotherapy, which has far fewer side effects than run-of-the-mill chemo.  

Unfortunately, her story is the exception. Throughout the country, insurers increasingly shun the most advanced cures, treatments and tests because they don't want to foot the bill. This short-term focus on profits is callous—and counterproductive. Denying patients the best health care leaves them sicker, ultimately raising insurers' expenses.

In recent years, insurers have replaced fixed co-pays—say, $20 at the pharmacy—with "co-insurance," where patients are asked to pay a percentage of an advanced medicines' total cost. Insurers know many patients can't afford these payments and will ask their doctors for less expensive—and often, less effective—medicines.

Insurers have also embraced "fail first" policies to limit patients' access to cutting-edge treatments. Such policies require patients to use cheaper treatments first. Only if those treatments fail—and patients get sicker—do insurers pony up for cutting-edge drugs.

Increasingly, insurers are also refusing to pay for some treatments unless a doctor first seeks permission before writing a prescription. Most drug regimens should begin immediately, so waiting days or even weeks for an insurer to approve a doctor's prescription simply isn't tenable.  

Consider the case of Angela and Nate Turner, both addicted to opioids. Their doctor sought insurer permission to prescribe a treatment that lessens drug cravings. As the insurer delayed, precious time passed. Angela became violently ill from withdrawal after waiting for three days. After waiting five days, Nick succumbed to his urges and used heroin.  

Insurers are even reducing coverage of preventive care.

For instance, insurers in New Jersey, Tennessee and North Carolina have stopped covering the only genetic screening test approved by the Food and Drug Administration. Such tests scan for genetic mutations that often lead to hereditary breast or ovarian cancer. The insurers now cover only cheaper, lower-quality genetic tests, none of which have proved to be as accurate.

The less-precise tests could raise a woman's risk of receiving a false positive, a test result that incorrectly concludes she's likely to get cancer and should undergo expensive preventive surgery. Worse, lower-quality tests could also lead to false negatives. Women would incorrectly think they have little risk of cancer and opt out of preventive surgeries that could save their lives.

Subpar insurance harms patients' financial health too. Using a better medicine or genetic test from the start prevents lost productivity and needless hospitalizations. In fact, for every dollar spent on newer advanced medicines, non-drug medical spending drops by more than $7.

Insurers know that better preventive care is ultimately cost-effective. But they hesitate to make large up-front investments in patients' health since people frequently switch insurers. Companies don't want to spend heavily—and jeopardize their sacred quarterly results—to save their rivals money a few years down the road.

But this short-term thinking won't save them in the long run. If the overall pool of insured people becomes sicker, insurers' expenses will increase. Year-to-year fluctuations obscure the fact that insurers are just recruiting each other's sick people.

Under the Affordable Care Act, insurers can't resort to their old practice of flat-out denying people coverage. And their new strategies of restricting access to the best treatments only make people sicker, delaying the inevitable expense of covering their care.

Health care economists tout "precision medicine"—the strategy of giving patients treatments and tests uniquely suited to their health and genetic backgrounds—as a way to ultimately prevent and cure illnesses and curb health care spending. But precision medicine works only if insurers pay for the treatments doctors recommend.

If insurers want to help their customers, and their own bottom lines, they need to think long term and cover treatments that prevent and wipe out diseases.

Peter J. Pitts, a former FDA associate commissioner, is president of the Center for Medicine in the Public Interest.

Amundsen turns frosty on biosimilars

  • 09.16.2016
  • Peter Pitts
When it comes to biosimilars, where is True North?

An interesting new paper on the value of biosimilars from the folks at Amundsen Consulting. The paper is appropriately titled, “Who Saves?” The answer isn’t what patients want to hear.

Per the report:

“It may be difficult for patients, physicians, and advocates to understand the value of biosimilar therapies: Because patient costs are not likely to decline, use of biosimilars may be especially difficult to rationalize for those patients who are already gaining clinical benefits from innovator therapies. Without the realization and effective communication of how payers will use savings to support patient care or reduce cost, the value of biosimilars to the U.S. healthcare system will be limited ... Therefore, it is critical that the savings recognized by other parties translate into meaningful improvements in access or new pro­grams/initiatives that advance patient care. Moreover, they need to be communicated to patients and plan sponsors effectively."

The complete paper can be found here.
 
My colleague Peter Pitts has penned an eloquent response to the UN Secretary General’s High Level Panel on Access to Medicines Report. 

The Report is entitled: Promoting Innovation and Access to Health Technologies.  But that’s false advertising.  The report recommends shredding patent protection for new medicines and claims that the cost of medical innovation should have nothing to do with prices.  

I am not surprised.  The recommendation has nothing to do with the facts.  Rather, the panel claims that there is "policy incoherence between the justifiable rights of inventors, international human rights law, trade rules and public health.”

Really?   Seems to me that the thousand-fold improvement in public health worldwide is the result of the commercialization of medical inventions.  As Nobel Prize winning economist Angus Deaton has observed: “Progress in health has been as impressive as progress in wealth. In the past century, life expectancy in the rich countries increased by thirty years, and it continues to increase today by two or three years every ten years. Children who would have died before their fifth birthdays now live into old age, and middle-aged adults who once would have died of heart disease now live to see their grandchildren grow up and go to college. Of all the things that make life worth living, extra years of life are surely among the most precious.”

But the UN panel breezes through how this progress has been achieved – and how countries such as Venezuela has starved its people -- to argue that inequalities of access require redistribution and confiscation of private property.    

To be sure, progress has opened up inequalities as new medicines originated in America (for the most part) and were consumed there first at launch prices that put such drugs out of reach for much of the world. As Professor Deaton notes: “These “health inequalities” are one of the great injustices of the world today... (But) when new inventions or new knowledge comes along, someone has to be the first to benefit, and the inequalities that come with waiting for a while are a reasonable price to pay. “

That is, eliminating inequality is less important than eliminating disease or poverty. And in my opinion the UN panel’s ideas and ideology should be judged by whether or not it will increase absolute well-being not whether it makes Paul Krugman happy. 

The idea that letting rogue states violate IP will somehow increase well-being should have died with Venezuela, Argentina Brazil, India or Thailand.  There is no empirical evidence that state regulation promotes betterment.  On the contrary, the countries pushing for greater government control over private ideas are also societies where the extreme inequality of power strangles growth, including Russia and China. 

Not surprisingly, in such countries the confiscation of IP is just another tool for rewarding rich and powerful interests.  Government production of medicines through compulsory licensing with contracts to produce and counterfeit US developed innovations.  As Deaton notes: “Powerful and wealthy elites have choked off economic growth before, and they can do so again if they are allowed to undermine the institutions on which broad-based growth depends.”

The UN Panel is not simply assaulting patent protection.  It is attacking, as the brilliant economist Deirdre McCloskey observes, two levels of ideas: the ideas in the heads of entrepreneurs for the betterments themselves (the electric motor, the airplane, the stock market); and the ideas in the society at large about the businesspeople and their betterments (in a word, that liberalism).

The UN Panel on Patent Expropriation is, to paraphrase McCloskey “obsessed with first-act changes that cannot much help the poor, and often can be shown to damage them grievously, and are obsessed with an angry envy at the consumption of the very rich. They are willing to stifle, through the generation and commercialization of ideas that lead to greater well-being the trade-tested betterments that in the long run have gigantically helped the poor.”

This assault on health innovation is not only an attack on American productivity, it is a direct hit on the access to medicines the panel professes to promote. 


 

Forrest Gump for UN Secretary-General

  • 09.14.2016
  • Peter Pitts
Just when you thought it was impossible for the United Nations to be less relevant comes The Report of the United Nations Secretary-General’s High-Level Panel on Access to Medicines.

What’s the UN’s answer to broader access to medicines? Doing away with patents. Disregarding intellectual property. Compulsory Licensing. A triple-play of nonsense and unintended consequences. But we’ve heard it all before.

The UN is living proof of HL Mencken’s famous maxim that, “For every complex problem there is an answer that is clear, simple, and wrong.”

The focus of the UN report is on new, on-patent medicines, but the report ignores the WHO’s model Essential Drug List. Why? Because it doesn’t fit the narrative of “Big Pharma as the enemy.” After all, very few of the 400 or so drugs deemed essential are new, or patented or were ever patented in the world’s poorest countries.  In category after category, from aspirin to Zithromax, in almost every case and in almost every country, these medicines have always been (or have been for many years) in the public domain.  That is, the medicine is fully open to legal and legitimate generic manufacture.

And yet, they are not readily available.

There are important implications for the world’s poorest patients. If these patients had reliable and affordable access to these several hundred essential medicines, all available theoretically as multi-source, that is from generics companies, then global mortality and morbidity might be cut as much as 10-20% — a huge gain for populations around the world. Given the potential hugely positive impact on access to medicines, any reasonable person might ask why doesn’t a body that largely represents the needs and desires of the Developing World address this issue?

The UN report isn’t a serious attempt to address access. It’s just another political broadside against patents and intellectual property. For shame.

Perhaps the next Secretary General of the United Nations should be … Forrest Gump.

Neatly timed to coincide with the UN’s screed comes Colombia’s Declaration of Public Interest to impose additional price cuts on the Novartis’ Gleevec. This decision has the potential to result in higher spending on lower-quality generics. Here’s how PhRMA’s Brian Toohey directly addresses the issue,

“There continues to be no legitimate reasons for Colombia to enforce a declaration of public interest for the product in question. This medicine is being provided to all Colombian patients who need it and almost half of the patients needing the drug are taking a generic version. In addition, the medicine has been sold in Colombia at a price negotiated and agreed to by the Colombian Government under its existing pricing system and there is no apparent shortage or evidence of other access issues. The Colombian Government’s actions are therefore without merit.

“Biopharmaceutical innovators support strong national health systems and timely access to quality, safe and effective medicines for patients who need them. Ad hoc price cuts are not effective or sustainable ways to improve access or achieve other critical public health goals. Pricing systems should be based on transparent rules and fair processes that provide business certainty for pharmaceutical innovators.

“The enforcement of a declaration of public interest as a mechanism to impose superfluous price controls sets a harmful global precedent, undermining the incentives that enable high-risk research and development investments in life-saving medical innovation and a host of other cutting-edge industries.

“The way to achieve access to medicines is not through compromising incentives for innovation but by leveraging the collective abilities, strengths and resources of all stakeholders to improve health outcomes.”

Bravo.
Allergan CEO Brent Saunders published a social contract with patients on his corporate blog.   In doing so, Saunders demonstrates that the first question for a leader always is: 'Who do we intend to be?' Not 'What are we going to do?' but 'Who do we intend to be?

Saunder's begins by noting that large price increases undermine the mission of medical innovation:

“Basically, in this social contract patients understood that making new medicines required significant investment.   At the same time companies, doing the hard, long and risky work of bringing new medicines to market, understood that they had to price medicines in a way that made them accessible to patients while providing sufficient profit to encourage future investment.  It was designed to be a win-win-win.  New medicines for patients.  Lower overall cost or damage of disease.  An appropriate return on capital for those taking risk by investing time and talent in the arduous and uncertain task of developing new treatments.

Those who have taken aggressive or predatory price increases have violated this social contract!

I don’t like what is happening, and despite the fact that it is hard to speak out publicly on this, now is the time to take action to spell out what this social contract means to me.  By doing so, I am conveying to my Allergan colleagues that we must keep this social contract in mind as we make business decisions that ultimately improve wellbeing, and as a result, address the hopes others place in us
.”

Saunders first observes that “large payers making decisions that may limit patient access to our medicines in favor of a competitor based on the latter’s willingness to pay more rebates.  In order to ensure that patients and physicians have access to a full array of medical options, we believe that these intermediaries should have open access to formularies whenever possible. “

In the main however, he discusses what his company will do to reinvigorate the social contract with patients: 

We commit to these responsible pricing ideals for our branded therapeutics.
•    We will price our products in a way that is commensurate with, or lower than, the value they create by mitigating or avoiding the need for other treatment modalities or providing better quality of life to those patients without other treatment options.
•    We will enhance access to patients.  This means that Allergan will enhance our patient assistance programs in 2017 to match the current industry leader(s).
•    We will work with policy makers and payers to facilitate better access to our medicines.
•    We will not engage in price gouging actions or predatory pricing.
•    We will limit price increases.  Where we increase price on our branded therapeutic medicines, we will take price increases no more than once per year and, when we do, they will be limited to single-digit percentage increases.  Our expectation is that the overall cost of our drugs, net of rebates and discounts, will not increase by more than low-to-mid single digits percentages per year, slightly above the current annual rate of inflation. 
•    We will not engage in the practice of taking major price increases without corresponding cost increases as our products near patent expiration. While we have participated in this industry practice in the past, we will stop this practice going forward.  Where new regulatory requirements impose added costs, we will seek to reflect those costs in our pricing.
•    We commit to providing an aggregate view of the net impact of price on our business at least annually.


Three quick observations:

1.    Every biopharmaceutical company should take or make the same pledge.   Failing to do so is tantamount to siding with price gouging. 
2.    To limit out of patient of pocket costs Allergan will consider taking the rebates now pocketed by PBMs and insurers and give them directly to patients in the form of increased patient assistance. Other biopharma companies should make the same pledge.
3.    The Allergan social contract includes ensuring that the increased cost of government regulation not be passed on to consumers whenever possible and to limit price increases net of rebates.  See point 2 for how this will be done. 

If most drug companies adopt and live up to the Allergan social contract it would be the most disruptive and positive step the industry has ever taken to establish its value.    

Ultimately, biopharmaceutical companies will need to replace the current business model in which hundreds of billions in rebates go to corporate profits, not patients and in which so-called drug value framework builders are focused on driving down drug prices to increase rebates under the guise of making drugs affordable to patients.   

Hence, the social contract developed by Brent Saunders is also a call to replace a business model that has become financially and morally unsustainable.  To increase and accelerate access now, companies have to reward insurers and PMBs less and help patients more by reducing the cost (and increase in cost) of medicines.  Otherwise any social contract with patients will be an exercise in hypocrisy.  

FDA's Seven Year Itch

  • 08.31.2016
  • Peter Pitts
Almost seven years to the day of the FDA's November 2009 two-day Part 15 hearing on social media, a new Part 15 meeting on off-label communications, Manufacturer Communications Regarding Unapproved Uses of Approved or Cleared Medical Products; Public Hearing

The 2009 affair was "The Super Bowl of Part 15 hearings." Attended by hundreds of interested stakeholders, many of who were skilled communications professionals. The FDA listened as speaker after speaker (including me) offered timely comments on the new frontier of social media. The FDA listened -- and then waited until June 2014 to issue draft guidance.

The big difference between that meeting and the November 2016 version is that there are already a slew of lawsuits that have seriously undercut the agency's authority in regulating off-label speech. Another important difference is that industry already has released its own guidelines. It's also important to note the meeting will take place immediately after national elections. If the folks at White Oak think this will deter attention, they are mistaken.

The agency is soliciting comments as to the ways communications from drugmakers regarding off-label use information are distinct, and whether they provide unique benefits compared to other sources. The announcement lays out eight lengthy sets of questions. Some specific ones are:

* What are the benefits for clinical decision making, research, coverage, reimbursement or other purposes if firms communicate to health care professionals, payers, researchers and patients information about off-label uses? Are there risks, and ways to mitigate these risks?

* To what extent do changes occurring in the health care system that give payers and formulary committees more influence on prescribing decisions provide incentives for firms to generate the necessary high-quality data demonstrate safety and effectiveness for off-label uses?

* What processes do firms use to determine whether information is scientifically appropriate to communicate to health care professionals about a product?

* What information should firms communicate to make audiences aware that the medical product is not indicated for a certain use and to distinguish between the approved uses of the medical product and the unapproved use?

The agency is asking a lot of excellent questions, but they've had a lot of time to ponder all of them already. The only thing that is clear is that no guidance on the topic will be forthcoming until well after a new President takes office -- and that could have profound implications on the direction of both agency thinking and timing.

It will surely be worthwhile, but can the sequel live up to the original?
CMPI

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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