You can ignore but you can not hide from Change in Pharma

A few years ago, contrary to the recommendations in Pharmaplasia and in the face of an industry-wide “patent cliff” and a rapidly changing healthcare market, the Pharma industry went on a binge of mega-mergers and multi-billion dollar acquisitions.  The book Pharmaplasia had identified large organizational size as not only challenging but as a liability in the evolving new healthcare market.  But “Big Pharma” wanted to get “Bigger” and it took years for Pharma to appreciate the need for change that had been recommended in Pharmaplasia“eliminate facilities, people, and support systems that no longer have a role in the evolving new healthcare market.”   Now, more recently, we have seen unprecedented downsizings, including elimination of facilities and people in massive restructurings at some Big Pharma.

Sure,  change can take time, especially in large public corporations.  The status quo and doing “what we have always done” is easier but it merely delays the inevitable and can give current frontline employees a false sense of accomplishment and security.

There are over 20 specific recommendations for change in Pharmaplasia that could accelerate a positive evolution for Pharma.  If these recommendations are being ignored by your company for now, it may be just a matter of time before you are affected?

With the information and recommendations in Pharmaplasia you can determine how the changing Pharma business model will affect you in this evolving new healthcare market.  Better yet, Pharmaplasia can help you determine how to  align yourself with these changes so you can participate in these positive changes and not be caught off guard by the inevitable.

Do Manipulative Pharmaceutical Sales Techniques still work?

I’m curious and seriously don’t know the answer to this as it’s been a while since I’ve been in the field.  As a VP of  Sales (admittedly long ago) our management team continuously looked for training programs and techniques to help our sales force be more effective in the physician’s office.   Some were pretty basic but others were just outright manipulative.  I guess in the days of “reach and frequency,”  “reminder detailing,” and unencumbered access we thought we were being cleaver and maybe they even worked to some extent.  At the same time, I have to believe some of these types of techniques also contributed to physician frustration, resentment, and ultimately denied access.

The reason I am asking the question now is because I still see pharmaceutical sales training programs offering what appear to be decade’s old sales techniques that border on manipulative.  Even back in the day when I was selling (admittedly even much longer ago) we learned about “closing the physician”, challenging them, and “getting them to commit.”     I now wonder how much damage these basic sales tenants, combined with a few “tricks,” did in terms of our relationship with physicians.   The reason I say this is because there is nothing more demeaning than to have a sales person try their manipulative sales pitch on you when you go to a store to buy something.  The automobile industry learned this the hard way.  You would think the pharmaceutical industry would have learned by now as well.

But, maybe these techniques and “tricks” still work.  What do you think?  More importantly, how do physicians feel about being sold this way?

Five Underappreciated Contributions from Pharma

In my previous post I pointed out the Five Irreparable Mistakes made by Pharma.  At the same time, many patients owe their health, if not their lives, to advances in drug therapy.  The good is often overshadowed by the unethical and greedy, if not illegal activities and behaviors that make the headlines.  Here are some of the “good” that have been mitigated by the bad:

  1. Treatments for most common diseases

Decades of drug discovery and development have filled pharmacies and our medicine cabinets with a broad range of treatments for many of the common diseases that can compromise our health today.  Most of these are now available as low cost generic drugs.  Sure there are diseases yet to be conquered and recent R & D productivity has been disappointing but we still have to appreciate the numbers of affordable drug treatments we have available to day.

  1. Technology acquisition and development

There is no doubt that without pharmaceutical industry funding and development expertise, we would not have seen the benefits from technologies that would have otherwise remained hostage to under-funding in university laboratories or small biotech companies.   Depleted pipelines in the past decade forced Big Pharma to abandon the NIH (not invented here) research mentality and aggressively pursue acquisition strategies.  To the benefit of patients around the world, Pharma pulled out their checkbooks, took the financial risks, and in many cases, over paid for promising but unproven technologies.

  1. Medicine for the poor

It is easy to criticize the pharmaceutical industry for their seemingly greedy pricing policies and taking advantage of their monopolistic hold on drug treatments.  It is also important, however, to recognize that the industry has done a lot to offset costs for the poor and elderly but even more importantly, making sure that the poor in developing countries have access to drugs they need.  OK, they could have done more and could do more but you can not ignore the free medicines, discounted treatments, and global in-kind disaster support.  And let’s not forget the often criticized sampling programs that often meant the difference between a patient taking medicine that they could not afford or going without treatment.

  1. Continuing Medical Education

Despite the concerns and criticism about whether or not pharmaceutical industry CME is or was promotional and biased, the contribution the industry has made to educating healthcare providers is undeniable. The pharmaceutical industry helped educate millions of healthcare providers not only about drug therapy but about the associated diseases.  While you might debate the quality of the programs, it is clear that academia and the medical profession will have a hard time replacing the numbers of programs and frequency of reaching the numbers of physicians exposed to educational programs by the pharmaceutical industry.  Even if you discount industry sponsored programs for their promotional, biased approach, at the very least, these programs encouraged and provided regular opportunities for healthcare providers to reflect on and do their own research about a broad range of drug therapies and diseases.

  1. Providing a venue for Scientific Excellence

The pharmaceutical industry has provided a fantastic place to train, develop, and apply scientific and technical expertise.  From research and development to manufacturing, science is at the foundation of operational performance excellence.   In the end, pharmaceutical industry training and application of science leads to products and programs that positively benefit mankind in ways that are unmatched by other industries, except perhaps, by healthcare itself.

So for all the notable greed-driven bad behaviors and mistakes that characterize the pharmaceutical industry today, we should not forget the good that has been done.  Unfortunately, the pharmaceutical industry had the chance to be the most respected and valued industry in the world while being embraced, not begrudged, as the most profitable.  Still a laudable goal but one that will take decades to recover from past indiscretions and to demonstrate consistency of good behaviors over time while delivering clinically meaningful breakthrough drugs that can satisfy the remaining unmet medical needs.

Five Irreparable Mistakes made by Pharma

Over the past several decades the Pharmaceutical Industry has made more than its fair share of mistakes but there are five that may be irreparable.   These mistakes that will be near impossible to correct, even in the long term.

  1.  Taking  manufacturing for granted

Pharma executives still don’t get it.   cGMP – compliant pharmaceutical manufacturing is difficult and requires expertise.  It also takes considerable investment to support the quality systems and to maintain operationally.  Pharmaceutical companies often looked to manufacturing for cost cutting opportunities disguised as challenges for operational efficiencies.  Some found outsourcing to be the answer to lower costs and a way to abdicate responsibility.  Others merely delayed repairs and maintenance, reduced labor costs (e.g., hire lower cost, inexperienced technicians and supervisors) or shortcut quality.  And now we wonder why the industry is plagued by seemingly endless episodes of FDA interventions, product recalls, and plant closures.

  1. Valuing “talent” and organizational savvy over expertise

Compensation has been driven by getting promoted, not by the expertise you brought to the table.  People skills, slick presentations, and “managing up” were critical success factors for corporate ladder climbing.  For some it was being lucky enough to be on a blockbuster product team or having a sales territory that embraced private practice over managed care.  Broad general management was valued more than individual contribution as an expert.  For example, wouldn’t you think that individuals who discovered the blockbuster products or figured out how to deliver them safely would be compensated as well as some of the executives who benefited financially from those discoveries?  Without expertise you don’t discover breakthrough products, you can’t consistently manufacture cGMP- compliant products, you can’t run efficient organizations, you can’t be a credible source of information, and you can’t rebuild public trust and confidence.

3.      R & D focus on “development “ rather than discovery research

Just find a patentable compound that has some disease modifying affect that is safe enough to get through the FDA and get it to market as fast as possible.  Fill the pipeline so the CEO can brag to Wall Street about the number of projects in the pipeline, regardless of their real clinical value.  But drug development, while expensive, is the easy part.  Drug discovery is the hard work.  I’m not talking about the simple  “hit and miss” screening for activity.  Finding that new breakthrough product with clinically meaningful benefits for patients requires multidisciplinary expertise, a comprehensive understanding of the underlying pathophysiology of the disease, and takes a long time to produce results.  Most of Big Pharma no longer has the expertise or “know how” to do drug discovery well or efficiently.

  1. Abusive customer practices

If unethical and illegal marketing and sales practices were not enough to erode public trust and confidence in the industry; abusive pricing practices have certainly done so. Desperate patients have been subjected to expensive therapies that, for many, caused more harm than good.  In many cases, the drug companies knew full well they were profiting not from helping patients but rather by putting patient health and safety at risk.  For all the good the industry has done, most patients and the healthcare community don’t care anymore because they no longer trust the industry.

  1. Wasteful spending

Lack of money was never a reason not to do something, including building organizational empires, ramping up sales organizations to tens of thousands of individuals, spending billions on ineffective Direct to Consumer advertising, and billions on promotional “medical education” lunches and dinners.  Tens of billions were spent on R & D that resulted in more “me too” products than true clinical breakthroughs.  And when a Big Pharma got desperate to show growth they spent tens of billions to acquired another struggling Big Pharma at a premium price only to dismantle the acquired company, pay exiting executives lottery size bonuses for getting the deal done, and promising investors long term better results which never came.  We’ll never know what good could have been done with the hundreds of billions of wasted money over the past couple of decades.

The cumulative impact of these mistakes is enormous.  And, as much as these seem like they could be fixed, the damage has been done. What’s most depressing is that the executives managing drug companies contributing to these mistakes were handsomely rewarded for these industry-destructive behaviors.

So, has the pharmaceutical industry done anything good?  We’ll look at that in the next post.

Less Regulation for the Pharmaceutical Industry

I have always been perplexed by the contention that we need less government regulation and more lenient regulatory enforcement in this country, including in the pharmaceutical industry.   Proponents of less government regulation often make their case by proposing to “get government out of private sector business and let the market decide.”  They point to the unnecessary costs, business hardships, and ineffectiveness of government regulation.

We hear about “too much government” until something happens.  Something bad enough to negatively affect a large number of people.  A financial system meltdown, security breach that compromises their safety, or when people take advantage of them through deceptive marketing practices or fraud.  You really hear about the need for more government when people die unnecessarily due to a faulty or poorly manufactured product or a blatant disregard for health and human safety.  Then you hear “where is the government?” “isn’t there a law against that?” and “I’m going to sue.”

For years I have wondered how compounding pharmacies that scale to the size of drug manufacturers could operated without FDA oversight of their manufacturing processes.  Just leave it to State Boards of Pharmacy to monitor.  How “nutritional supplements” could make the health claims pharmaceutical companies could not make without rigorous clinical trials and FDA approval.  Do they really do what is claimed and do no harm?  How device companies could merely claim their product was “substantially equivalent” to an already marketed product to get on the market.  Does it really matter that pharmacies can sell drugs over the internet?  Why should I need a prescription?  Does it matter that the pharmacy operates out of another country or that the drugs I get are counterfeit?  Should it really be ok to create billion dollar blockbuster prescription drugs by marketing them for unapproved claims for patients who might be putting their lives at risk for no benefit?  Less government regulation and limited enforcement make these all possible, today.

The pharmaceutical industry and healthcare market have proven that business enterprises and individuals will push the limits of the law and even ignore the law to make a buck.  That doesn’t mean we should just get rid of government regulation.  In fact, less government regulation and diminished enforcement merely create even more opportunities without negative consequences for fraud, for dangerously marginalizing manufacturing quality, and for the unscrupulous to take advantage of consumers, regardless of how informed or well educated the consumer might be.   Less government regulation and limited enforcement in a “buyers beware” market is not an acceptable commercial environment, especially for the pharmaceutical industry where patient health, safety, or lives are at stake.

So what’s my conclusion?  We need government agencies to do a better job of enforcing the laws that are already in place. We need them to work with law makers to eliminate laws that are no longer doing what they were intended to do when they were adopted.  We need law makers to do a better job of drafting legislation (have you ever read a page of Final Rules in the Federal Register?) to protect consumers.  But what we need most is for businesses and individuals to quit “gaming the system” with wealth building strategies based on taking advantage of lax regulatory enforcement and the unaware or mislead consumer.

Mindset Change Needed for Re-Engineering Pharmaceutical/Biotech R & D

In the previous post and in the book Pharmaplasia I suggest a need for a new approach to pharmaceutical R & D.  There are two essential elements to this proposal.

The first has to do with scientific and corporate integrity.  If the research scientists and corporate executives are not honest about what you have and what you have seen in drug development (if their priority is to protect the “potential“ of your compounds), you can’t make good decisions about your pipeline products.  Research scientists or corporate executives trying to preserve compound “potential” by strategically navigating around potentially damaging data, half-truth disclosures, or blatantly ignoring negative results and their implications can easily sabotage any corporate effort to produce a more robust pipeline with a higher probability of success.  The second essential element will be meaningless if this first element is ignored.

The second essential element:

Know more about your compounds before they go into Phase III clinical trials.

A more comprehensive basic science understanding of your compounds before they enter the clinic, especially expensive Phase III trials; can increase the probability of success.

For safety, this requires an aggressive exploratory preclinical program that “looks for toxicity” (going well beyond the regulatory requirements) and understanding what you find and what you see … not just being able to explain it away.

From an efficacy and safety perspective, if your compound affects one biologic system, what other systems does it affect? Have you really looked or have you been focused on “getting an indication?”   Do you have the basic science data to support the premise/hypothesis for efficacy (or comparative superiority)?  Have you scientifically challenged the premise with alternative outcome possibilities with data to support the different probabilities?   Or, is it still just a hypothesis you plan to prove in Phase III?  Are you sure you have the right endpoints for your Phase III trials?  Have they been sufficiently validated (high probability statistics) in Phase II studies?  By this I mean, have you done more than just a couple of regulatory required trials?  Have you looked at design alternatives that could affect outcomes for different endpoints options?

This strategy may take longer.

But, rather than how fast can you get how many compounds into patients and to the market, research teams and corporate executives need to shift their mindset to increasing the probability of success.  You can do this with a more comprehensive approach to preclinical research.  Thoroughly understanding how your products are going to perform in Phase III trials (or comparative trials) before the trials commence.  Eliminate the “surprises” with better, more comprehensive science around the products.  Be exhaustive in exploration, honest about the findings, inclusive of interpretations, and have better data to support the “go forward” premise or hypothesis.  This all may seem like a simplification but if you’re interested in a more elaborate discussion, I invite you to read about the need for pharmaceutical R & D change and recommendations for change in Pharmaplasia.

Sure there will still be failures, but they should be fewer.  But perhaps the biggest benefit from this approach is the potential for new discoveries of safer, more effective products.  Better diagnostics, more definitive efficacy benefits (maybe we are looking at the wrong endpoints), and advances in the understanding of human biology await this new Pharmaceutical R & D model.  Not to mention, less contentious regulatory reviews, renewed hope for patients with difficult to treat diseases, and more certainty for the investment community.

Why so many Surprising Disappointments from Pharmaceutical R & D?

FDA rejections of new drug applications (insufficient efficacy or safety data), totally unexpected drug failures in Phase III trials,  bewildering “no significant differences” demonstrated in comparative trials, eye opening safety issues in late stage trials or raised by FDA Advisory Boards.  In many cases, negative results sufficient to delay approval if not “kill the drug.”

Along with these stories come the unscientific rationalizations of failures. “That’s drug development.”  “High risk, high reward.” “Biology is complicated.” “Diseases we are trying to treat today are far more complex.”

These are not new headlines for the pharmaceutical industry.  In fact, and unfortunately, they have almost become a cultural industry expectation. Patients ride the roller-coaster of hope and disappointment while investors, also frustrated, keep hoping for that occasional “big win” that makes it all worthwhile.

The pharmaceutical and biotech industries have to find a better R & D model before patients lose faith and investors no longer feel that the “drug discovery and development lottery” is worth playing.

How many more times can Big Pharma place big bets on “promising“ compounds with limited “proof of concept” only to find out they have been sold worthless technology that can’t even make it through a traditional development program to gain market approval?

How many Pharma pipelines boast the number of compounds in development merely to demonstrate that they have something worth investing in, while knowing full well most of the compounds have little or no chance of really making it to market or producing a profit?

How many compounds in these Pharma pipelines (or biotech compounds for that matter) have been strategically developed so as to embellish the efficacy “potential” without exposing or exploring the design flaws that might compromise this “potential?”  How many of these compounds have been carefully tested so as to avoid any suggestions of toxicity that might be difficult to explain or might raise concerns during a “Big Pharma due diligence” (for biotech) or worse, during a regulatory review?

But many of you might be thinking…well that’s just the way pharmaceutical and biotech R & D is.  Well, you’re right… it is and it has worked for decades when the benefits of drug treatment (versus no treatment) outweighed the risks and the market was far more receptive to paying for mediocre “follow-on” products?

Find a compound with biologic activity (remember “get a hit in high throughput screening?”), see if it causes any “apparent toxicity” (do the regulatory required testing but don’t look too hard beyond that) in a few animal models.  Do a quick Phase I trial to see if it causes any “apparent toxicity” in a few volunteers.  Your objective is to get into and out of Phase II (not to really understand what happens in Phase I or II).  Now, pick a dosage schedule and the easiest, fastest indication to establish a quick proof of concept. Then, if you’re a biotech company, find a Big Pharma to buy your compound and/or your company.  If no buyer, get more investment to start a Phase III trial.  If you’re a Big Pharma, push it into full-blown Phase III clinical trials as fast as possible on a timeline that shows investors your “quick to market” development strategy and then “hope for the best.”

The problem is that this historical Pharmaceutical/Biotech R & D model is no longer viable.  So what has to change?

Five Predictions for the Pharmaceutical Industry

I don’t pretend to have a crystal ball but trends and evolving patterns can lead one to predict pretty accurately what is going to happen in the future.  As the predictions in Pharmaplasia have pretty much come to pass and continue to play out,  I decided to take another shot at formulating what appears to be in store for the pharmaceutical industry over the next five to ten years.  So here goes…

  • Big Pharma investors will become less tolerant of multi-billion dollar mega-acquisitions of companies and technologies that benefit executives, bankers, and lawyers but do little to improve the acquiring company.  Investors will no longer accept excuses when these overpriced deals don’t deliver on the expectations and promised returns.
  • Biotech investment will continue to decline as investors become increasingly rigorous in their due diligence, identifying true innovation with meaningful clinical potential rather than investing hopefully in purported innovation hyped by articulate CEOs looking to win the “buy-out lottery.”  With the first prediction in play, there will be fewer “buy-out lotteries” to be won.
  • The continued product focus of pharmaceutical R& D is leading to a prolonged period of fewer truly innovative clinically important new treatments.  The acquisition opportunities for development-ready, truly innovative technologies in small biotech companies will slow dramatically.   And,  merely shooting at “disease targets” with chemistry, without a comprehensive understanding of the pathophysiology of disease, leads to a “hit and miss” mentality, lacking in appreciation for the complexities of human biology.
  • The market will continue to experience significant drug shortages until the healthcare market is willing to pay prices that support high quality cGMP-compliant manufacturing and the pharmaceutical industry (including generic drug companies) realizes that high quality cGMP-compliant manufacturing is a critical success factor, important enough to make the necessary investments to consistently sustain it.  This is difficult for Pharma executives because manufacturing has always been the place to look for operational cost cutting and investment in manufacturing expertise and systems is not as “sexy” or newsworthy as placing a big bet on a “hyped up” new drug technology to excite Wall Street analysts.
  • Unfortunately, the pharmaceutical industry will not have the necessary experience base or leadership to navigate this perilous journey.  Downsizings, retirements, and industry changing career choices have diminished the necessary operational (think research and manufacturing) experience and expertise from the industry.   And, those who have survived and aspire to take on leadership roles have mentored during a period characterized by “me too” drug and acquisition driven R & D, questionable (if not illegal) marketing and sales tactics, and a corporate priority for investor interests over patient well-being.   Not exactly the development track you’d prescribe given the organizational challenges  and the complexities of the evolving healthcare market these new leaders will face.

Realistically facing the prospects for the future can help identify opportunities for changing what isn’t working and developing plans to take corrective action, if necessary.  If you don’t believe my predictions are valid, or you believe I have been to negative, then there is nothing to change.  Big Pharma can continue doing what it is doing.   Seems to me  that’s been the consensus position for some time now.

Completely Eliminating Pharmaceutical Company Financial Conflict of Interest

A guest post in Forbes by Dr.Tom Yates, a UK-based physician, challenges the disclosure statements in two review articles on anticoagulant therapies published in the September 2011 edition of the Quarterly Journal of Medicine.  Dr. Yates also seems to dismiss peer-review as a safeguard and essentially suggests that despite disclosure statements and the peer-review process, any funding by sponsors (pharmaceutical companies in this case) of authors or for editorial assistance for review articles will bias the information presented and will result in an unbalanced assessment of the therapies being evaluated.  He doesn’t critique the papers or their conclusions, admitting that that he is “not an anticoagulant expert.”

In the Forbes blog published letter response to the journal, Dr. Yates’ expectations regarding conflict of interest and the potential for bias are reflected in the following statements:

“In answer to Prof Hobbs’ question [2], I believe it is important that clinicians are able to access review articles on this topic. However, they should be written by authors who have no financial relationship with the companies who make the products under discussion.”

I don’t believe Dr. Yates is alone in his thinking.  I do wonder, however,  if  Dr. Yates and his like-minded colleagues have considered the practicalties of completely eliminating the potential for financial conflict of interest.

  1. There are few therapeutic area relevant clinical experts (not just those who are self proclaimed) who could meet his expectations for independence.    As suggested in his statement above, the only people who could publish review articles are those who have never received any financial support from a pharmaceutical company that has a product in the therapeutic category being reviewed.  That would have to include pharmaceutical company sponsored clinical study investigators, consultants, and advisers.
  2. Only clinical studies that have been independently funded (self funded, government funded, or funded by an advocacy group that accepts no pharmaceutical industry support) could be included in the publication of therapeutic reviews. To accept results from pharmaceutical industry funded studies done by investigators supported by the pharmaceutical company in a review would further propagate the conflicts of interest and biases inherent in the original publications.
  3. Only unconflicted, qualified authors (no potentially biasing financial support from any benefiting organization, be it pharmaceutical companies or therapeutic area advocacy group) could initiate the drafting and publishing of reviews and they would have to seek their own editorial support (including graphics and formatting) at their own personal expense.
  4. Peer reviewers for journals would have to be held to the same independence and conflict of interest standards as they have the potential to introduce their personal biases in their feedback and commentary during the peer review process.   Perhaps then these conflicts would also have to be disclosed in the review article as well.

Here are some things to think about in the context of espousing Dr. Yates’ position:

  1. There are probably too few independently funded clinical studies that are large enough to adequately provide data to do a meaningful review for any therapeutic category or class of drugs.
  2.  “… authors who have no financial relationship with the companies who make the products under discussion” probably don’t have sufficient, statistically relevant, independently funded, personally developed  “controlled clinical data” to support their “independent conclusions.”  Peer-review would have to eliminate any inferences or conclusions that reflect personal biases or opinions from their anecdotal clinical experience that are not supported by statistically relevant clinical data.
  3.  As a result of points 1 and 2, very few review articles could be published.  In fact, I’m not sure there has ever been a clinically helpful therapeutic class review article that is completely void of financial conflict of interest and bias as suggested by Dr. Yates.

Unfortunately, history has demonstrated that we can no longer rely on the integrity of investigators, authors, peer reviewers, and editors to assure us that the implications and conclusions in a review article are valid and were not financially influenced.  Therefore, we have to depend on the disclosure statements and rigorous peer-review to mitigate the potential for financial conflict of interest and bias in scientific and medical publications.

Completely eliminating financial conflict of interest might be impossible and even if achieved has its own negative consequences. You would get reviews done by unconflicted experts who have little or no personal,  controlled clinical experience with the products discussed.  This being the case you really have to wonder “are the experts really experts?”   Their conclusions could only be based on their personal interpretation of financially biased industry supported clinical studies, perhaps some small self funded statistically meaningless studies, or worse, their own anecdotal clinical experience.  Where else would they get the data to support their conclusions?

As a result,  I still believe that honest,  full disclosure and rigorous peer review are better solutions than trying to completely eliminate financial conflict of interest.

How to Stop “Off-Label” Marketing and Sales of Prescription Drugs

I’m a little tired of reading about “off-label” promotion of prescription drugs, especially in the context of whistleblower instigated fraud cases and lawyer/patient driven product liability cases.  I’m not a lawyer but here are some solutions that would discourage inappropriate “off-label” promotion and would consume far fewer resources and certainly cost a lot less than is being spent now on litigating these types of offenses.

First, Pharma companies should not promote products for uses that are not approved by the FDA.  If a company is found guilty of “off-label” promotion, in addition to any corporate fines (which should equal total product revenues during the time of illegal promotion) , responsible individuals should be held legally accountable and convicted, with personal fines, disgorgement of incentive compensation during the time of illegal activities, and even incarceration if warranted.  No corporate settlements.  It is very likely that criminally charged front line employees directed or even trained to promote for off-label uses may be more than willing to offer up and provide evidence against culpable higher level executives who encouraged or approved of the promotion.  I’m pretty sure this would increase executive management oversight to ensure compliance.

To remove the financial incentives for “off-label” promotion, government programs (Centers for Medicare and Medicaid Services and states) should not reimburse for unapproved uses of prescription drugs.  If the patient wants to pay for the unapproved use of a prescription drug that a physicians has prescribed, that should be their choice.  At the same time, that choice carries the liability that if something should go wrong; the only legal recourse for the patient should be to hold the prescribing physician and perhaps their healthcare provider accountable.  Because “off-label“ use is an informed decision, neither the patient nor the physician (or healthcare provider system) could sue the pharmaceutical company for any negative consequences resulting from the unapproved use.  Physicians who prescribe for unapproved uses but post a diagnosis that aligns with approved uses just so the patient can get it reimbursed would face fraud charges and be held personally liable.  Similarly, there would be no need for federal or state litigation against pharmaceutical companies for False Claims that inappropriately causing taxpayers to fund unapproved uses.

If physicians and patients have made a choice to use a product “off-label” and private payers (insurance companies, employers, or PBMs) choose to pay for the unapproved use then they should assume the same liabilities as stated above.  They are making an informed decision and the payer is agreeing with that choice by reimbursing for the unapproved use.  The patient could sue the prescribing physician, healthcare system, and perhaps the payer, but they would have no legal recourse against the pharmaceutical company should a harmful event occur from the unapproved use.

But what about all the “medically established” unapproved uses in treating things like cancer?  The same rules and legal liabilities should apply.  Physicians have the choice to prescribe, patients have the choice to take, and payers have the choice to reimburse for the unapproved use if they want to assume the liabilities with the inability to sue the pharmaceutical company.  If the medical experts, patient advocacy groups, or government programs and insurance companies feel a prescription drug should be approved and reimbursable for a particular use, they should petition the FDA and submit their clinical proof of efficacy and safety to obtain an FDA approved label claim for the product.

While preserving physician, patient, and payer choice these recommendations remove a major financial incentive (reimbursement) for pharmaceutical companies and increase the legal consequences for individuals who inappropriately promote for off-label uses of prescription drugs.  More importantly, it appropriately shifts product liability for unapproved uses to healthcare providers and payers.

Transforming Pharmaceutical Companies in an era of Healthcare Reform