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Medical Innovation: The Dream of More Cures and More Industry Success

Can we, as a society, stimulate medical innovation? If so, how? These are key questions facing Congress as it considers amendments to the FDA user fee reauthorization legislation. The answers are of central importance to FDA, patients and industry. So far, most of the “solutions” being considered by Congress (legislatively) and FDA (administratively) are worthwhile and likely to have a positive impact over time.

However, in FDA Matters’ view, the challenge of stimulating medical innovation mostly lies outside the policy sphere. Instead, achieving more cures and more industry success requires substantive and attitudinal changes inside the research and development process itself.

Simply put, most of what can be done externally to stimulate medical innovation is important, but marginal. The biggest changes must come from industry and academia.

  New biomedical knowledge   is being generated every day, some of it quite extraordinary. The challenge is aligning that knowledge into safe and effective medical products. That process requiring inspiration, ingenuity, luck, capital and incredible amounts of hard work. Unsurprisingly, success is hard to achieve.

Regulatory agencies, legislatures, patients, consumers and payers all have a role to play and can definitely influence the success of the research and development process and, thereby, stimulate medical innovation. However, all of those efforts come to naught unless there is:

  • an initial discovery or insight from researchers and inventors
  • followed by carefully planned development and clinical trials
  • concluding with proof that a medical product is safe and effective for its intended use.

 It is the researchers, development teams, product managers, and corporate executives, along with investors and shareholders, who hold the key to medical innovation.

Numerous CEO surveys say otherwise, with a plurality and sometimes a majority asserting that FDA is a major obstacle to their company’s success. However, these CEO’s are hardly objective judges of their own product pipeline. Very few ever acknowledge that regulatory concerns might be justified.  When faced with slow-moving projects, failed trials and agency rejections, the natural response of many is to blame FDA.  

Reality can be hard for CEO’s to accept. Medical innovation is a tough business, as witnessed by the startlingly large numbers of drugs (and to a lesser extent, complicated devices) that don’t survive late stage development. However, with a few exceptions, the inability to prove safety and/or efficacy–not FDA policy or reviewers– are the primary cause of product failures.

This does not absolve FDA, just places the focus on the companies, where it belongs. For its part, FDA knows it can do better and is committed to supporting medical innovation For example, the agency has already agreed (on its own initiative and through support of legislation) that:

  • Some medical products should be moved along faster in the process than they are now.
  • More early-stage meetings with companies would reduce late-stage problems.
  • Greater flexibility is needed with particularly promising compounds.
  • Areas of unmet medical needs and where trials are particularly challenging should be given more attention.  
  • Development of new methodologies, such as  adaptive trials and proper use of patient-reported outcomes, is a key component of advancing regulatory science.

FDA and Congress seem to be doing a good job of working on these issues. We will see the results in the user fee reauthorization process and other amendments, as well as new policies and pathways being developed at FDA. Hopefully, we will also see Congress support these activities through increased appropriated funding of FDA.

These external changes planned by Congress and FDA are valuable in their own right and will certainly result in some good approvals that might not otherwise have occurred.

However, stimulating a significant increase in approvals of medically-innovative products requires industry to: support more biomedical and bioengineering discoveries, carefully plan development, and achieve proof of safety and efficacy. This past week, the CEO of Eli Lilly, which faces patent expirations and generic competition, stated “I don’t think we can save [cost-cut] our way out of the enormous challenge we face. The best course is to maintain our focus on advancing our pipeline.” 

Exactly so. While FDA and Congress are doing their part, our focus shouldn’t wander too far from where it belongs. The key to medical innovation is better and smarter drug and device development. Nothing will help patients or companies if medical innovators don’t innovate.


4 Responses to “Medical Innovation: The Dream of More Cures and More Industry Success”

  1. One reader made a useful distinction between regulatory policy (what my column was about) and public policies that affect intellectual property and financial considerations. He asked: “What about IP protection and other exclusivity to allow companies to recoup investment? Other incentives? Look at the drug shortages – many of which seem to stem from companies who’ve lost their financial incentives. Academia might benefit from rewards that align with national and international health priorities. These would likely be linked to policies.”  I would agree with the reader that these are areas where public policy definitely impacts medical innovation.

  2. I highly recommend a current article in Forbes entitled: "Culture as a Culprit of the Pharma R&D Crisis." It can be found at: http://www.forbes.com/sites/brucebooth/2012/04/19/culture-as-a-culprit-of-the-pharma-rd-crisis/?goback=%2Egde_148555_member_109550482.

    Remarkably, the author, Bruce Booth, manages to analyze drug development problems and possible solutions without once mentioning FDA as a culprit. Even better, he has good ideas that any company can implement if they have the will to do so. I resonate particularly with his comment about reducing meetings–I have clients who spend their entire day in meetings…leaving me to wonder when they get their work done.

    Coming from a completely different perspective, he reaches the same conclusion that I do:  The key to medical innovation is better and smarter drug and device development. Nothing will help patients or companies if medical innovators don’t innovate.

  3. VeronikaValdova says:

    Analysis of large data sets in pharmaceutical industry should be structured. If a study design does not pass the "rule of thumb" test, there is something seriously wrong and it is time to start thinking about the whole process of …thinking.

    That's what meaningful analysis is all about. Maybe that is the reason why new drugs are so expensive – that nobody dares to challenge flaws in projects in the beginning. It is always less expensive to halt a non-viable project sooner rather than later. In the end, when a major problem is discovered, it already swallowed so much money that the person who challenges its validity and purpose risks his/her job or even career. Design flaws rarely disappear by themselves.

    This is a great resource from a different profession but can be transferred in pharmaceutical industry perfectly well.

    David T Moore: Critical thinking and intelligence analysis, NDIC, 2007. It is about reasoning, evidence, methodology, and validity of thought process. The stakes are high – when pharmaceutical and medical professionals "screw up", their clients and customers die.

    David T Moore: Sensemaking, NDIC, 2011. Who can afford to be wrong? How to make better judgments, based on thinking skills and not gut feelings and perceptions.

    From my experience, critical thinking is not a skill highly valued in teams in pharmaceutical industry (specifically pharmacovigilance), as these people are often perceived by management and even their own colleagues as "troublemakers" who tend to "rock the boat". Group-think is dangerous, expensive, and potentially deadly.

  4. Ray Perkins says:

    Your concerns over the origins of medical innovation echos mine for our culture generally.  Our wealth and past successes appear to have fostered both a complacency and fear of loss that is antithetical to innovation.  After all, despite any complaints we might have, most of us survive and some of us succeed within our current systems.  As a general treatment on this subject I recommend the work of Nassim Nicholas Taleb embodied in his "The Black Swan:  The Impact of the Highly Improbable."  In this work Taleb, a former wall street "tech," makes a convincing case that it is the unexpected that ultimately defines subsequent norms.  He specifically expounds on this observation in his model for biomedical research.
    Taleb asserts that, for example, the source of a cure for a disease such as cancer cannot be predicted a priori.  This would seem to be a perfectly rational starting point, except that such thinking excites natural human aversion to apparent chaos.  In the Taleb model for research funding, the inherently unpredictable nature of nature is acknowledged by a system that funds 10,000 research projects regardless of their likelihood of success as measured by current thinking.  Compare this model to that in practice in which thousands of closely related projects are funded, the vast majority of which are small variations on an acceptable theme.  Call this the "Quiet Solution" model for research support.  The "Quiet Solution" model of research funding may provide comfort in its apparent wisdom, but its abysmal track record in, for example, the failure of modern drug development argues for fundamental change.
    Repeating the same actions and expecting new outcomes is clearly wrong-headed thinking.  Industry, funding institutes and academia must find a way to support the search for the "Black Swans."  This may include, for example, a lottery type of financial and materiel support for what many might perceive as wacko science fostered by wacko thinkers, especially non-academic wacko thinkers. 
    But it's not really wacko thinking at all, simply a hedge that 99% of us could be wrong and that the wacko 1% will someday redefine common knowledge.

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