Translation, innovation, regulation: Responding to biopharma's key challenges

Article Highlights

  • Disease-focused foundations reflect the voice and concerns of the patient, bring energy and urgency that big pharma lacks

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  • Helping patients take their medications correctly benefits both patients and drug companies alike

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  • VC firms avoid investing in health products requiring regulatory interaction because the risk, uncertainty aren’t worth it

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Perhaps it's the sweet California air, but the pervasive (though not universal) pessimism in biopharma these days is really bumming me out.  Consequently, I'd like to discuss three potential responses to difficult industry problems.

Problem  1: "Valley of Death" between academia and industry

Response (discussed at length in this recent Atlantic contribution): Perhaps there's a lesson to be found in disease organizations (such as the Myelin Repair Foundation) that are focused on the translational space, and not only facilitate collaborations but actually do some of the critical enabling work required for successful translation, such as validating and robustifying promising but preliminary preclinical models and candidate biomarkers. 

The explicit goal of such translational research is to grease the skids, and ultimately enable companies to more efficiently assess and progress promising compounds.  It's basically taking the experimental medicine/translational medicine function out of pharma and running it out of an organization with an impassioned and enduring commitment to the problem. 

I've long been impressed with the potential transformative power of disease-focused foundations (see here and here), as these organizations tend to viscerally reflect the voice and concerns of the patient, and thus bring a tremendous energy and an impassioned urgency that traditional big pharmas tend to lack.  I also believe deeply in the transformative power of entrepreneurial passion, and the role of clinical champions.

Problem 2: Stagnant biopharma innovation (as measured by NMEs - see here)

Response: Fact - effective new medicines are urgently needed for a range of serious medical conditions, and efforts to generate such products represent an essential, shared goal of patients, physicians, researchers, and medical products companies. 

Yet, I worry that in defining biopharma innovation solely as NME generation, we may be inappropriately narrowing our vision, and missing additional profound new opportunities to significantly enhance the health and lives of patients.  Meaningfully improving behavior and choices could offer significant benefit - the challenge for traditional biopharma companies is figuring out both how to deliver the benefit and capture the value.

Tackling this dual challenge will be difficult but not impossible.  Just helping patients take their currently prescribed medications (or even fill their issued prescriptions) could benefit both patients and companies alike.  Similarly, an inspired effort to understand and characterize a patient's experience with illness could uncover important opportunities for incrementally better drugs to provide impactful benefit, and provide a mechanism to more easily quantify the resulting benefit. 

While most biopharmas (and arguably, most funders of medical research) understandably focus on elucidating the underlying biology of disease, it's interesting to contemplate the impact of instead allocating significant resources towards investigators focused on behavior and decision-making - as well as on the more nuanced exploration of a patient's experience with illness.  To be sure, patient interviews are common - but usually as part of commercial market research, occurring towards the end of medical product development, rather than part of the underlying R&D process, and launched at outset.

Problem 3:  Regulatory concerns stifling innovation

Response:  Everyone recognizes there's a tense dynamic between those developing medical products and those responsible for regulation.  What I hadn't appreciated until recently was the extent to which these concerns have impacted Silicon Valley investment in innovative technologies.  I'm stunned by just how many of Sand Hill Road's most respected (and brashest) technology-oriented VC firms explicitly avoid investing in any health product that requires regulatory interaction; the risk, the aggravation, and the uncertainty are just felt not to be worth it.  Many tech VCs also express concern about products for hospitals, or other major stakeholders - my sense is that the much-discussed complexity of the current health system has made many potential investors leery.

Two thoughts:

(1)   This reminds me a lot of a famous study about risk, which described a phenomenon called the "Uncertainty Effect" (described here).  The basic idea is that the presence of uncertainty causes us to disproportionately undervalue opportunities.  (Some recent work questions the original conclusion - see here.) 

Arguably, the reflexive aversion of many tech VCs to the complexities of the health space reflects an exaggerated concern about the risk, and the proportional underestimation of the potential opportunity.

(2)   I'm also reminded of the frequent advice dispensed by one of my most cherished mentors, Bill Crowley, a professor and distinguished clinical researcher at MGH.  When a subject for a study would be difficult to recruit, or aggressively question the protocol design or rationale, Bill invariably viewed this not as problem but as an opportunity.  The patients who seemed the most demanding, Bill said, were often the ones who would stick with you the most, and would turn out to be the most invested; in contrast, those who signed up demurely often were the ones who dropped out first.

The point is that obstacles are often opportunities, and beyond that, if you invest the time and resources to surmount an obstacle, you are rewarded with an opportunity not accessible to others. 

Implication: perhaps the exceptional time and investment that life-science companies and life-science VCs have made in understanding the great complexities of the healthcare space could be more effectively leveraged than they might appreciate.  If the tech investors who by all rights should be funding the solutions are going to essential cede the space because dealing with regulators and multiple stakeholders stresses them out, someone should take advantage of this opportunity. 

How delicious it would be if the prodigious investment big pharma companies have made in understanding regulators and payors could be repurposed, and this capability used to identify. refine, and even help launch the most promising new technology opportunities.  Perhaps it's time for biopharma companies, or at least biopharma VCs, to ask, in the immortal words of Curt Schilling: "Why not us?"

 

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About the Author

 

David
Shaywitz

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