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David Shaywitz and Mathai Mammen

The next killer app

Online communal wisdom should be harnessed for drug development

By David Shaywitz and Mathai Mammen
January 23, 2011

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NEW DRUGS are subject to exhaustive critical scrutiny, yet there has never been a corresponding effort to collect reports of drugs delivering unexpected benefits. If open innovation can lead to the creation of the world’s most complete encyclopedia, a top-selling cleaning duster, and the game Angry Birds, it’s time to ask whether this approach might be used to capture the exceptional untapped value associated with existing drugs, and to power the discovery of important new medicines.

Half a century ago, astute clinicians noticed that patients receiving the new anti-tuberculosis drug iproniazid experienced an enhanced sense of well-being, a chance observation that led directly to the development of antidepressants and the birth of psychopharmacology. More recently, an unexpected side effect reported by patients in a clinical trial of a drug for chest pain led to the development of Viagra.

These stories of medical discovery may not be so unusual. The idea of using a leukemia drug for stomach cancer, an anti-seizure drug for nerve pain, a cancer drug for gout — all were impactful suggestions proposed by physicians or researchers outside the company developing the drug.

In the age of Facebook and Google, it seems there should be a better, more systematic way of harnessing this communal wisdom and cultivating this sort of medical discovery. Enter open innovation.

Open innovation describes a range of approaches for sourcing ideas externally. These approaches share an appreciation for the richness of ideas and experiences in the world as a whole.

The remarkable power of the open innovation approach and mindset is obvious to anyone who has used Wikipedia, shopped for an iPad app, or used a Swiffer duster — a product developed through Procter & Gamble’s “Connect and Develop’’ open initiative.

A thoughtfully constructed open innovation platform built around existing medications could offer enormous value in the development of new therapeutics. Consider a community where patients and physicians share their experiences with a specific drug, contributing their expertise for the same reasons that lead so many people to offer Amazon reviews, Wikipedia revisions, and Twitter tweets.

A critical mass of participants could identify the unexpected benefits of a medication, and elicit suggestions for new uses. Some of these individual insights might lead to exploratory research studies, generating data that could be deposited in open-source fashion so that others could leverage and learn from it. If these early studies seem promising, rigorous company-sponsored clinical trials could then be conducted to appropriately validate the proposed new indication.

Many of the required components currently exist separately in some form — physicians discuss cases on the websites Sermo and Ozmosis; patients discuss their experiences on PatientsLikeMe and on many disease-oriented websites; while open-access data repositories are offered by both governmental organizations, such as the NIH, and non-profit groups, such as Sage Bionetworks. Yet, there remains a need for a common forum, a community where patients and physicians can interact and iterate, motivated by a desire for understanding, and grounded in a common set of accessible data.

Why aren’t drug development companies trying to set up such open innovation communities to explore potential new applications for their products? The short answer is: they’re not allowed. As useful as it might be to introduce an open innovation platform, any attempt to explore potentially unexpected benefits of a product would almost certainly be critiqued by regulators and industry scolds as “off-label promotion,’’ subjecting the offending company to significant fines and its officers to profound legal consequences.

Companies are also hesitant about seeking any additional knowledge beyond the bare minimum needed to garner approval and fulfill safety obligations, for fear of attracting too much attention from overeager regulators, hammers in search of nails, a phenomenon research analyst Jack Scannell refers to as the “Narrow Clinical Search Problem.’’

To foster the discovery of important new medicines, regulators will need to re-envision themselves as vital enablers of intelligent drug development, and consider the many benefits — both established and unrecognized — of new medications, as well as the real harms of suppressing innovation.

The very quantity and visibility of information generated by an open innovation platform might paradoxically provide the greatest hope, as the sheer abundance of information will emphasize the need not to overweigh any individual data point, while the transparency of data will ensure statistically suspect analyses are appropriately challenged.

Ultimately, of course, the success of an open platform will rest with the community of users themselves. If patients and physicians volunteer experiences in an open and honest fashion, and engage in interactive conversation, the result could be a remarkable opportunity to learn from existing drugs in an unprecedented fashion, accelerating the discovery of powerful new therapies.

A killer app? No, but potentially, a life-saving one.

Dr. David Shaywitz and Dr. Mathai Mammen are employed by Theravance, Inc., a San Francisco- based biopharmaceutical company. Their views do not express the views of Theravance.