Orphan Drug Franchises Best Left to Mature Alone, Rather Than as Part of Large Organization, Says Bionest Partners

New In Vivo Article Points to Highly Patient-Centric Approach Required for Rare Disease Success Compared to Traditionally Structured Pharmaceutical Business Model

NEW YORK & PARIS--()--Orphan Drug franchises perform better if kept separate from traditional pharmaceutical/biotech organizations due to the different cultures needed for successful product commercialization, says Alain J. Gilbert, co-chairman of global strategic consulting firm, Bionest Partners. Commercial success in the rare disease field depends on a highly patient-centric approach that connects effectively with rare disease patient communities at a senior management level and focuses on access and interactions with thought leaders and physicians, rather than on selling drug features and benefits. Large companies can rarely afford such focused engagement for a small product line and face organizational structures and pressures that make alignment with a successful rare disease model difficult.

An in-depth analysis of the business model of successful rare disease franchises and recommendations for larger firms seeking to engage in such initiatives is the subject of a new publication by Gilbert and co-authors, Anne-Sophie Demange and Mark Ratner in the February issue of In Vivo.

“While a number of large, established pharmaceutical firms have been interested in drugs for rare diseases, either through their own R&D efforts or through in-licensing, few to date have achieved notable success,” said Mr. Gilbert. “Effective marketing of rare disease drugs is very patient-focused -- it requires finding the patients who need treatment, getting them on therapy quickly, and helping with country by country reimbursement. Moreover, it involves building close and trusted relationships with thought leaders, clinical investigators, physicians and patient advocacy groups at a high level of senior management commitment. In contrast, a number of issues and rigid organizational structures can prevent large companies interested in rare diseases from performing with the flexibility and creativity needed for success.”

The authors say that the large size of a pharmaceutical company often comes with standardized practices, slower decision-making, and legal corporate compliance policies that can greatly limit patient contact at meetings and other settings. The best model to date for large companies building rare disease franchises has been one in which an existing rare disease company has been acquired and left alone to interact with the rare disease community.

“We believe that while orphan drug franchises are indeed valuable, they are not for everyone,” added Ms. Demange. “Broad-based traditional pharmaceutical companies should let these orphans live alone -- at least until they reach a level of maturity to be able to withstand the structural organizational pressures of big company life.”

About Bionest Partners

Bionest Partners is a strategic life sciences consulting firm focused on Pharma, Biotech, Medical Device and Diagnostics. We help clients navigate complex product development and commercialization issues in order to drive growth, create value and gain competitive advantage. We also offer our expertise and analytic skills to support investors with due diligence assignments. Our deep content experience, combined with our collaborative and hands-on approach results in practical solutions that drive internal alignment at the corporate, portfolio, and brand levels. We operate globally, with corporate offices in New York, NY and Paris, France. For more information on our team and services, please visit our website at http://www.bionest.com.

Contacts

Bionest Partners
info@bionest.com
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Release Summary

An in-depth analysis of the business model of successful rare disease franchises in biopharma is the subject of a new publication in In Vivo by Bionest Partners called "Orphans Should Live Alone."

Contacts

Bionest Partners
info@bionest.com
or
For Media:
Bioscribe, Inc.
Joan Kureczka, +1-415-821-2413
Joan@bioscribe.com
or
Nicole Litchfield, +1-415-793-6468
Nicole@bioscribe.com