BIO Investor Forum – Raring to Go: The Race to Treat Ultra-Rare Diseases

The signing of FDASIA by President Obama in July reinforces the country’s commitment to fostering innovation for rare diseases. With a record 460 medicines in late-stage clinical trials, biopharmaceutical companies have embraced rare disease medicine and are rapidly developing the pipeline. In anticipation of an even more compelling opportunity, many companies are taking a step beyond and focusing on ultra rare diseases.

Last week’s 11th Annual BIO Investor Forum in San Francisco hosted a panel, Raring to Go: The Race to Treat Ultra-Rare Diseases, which provided an in-depth analysis of this thriving market opportunity by featuring companies specializing in the ultra rare disease market alongside clinical and policy / regulatory experts in the field.

Moderated by Ellen Licking, Senior Writer & Analyst, Real Endpoints, the panel opened with a focus on the legislation and the regulatory environment. Licking pointed out that there has been a recent uptick in interest on rare diseases which has led to an increase in deal-making. With the recent passage of FDASIA, there have been new opportunities identified for expediting review and approval of rare disease drugs, including accelerated approval, expanded reviewer training and priority review voucher programs to include pediatric rare diseases.

E. Cartier Esham, PhD, Senior Director, Emerging Companies, Health and Regulatory Affairs, BIO was quick to point out the PDUFA technical agreement and FDASIA legislation passed this summer, includes provisions that will  provide training for reviewers to improve communications between FDA and sponsors during drug development by 2014, an expanded accelerated approval pathway, a new  breakthrough therapies designation, and a provision to allow the FDA the ability to access external experts – all of which should be beneficial to sponsors developing treatments for rare diseases. Nick Leschly, CEO, bluebird bio, commented, “Our experience has been that the FDA is very open-minded, they want us to educate them.”

The conversation next turned to the importance of patient and advocacy communities. The panelists agreed that parents must become active and help collect data. Leschly suggested that companies conduct a retrospective study based on data, since ‘there is a lot of haze in scores’. The FDA wants companies to show that the likelihood problems will occur is very low.

Hans GCP Schikan, PharmD, CEO, Prosensa, believes we have an obligation to set up registries to better understand data. Due to the lack of content with small population numbers, there is a need to understand the diseases and future developments as there are few experts in the rare disease field.

Since there are inherent challenges with collecting good clinical data, Emil Kakkis, MD, PhD, Chief Executive Officer & President, Ultragenex Pharmaceuticals recommended an ‘approve early, monitor long approach’ to expedite cures and treatments and ensure safety through post approval studies.

Dr. Kakkis helped concluded the panel with his observation that the best approach to tackling rare diseases is to approve early and monitor for a long period of time. Schikan summed it up best when he asserted that the existing pricing system is not sustainable unless the entire paradigm is changed – longer patents and lower development costs should be the goal.

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