We must do everything in our power to ensure that patients have access to innovative new therapies.
In the biopharmaceutical industry, this refrain is as familiar as it is true. For those of us at companies committed to developing potentially transformative medicines to treat any of the approximately 7,000 recognized rare diseases—93% of which have no FDA-approved treatment—this call to action has never felt more attainable, yet at the same time, more at risk.
As legislators contemplate a range of access and pricing reforms in this era of rising costs and constrained resources, there is a considerable temptation among legislators to paint our industry with a single broad brush. Such one-size-fits-all policy approaches are a very real threat to the innovation that fuels the hopes of the small but needful patient populations we endeavor to serve. Further, such policies fail to recognize or reward the level of risk that smaller, rare-disease-focused companies—historically, the companies best suited to producing true advances in their chosen spaces—incur through much of their existence.
Before becoming CEO of Acceleron, I spent more than 20 years in classic “big pharma” at a multinational corporation with a vast portfolio of marketed drugs to treat prevalent diseases across a breadth of therapeutic areas. Revenues supported nearly 100,000 employees worldwide and funded a robust research pipeline. This is most certainly not the business model my rare disease counterparts and I execute on. Many of us are in the pre-clinical, clinical or early commercial stage, relying almost entirely on investors for funding as we seek meaningful therapeutic advances. Many of us may only have one or two seemingly viable assets, a precarious state in an industry in which failure is far more often the rule rather than the exception.
In my four and a half years at Acceleron, I have had to make the difficult but necessary decisions to terminate three pipeline candidates, one in oncology, and two in neuromuscular disease. While we were not forced to downsize—an all-too-familiar and painful cost for many in our line of business—perhaps the most difficult aspect of these decisions was the palpable disappointment among the patients who had participated in our trials and for others who were so desperate to see these programs succeed.
Unfortunately, many of the policy proposals being considered in Washington, D.C., and in state capitols fail to take into account the unique circumstances of small biopharmaceutical/biotech companies, especially those committed to discovering and delivering transformative therapies to patients living with rare diseases.
To be clear, an articulation of the need for policies that foster and protect our ability to innovate is not an endorsement of the status quo. It is a societal and industry imperative that we distinguish the transformational from the incremental and incentivize the development of—and access to—first-in-class/best-in-class therapies for patients with few if any options for treatment. At the same time, we in industry must commit to being responsible partners within the rare disease and greater healthcare ecosystems.
We have much work to do in this regard, but as one tangible step forward, I’m proud that Acceleron is one of the founding members of the recently formed Rare Disease Company Coalition, whose mission is to educate and advocate for scientific innovation on behalf of rare disease patients. I look forward to collaborating with the Coalition, MassBio, other like-minded organizations, and my counterparts at life science companies large and small in pursuit of sustainable public policy and regulatory solutions that preserve and expand access to groundbreaking therapies for the one in 10 patients around the world living with a rare disease.
Habib Dable joined Acceleron in December 2016 as the company’s President and Chief Executive Officer, and as a member of its Board of Directors. Prior to Acceleron, he served as President of U.S. Pharmaceuticals at Bayer AG. During his 22-year tenure there, Mr. Dable held positions of increasing responsibility. In addition to President of U.S. Pharmaceuticals, he also served as Executive Vice President, Global Head Specialty Medicine; Vice President, Ophthalmology, Global Launch Team Head, EYLEA®, Global Head, Neurology, and Ophthalmology; Vice President, Regional Head, Hematology and Cardiology; Director of International Sales and Marketing, Biologics Research; and Head of Strategic Planning, Pharmaceuticals, Japan.