By: Habib Dable, CEO of Acceleron Pharma, a company member of the Rare Disease Company Coalition
Originally published on MassBio.org (July 20, 2021)
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.
Continue reading at MassBio.org