Bipartisan measure can expedite development of therapies for rare diseases
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These days there are not many issues which garner widespread bipartisan, bicameral agreement within the corridors of the United States Capitol; yet the urgent need to encourage the development of better drugs and treatments for patients with rare diseases has proven to be such an issue. After all, we all know someone impacted by a rare medical condition-because rare diseases are not a rare problem. A rare disease is defined as one impacting fewer than 200,000 people in the U.S. An estimated 30 million Americans suffer with one of the 7,000 known rare diseases, and 95 percent of these conditions currently have no FDA approved treatment or cure. Traditional regulatory processes have become more complex involving combinations of therapies, genomics, novel diagnostic tests, multi-systemic diseases, small patient populations, and precision medicine. As a result, numerous parts of the regulatory system need to cohesively work together. The development process for a rare disease drug can take an average of 15 years, which leaves rare disease patients and their families waiting without access to life-changing or even life-saving treatments. Further complicating the situation is that when new therapies for rare diseases are finally approved under the current structure, patients often face unnecessary delays and barriers to access, resulting in avoidable health deterioration. Tragically, many of these patients cannot afford to wait decades for help.

In 2016, Congress came together in near unison to enact the 21st Century Cures Act to accelerate development and access to innovative treatments and cures for those with rare diseases. Now, nearly five years later, leaders from both parties in the House and Senate are once again working feverishly together to pass the Speeding Therapy Access Today Act (STAT) of 2021. This important legislation will enact targeted, impactful, and attainable policy reforms at the FDA to accelerate development of therapies across the spectrum of rare diseases and disorders and facilitate patient access to such therapies. Specifically, the STAT Act will:

  • Improve rare disease coordination, stakeholder engagement, and policy development within FDA
  • Inform rare disease policies and actions by creating a Rare Disease and Condition Drug Advisory Committee,
  • Fund regulatory science and related activities to support development of therapies to treat very small rare disease populations, and
  • Strengthen rare disease patient access to FDA-approved therapies in both public and commercial plans through enhanced FDA and Centers for Medicare and Medicaid Services coordination, proactive engagement of payers, and specific actions intended to strengthen Medicare and Medicaid beneficiary access to novel therapies.

These objectives are accomplished through the creation of a Center of Excellence within the FDA to improve coordination and expedite approval of rare disease drugs. First authorized under the 21st Century Cures Act, the Center of Excellence model has proven effective for rapidly stimulating the development of drugs to treat cancer within the past three years. Last year, the FDA announced a second Center of Excellence focused on digital health, further building on its success. The goal of the STAT Act is to replicate the Agency’s success with this model for the rare disease community. Most importantly, the FDA reforms contained in this legislation will provide hope to the millions of patients and families who are coping with a rare disease. As co-chair of the Congressional Rare Disease Caucus in the House, I am proud to be leading this patient-centered, bipartisan initiative, and encourage my colleagues to join us in this fight.

Bilirakis represents the 12th District of Florida.