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Pelosi’s radical plan would leave ALS patients behind

Tom Wilbur   |     October 15, 2019   |   SHARE THIS

Last month, House Speaker Nancy Pelosi unveiled a radical drug pricing plan, H.R. 3, that could jeopardize the development of innovative treatments for some of the most challenging diseases and leave U.S. patients behind. For example, researchers are focused every day on finding new treatments and cures for amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig’s Disease), but Pelosi’s plan could threaten future treatments for ALS by imposing government-set prices on critical medicines, creating increased uncertainty and eroding incentives for investment into risky research and development (R&D).

ALS is a devastating and debilitating disease for which there is currently no cure, due in part to substantial scientific complexities and largely because the disease is still not well understood. Tragically, every 90 minutes someone in the U.S. is diagnosed with ALS, affecting between 14,000 and 15,000 Americans in total. That means between 5,000 and 6,000 newly diagnosed people and their family members are told there is no treatment or cure, each year. To date, developing effective medicines to treat ALS has proven uncertain and difficult: 

  • Currently, only four medications have been approved by the Food and Drug Administration (FDA) to treat symptoms of the disease, with the newest agent being the first approval in over 20 years.

  • None of the available products offer a cure to this fatal disease that kills 90% of patients within 10 years of diagnosis.

  • Between 1995 and 2019, 28 medicines in clinical trials to treat ALS failed to make it to market.

  • 60% of these failures occurred late in the clinical trial process, at Phase II or later; 29% occurred at Phase III or later.

Additionally, the R&D process is a costly one. Before biopharmaceutical companies even reach the clinical trial stage, they have made substantial investments to better understand the nature and potential causes of a disease and to complete FDA-required non-clinical studies – in addition to the costs of finding and screening any number of potential drug candidates that never make it out of the lab. Though progress has been made, particularly in the molecular understanding of this disease, much remains unknown. The unique complexities of this disease create additional challenges to the already difficult, costly and timely research and development for effective ALS treatments.

While critics have said otherwise, research shows that “it is simply not true that government can impose significant price controls without damaging the chances for cures.”

The Pelosi plan could destabilize incentives for the development of new treatments for ALS, attempting to diminish hope that patients and caregivers have resiliently held on to for the future of medical innovation. Instead of blowing up the current system, policymakers should pursue practical, bipartisan solutions that prioritize lowering out-of-pocket costs for patients.

Learn more about Speaker Pelosi’s drug pricing plan here.

Take action and tell Congress to stop Speaker Pelosi’s plan here.

Tom Wilbur

Tom Wilbur Tom Wilbur is a director of public affairs at PhRMA focusing on the organization’s federal advocacy priorities including intellectual property and Medicare Part D. Prior to joining PhRMA, Tom worked in national and state politics for nearly a decade, most recently on Capitol Hill as a strategic communicator and campaign manager. Tom is a proud Michigander and in his spare time enjoys reading, live music, and spending time with friends and family cheering on Detroit sports teams.

Topics: Research and Development, ALS, Pelosi Plan

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