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Pelosi’s radical plan would leave patients with sickle cell disease behind

Tom Wilbur   |     November 14, 2019   |   SHARE THIS

Sickle cell disease (SCD) is the most commonly inherited genetic blood disorder, affecting nearly 100,000 children and adults in the United States. Three million Americans carry the sickle cell trait and it is nearly 25 times more common in people of African American descent. The disorder, which can cause irregularly shaped blood cells to get stuck in narrow vessels, inflicts substantial pain on patients. The resulting reduced oxygen flow creates life-threatening complications such as difficulty breathing, chest pains, blood clots and stroke.

House Speaker Nancy Pelosi’s recently unveiled radical drug pricing plan, H.R. 3, would directly impact patients with SCD. The proposal imposes government price setting for innovative medicines, creating increased uncertainty and destabilizing incentives for investment in research and development (R&D). This is especially true in areas where there is a high level of scientific regulatory uncertainty, like R&D for SCD treatments.

In the 1970s, life expectancy for people with SCD was just 14 years. Now, because of improved screening and therapeutics, most people with SCD are able to live with the disease into adulthood, yet average life expectancy for people with SCD may be up to 25 years lower than those without the disease. Researchers have made great strides, but SCD remains scientifically complex and developing effective medicines has proven uncertain and difficult:

  • Since the late 1990’s, there have been 20 failures to develop a drug that can treat SCD.
  • 45 percent of the failures occurred in Phase II or later.
  • 15 percent were in Phase III when they failed – the lengthiest and costly phase of the clinical trials process.

Despite these setbacks, researchers have discovered new scientific breakthroughs and insights that have led to nearly 20 promising investigational medicines in the pipeline, including a potentially curative gene therapy. This robust pipeline offers hope for patients with SCD, but as of today, there are only three approved drugs for treatment of SCD symptoms.

Biopharmaceutical researchers are working hard every day to improve the quality of life for patients with SCD, but the continued investment is being threatened by proposals like H.R. 3. While critics have said otherwise, research shows that “it is simply not true that the government can impose significant price controls without damaging the chances for cures.”

The Pelosi plan would destabilize incentives for the development of new treatments for SCD, diminishing hope for patients and caregivers relying on future medical innovation.

Policymakers should pursue practical, bipartisan solutions that prioritize lowering out-of-pocket costs for patients. Pelosi’s proposed plan leaves patients behind, jeopardizes the development of innovative treatments and is the wrong approach to control drug pricing in the United States.

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, Rare Diseases, Pelosi Plan

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