Amyloidosis Foundation leader calls for reform in drug pricing for rare disease patients

During the recent Health Policy - Behavioral Health Committee meeting in Michigan, a pressing issue regarding the treatment of rare disease patients was brought to light. Advocates emphasized the critical role of the 340B drug pricing program, which is designed to provide financial relief and necessary care for these vulnerable individuals.

The 340B program allows safety-net providers, such as hospitals and clinics, to stretch their resources to serve underserved populations, including those with rare diseases. However, concerns were raised that hospitals are not passing the savings from this program onto patients. Instead, they continue to charge full price for medications while profiting from the discounts they receive. This practice undermines the very purpose of the program, leaving many rare disease patients without the financial support they desperately need.

Advocates highlighted the unique challenges faced by rare disease patients, including the need to travel long distances for specialized care, as there are few centers of excellence available. This situation exacerbates their already high medical costs. To address these issues, it was suggested that hospitals establish charity care programs to ensure patients can access necessary treatments without facing financial hardships.

The discussion underscored the importance of reforming the current system to better support rare disease patients. While the 340B program has the potential to provide significant discounts, the existing framework is failing to deliver the intended benefits to those who need it most. Moving forward, stakeholders are urged to prioritize patient perspectives in any proposed reforms to ensure that the financial barriers to accessing care are effectively addressed.