Treatments for rare diseases at risk, due to the decline of plasma donations
Representatives of patient advocacy groups, expert physicians, thought leaders, and representatives from the U.S. Food and Drug Administration (FDA) Center for Biologics Evaluation and Research came together on December 9 at a roundtable sponsored by the Plasma Protein Therapeutics Association (PPTA) to discuss the impacts on people with rare diseases if not enough plasma is available to manufacture lifesaving plasma-derived therapies. These therapies are often the only treatments available for people with primary immune deficiencies, bleeding disorders, Alpha-1 Antitrypsin deficiency, hereditary angioedema, and certain neurological conditions.
Roundtable participants stressed the continued urgent need for plasma donations. Reports vary, but plasma collectors experienced significant declines in collections due, in part, to the impacts of social distancing measures and other mobility restrictions caused by the COVID-19 pandemic. Considering the complex manufacturing of plasma-derived therapies can take 7-12 months, any decline in plasma donations could impact patients’ ability to access their lifesaving therapies. This sharp decline in plasma collections could cause more significant challenges in coming months.
“Imagine enduring this year while living with a rare disease whose treatment is dependent on a raw material that has declined precisely because of the pandemic,” said Amy Efantis, PPTA’s President and CEO. “Source plasma is essential, so our concern for meeting clinical need is not based on reimbursement or supply chain disruption, but raw material.”
Efantis went on to stress that individuals can help ensure patients continue accessing their therapies – first and foremost, all eligible and healthy adults should consider donating plasma and spread the word about the need for plasma via social media. Additionally, PPTA is asking individuals to contact their local policymakers about the need for plasma donation and to support calls for the coexistence of public and private plasma collection programs.
Director, Global Communications