USA – FDA to form task force, public-private partnership on rare neurodegenerative diseases

The US Food and Drug Administration (FDA) released a five-year action plan for accelerating drug development for rare neurodegenerative diseases, such as amyotrophic lateral sclerosis (ALS), as well as improving access to novel therapies.

“The effects of rare neurodegenerative diseases are devasting, with very few effective therapeutic options available to patients,” Robert M. Califf, MD, FDA Commissioner, said in a statement. “This action plan, especially including the use of public-private partnerships and direct involvement of patients, will ensure the FDA is working toward meeting the task set forth by Congress to enhance the quality of life of those suffering by facilitating access to new therapies.”

The action plan, which implements provisions of the Accelerating Access to Critical Therapies for ALS Act (ACT for ALS), includes establishing the FDA Rare Neurodegenerative Diseases Task Force and a public-private partnership for rare neurodegenerative diseases. Both of these activities would begin in Fiscal Year 2022. The action plan also calls for developing disease-specific science strategies throughout the next five years…