FDA Unveils Five-Year Plan for Rare Neurodegenerative Disease Research
Under a new five-year action plan for accelerating the development of treatments for rare neurodegenerative diseases, the FDA will focus on incorporating new technologies and innovative trial approaches beginning with the study of amyotrophic lateral sclerosis (ALS).
According to the agency, the goals of the five-year strategy include encouraging the use of digital health technologies and decentralized approaches in ALS trial design, ensuring expanded access is accounted for in development programs and improving the diversity of ALS trials by tackling barriers and burdens for underrepresented groups.
The agency also said it will work to improve the characterization of ALS pathogenesis and natural history, including by researching predictive and prognostic biomarkers and by quantifying how the disease progresses.
Under the plan, a task force will develop disease-specific scientific strategies over the next five years and “leverage ongoing FDA regulatory science efforts” in the therapeutic area. The agency also pledges to work with patient advocacy communities to ensure the patient voice is included in discussions.
In addition, the agency will work to identify promising therapeutic candidates earlier to optimize their trial designs, open access, streamline operations and cut the cost of drug development.
Read the FDA’s plan here: https://bit.ly/3unRhgj.