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FDA Issues Action Plan for Rare Neuro Diseases

Man in wheelchair


24 June 2022. The Food and Drug Administration published its blueprint to encourage new treatments for rare neurodegenerative disorders, including ALS. FDA says its five-year action plan seeks to promote more innovative medical products addressing amyotrophic lateral sclerosis or ALS and other rare degenerative diseases of the nervous system, as specified in legislation enacted late last year.

The Accelerating Access to Critical Therapies for ALS, or ACT for ALS bill calls for FDA to create a five-year plan to encourage new drug and medical device development addressing rare neurodegenerative diseases, such as ALS. The law also asks FDA to increase access to investigational therapies, and provide financial support in grants or contracts to cover costs of research for prevention, diagnostics, and treatments for these diseases. In addition, the law calls for FDA to work with National Institutes of Health to establish a public-private partnership, with funding through cooperative agreements or contracts, to advance understanding and new therapies for ALS and other rare neurodegenerative disorders. The bill authorizes $100 million over five fiscal years for these measures.

ALS, also known as Lou Gehrig’s disease, is a progressive disorder where neurons in muscles start wasting away, and no longer send or receive signals from the brain or spinal cord. As these nerve cells stop working, muscles in the limbs, followed by speech and breathing muscles, begin weakening and eventually stop functioning. Most people with ALS die of respiratory failure. According to Johns Hopkins University, ALS affects some 30,000 people in the U.S., with 5,000 new cases reported each year.

Fewer biomarker targets and and preclinical models

“We recognize the urgent need for new treatments that can both improve and extend the lives of people diagnosed with these diseases,” says FDA commissioner Robert Califf in an agency statement. “To face that challenge and to accelerate drug development, we need innovative approaches to better understand these diseases while also building on current scientific and research capabilities.”

FDA’s action plan notes that ALS and other rare neurodegenerative diseases are difficult to study because of their small numbers of occurrences, and thus have few new therapies in current pipelines. Molecular drivers and mechanisms of these disorders, says the agency, are less understood, with fewer biomarker targets and and preclinical models, resulting in fewer new diagnostics or drugs, and lower success rates in clinical trials for new product candidates.

FDA’s plan includes a science strategy to address these knowledge gaps for ALS. The agency says it plans to support research that better defines and quantifies the progression of ALS in the body and reveals more biomarkers for predicting disease progression. The science strategy also proposes more flexibility in clinical trial design, including decentralized studies and digital data gathering, and improved access for patients to reduce their time and cost. Better access to clinical trials, says FDA, can also provide greater access to therapies in development. Similar science strategies are planned for other rare neurodegenerative disorders.

For the immediate future, FDA is establishing with NIH a public-private partnership for rare degenerative diseases to encourage new treatments and better tools for drug development. And FDA is setting up an internal rare disease taskforce to coordinate actions related to new therapies among its drug, biologics, and orphan products offices. In May, the agency began a program to accelerate development of treatments for rare diseases, which will establish a working group for neurodegenerative disorders.

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