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The SPSRF Staff

National Academies Report Calls for Faster Approval of Treatments for Rare Diseases

National Academies report highlights recommendations to accelerate the approval of rare disease treatments, offering hope for faster therapies for conditions like SPS
 

The US National Academies of Sciences, Engineering, and Medicine have released a landmark report aimed at improving the development and approval processes for treatments of rare diseases, such as Stiff Person Syndrome (SPS).


This comprehensive review makes significant recommendations that could pave the way for faster approvals and more effective treatments for rare diseases across the globe.


Recommendations for the development of rare disease treatments.

The US National Academies of Sciences, Engineering, and Medicine have released a landmark report aimed at improving the development and approval processes for treatments of rare diseases, such as Stiff Person Syndrome (SPS).


This comprehensive review makes significant recommendations that could pave the way for faster approvals and more effective treatments for rare diseases across the globe.


 

This new report provides recommendations for enhancing and promoting disease drug development and treatment by improving engagement with people affected by a rare disease.


 

One key takeaway from the report is the emphasis on using Alternative and Confirmatory Data (ACD), which includes patient-reported outcomes, natural history studies, and patient registry data. This approach is particularly relevant to rare diseases like SPS, where traditional clinical trials may be challenging due to the small patient population. By incorporating ACD, the FDA and the European Medicines Agency (EMA) can potentially accelerate the approval process for much-needed treatments.


The report further advocates for enhanced international collaboration, particularly between the FDA, EMA, and other regulatory bodies. This cooperation would facilitate data sharing and streamline the development of rare disease treatments. For SPS patients, caregivers, and advocates, this development offers hope that the time to market for new therapies may significantly decrease.


Although the report does not specifically address SPS treatments, its recommendations create a framework that can benefit the entire rare disease community, including those affected by SPS. The full report is freely accessible and contains critical insights that could shape future policy and research efforts.


For more details, you can read the full report here.

 

For media inquiries, please contact:

Neil Mathis

Marketing and Communications

The Stiff Person Syndrome Research Foundation

 

About SPSRF

The Stiff Person Syndrome Research Foundation is dedicated to advancing research, raising awareness, and providing support for those affected by SPS. Our mission is to find a cure and improve the quality of life for patients through raising SPS awareness, funding SPS research, and providing community support and education.

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