Gene And Cell Therapy Products Community

FDA CBER and CDER divisions posted Rare Disease Evidence Principles (RDEP) on 3Sep25 (https://lnkd.in/eYQ_qhq2). These principles are to "facilitate the approval of drugs to treat rare diseases with very small patient populations with significant unmet medical need and with a known genetic defect that is the major driver of the pathophysiology." Listed on the page are the 'Eligibility criteria' for the disease and therapy to come under the RDEP:

"Sponsors may apply for the RDEP process at any time prior to the launch of a pivotal trial if the investigative therapy is specific to the correction of the genetic defect in question (i.e., either correcting the gene or is a replacement of an essential physiological protein that is otherwise deficient due to the gene defect) and meets the following criteria:

• the drug is for a very small, rare disease population or subpopulation (e.g., generally less than 1,000 persons in the United States);
• the drug is intended to treat a known, in-born genetic defect that is the major driver of the pathophysiology;
• the clinical course of the disease is progressive deterioration in function leading to rapid and/or significant disability or death in a relatively short period of time; and
• there are no adequate alternative therapies that alter the course of the disease."

For more details see the linked page.

#fda #RareDiseaase

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Mark Arnold Ph.D., FAAPS
Westampton, NJ
[email protected]
Bioanalytical Solution Integration
LinkedIn: https://www.linkedin.com/in/markearnoldphd/
Website & Blog: Bioanalysis & Biomarkers <bioanalysisandbiomarkers.blogspot.com>
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</bioanalysisandbiomarkers.blogspot.com>

Thank you for sharing this, Mark.

Yan

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Yan Ni Ph.D.
Executive Director
Passage Bio
Philadelphia PA
[email protected]

Disclaimer: Opinions expressed are solely my own and do not express the views or opinions of my employer.
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Original Message:
Sent: 09-04-2025 08:24
From: Mark Arnold
Subject: FDA Rare Disease Evidence Principles (RDEP) to facilitate therapeutic development

FDA CBER and CDER divisions posted Rare Disease Evidence Principles (RDEP) on 3Sep25 (https://lnkd.in/eYQ_qhq2). These principles are to "facilitate the approval of drugs to treat rare diseases with very small patient populations with significant unmet medical need and with a known genetic defect that is the major driver of the pathophysiology." Listed on the page are the 'Eligibility criteria' for the disease and therapy to come under the RDEP:

"Sponsors may apply for the RDEP process at any time prior to the launch of a pivotal trial if the investigative therapy is specific to the correction of the genetic defect in question (i.e., either correcting the gene or is a replacement of an essential physiological protein that is otherwise deficient due to the gene defect) and meets the following criteria:

• the drug is for a very small, rare disease population or subpopulation (e.g., generally less than 1,000 persons in the United States);
• the drug is intended to treat a known, in-born genetic defect that is the major driver of the pathophysiology;
• the clinical course of the disease is progressive deterioration in function leading to rapid and/or significant disability or death in a relatively short period of time; and
• there are no adequate alternative therapies that alter the course of the disease."

For more details see the linked page.

#fda #RareDiseaase

------------------------------
Mark Arnold Ph.D., FAAPS
Westampton, NJ
[email protected]
Bioanalytical Solution Integration
LinkedIn: https://www.linkedin.com/in/markearnoldphd/
Website & Blog: Bioanalysis & Biomarkers <bioanalysisandbiomarkers.blogspot.com>
------------------------------
</bioanalysisandbiomarkers.blogspot.com>