With the advances in genomics, proteomics, lipidomics and metabolomics, we are understanding so much more about the fundamentals of diseases. However, we still have more to do with rare diseases and in this final guidance, the FDA is providing detailed points to consider and as always recommends meetings with the Agency.
From the Introduction:
"The purpose of this guidance is to assist sponsors of drugs for the treatment of rare diseases in conducting efficient and successful drug development programs. The statutory requirements for marketing approval for drugs to treat rare and common diseases are the same and issues discussed in this guidance are encountered in other drug development programs. These issues are frequently more difficult to address in the context of a rare disease for which there is often limited medical and scientific knowledge, poorly understood natural history data, sample size constraints, and lack of drug development experience."
The guidance includes sections on nonclinical animal studies and under the clinical studies section, subsections on: natural history, dose selection, biomarker selection, study design considerations, evidence of effectiveness and efficacy endpoints, as well as safety concerns. Additionally, the content includes guidance on pharmaceutical quality and considerations for the patient (including pediatric patients), care providers and disease advocates.
#fda #RareDiseaase #Guidance
https://www.fda.gov/media/119757/download
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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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