Rare diseases
For Rare Disease Week the FDA Center for Drug Evaluation and Research (CDER) published a report following interviews with stakeholders in the rare disease drug development community to better understand the unique challenges in bringing rare disease products to market, such as challenges related to nonclinical studies, dose-finding, natural history studies and registries, novel endpoint and biomarker development, clinical trial design and analysis, and regulatory considerations.
See also:
Accelerating Rare disease Cures (ARC) Program
Device manufacturing
Following an increase in submissions containing unreliable data generated by third-party test laboratories, the FDA is calling upon the medical device industry to be vigilant and proactive about ensuring the integrity of all data they include in medical device submissions:
Fraudulent and Unreliable Laboratory Testing Data in Premarket Submissions: FDA Reminds Medical Device Manufacturers to Scrutinize Third-Party-Generated Data. This trend has resulted in the FDA being unable to reach a substantial equivalence determination or otherwise authorise marketing for medical devices whose submissions include such data.
Draft guidance out for comment
With the release of the draft guidance titled Use of Data Monitoring Committees (DMC) in Clinical Trials the FDA intends to provide recommendations to help sponsors of clinical trials determine when a DMC would be useful for clinical trial monitoring and what procedures and practices should be considered to guide their operations.
Submit comments by 15 April 2024.
Draft guidance out for comment
FDA’s guidance on Advanced Manufacturing Technologies (AMT) Designation Program provides recommendations to stakeholders interested in participating in the program.
The guidance outlines the eligibility criteria for AMT designation, the submission and assessment process for requests, the benefits of receiving an AMT designation and includes a question and answer (Q&A) section to cover additional details about key concepts important for program utilization.
Submit comments by 13 March 2024
Final procedural guidance released
The following guidance provides information and Q&As on the implementation of FDA’s regulations on
charging for investigational drugs under an investigational new drug application (IND) for the purpose of either clinical trials or expanded access for treatment use (21 CFR 312.8)
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