Orchard Therapeutics CEO Dr. Bobby Gaspar, M.D., Ph.D. is one of only a handful of gene therapy leaders to see the light of market-approved therapy. Here, he offers advice for those seeing manufacturing, regulatory, and reimbursement challenges for the first time.

Mistakes in batch records can have considerable consequences for the release of medicines in terms of delays and rejections. Errors can be minimized through the implementation of electronic batch records. However, with any electronic system within the pharmaceutical industry, the requirements of current good manufacturing practice (cGMP), including those of data integrity, need to be met. 

This article is the first in a three-part series based on information from the new guide Incorporation of Pharmaceutical Excipients into Product Development using Quality-by-Design, published by the International Pharmaceutical Excipients Council Federation. Topics covered include measuring the impact of excipient variability on finished product quality and more!

In its latest move to encourage the broader adoption of advanced manufacturing technologies, the FDA has entered into a memorandum of understanding (MOU) with the National Institute of Standards and Technology (NIST) that combines the strength of FDA’s regulatory expertise and NIST’s globally recognized precision characterization and standards. The MOU is significant because it takes the next step in providing much-needed guidance and resources to the industry.

Published in January 2021, the FDA COVID-19 Pandemic Recovery and Preparedness Plan (PREPP) Initiative Summary Report is the culmination of work performed by the FDA’s PREPP Initiative, which launched in April 2020 to examine lessons learned from the agency’s response to the global COVID-19 pandemic. This article provides a brief overview of the report’s overarching themes and highlights key regulatory insights for manufacturers in light of two action areas in particular.

With a small number of approved cell therapies/ex-vivo gene therapies, and with those approved therapies reaching small patient populations, there is not a proven strategy to answer the questions about large-scale commercial manufacturing. The developers of these processes are taking different strategies for commercial manufacturing as they weigh several factors.

It is time to consider augmenting your validated production processes, including processing, packaging, and labeling, with continuous process monitoring using control charts to ensure continued compliance with established specifications and requirements. 

Cleaning validation continues to be a hot topic during regulatory inspections and industry discussions. The cleaning required in an active pharmaceutical ingredient (API) plant between one manufacturing process and the next can present a huge challenge, particularly when multipurpose plants are used.

The FDA posted two unique warning letters that all pharmaceutical and API firms, regardless of their product category, should consider and evaluate. These first-of-a-kind warning letters represent a renewed emphasis on both alternatives to on-site inspections and the importance of purchasing controls and supplier management.