The true effect of an intervention is often not seen until real-world usage takes place, but with such a delay between R&D and healthcare delivery, how can the industry close the gap? And what is needed to deliver more effective interventions that patients really want?

While the designation for what constitutes a combination product is clearly defined by the FDA, industry continues to push the boundaries for disease treatment with new and innovative breakthrough drug therapies. As regulatory professionals, the challenge we face is to align these new therapies with application-filing strategies including investigational new drug applications, new drug applications, biologics license applications, and medical devices.

Getting to this point has been a multi-year progression of technological development and regulatory evolution by both the market and government.

In July 2018, the FDA unveiled a Biosimilar Action Plan (BAP) intended to “facilitate the efficient development and approval” of biosimilar products. When FDA Commissioner Scott Gottlieb introduced the BAP, he emphasized the importance of building a market for biosimilar products and expressed concern that the market is not yet established.

With the 2018 Compounding Policy Priorities Plan, the FDA has established a clear pathway for advancement of policies for traditional compounding pharmacies and associated outsourcing facilities. The agency intends to implement the strategies while ensuring access to medications for the critical patient population. It is acting under the belief that a growing number of organizations are trying to get into the large drug manufacturers’ domain while operating under pharmacy licenses.

Are you ready to give the FDA more? “What?” you ask, as a manufacturer of API and finished goods. Are you ready to give them more data and information about your manufacturing process? You think, “Don’t they get all the necessary information from me during their inspections and from my filings?” Well, the answer is yes and no.

The draft guidance document provides recommendations regarding the testing for RCR during the manufacture of retroviral vector-based gene therapy products, and for follow-up monitoring of patients who have received retroviral vector-based gene therapy products.

The FDA recently issued for public comment six draft guidance documents intended to serve as part of a modern, comprehensive framework for how CBER will help advance the field of gene therapy. This article summarizes the first of those draft guidances, Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs).

Writing effective IQ/OQ/PQ protocols is a must for following the regulations required by the FDA for equipment, systems, and utilities to demonstrate suitability for the intended use and to operate according to their design and functional specifications. In order to prove the requirements are met, qualification protocols have to be written and followed.