New FDA Guidance Documents: Severely Debilitating or Life-Threatening Hematologic Disorders/ Developing Depot Buprenorphine Products/ ICH Q7A Questions & Answers

1. Severely Debilitating or Life-Threatening Hematologic Disorders: Nonclinical Development of Pharmaceuticals; Draft Guidance for Industry

The purpose of this guidance is to provide information to assist sponsors in the design of an appropriate program of nonclinical studies for the development of pharmaceuticals used to treat patients with severely debilitating or life-threatening hematologic disorders (SDLTHDs).  

While FDA has guidance for oncology indications (most of which are considered severely debilitating or life-threatening diseases) and for rare diseases (which include some SDLTHD conditions), FDA has no guidance to facilitate nonclinical development specifically for pharmaceuticals used to treat nononcology patients with SDLTHDs.  A streamlined approach to drug development is necessary to allow patients with SDLTHDs earlier and continued access to new and potentially effective therapies.

2. Opioid Dependence: Developing Depot Buprenorphine Products for Treatment - Guidance for Industry

This guidance reflects the Agency’s current thinking regarding drug development and trial design issues relevant to the study of depot buprenorphine products (i.e., modified-release products for injection or implantation). This guidance focuses on the development of depot buprenorphine products for which submission of a new drug application (NDA) through the pathway described in section 505(b)(2) of the Federal Food, Drug, and Cosmetic Act (FD&C Act) may be appropriate. 

3. Q7 Good Manufacturing Practice Guidance for Active Pharmaceutical Ingredients: Questions and Answers

The FDA is announcing the availability of a final guidance entitled “Q7 Good Manufacturing Practice Guidance for Active Pharmaceutical Ingredients:  Questions and Answers.” The guidance, which was prepared under the auspices of the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH), addresses questions about implementation of FDA’s guidance on good manufacturing practice for active pharmaceutical ingredients (APIs) (ICH Q7).  The Q&A guidance is intended to clarify uncertainties due to the interpretation of certain sections of ICH Q7 and to help ensure that all APIs meet the standards for quality and purity they purport or are represented to possess.  

FDA publishes ICH guidelines as FDA guidances.  The final guideline was submitted to the ICH Assembly and endorsed by the regulatory agencies in June 2015. 

This guidance reflects just one element in FDA’s work with regulatory authorities and industry associations from around the world to promote international harmonization of regulatory requirements under the ICH. One of the goals of harmonization is to identify and reduce differences in technical requirements for drug development among regulatory agencies. FDA is committed to seeking scientifically based harmonized technical procedures for the development and manufacture of pharmaceuticals.