Chemistry, Manufacturing, and Control Information for Human Gene Therapy Investigational New Drug Applications
This draft guidance is aimed at giving gene therapy developers recommendations on how to assure product safety, identity, quality, purity and potency with new gene therapies.
The 54-page guidance is organized with a similar structure as the Common Technical Document, and applies to gene therapies that are used in combination with another drug or device.
Long Term Follow-up After Administration of Human Gene Therapy Products
Because of the uncertainty involved with these novel therapies, the FDA has created guidelines for companies to follow when conducting post-approval trials. These standards detail which product characteristics, patient-related factors and clinical data should be studied to create the kind of robust, post-market follow-up needed to better understand the impact of new gene therapies, and help ensure long-term patient safety.
The days of FDA inspectors climbing into full-body Tyvek suits and carefully monitoring manufacturing facilities are far from over. But this year, the efforts to modernize and streamline the process reached a critical new turning point.
New Inspection Protocol Project
Since the New Inspections Protocol Project (NIPP) was passed in 2015, the FDA has been working to bring facility inspections into the digital age. According to the agency, NIPP aims to shift to data-based tools to “facilitate the collection of information during the inspection in a standardized format.”
“The electronic inspection protocols enable semi-automated generation of Establishment Inspection Reports for pre-approval and surveillance inspections…and are expected to save investigators time in compiling data, which will also help with efficient analysis of the inspection,” the FDA said in an emailed statement.
In 2018, the agency announced that NIPP was moving beyond the pilot testing phase and is now being implemented in certain facilities. The agency chose aseptic facilities as its first targets because of the higher safety risk involved in sterile manufacturing.
The FDA is now working on expanding the protocols to cover unit operations with other dosage forms and plans to have NIPP ready for full implementation by the end of 2020.
Cleaning up Opioid Addiction
Much like the pharma industry as a whole, the FDA has been a target for criticism about its lack of action towards preventing the rise of opioid addiction. But in recent years, the agency has kicked its efforts to address the ongoing crisis into high gear.
In 2018, the FDA released a string of new regulatory standards in guidance documents that address various parts of opioid manufacturing and consumption with the goal of reducing the number of new addiction patients, treating current opioid addicts and continuing to support adequate relief for pain patients.
In September, the FDA approved new rules that extend the Opioid Analgesic REMS guidelines for long-acting opioids to immediate release (IR) narcotics as well. The new rules will require manufacturers of IR opioids to provide training to healthcare professionals about safe prescribing practices for IR opioids and prompt them to consider alternatives.
Transforming Pain Med Development
In August, the FDA announced that it plans to withdrawal its analgesic guidance document, which was released in 2014. Calling the existing guidance too broad, the agency said it will be releasing a series of new guidance documents through 2019 that will push drugmakers in the direction of developing new treatments with a limited indication for a specific type of pain.
The FDA also launched an innovation challenge to spur development of medical devices and other alternative treatments to opioids.
A Final Note
This, of course, is just a glance at some of the high points from the year. Although the FDA had many regulatory accomplishments such as tackling opioid addiction and promoting generics competion and gene therapies, there is still much to be done. As 2018 comes to a close, regulators will have their hands full keeping pace with pharma innovations and promoting healthier, more affordable medicines in the year ahead.