The US Food and Drug Administration (FDA) has published new draft guidance around the development and application process for new cell or gene therapies (CGTs).
The draft is an extension of previous guidance published in January 2020 on the chemistry, manufacturing and control information for human gene therapy investigational new drug (IND) applications.
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The 44-page long document answers frequently asked questions (FAQs) on IND quality and submission, meeting types, product development considerations and conducting both non-clinical and human studies.
The guidance covers a variety questions, including on FDA recommendations around pre-biologics license application (BLA) meetings and on safety considerations for short- and long-term monitoring in trials of investigational CGT products.
On pre-BLA meetings, it said that “in an effort to mitigate review delays, the Agency strongly recommends sponsors schedule a pre-BLA meeting with their review team in OTP to help ensure all information, data and analyses necessary to support review are included in the BLA submission.” It noted that the “exchange of information about a proposed marketing application” reduces delays often associated with the initial review of a marketing application.
On safety, the document emphasised the need for the short-term close monitoring of subjects, outlining: “During and immediately following product administration, there should be intensive safety monitoring with frequent monitoring of vital signs, physical examinations, laboratory studies, radiologic evaluations and other relevant studies as warranted.”
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By GlobalDataIn the longer term, it says the FDA “advises sponsors to observe subjects for delayed adverse events for as long as 15 years following exposure to the investigational product”.
The document also covers recommendations regarding trial design and the selection of endpoints, including for surrogate endpoints. It explains what sponsors should consider when using a surrogate endpoint and considers the appropriate use of biomarkers as such.
The FAQ draft represents part of the FDA’s response to the sixth reauthorisation of the Prescription Drug User Fee Act (PDUFA VII), which resources the FDA. Within the guidance, the FDA restated its “commitment to increase efficiency in the development of CGT products”, which it said continue to grow at a fast rate in the US, with a range of products already on the market and more in development.
Recent analysis of drugs tracked by GlobalData identified that over the past decade, a total of 14 CGTs were approved across various therapy areas in the US. Of these, over 70% were approved within the last five years.
GlobalData noted in February: “The opportunity for the approval of more CGTs remains high,” pointing out that 694 CGTs were in development across various therapy areas in the US at the time.
GlobalData is the parent company of Clinical Trials Arena.
