Officials from Health Canada and the pharmaceutical industry addressed plans to develop harmonized guidelines on cell and gene therapies and on the design of pharmacoepidemiological studies that use real-world data (RWD) to assess new medicines at the International Council for Harmonisation (ICH) regional meeting sponsored by the US Food and Drug Administration (FDA) and Health Canada.
Kathleen Francissen, global head of PT cell and gene therapies at Genentech, and Melissa Kampman, manager of the marketed health products directorate for Health Canda, provided an update on these plans as well as timetables for released harmonized documents at the meeting, which was held virtually on 22 February.
Francissen, who is the rapporteur for the ICH Cell and Gene Therapy Discussion Group (CGT DG), discussed the need for harmonized guidelines in this space. There have been “tremendous advances” in the development of cell and gene therapies over the past several years, with more clinical trials being conducted globally, she said. There are 1,400 advanced therapies in clinical trials around the world. Currently, 5,800 patients have been dosed with AAV-based gene therapies while 38,000 patients have been dosed with CAR T-cell treatments.
Despite the growth of this sector, current ICH guidelines for traditional biologics do not fully address the unique characteristics of ATMPs, as the non-clinical, clinical, and manufacturing development of ATMPs are uniquely complex. “It is important to converge on a science-based regulatory framework across all the regions” for these products, Francissen said.
The Biotechnology Innovation Organization (BIO) proposed the formation of the discussion group last year, and the remit of the group was endorsed by the ICH Management Committee in May 2023. A kickoff meeting was held October 2023, and the work plan was endorsed in December 2023.
Francissen said that “this is a technical discussion forum rather than an expert working group, so our remit and deliverables are a bit different than the expert working group. Rather than revising an ICH guideline, we discuss the scientific and technical issues related to ATMP development, and our ultimate deliverable is to have a strategic roadmap rather than a revised guidance.”
Over the next year, the discussion group plans to release a roadmap of potential areas of harmonization and to recommend revisions to existing guidelines or whether to develop new guidelines specific to CGT products. The roadmap is anticipated to be released in June 2025.
Need for harmonized RWD guideline
Kampman provided an update on the work being done under ICH M14, which aims to provide a multidisciplinary guideline to harmonize post-approval non-interventional studies on drugs, vaccines and other biologics using real word data.
She stated that “epidemiology has long been a mainstay of postmarketing pharmacovigilance and safety evaluation. We as regulators have long recognized their utility and have written a number of recommendations on these studies for regulatory purposes. We also recognize that the availability of real-world data also continues to increase steadily worldwide. That being said, there is no internationally harmonized guidance, and this results in issues of inefficiencies for both sponsors and regulations.”
Kampman said that ICH recognized the benefits to having a harmonized guideline in this space to reduce review inefficiencies and issued a reflections paper to develop such a guideline in June 2019. The ICH M14 reflection paper also proposed the establishment of a pharmacoepidemiology discussion group (PEpiDG) to conduct a gap analysis to evaluate the guidelines of Health Canada, FDA, and other regulators to identify gaps in the regulations and identify potential areas for harmonization.
The group issued a draft guideline for internal comment in September 2023, and received feedback from 615 stakeholders. A revised document to incorporate these comments was issued internally in January 2024. Plans are to have Step 2a document released by April 2024, and to adopt a Step 4 guidance in May 2025.
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