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ASH-FDA Collaboration: Workshop on Regulatory Science in Hematology

December 11, 2022

ASH, in collaboration with the U.S. Food and Drug Administration (FDA), held its inaugural workshop on regulatory science in hematology at ASH headquarters in Washington, DC. The workshop took place in late October and was co-chaired by Dr. Grzegorz Nowakowski, vice chair of the ASH Clinical Trial Innovation Subcommittee, and Dr. Richard Pazdur, director of the FDA’s Oncology Center of Excellence. Twelve ASH members from institutions across the United States with expertise in pediatric, adult, malignant, and classical hematology were selected from among 40 applicants.

ASH News Daily connected with Dr. Yi Lin, who participated in the workshop, to get the inside story. Dr. Lin is an associate professor of medicine in the Division of Hematology at Mayo Clinic, and chair of the Cell Therapy Cross-Disciplinary Group and Enterprise Leader of Cancer Regenerative Biotherapeutics.

Please share with us the salient/ standout features of the ASH-FDA collaborative workshop on regulatory science.

Dr. Lin: The workshop opened with inspiring presentations from Co-Chairs Dr. Nowakowski and Dr. Pazdur and closed with roundtable discussion from ASH and FDA leadership. Members of the FDA shared their career paths that led them to work at FDA and were genuinely interested in hearing the clinical and patient-oriented perspectives brought forward by the participants. It helped “humanize” the FDA from being seen as the regulatory agency to individuals who want to work with clinical and translational investigators to bring effective therapies to patients safely.

We spent two days delving into clinical trials and review/ approval consideration for drugs, biologics, and devices with staff from the FDA Oncology Center of Excellence, Center for Biologics Evaluation and Research, and Center for Drug Evaluation and Research. Members of the FDA gave a concise overview on topics such as investigational new drug and device filing and early- and latephase or registration trial designs. These talks were followed by ample time for discussion and question-and- answer sessions with a panel of FDA experts in clinical issues, statistics, pharmacology/toxicology, and other relevant areas.

We worked in small group sessions with FDA staff to review the filing materials for drugs recently approved by the FDA, as well as cell therapies and devices for hematologic diseases. This included timely review of contemporary therapies that are highly relevant to hematologists, and also highlighted the need for regulatory science to evolve and adapt to address novel mechanisms of action and/or toxicities such as those seen with targeted therapies, antibody drug conjugates, and genetically engineered cell therapy.

Is there any advice you can provide members wishing to get involved with ASH initiatives in this sphere?

Dr. Lin: In Dr. Nowakowski’s closing remarks at the workshop, he stated, “This is your society … You can make it what you want it to be.” This workshop was a culmination of ASH members and committee leaders seeing a need and value to engage with the FDA to discuss the considerations and needs of regulatory medicine to address the evolving landscape of novel therapies.

I applied to participate in this workshop because the agenda resonated with my own experience as a principal investigator conducting clinical trials and in my role at Mayo Clinic Cancer Center, overseeing the clinical research portfolio as the chair of the Cell Therapy Cross-Disciplinary Group and as Enterprise Leader of Cancer Regenerative Biotherapeutics. I found the workshop to be an invaluable opportunity to discuss these timely topics with the FDA, and it was also extremely helpful to hear the perspectives of other participants in different stages of their careers, and focusing on different hematologic conditions, including classical hematology.

This is a great example of how every member has something to contribute, and the engagement of the participants really elevated the discussions and the workshop experience overall. I hope that all ASH members will actively look for opportunities to participate in workshops and committees in their areas of interest.

Are there upcoming ASH events that you are excited about?

Dr. Lin: One of the topics that came up in several sessions at the ASH-FDA workshop was inadequate inclusion of diverse patient populations in trials that are reflective of the patients affected by those conditions. This is a priority for both the FDA and ASH. ASH has the Committee on Diversity, Equity, and Inclusion, and is also looking to incorporate these considerations in their scientific committees. These are great initiatives to not only empower a more diverse community of researchers but also to make scientific advances more relevant to the patients we treat.

Finally, being a researcher in novel immunotherapy, I am excited to see a great program taking place in March 2023 — the ASH Summit on Immunotherapies for Hematologic Diseases. The summit will cover many different aspects of novel immunotherapy, including new strategies, manufacturing and clinical trials considerations, and management of toxicities.

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