Mitigating deficiencies in evidence during regulatory assessments of advanced therapies: a comparative study with other biologicals
Open accessAbstract
Advanced therapy medicinal products (ATMPs) comprising cell therapy, gene therapy, and tissue-engineered products, offer a multitude of novel therapeutic approaches to a wide range of severe and debilitating diseases. To date, several advanced therapies have received marketing authorization for a variety of indications. However, some products showed disappointing market performance, leading to their withdrawal. The available evidence for quality, safety, and efficacy at product launch can play a crucial rule in their market success. To evaluate the sufficiency of evidence in submissions of advanced therapies for marketing authorization and to benchmark them against more established biological products, we conducted a matched comparison of the regulatory submissions between ATMPs and other biologicals. We applied a quantitative assessment of the regulatory objections and divergence from the expected data requirements as indicators of sufficiency of evidence and regulatory flexibilty, respectively. Our results demonstrated that product manufacturing was challenging regardless of the product type. Advanced therapies displayed critical deficiencies in the submitted clinical data. The submitted non-clinical data packages benefited the most from regulatory flexibility. Additionally, ATMP developers need to comply with more commitments in the post-approval phase, which might add pressure on market performance. Mitigating such observed deficiencies in future product development, may leverage their potential for market success.
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Decentralized manufacturing in multiple small facilities is appealing, but new models of production scaling that rely on this approach remain a challenge to regulate.
“Decentralized manufacturing in multiple small facilities is appealing, but new models of production scaling that rely on this approach remain a challenge to regulate (137-139).”
From Scalable Manufacturing of CAR T Cells for Cancer Immunotherapy (2021) · cites this paper as [139]
The Discussion describes manufacturing and logistics constraints and regulatory objections for advanced therapies, but does not address decentralized production across multiple small facilities or the appeal and regulatory difficulty of scaling that model. Searches for decentralized manufacturing, scale-out, and multiple facilities found no passage making the attributed point; page numbers were unavailable in our copy. (LLM, conf=0.72)
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- Authors
- Elsallab, M., Bravery, C. A., Kurtz, A., & Abou-El-Enein, M.
- Affiliations
- Berlin-Brandenburger Centrum für Regenerative Therapien (DE)
- ORCID
- Elsallab, Magdi
- Venue
- Molecular Therapy - Methods & Clinical Development
- Publisher
- Elsevier BV
- Published
- 2020-09
- Volume
- 18
- Pages
- 269-279
- ISSN
- 2329-0501 (ISSN-L)
- PMC
- PMC7327881
- OpenAlex
- W3033248840
- Cited by
- 59
- References
- 40
- Citations by year
- 2026: 11 · 2025: 12 · 2024: 4 · 2023: 3 · 2022: 11 · 2021: 16 · 2020: 2
- FWCI
- 4.81
- Type
- journal article
- Language
- en
- Open access
- gold
- doi.org
- Licence
- Locations
- Molecular Therapy — Methods & Clinical Development (publishedVersion, cc-by-nc-nd)
- PubMed Central (publishedVersion, cc-by-nc-nd)
- PubMed (publishedVersion)
- DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion, cc-by-sa)
- Funders
- Wellcome Trust
- Arab-German Young Academy of Sciences and Humanities
- Bundesministerium für Bildung und Forschung
- Northeastern University
- McGill University
- Topics
- Health Sciences › Medicine › Physiology › Biomedical Ethics and Regulation
- Social Sciences › Economics, Econometrics and Finance › Economics and Econometrics › Pharmaceutical Economics and Policy
- Health Sciences › Health Professions › Health Information Management › Healthcare Regulation
- Keywords
- advanced therapy medicinal products, regulatory evaluation, drug approval, cell therapy, gene therapy, tissue-engineered products, regulatory flexibility, product withdrawal
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