This collection examines the development, manufacture and regulation of cell and gene therapies. Topics include hospital exemptions, clinical manufacturing, human-origin starting materials, orphan exclusivity, regulatory sandboxes and health technology assessment.
These articles provide general information and are not legal advice.
The EU Pharma Package turns the hospital exemption for cell and gene therapies from a national carve-out into an approval notified to the EMA, with annual data on use, safety and efficacy, while Switzerland drafts a narrower exemption of its own.
Cell and gene therapies developed under hospital exemption face uncertain boundaries. Scale, geography, and commercial intent determine where the authorization line falls.
For a cell or gene therapy made under good manufacturing practice (GMP), the EU authorizes the finished product, while Switzerland may authorize the manufacturing process instead. They diverge on the hospital exemption, cross-border cell tracing, process changes and contract manufacturing.
From 7 August 2027 the EU Regulation on substances of human origin replaces the Blood and Tissues and Cells Directives, extending EU oversight of donated starting materials up to the manufacturer's door and requiring US cell and gene therapy sponsors to repaper supply agreements.
EU orphan exclusivity will run on one clock per holder and active substance, and new orphan indications can extend it by a year at most twice. The two-year pediatric orphan reward will end, and earnouts keyed to orphan exclusivity will track a changed right.
A statutory sandbox in the EU Pharma Package lets the Commission adapt authorization requirements for products that cannot meet the standard rules, such as cell and gene therapies and AI-enabled medicines. FDA expedited programs speed a fixed pathway; the sandbox changes the requirements themselves.
The EU Health Technology Assessment Regulation has required a Joint Clinical Assessment for new oncology medicines and cell and gene therapies since 12 January 2025; it harmonizes the clinical dossier, not national pricing, and the outcome turns on comparators chosen at trial design.
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