Policy brief: Point-of-care manufacture of advanced therapies: readiness measures

This policy brief highlights readiness measures that can be taken by three groups of stakeholders to implement point of care manufacture at a larger scale.

Advanced Therapy Medicinal Products (ATMPs), such as cell and gene therapies, are promising for treating rare diseases, as well as some more prevalent conditions such as severe burns or diabetes. In some cases, it is necessary to adopt models where ATMPs are produced in clinical settings and immediately delivered to patients (point-of-care manufacture). This could enlarge the range of innovative therapies made available in the NHS, but also brings about considerable challenges. This policy brief explores some of those challenges and sets out a range of readiness measures that different stakeholders can take to prepare - including healthcare system and hospital staff (including NHS Trusts, clinical centres, commissioning groups), companies, and regulatory agencies.

Key recommendations

To implement point-of-care manufacture at larger scale, some readiness measures would be beneficial for the following groups:

Hospitals would benefit from training staff (including professionals who may act as assistants to Qualified Persons), adjusting infrastructure (including storage space and stem cell laboratories), reviewing the standards generally used by the industry, and updating governance frameworks.
Companies would benefit from enhancing transportation systems for biological materials and reagents, developing data management systems for use in hospitals, and engaging in dialogue with regulators to establish processes for therapy release.
Regulators would benefit from strengthening horizon scanning activities and seeking proportionate regulatory assessments for manufacturing systems that will be highly flexible.

Download the Point-of-care manufacture of advanced therapies: readiness measures for hospitals, companies, and regulatory agencies brief [PDF]