OBJECTIVES: To review current processes for reimbursement of ATMPs in the UK and consider mechanisms to improve patient access to these therapies.

METHODS: Structured desk research was conducted to assess the health technology assessment (HTA) status and reimbursement of products classified by the European Medicines Agency (EMA) as ATMPs in the UK. A review of decision drivers and consideration of potential policy solutions to support wider access was undertaken.

RESULTS: Appraisal processes in the UK are no different for ATMPs than non-ATMPs. The only determinant of which reimbursement process applies to an ATMP is the size of the eligible patient population. Eight ATMPs have received marketing authorisation in Europe, seven launched commercially, but four were subsequently withdrawn from the market due to low uptake. Imlygic was recommended for restricted use by NICE with a patient access scheme, ChrondoCelect was not recommended by NHS England and Strimvelis is scheduled for a NICE Highly Specialised Technology appraisal. Provenge was not recommended by NICE and the company has subsequently withdrawn marketing authorisation. Scottish Medicines Consortium has not appraised any ATMPs.

CONCLUSIONS: Despite UK Government rhetoric in support of gene therapies and innovation, there has been limited access to ATMPs on the NHS. Although the EMA has introduced measures to support early regulatory approval of ATMPs, premature data combined with high costs appear to deter companies from applying to UK HTA bodies. Only one ATMP has received reimbursement. Initiatives to support the life sciences industry and gene therapies research need to be backed up with flexible HTA assessments. Early negotiations between companies and NHS bodies should be used to identify managed access agreements, limiting the NHS financial risk whilst allowing patient access to innovative products. Where products deliver a cure following a single use or single course of treatment, longer-term payment plans may help to manage NHS budgets.