OBJECTIVES: The use of real world evidence (RWE) in regulatory decision-making may play an important role for personalised medicine such as advanced therapy medicinal products (ATMPs), particularly where ‘gold standard’ randomised control trials may be deemed unethical or impractical, or to provide context within standard care. This is an overview of the RWE used in HTA decision making for ATMPs within England.

METHODS: European Public Assessment Reports (EPARs) published by the European Medicines Agency were reviewed to identify all authorised ATMPs. A separate search was then carried out to identify which of these were assessed by The National Institute for Health and Care Excellence (NICE). Committee discussions were reviewed to ascertain the evidence need or potential solutions offered by RWE, as well as the benefits and challenges of using RWE in NICE appraisals.

RESULTS: Nine ATMPs were identified as undergoing technology appraisal by NICE. All but two referenced the consideration of RWE in the committee discussion. ATMPs commonly utilised open-label single arm studies, with supportive comparator data via RWE. Elusive long-term efficacy or safety data and small patient numbers were referenced as examples of a common evidence need for RWE. Comparator group evaluations were deemed to be challenging and were confounded by differences in reported outcomes and baseline prognostic factors, such as heterogenous populations and prior treatment. Confidentiality issues were also shown to restrict the availability of RWE sources. Despite such limitations, the approach taken by the submitting company was often highlighted as the best available evidence for such complex products and therefore, appropriate for decision-making.

CONCLUSIONS: Utilising RWE within ATMP decision-making remains a challenge. RWE can provide additional safety and efficacy considerations for ATMPs, adding therapeutic value and supporting positive reimbursement decisions.