Spinner DS, Fernandez MM. Health outcomes and economics research for regenerative medicine and cellular therapies: lessons from a multi-market health technology assessment and reimbursement review. Poster presented at the 2011 ISPOR 14th Annual European Congress; November 2011. Madrid, Spain. [abstract] Value Health. 2011 Nov; 14(7):A337-8.

OBJECTIVES: Regenerative medicines, which include cellular and gene therapies, offer to shift the focus of healthcare from one of palliative care to curative treatment. Because these technologies are novel, more complex than standard biopharmaceuticals, and often costly, they are anticipated to face heavy scrutiny for market access and adoption. The objective of this analysis was to evaluate published HTAs and reimbursement policies on regenerative medicines for select global markets, compare them to existing biopharmaceuticals, and evaluate lessons for HEOR and market access planning.

METHODS: A search of HTAs and reimbursement policies from Australia, Canada, France, Germany, Sweden, the UK (UK) and the United States (US) was conducted to identify reimbursement recommendations and key HEOR considerations for this new field. A review of the literature, including . the Cochrane Library and PubMed was also conducted using relevant MeSH terms and text words to identify additional reimbursement and HEOR issues associated with regenerative medicines.

RESULTS: Although considered a nascent field, over 40 HTAs and coverage policies on cellular therapies and regenerative medicines were available from the key Australian, European and North American HTA agencies and payers considered in the assessment. Many of HTAs and coverage policies identified gaps in effectiveness data, particularly the lack of compelling comparative trials and long-term outcomes, and uncertainty surrounding cost-effectiveness.

CONCLUSIONS: While HTAs and payer policies on regenerative medicines reflected decision factors commonly associated with biopharmaceuticals, other key market access factors beyond clinical- and cost-effectiveness were identified that are specific to this field. These factors include involvement of multiple reimbursable technologies in cell extraction, processing and administration; cell handling steps that may engage multiple healthcare budgets; and requirements to characterize the value of the entire procedure versus the regenerative medicine alone. Key considerations for HEOR and market access planning are considered.

Share on: