Faulkner E, Spinner DS, Fernandez M. Health outcomes and economics research for cellular therapies and regenerative medicines: lessons from a health technology assessment and reimbursement analysis in the United States. Poster presented at the 2011 ISPOR 16th Annual International Meeting; May 25, 2011. [abstract] Value Health. 2011 May; 14(3):A23.

OBJECTIVES: Cellular therapies and regenerative medicines, are poised to have the same paradigm-shifting influence on healthcare as monoclonal antibodies (mABs) and personalized medicine. While these therapies hold similarities to conventional biopharmaceuticals, they also differ in material ways including attributes of both medical devices and pharmaceuticals; use of multiple procedures to prepare and deliver cells; and the potential to cure some diseases. Because of their complexity, these technologies are also anticipated to be costly and face heavy scrutiny of value. The objective of this research is to evaluate recent reimbursement policies on regenerative medicines, compare them to current biopharmaceuticals, and evaluate lessons for health economics and outcomes research (HEOR) and reimbursement planning.

METHODS: A search of US HTAs from the Centers for Medicare and Medicaid Services, the Agency for Healthcare Research and Quality (AHRQ), the BlueCross BlueShield Technology Evaluation Center and publicly available commercial payer coverage policies was conducted to identify reimbursement recommendations and supporting rationale. 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 issues associated with regenerative medicines.

RESULTS: Although a nascent treatment area, over 15 technology assessments and coverage policies on regenerative medicines were available from US HTA agencies and payers. Different from most other technologies, some non coverage positions have been established prior to the emergence of specific technologies or supporting evidence. The literature relevant to HEOR issues on regenerative medicines is particularly limited to date.

CONCLUSIONS: While payer policies on regenerative medicines reflected decision factors commonly associated with biopharmaceuticals, other factors beyond conventional biologics were identified. These factors include special evidentiary considerations for cell processing steps, influence of the evidence-base supporting multi-procedural steps on reimbursement, and evaluation of the entire procedure vs. the biopharmaceutical alone. Key considerations for HEOR are discussed.

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