Php66 Demonstrating “Disease Modifying Therapies”? An Hta Perspective

Abstract

ysis, logistic analysis, survival analysis, and recursive partitioning decision analysis were used to estimate the relationship between the financial impact of a new drug indication and the probability of its reimbursement. The multivariable analyses controlled for other clinical and economic variables that have been shown to be correlated with the probability of reimbursement, including the cost per qualityadjusted life-year gained. RESULTS: In all analyses, financial impact was a significant predictor of the probability of reimbursement. For example, in the logistic analysis, the odds ratio of reimbursement for a drug submission with a financial impact greater than A$10 million compared with A$0 or less was 0.12 (95% confidence interval [CI]: 0.03-0.55); the odds ratio of reimbursement for a drug submission with a financial impact greater than A$0 up through A$10 million compared with A$0 or less was 0.16 (95% CI: 0.04-0.60). Similar results were obtained in the survival analysis. In the recursive partition decision analysis, the first split of the data was for submissions with a positive financial impact compared with those with a negative financial impact. CONCLUSIONS: In Australia, financial impact on the health care system is an important determinant of whether a new drug is recommended for reimbursement, even when cost-effectiveness estimates and other clinical and economic variables are controlled.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 noncoverage positions have been established prior to t...