OBJECTIVES: There are many definitions of regenerative medicine offered by regulatory agencies, health technology assessment (HTA) authorities, and professional societies, which may complicate efforts by those conducting health economics and outcomes research (HEOR). The objective of this study was to propose a classification system for regenerative medicine that is standardized based on characteristics potentially more likely to be considered in HEOR.
METHODS: For the purposes of this research, regenerative medicine was assumed to include cell and gene therapies and tissue-engineered products. A targeted review of documentation from government agencies, HTA authorities, and professional societies, as well as published literature was conducted, and a classification system was developed to support HEOR.
RESULTS: The classification system is based on therapeutic strategy and, within that, technology type. Examples of therapeutic strategies include treatment for an inherited single-gene disorder, enhancement of the immune system, and tissue regeneration. As an example of technology type and subtypes, gene therapies can be subclassified into gene addition, correction, silencing, or elimination. To demonstrate the classification system, a subset of regenerative medicines was classified. Treatments considered included, for example, voretigene neparvovec (Luxturna), classified as having a therapeutic strategy of treating an inherited single-gene disorder (blindness), with the technology type and subtype being gene therapy—addition (delivered via a viral vector). Another example is tisagenlecleucel (Kymriah), classified as having a therapeutic strategy of enhancing the immune system (to fight cancer), with the technology type and subtype being gene therapy—modification (of T-cells). A final example includes Carticel, classified as having a therapeutic strategy of tissue regeneration (to regenerate cartilage), with the technology type and subtype being cell therapy—minimally manipulated autologous cartilage cells.
CONCLUSIONS: Classification of the therapy strategy, as well as the technology, should be taken into consideration to distinguish cell and gene therapies and their HEOR implications.
