The EU’s “hospital exemption” rule permits the manufacture and use of CAR-T therapy in academic centers, with member state regulatory oversight. This has been proposed as an alternative to the commercial model for CAR-T development, with global clinical trials and centralized regulatory approval, manufacturing and pharmacovigilance.

HTA methods for evaluating oncology survival benefit were introduced when most drug treatments were cytotoxic regimens for advanced disease, delivering moderate survival benefit. Immunotherapies now offer the potential for transformative benefit in many indications. Adjuvant drug therapies with curative intent in earlier disease stages are also expanding. The increased diversity in survival benefit delivered by new technologies adds complexity and presents new challenges for HTA evaluation of oncology therapies. 

The last 5 years has seen a dramatic evolution in treatments for multiple myeloma. Previously, bortezemib, lenalidomide and pomalidamide, in combination with steroids and used sequentially, was the mainstay of treatment for transplant ineligible patients. With the introduction of carfilzomib, panobinostat, elotuzumab, daratumumab and ixazomib as add-ons to existing therapies, there is now a much large number of possible combinations and sequences.