18th July 2019

With the increasing demands being placed on HTAs in Europe, a methodology allowing for a valid assessment of the clinical effectiveness and costs for indications targeting small patient populations and orphan conditions, such as ALL, at the time of a reimbursement decision is vital. This is particularly true when the costs of these interventions are significantly high, such as in the case of CAR-T, where costs exceed US$375 000. By extracting RWD from live hospital EHRs, we were able to demonstrate a novel methodology that allows us to move beyond the use of proxies for clinical effectiveness measures extrapolated from clinical trials to obtain an assessment of the long-term outcomes and total costs of new therapies, such as CAR-T, compared with the next-best legacy treatment option.

Photographer: Al Drago/Bloomberg © 2019 BLOOMBERG FINANCE LP Publish date: June 21, 2019 By John LaMattina, Contributor for Forbes Azar claims that HHS can reduce spending on Medicare Part B drugs with little impact on biopharmaceutical R&D investment. Duane Schulthess, Managing Director at VitalTransformation, questioned Azar’s claim on this. As he delved into specific compounds on Azar’s list of 27, he found some startling data. One striking case is with Biogen’s Multiple Sclerosis drug, Tysabri.