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Making Real World Data Real – New RWE Methodologies for HTAs
With the increasing use of accelerated regulatory tools like the Food and Drug Administration’s breakthrough designation, there are mounting challenges for European health technology assessors (HTAs) to make an accurate assessment of the long- term value and performance of many new therapies. Data presented in evidence is often extrapolated from outcomes six months or less, and what’s good enough for regulatory approval often doesn’t meet the needs for comparative effectiveness, value, and outcomes measures for HTAs. Vital Transformation’s recent BMJ publication demonstrates a novel, practical approach to harnessing electronic health records and RWE to help solve this problem. However, access to robust deidentified patient records in Europe is key.
Are CAR-T therapies living up to their hype? A study using real-world data in two cohorts to determine how well they are actually working in practice compared with bone marrow transplants GalleryAre CAR-T therapies living up to their hype? A study using real-world data in two cohorts to determine how well they are actually working in practice compared with bone marrow transplants
Are CAR-T therapies living up to their hype? A study using real-world data in two cohorts to determine how well they are actually working in practice compared with bone marrow transplants
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.
Azar’s Plan To Tie U.S. Drug Prices To Foreign Ones Will Impact Investments In R&D
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.
The International Pricing Index: What will be the Impact on Patients, Outcomes, and Innovation?
According to research from the US Health and Human Services, US tax payers pay 1.8 times the international price for medicines and are ‘subsidizing’ global healthcare. In order to combat this imbalance, the Trump administration is proposing the “International Pricing Index” (IPI) payment model to reduce reimbursement levels for the Centers for Medicare & Medicaid Services (CMS). Following Vital Transformation's research on investment success factors for biotech companies released at the European Health Forum Gastein, Vital Transformation presented original research modelling the potential impact both short and long term for patients, payers, and industry for such proposals, and show how the IPI can impact access and innovation broadly. As well, the panellists placed the IPI into the context of other models currently being investigated internationally such as value based payments, managed entry agreements, and adaptive pathways.