FDA Cancer Office Taps Syapse for Real-World Evidence Development
(8/14, Conor Hale, Fierce Biotech) reports “...‘Real-world evidence from well-designed studies meeting appropriate data quality standards can help to inform decision-making and provide information regarding the impact of new therapies in real-world patient populations, particularly those not represented in clinical trials,’ [FDA associate director for oncology regulatory science and informatics Sean Khozin] added. ‘This is especially critical in precision medicine, where understanding all of the factors that may drive safety and response is both imperative and difficult to capture at scale using traditional clinical trials.’” Full
ICER Reviews Duchenne: Exondys 51 Lacks Net Health Benefit; Emflaza Fairs Better
(8/13, Pink Sheet) reports “...[ICER President Steve Pearson] was forced to walk a difficult line during the meeting – asking tough questions on long-term value to elicit a well-rounded discussion while expressing appropriate sympathy to the parents at the table. ‘Value is a tricky word, because to a patient or a family, value is intrinsic in the benefits – or even the hope – that they can get from a new treatment,’ he said. ‘Votes here are not intended to say that patients aren't important. This is a hard-edged look,’ he acknowledged, noting that even an ‘extraordinarily successful treatment can still be overpriced.’” Paid Subscription Required
Video: Dr Jeroen Jansen Discusses Value-Assessment Questions Posed by Curative Treatments
(8/14, The American Journal of Managed Care) “With the increasing prevalence of more expensive, potentially curative therapies, there comes the question of can we still afford expensive interventions that are that valuable, explained Jeroen Jansen, PhD, lead scientific advisor, Open-Source Value Project, Innovation and Value Initiative.” View Video
COTA Collaboration: Helping FDA Figure Out What's Possible and What's Not in Embrace of Real-World Evidence
(8/13, Mary Caffrey, Evidence-Based Oncology) reports “...In an interview with Evidence-Based Oncology, [COTA chief medical officer Andrew Norden, MD, MPH, MBA] emphasized that COTA is not looking to eliminate RCTs. But by unlocking the secrets of the data sets in EHRs, real-world data can ‘extend’ the findings of the RCT, as Norden puts it, and offer researchers, clinicians, and the FDA insight into how drugs work in populations that don't find their way into clinical trials.” Full
A Patient-Centered Model of Congenital Thrombotic Thrombocytopenic Purpura Symptoms and Effects
(8/14, Sonali Gattani, Hematology Advisor) reports “In order to get an in-depth understanding of patient experience, researchers developed a conceptual model of congenital thrombocytopenic purpura (TTP) that described its symptoms, effects, and disease burden. They reported their findings in The Patient — Patient-Centered Outcomes Research.” Full
Patient Experience with Congenital (Hereditary) Thrombotic Thrombocytopenic Purpura: A Conceptual Framework of Symptoms and Impacts
Abiola O. Oladapo, et al.
July 30, 2019, The Patient - Patient-Centered Outcomes Research
Barriers and Facilitators to Conduct High-Quality, Large-Scale Safety and Comparative Effectiveness Research: The Biologics and Biosimilars Collective Intelligence Consortium Experience
Lockhart CM, et al.
August 11, 2019, Pharmacoepidemiology and Drug Safety