News from Tuesday, September 19, 2023
Articles
Real-World Data Or Studies Of Competitor Drugs Can Serve As Confirmatory Evidence For US FDA
(9/18, Brenda Sandburg, Pink Sheet) reports “...Draft guidance describes seven types of confirmatory evidence that can be used with one adequate and well-controlled clinical study to demonstrate substantial evidence of effectiveness, including evidence from expanded access, real-world data, and studies of other drugs in the same class.” Subscription Required
FDA's Single-Trial Guidance Calls for the Good, Bad and Ugly Data to Support Effectiveness
(9/19, Annalee Armstrong, Fierce Biotech) reports “...To show confirmatory evidence, drug sponsors have plenty of options and they may not have to reinvent the wheel to find it. The FDA provided some examples...Options include efficacy evidence from a previously submitted or approved indication for a drug; preclinical data from an established animal mode; evidence from therapies in the same pharmacological class; natural history data; real-world data or evidence; and data from an expanded access program.” Full
Paxlovid and Lagevrio Benefit COVID Outpatients in Omicron Era
(9/19, Diana Swift, Medscape) reports “...‘The practice points only address [whether] treatments work compared to placebo, no treatment, or usual care,’ cautioned Linda L. Humphrey, MD, MPH, MACP, chair of the ACP's Population Health and Medical Science Committee and a professor of medicine at Oregon Health and Science University VA Portland Health Care System. The ACP continues to monitor the evidence. ‘Once enough evidence has emerged, it will be possible to compare treatments to each other. Until that time we are unable to determine if there is an advantage to using one treatment over another.’” Subscription Required
Dr Ryan Haumschild Discusses Payer, Provider Perspectives on Prior Authorization in Rare Diseases
(9/19, Pearl Steinzor, The American Journal of Managed Care) reports “...[Ryan Haumschild, PharmD, MS, MBA:] As we're evaluating rare diseases, we need to look at the value they bring. We need to look at value not just on acquisition cost, we need to look at it from total cost of care perspective or quality of life perspective, or even looking at the ICER and the quality to say, ‘Does this meet the minimum threshold?’ And if it doesn't, we need to have those open conversations with providers. Maybe there might be a sub-population that may benefit the most, but not all populations are going to benefit. And be very clear about that.” Full
Press Releases
Cristina Murray-Krezan Will Chair the PCORI Clinical Trials Advisory Panel
(9/19, University of Pittsburgh) “Cristina Murray-Krezan, associate professor of medicine and clinical and translational science at Pitt, was selected by the Patient-Centered Outcomes Research Institute (PCORI) board of governors as chair of the organization’s Clinical Trials Advisory Panel. The panel advises those conducting research through the PCORI Methodology Committee on the selection, research design, implementation, and technical issues of clinical trials for patient-centered outcomes. Murray-Krezan has served on the committee for three years and served as co-chair in 2022-23.” Full
Curta Selected as Partner to the Peterson Health Technology Institute (PHTI) to Provide Independent Health Technology Assessment Evaluations of Innovative Digital Health Technologies
(9/19, Curta Press Release) “...As a leader in the field of health technology assessment, Curta was selected to bring learnings from the from decades of experience in comparative and economic assessment to the evaluation of digital health technologies. ‘Due to Curta's unique and deep understanding of HEOR and HTA in the US, we make the perfect partner to PHTI for this important and precedent-establishing program to evaluate digital health technologies under the ICER-PHTI Assessment Framework for Digital Health Technologies.’ –Lisa Bloudek, PharmD, MS, CEO, Curta.” Full