Measuring the novelty of new drugs

All of us want new, improved medicines. Drugs that provide incremental benefits (often called “me too” drugs) are good, but truly revolutionary therapies are even better. However, how does one separate how “novel” a treatment is? Is there a way to quantify this? In fact there is. One way to do this is based on…

CMS = HTA?

The U.S. is one of the the few developed nations without a government-run health technology assessment (HTA) body. Or are they? A recent perspective in the New England Journal of Medicine by Peter Neumann and Sean Tunis argues that the Centers for Medicare and Medicaid Services (CMS) already is serving as a de facto HTA…

CMS and outcomes-based pricing for cell and gene therapy

New cell and gene therapies offer the promise of revolutionizing care for patients with genetic diseases. Many of these diseases impact kids and about a third of children in the US are insured by Medicaid or the Children’s Health Insurance Program (CHIP). The government faces a challenge: cell and gene therapies are potential breakthrough therapies…

ChatGPT summary of Congressional Testimony on “Innovation and Patient Access”

On May 10, the House Ways and Means Committee held a hearing on “Examining Policies that Inhibit Innovation and Patient Access“. You can view the testimony here. I summarized submitted Congressional Testimony with the help of ChatGPT below. Actual testimony submitted is also referenced below as well. Mr. Tony Gonzales, National Early-Stage Advisor, Alzheimer’s Association.…

Health insurance: Incentivizing overspending or fueling innovation?

Based on a study by Frankovic and Kuhn (2022), the answer is both. However, the value of increased innovation–as measured through longevity gains–more than offset inefficiencies from overspending. Specifically, the authors use an overlapping generations model where individuals can purchase health insurance and medical progress depends on health care sector return on investment. The authors…

Challenge in HTA for gene therapies

Last month, the Office of Health Economics published a report titled “Health Technology Assessment of Gene Therapies: Are Our Methods Fit for Purpose?” I summarize some of the key challenges and solutions below. Challenge #1: Initial assessment of clinical effectiveness. Since gene therapies often target rare disease, the sample size from clinical trials is often…