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…

FDA guidance on patient-focused drug development

FDA wants drugs to be more targeted to factors that impact patient’s lives in ways that they care about. To achieve this goal, last month FDA released a third guidance document on patient focused drug development generally applicable to a variety of clinical outcome assessments (COAs), including patient-reported outcome (PRO), observer-reported outcome (ObsRO), clinician-reported outcome…

COVID-19 news and stats

Some COVID-19 news updates. 66.5% of the world population has received at least one dose of a COVID-19 vaccine. 12.09 billion doses have been administered globally, 5.74 million COVID-19 vaccines are administered each day. Only 17.6% of people in low-income countries have received at least one dose. There have been approximately 550 million cases of COVID-19…

Effect of pharmaceutical innovation on longevity

Using data from 2006-2018 across 26 high-income countries, Lichtenberg et al. (2022) measures the relationship between pharmaceutical innovation and longevity. Innovation is measured by “mean vintage…of the drugs used to treat each disease in each country” where vintage is defined as the year of worldwide launch. For the U.S., the authors found that: …the diseases…