How much do orphan drugs cost?

The answer is provided in a recent white paper by Pearson, Schapiro and Pearson (2022). They use IQVIA data through 2020 across 389 drugs treating 1.8 million individuals. The study finds that while 5% of drugs with an orphan indication cost more than $500,000 per year, these drugs make up only 0.08% of all patients…

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…

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…

How can physicians earn a ‘gold card’?

Prior authorization is a pain. A paper by Howell et al. (2021) found that the total cost of various utilization management schemes is $93.3 billion of which physicians devote $26.7 billion worth of time to navigate utilization management systems. Payers would argue that prior authorization helps to control costs and stop the prescribing of unnecessary…

The rise of prior authorization

While many patients and physicians feel that it is increasingly difficult to get insurance to cover new treatments, a JAMA Viewpoint (Resneck 2020) demonstrates this trend numerically.  Prior authorization requirements increased from 8% to approximately 24% of covered drugs on Medicare Part D plans between 2007 and 2019.1 In 4 therapeutic classes (including antidepressants, autoimmune disease…