Unbiased Analysis of Today's Healthcare Issues
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…
If you were a drug manufacturer, you naturally would want to focus your research on more common, highly prevalent diseases. If there are more patients with the disease, that means more drugs that can be sold. In recent years, however, the number of so-called “orphan drugs” has increased. In the 1970s, only 10 orphan drugs…
That is a title of a new white paper from FTI Consulting and Alexion. The key findings from the article are: HTA practices are designed to be used for medical technologies and pharmaceuticals that treat commonly-occurring diseases, and are not fit to determine the value of orphan drugs for rare disease, which face unique challenges.…
Last month, the Government Accountability Office released a report on the cost of rare disease. The report is interesting, and especially interesting is Appendix 1 which has a detailed list of studies of the cost of rare disease for about 50 rare diseases. Below are some highlights: Currently, there are no treatments or cures for…
This is the question posed by a recent article by Lopata et al. (2021). The authors surveyed 26 payers (e.g., leaders of national, regional, local, Medicaid, and Medicare plans; payer components of integrated delivery systems; and pharmacy benefit managers (PBMs) and 11 employers (e.g., large employers, employee benefit consultants, employer coalitions). Respondents were informed that…
This is the question a paper by Chambers et al. (2019) aims to answer. The authors analyzed coverage decision of 204 drugs (or 409 unique drug–indication pairs based on information from the Tufts Medical Center Specialty Drug Evidence and Coverage (SPEC) database. This database contains coverage information for 17 or the top 20 health plans…
Drugs known as “orphan” drugs are those used to treat rare disease. However, how “rare” is rare disease? In Europe, a rare disease is one affecting 5 or fewer people per 10,000 residents. In the U.S. this figure is less than one in every 1,500 residents. Although each rare disease is–by definition–rare, about 350 million…
How can policymakers incentivize innovators to invest in new treatments for rare diseases? One solution policymakers invoked was enacting the Orphan Drug Act of 1983 which provided a number of benefits–including lower tax rates–for innovators who created drugs to treat rare diseases. Was it effective? According to a paper by Miller and Lanthier (2016), the…