Unbiased Analysis of Today's Healthcare Issues
A study by Fermaglich and Miller (2023) evaluated trends in orphan drug designations and approvals after the Orphan Drug Act of 1983 was passed in the US. The authors use FDA data to evaluate between 1983 and 2022 and find that: Over the 40 years of the ODA, 6,340 orphan drug designations were granted, representing…
Why is it so hard to estimate the value of orphan drugs indicated for the treatment of rare diseases? There are a variety of reasons, but a scoping review by Grand et al. (2024) provides a nice summary of these issues. Key challenges include small sample sizes for nearly all parameters and lack of data…
That is the topic of a recent white paper from the Innovation and Value Initiative titled “Valuing Rare Disease Treatments in Healthcare: Real Experience, Real Impact“. The report notes a number of challenges in assessing treatment value in rare disease (see my white paper “Challenges in Preserving Access to Orphan Drugs Under an HTA Framework”…
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…
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.…
Check out an event this Friday, December 10, at 10am EST/7am PST on at The Hill on Rare Diseases: A Conversation on Value Assessments. A summary of the topic and the speaker list are below. One in 10 Americans suffers from a rare disease—that’s 30 million Americans who need access to treatment. According o the NIH,…
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…
How do you incentivize pharmaceutical companies and researchers to develop new drugs for rare diseases? Drug development is expensive and–by definition–rare diseases have a small market. One solution to this problem is that governments have used orphan drug policies to provide monetary incentives for innovators to research drugs for rare diseases. But which countries have…
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…