Unbiased Analysis of Today's Healthcare Issues
The Times reports that the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) is revising it’s approach to how it approves new medicines that treat rare diseases. How? Principally by allowing for single arm trials with real-world ‘digital twin’ controls as well as expediting the approval process. The new plan, which is being launched on…
Check out my new publication with co-authors Suhail Thahir, Alexa Klimchak, Ivana Audhya, Lauren Sedita, and John Romley titled “Quantifying the Insurance Value for Rare Diseases: Duchenne Muscular Dystrophy” in AJMC. The abstract is below. Objectives: To quantify the magnitude of an ISPOR novel value element, insurance value, as applied to new treatments for a rare,…
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…
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,…