Traditional knowledge believes that orphan drugs treat rare (according to definition) and more serious diseases. Because they treat diseases with important unmatched needs, their health benefits per person are large. But are they really? Does traditional knowledge align with evidence?
To answer the question of himself and colleagues in FTI consultation, including co-author Mary Steel-Edgoganan, a study was done to answer this question. Our white paper “Determining the value of orphans and IRA only orphan exclusion” applied a 4 -stage approach:
- Step 1: Identify FDA approved drugsAnalysis sees FDA approved between 2011-2024
- Step 2: Identify CEA StudiesWe examined the years of life obtained in identified medicines.
- Step 3: Remove dataWe measured the benefits of life year from the most relevant CEA study, and if we have been achieved based on the basic survival and estimated danger rate, then brought to affect.
- Step 4: Statistical analysisWe compared to the song benefits for orphan drugs and non-miscreants. We also examined the possible health benefits at risk by not giving complete exemption to orphan drugs with Medicare Drug Price Dialogue.
Based on this approach, we found that:
- Orphan drugs have more effect on the patient’s survival 4 times bigger Compared to existence
Benefits of non-non-active drugs. - Orphan drugs with many rare disease signals provide more and more 3 times
Survival profit As non-unknown drugs. - Because many orphans treat many rare and non-dough diseases, by removing them
From ‘the only orphan’ exclusion, 58% orphans Medicare will be eligible for drug price
Conversation. - If the sole orphan exclusion has stopped all orphans with several indications
Developed, more 16.7 million life year Will be lost in the next 15 years.
You can read full white paper Here,
