Did you know:
- More people, collectively, suffer from a rare disease than HIV + AIDs combined
- Accelerated Approval has only been used once for a rare disease treatment (since 1992)
- Accelerated Approval has been used dozens of times for HIV and AIDs
The typical drug approval process involves requires that companies prove a clinical benefit before it can be marketed. This may take many, many years.
This is bad because:
- It prevents many patients from gaining access to potentially beneficial treatments
- It causes the development cost of new treatments to be high… so high that many extremely rare diseases just aren’t economically viable for treatment
Accelerated Approval involves granting tentative approval for a promising drug based on a biomarker versus clinical outcome.
Example:
To get initial approval to market the drug – in the case of Cancer, the drug would simply need to show that it shrinks a tumor rather than extending the life of the patient. Post-market surveillance is used to determine clinical efficacy.
Of course, this approach has many applications outside of cancer. In the case of many rare genetic diseases, the underlying mechanism can be isolated to a single genetic defect and biological pathway. I am told that this makes the identification of biomarkers much easier.
In contrast to the classical approach, accelerated approval has the following advantages:
- It gets drugs to as many patients as quickly as possible
- It is grounded in science, assuming appropriate biomarkers are chosen
- It reduces drug development cost by over 60%
The Ultra-Orphan Lifesaving Treatment Act of 2012 will help make accelerated approval available to rare disease. Only a small percentage of rare diseases have treatment and this could be a game changer.
You can help.
Ask your patient group to sign on to the Congressional support letter
Talk to Julia Jenkins at patientadvocate@kakkis.org – Remember, you don’t have to be the a big foundation to sign on. We need as many patient groups as possible to add their name to the letter.
Want to learn more? Watch the video PSAs:
