Oh boy, that's a complex question. I will let Dr. Levine chime in if he wants to supplement my answer.
We hear a lot from advocates for patients for rare diseases' pharmacoeconomic value. This is a very esoteric topic, but it shouldn't have application in this space because there are unique features of these products that make them not amenable to that type of analysis. When I meet with my counterparts in other countries around the world, and with health technology authorities in particular who do this type of analysis on behalf of payers and governments worldwide, they take issue with that argument.
I will give you an example of the point I'm trying to make. The U.K. sets pharmacoeconomic value thresholds. If a drug costs more than a certain amount for one quality-adjusted life year, the U.K. health authority won't reimburse it unless the price comes down. There are varying thresholds that apply to different drugs. Rare disease drugs have a higher threshold than regular drugs.
At some point, people became very concerned that a lot of the new oncology drugs, for rare diseases in particular, weren't meeting these thresholds—even the more elevated thresholds. The U.K. decided to create a special fund just for cancer drugs and forgo the pharmacoeconomic value assessment for these drugs.
I think the fund started off with $1 billion. It was quickly exhausted. Authorities soon realized that health outcomes for cancer patients hadn't improved an iota over that period. In fact, they probably suffered a lot more side effects as a result of allowing medications on the market that didn't have good clinical data and weren't able to establish significant value in terms of their cost effectiveness.
I'm wary of putting these medications in their own little bucket. I'm very sympathetic to people who are concerned about the impact of these changes on rare disease medications, but that's one of the reasons we introduced that $12-million annual revenue insulatory mechanism that I alluded to earlier.
Dr. Levine has more experience day to day as a clinician dealing with these types of medications. Do you have something you want to add?