The Colorado Prescription Drug Affordability Board (PDAB) selected five prescription drugs for its first affordability reviews at its Aug. 4th meeting.
The drugs and their primary treatments that will enter affordability reviews and be considered for upper payment limits (UPL) are as follows:
- Enbrel (rheumatoid and psoriatic arthritis)
- Genvoya (human immunodeficiency virus)
- Cosentyx (plaque psoriasis and psoriatic arthritis)
- Stelara (Crohn’s disease)
- Trikafta (cystic fibrosis)
The selections were made from the Prescription Drug Affordability Advisory Council’s (PDAAC) list of 50 prioritized drugs last month, which included selections based on categories of highest average paid price per person per year, highest patient count, and highest wholesale acquisition costs (WAC).
During the public comment period at that meeting, multiple members from the Colorado cystic fibrosis (CF) and rare disease community urged the board to remove Trikafta from review consideration over concerns that upper payment limits would impact market access to the drug in the state.
Trikafta was approved in 2019 to treat CF and is the only effective medication on the market, with no biosimilars or therapeutic equivalents available.
Patrick Sosnay, senior medical director of Vertex Pharmaceuticals, the manufacturer of Trikafta, urged the board during public comment not to apply UPL to “truly transformative rare disease medication” such as Trikafta where patients in the state already have broad access.
“[Trikafta has] reduced the risk of hospitalization by 74%. It’s reduced the risk of death by 72% compared to immediately before approval. Trikafta is projected to increase the median survival for this disease by 37 years when it’s initiated in the youngest eligible ages.”
— Sosnay
Numerous beneficiaries of the drug also advocated on behalf of themselves, airing concerns price limits could impact access to this and future cystic fibrosis treatments and criticizing PDAAC’s selection of Trikafta even after making the recommendation of delaying consideration of drugs for rare diseases.
PDAB chair Dr. Gail Mizner assured those in attendance that the board would take the public’s interests into consideration in its selection and review, saying the reviews were a process of fostering a better understanding of the cost mechanisms.
“I want to reassure the public that we are not political appointees. Our [members] were appointed for our expertise—two highly experienced clinicians, two highly experienced pharmacists—all four of us deal on a daily basis with patients with rare diseases [and] high cost of medication. All of those concerns are ones that are extremely important to us. We would never want to put access at risk …
I also just want to emphasize that if for some reason a rare drug should get on our list of medications to be reviewed for affordability, that in no way means that that drug would be in the second process—that we would decide to put an upper payment limit on it. So I just want to try to calm the public and our other speakers that the board is being very deliberate and very careful about our decisions. And we’re fully aware of the concerns that you’ve outlined and very much appreciate you having outlined them for us.”
— Mizner
During the board’s deliberations, it maintained the selection criteria and PDAAC recommendations in determining the top five drugs to approve for reviews, which will include Trikafta. Members agreed its exorbitant pricing and patient count warranted a deeper analysis of the data.
PDAB staff has initiated its stakeholder engagement and published a selection guide for the submission of larger data sets for the reviews this week, which will be staggered to review three drugs initially and the remaining two in the subsequent stage.
PDAB’s next scheduled meeting on Sep. 15th will examine the data and consider factors like rebates, current WACs, and the change in WAC in determining which selections will be assigned UPLs.