Weekly Policy Blog: PDAB Deems Trikafta “Not Unaffordable” in First Affordability Decision

On Friday, December 8, the Colorado Prescription Drug Affordability Board (PDAB or Board) completed its first affordability review and unanimously voted that the selected drug, a cystic fibrosis drug called Trikafta, is “not unaffordable for Colorado consumers.” Trikafta is therefore not eligible for the imposition of an “upper payment limit” by the Board. 

The PDAB also reviewed preliminary data presented by staff on the next two drugs under review, Genvoya (for HIV) and Enbrel (for autoimmune diseases), and plans to vote on their affordability, and then potentially on whether to set an upper payment limit, in February.  

The PDAB’s December 8 Meeting 

In the first two hours of the December 8 PDAB meeting, PDAB staff reviewed the draft Affordability Review Report for Trikafta with the Board and allowed Board members to ask questions and provide thoughts and feedback. In addition to PDAB staff, Benjamin Rome, M.D., from Harvard’s Program On Regulation, Therapeutics and Law (PORTAL) was also on hand to help answer Board members’ questions. Following about ten minutes of discussion by the Board about their overall thoughts and impressions regarding the affordability of Trikafta and the stakeholder engagement process, a brief public comment period was allowed, during which ten individuals were permitted to provide two-minute public comments. 

The Board then decided that no further deliberation was necessary before taking a vote on the affordability of Trikafta. Board member Justin VandenBerg, Pharm.D., made the following motion: “The use of Trikafta, consistent with the labeling approved by the FDA or with standard medical practice, is not unaffordable for Colorado consumers.” Board member Sami Diab, M.D., seconded the motion and the Board proceeded to vote unanimously to approve the motion. 

As quoted by Ed Sealover in the Colorado Chamber of Commerce’s The Sum & Substance blog, PDAB chairwoman Gail Mizner, M.D., reflected on the Board’s decision by saying: 

“This is an extraordinarily expensive drug … But it is also an extremely beneficial drug, a miraculous drug that is making a big difference in Coloradans’ lives … At this point, it does not seem unaffordable. And I would be willing to look at it again if that changes.” 

The PDAB then went on to discuss the scheduling of upcoming meetings and staff recommendations for updating rules and policies. Staff plan to suggest potential changes for the Board to consider for the next cycle of affordability reviews (after its first five reviews). Staff plan to present draft redlined versions of the following documents for the Board’s consideration in future meetings: 

  • Policy 04 – Affordability Reviews: “Staff anticipate most proposed changes will be regarding manners, methods, and timelines regarding gathering patient and caregiver input.” 
  • 3 CCR 702-9 Rule Part 3 – Affordability Reviews: “Staff will draft a proposed change that would allow the board to consider a prescription drug’s orphan status during the selection state (in addition to the current requirement to consider this information as an affordability review is being conducted).” 

The December 8 PDAB meeting also included a presentation and discussion of the preliminary affordability review data for Genvoya and Enbrel after CBSA and other stakeholders expressed serious concerns about the PDAB’s inconsistent approach to the preliminary data review process for drugs under affordability review in the first cohort. While we are pleased that the timeline for the second and third drugs was pushed back to follow a process similar to that used for the first drug, we are disappointed that the PDAB’s December 8 agenda did not allot as much time for the presentation and discussion of preliminary data for each or those drugs as the PDAB utilized for Trikafta. It was clear from the PDAB’s discussion of preliminary data for Trikafta that more time is both beneficial and needed. 

CBSA’s Reaction and Continuing Concerns About Upper Payment Limits 

Following the December 8 PDAB meeting, Amy Goodman, Colorado BioScience Association Vice President & Counsel for Policy + Advocacy, said: 

“Today’s Prescription Drug Affordability Board decision gives cystic fibrosis patients peace of mind that government-imposed price caps won’t limit their access to Trikafta, a drug they say has been life-changing. Government price caps are the wrong solution for patients, who are worried about the serious, unintended consequences of price caps on medications. Government price controls won’t reduce patient out-of-pocket costs. They limit choice and could reduce investments in new medicines.”    

CBSA shares the PDAB’s goal of improving the affordability of medicines for patients, but we remain concerned about the possibility of any future decision to deem a drug unaffordable or to set a state-mandated UPL. CBSA will continue to work closely with BIO, PhRMA, and member companies as the PDAB continues with its next affordability reviews. We oppose the creation and enforcement of UPLs that we believe will lead to negative unintended consequences on patient access to medicines, on healthcare providers prescribing medicines, and on the market for innovative biopharmaceutical products. 

Draft Affordability Review Report for Trikafta 

In advance of the December 8 PDAB meeting, PDAB staff released a draft Affordability Review Report for Trikafta on December 1. The 588-page report contained a 23-page summary report and 16 appendices, but it did not contain any conclusions about the affordability of Trikafta. The report is divided into three profiles: 

  • Therapeutic and Utilization Profile: The Therapeutic and Utilization Profile includes information about Trikafta’s clinical efficacy and the people who use it. This section provides information regarding Trikafta’s indication, utilizer profile, health equity impact, and therapeutic alternatives. 
  • Price and Cost Profile: The Price and Cost Profile includes information on what different entities on the prescription drug supply chain charge for Trikafta, as well as what different entities pay for Trikafta. This profile also contains information on Trikafta’s financial effects on health, medical, and social service costs. 
  • Access to Care Profile: The Access to Care Profile examines potential access to care concerns related to Trikafta and whether there is evidence that the causes of access to care concerns may be related to Trikafta’s price or cost. This profile includes an examination of potential relationships of changes between utilization, price, and costs as well as information on safety net providers, utilization management requirements, and health benefit plan design. 

Next Steps 

Although timelines have repeatedly changed throughout the PDAB implementation process, as of right now, the next steps in the process are as follows: 

  • December 15 (time TBD): The PDAB will meet to adopt the final Affordability Review Report for Trikafta (including Board member edits, deliberation summary, and Board vote). 
  • January 25, 9:00 am MT: Prescription Drug Affordability Advisory Council (PDAAC) Meeting 
  • February 5, 7, or 16 (date TBD), 10:00 am – 2:00 pm MT: This PDAB meeting will include deliberations and votes on the unaffordability of Enbrel and Genvoya. We expect the PDAB will hold a follow-up meeting about a week later to finalize the Affordability Review Reports for Enbrel and Genvoya. If they deem either drug unaffordable, that follow-up meeting would also include a vote on whether to begin rulemaking to establish a UPL for the drug (which would include two rulemaking hearings to discuss the precise amount of the UPL and then promulgate the exact UPL). 
  • March 15, 10:00 am – 2:00 pm MT: Preliminary affordability review data for Cosentyx and Stelara, the two drugs in the second cohort, will be presented by PDAB staff and discussed by the Board. 
Categories: CBSA News