As policymakers are evaluating proposals to address the costs of prescription drugs, patient advocates and health policy experts warn against using international reference pricing or cost-effectiveness analyses based on the discriminatory QALY. That was the theme of this year’s 10th Annual PIPC Forum, held in Washington, DC on Monday, Dec. 16 2019. 

​The forum featured remarks from PIPC’s chairman, the Honorable Tony Coelho, two panels of patient advocates and policy experts, and videos featuring the stories of foreign patients’ struggles to access needed therapies.

​Speakers on the Forum’s distinguished panels included:

• Dr. Lakiea Bailey, Sickle Cell Community Consortium
• Christina Hartman, The Assistance Fund
• Susan Peschin, Alliance for Aging Research
• Ana Torres-Davis, National Council on Disability
• Siri Vaeth, Cystic Fibrosis Research, Inc.
• Joel White, Council for Affordable Health Coverage

A Decade of PIPC

Chairman Coelho opened the event by acknowledging the decade of great work PIPC has done and describing the journey it has taken over the past ten years.  He noted that though the battlegrounds have changed, the battles haven’t. When PIPC started it advocated successfully for PCORI to be established as a truly patient-centered entity with a ban on the use of cost-effectiveness research. Ten years later, we are, again, fighting against the use of cost-effectiveness in our nation’s healthcare system, this time in the form of how we determine pricing and coverage for prescription drugs. He shared his concern about QALYs creeping into our system either through third-party cost-effectiveness analyses or international reference pricing. He then set the stage for a video featuring a Canadian girl with cystic fibrosis, Laura, and her mother. All of Laura’s doctors have recommended she be on a certain drug, Orkambi, but as Orkambi is not deemed cost-effective by her province, Laura is unable to access the drug unless her lung function declines by 20%, thus forcing her to get sicker in order to have a chance of receiving the recommended therapy.

Patient and Caregiver Reactions

PIPC Executive Director, Sara van Geertruyden, moderated the first panel of patients and caregivers gauging their reaction to the video they just saw and asking their feeling and concerns about cost-effectiveness and potential of an international pricing index to determine the cost of drugs in the United States. The panelists provided some deeply personal insights on patient access and the potential implications of importing international pricing.

• Siri Vaeth shared that as a parent of a child with cystic fibrosis she would be terrified to watch her own child endure the trauma Laura did. She noted that for a cystic fibrosis patient, 20% lung function decline can mean irreversible damage and commented that this type of system was divorced from humanity, prioritizing cost over a human life. She commented on the Canadian system in comparison to the United States noting that though our system is far from perfect, we do have access to cutting edge therapies, which can make all the difference when dealing with certain disease populations. Siri expressed her concern that the desire to base coverage decisions on cost-effectiveness was creeping into the American system and cited the example of her experience undergoing an Institute for Clinical and Economic Review (ICER) assessment for cystic fibrosis treatments. She noted that she was utterly appalled by ICER’s lack of true patient and provider engagement and the fact that they could be callous enough to acknowledge that the drugs improved patient quality of life but still rule they were not worth the cost.

 

• Dr. Lakiea Bailey reacted to the video by saying it was discouraging and cruel. She also highlighted economic access problems that sickle cell disease patients are currently having in the United States and highlighted how important it is to care for our most vulnerable. She encouraged the audience that we should be looking to solve access restrictions that currently exist in this country and should seek out our own unique American solution instead of importing other systems we know to be fraught with access issues. She also shared some of her recent experiences with sickle cell disease being in the spotlight due to the approval of two new therapies in the past month. She focused on the fact that there is much conversation going on about patients without them at the table, citing ICER and a recent New York Times piece. She noted that one of her organization’s goals is to identify patients and caregivers with expertise and knowledge of the realities of Sickle Cell Disease and get them to the decision-making table. She stressed the importance of meaningful patient participation and cautioned against tokenism.

 

• Christina Hartman expressed her shock and horror at the video from the perspective of a parent of a child with a rare disease. She also echoed Dr. Bailey’s sentiment that we need to find our own unique American solution to solve problems with affordability noting that we should be focusing on investing in treatments and access to care, not perpetuating a system where existing therapies cannot be accessed. Ms. Hartman emphasized that it should be a given that peoples’ lives have value, and that it is astounding that we are having a debate about the topic. She emphasized that policymakers can either facilitate access or be a barrier to care and she wants to see them listen to patients more deeply and make thoughtful decisions centered on patients.

 
Technical Health Policy Experts Discuss International Pricing and the QALY

Following the patient perspective, we had the opportunity to hear from distinguished health policy experts about the technical policy implications of using international pricing to set prices for drugs in the United States. Prior to delving into the discussion, Ana Torres-Davis presented a brief summary of National Council on Disability’s report, Quality-Adjusted Life Years and the Devaluation of Life with Disability, including its recommendation that the United States should refrain from attempts to model U.S. pricing after the pricing in other countries.

• Ana Torres-Davis spoke on behalf of National Council on Disability (NCD), an independent federal agency that advises the federal government, on NCD’s report, Quality-Adjusted Life Years and the Devaluation of Life with Disability. She shared the report’s finding that the QALY undervalues treatment for people with disabilities and is a discriminatory metric. She highlighted the fact that we can see real world examples of QALY based assessments limiting access to care for those with disabilities and chronic illnesses by looking to other countries that rely on the QALY, like the United Kingdom. She emphasized that the report found that QALYs should not be used in the United States and reviewed the report’s recommendations to the federal government, which included:
  • Congress should pass legislation prohibiting the use of QALYs in Medicare and Medicaid;
  • Congress should provide funding to research best practices on cost-effectiveness in Medicare and Medicaid that do not rely on the QALY;
  • The Departments of Health and Human Services (HHS) and Justice should issue guidance that federal disability law applies to federal health programs and all health programs receiving federal assistance and that payment decisions should not rely on anything developed using QALYs;
  • Centers for Medicare and Medicaid Services (CMS) should refrain from developing pricing models after other countries that use QALYs;
  • CMS should rescind their proposed rulemaking that promotes international reference pricing.

 

• Joel White opened his remarks by stating that using an international pricing index presents a false choice of affordability or access when in reality we have been able to achieve both in many other federal programs, citing Medicare Advantage as an example. He noted that we already have ample evidence that patient access is limited in other countries due to reliance on cost-effectiveness. He also focused on the fact that this type of government price setting of innovating treatments will pull investment away from rare conditions and unmet needs and push it towards profitable drugs that already exist. Policies driving more “me too” drugs will leave many patients with conditions that have few treatment options without any innovation, which will lead to increased hospitalization and decreased quality of life.

 

• Susan Peschin shared that it is important we all understand the history and context of the use of the QALY in international healthcare systems and explained that it began with the British use of the QALY in the 1960s with a goal of rationing care, as there was a huge influx in the need for transplants and dialysis. She explained that understanding this history is critical in understanding that the QALY is a tool designed to ration care. She shared that because of this baseline, any method of basing U.S. prices on those of other countries is inherently discriminatory. She spoke about the recently passed house drug pricing bill, HR 3, which uses international pricing to determine pricing with the U.S. She highlighted that including any softening language in the bill did not assuage her concerns about the policy, because the policy is inherently discriminatory. Ms. Peschin also focused on the innovation implications of international pricing, expressing concern that reliance on an international pricing index would lead to a drastic decrease in innovation as companies generally spend about 20% of cash flow on innovation, and if you cut that it leads to fewer treatments for patients.