Donna Cryer, JD, a liver transplant recipient and founder, president and CEO of the Global Liver Institute, is a passionate advocate for patients. She is a familiar presence at health care stakeholder meetings in Washington, DC, where she works to ensure that patients’ views, concerns and voices are incorporated into setting research priorities, engaging in health information technology and making clinical decisions. Most recently, she has turned her attention to the Institute for Clinical and Economic Review (ICER), an organization focused on cost-effectiveness reviews that is now considering how to assess the value of specific treatments for liver disease.
On the eve of ICER’s public meeting to discuss its evaluation of these treatments, National Pharmaceutical Council President Dan Leonard sat down with Ms. Cryer to ask her thoughts about ICER, value assessments and how patients can become more engaged in these evaluations.
Dan Leonard (DL): First, thanks for sitting down with me to discuss these timely issues. How would your community assess the value of new therapies that have come on the market in the past few years, particularly cures for hepatitis C and other major advances in the treatment of liver disease?
Donna Cryer (DC): Thank you, Dan, and to the National Pharmaceutical Council for this opportunity to discuss the important issue of the impact of value frameworks to the liver community and to all patient communities. On a scale of 1-10, the value of new treatments in liver disease in recent years has been a 10, because we have not only found a cure for one of the top causes for liver transplantation, liver cancer, and liver-related deaths, globally, but we have started to see an energetic ecosystem of collaborative and competitive research and drug development emerging for a host of liver diseases from Hepatitis B vaccines to the first treatment for primary biliary cholangitis (PBC), a rare autoimmune liver disease, to potential upcoming therapies for non-alcoholic staetohepatitis (NASH), which is closely related to the obesity and diabetes epidemics.
DL: How is the patient perspective captured and quantified in not just ICER’s, but in other value assessment frameworks?
DC: Unfortunately, for the most part, the patient perspective is missing from the ICER and other value frameworks. This is a tremendous problem, because when health care is decoupled from a focus on meeting the needs of patients, it fails to have value. ICER has involvement from a consumer organization and limited opportunities in its process for patient comment, but is overwhelmed by payer participation in the composition of its governing board, topic selection process and curation of evidence. Also, its methodology consistently misrepresents and excludes patient-centered evidence, patient-reported outcomes, and cost-offsets, perpetuating the myth of the average patient or standardized over personalized care. Other frameworks, with the exception of those now being developed by the National Health Council and Faster Cures, also lack patient and patient advocate engagement.
DL: On July 15, ICER will be holding a meeting in Portland, Maine, to review its draft report on treatments for two liver diseases. Do you feel your community will be heard and adequately represented in their findings?
DC: On July 15, ICER’s New England Comparative Effectiveness Public Advisory Council (CEPAC) will review PBC and NASH. The liver community will be heard and represented by myself and others privileged with the health, wealth, and time to travel to Portland, Maine. Whether our perspectives, information from sources such as the UK-PBC Stratified Medicine Registry, and concerns about methodological flaws will be fairly incorporated into the final report remains to be seen.
DL: One of the disease areas ICER is looking at—non-alcoholic steatohepatitis (NASH)—doesn't even have an approved treatment yet. What do you think about ICER conducting an evaluation in this area with little to no available evidence?
DC: There is no FDA-approved treatment for NASH, so there is no valid reason to evaluate this disease area at this time, except to serve as a chilling effect to those seeking to innovate in this space. Also, the PBC treatment has only been on the market for a few months. I served on the FDA advisory community that unanimously recommended approval in April. So I do not see what additional evidence could have been developed in the ensuring time to justify a different evaluation of safety, effectiveness, or “care value” than that of the experts on the FDA advisory committee.
DL: You personally are not only an advocate for people living with liver conditions, you are also a patient. How has your personal experience impacted your views on how framework developers are attempting to assign value to new therapies?
DC: As a patient who has been living with an autoimmune liver disease and a liver transplant for well over two decades, I am particularly disturbed that (1) the unique expertise and experience of patients, patient function, return to productivity, patient benefit over time, total patient costs and cost-savings, factors that ease patient burden of adhering to a therapy, etc. have not been the central driving principles of value framework development and that (2) Quality-Adjusted Life Years (QALYs) are a primary mode of determining value. QALYs presume that anything less than perfect health makes you a less valuable person or assumes that you have a less valuable life. Unless you are training for the Olympics, physical perfection is not a determinant of the value of your life – either your appreciation for it or your contribution to it.
DL: Following the ICER meeting, what would you view as a positive outcome for your community and assessments of liver disease?
DC: A positive outcome for this particular ICER meeting on PBC and NASH would be for NASH to be completely tabled, and PBC considered correctly in its context as a rare disease disproportionately affecting women in their prime family-raising and work years having the first new treatment in 20 years, the only treatment for some patients, and the devastating disease progression that happens to the majority of these women without treatment options. Moreover, a positive outcome would entail being formally invited and integrated into the entire ICER process for all future consideration of liver-related diseases.
DL: Thanks for joining me.
To learn more about Ms. Cryer and her work, visit the Global Liver Institute’s website.
Mark your calendars: NPC will bring together health care stakeholders on September 29 in Washington, DC, for a conference, “Assessing Value: Promise and Pitfalls,” to examine value assessment frameworks. To register to attend in-person or watch live online, visit NPC’s website.