PCORI to incorporate cost but not cost-effectiveness in its new principles

Created by the Affordable Care Act (ACA) in 2010, the Patient-Centered Outcomes Research Institute (PCORI) has funded a wide variety of research comparing different treatments to one another. One thing PCORI was forbidden from funding, however, was cost-effectiveness analyses. Section 1181 of the Social Security Act, however, has expanded the scope of the types of outcomes PCORI can look at including some cost outcomes. As PCORI outlines in its Proposed “Principles” for the Consideration of the Full Range of Outcomes Data:

Research shall be designed, as appropriate, to take into account and capture the full range of clinical and patient-centered outcomes relevant to, and that meet the needs of, patients, clinicians, purchasers, and policy-makers in making informed health decisions. In addition to the relative health outcomes and clinical effectiveness, clinical and patient-centered outcomes shall include the potential burdens and economic impacts of the utilization of medical treatments, items, and services on different stakeholders and decision-makers respectively. These potential burdens and economic impacts include medical out-of-pocket costs, including health plan benefit and formulary design, nonmedical costs to the patient and family, including caregiving, effects on future costs of care, workplace productivity and absenteeism, and healthcare utilization

However, no one should mistake PCORI for a health technology assessment (HTA) body. In fact, PCORI is prohibited from conducting cost-effectiveness research.

The authorizing law includes the limitation that PCORI “not develop or employ a dollars-per-quality adjusted life year (or similar measure that discounts the value of a life because of an individual’s disability) as a threshold to establish what type of health care is cost effective or recommended.” Moreover, PCORI is not permitted to “mandate coverage, reimbursement, or other policies for any public or private payer.”

PCORI’s does, however, have a new set of principles for better measuring a broader set of stakeholder outcomes. These include:

  1. PCORI-funded research may consider the full range of outcomes important to patients and caregivers, including burdens and economic impacts. These costs may include patient costs (e.g., time in hospital, time home from work or usual activities, cost/time for transport, childcare costs, out-of-pocket costs), caregiver costs (e.g., hours spent caregiving, foregone wages), health care utilization (but not cost), and medical cost offsets.
  2. PCORI-funded research may consider the full range of outcomes relevant to other stakeholders, when these outcomes have a near-term or longer-term impact on patients. Relevant economic impacts can include treatment cost (medication cost, staff time), utilization (without associated cost), cost offsets (e.g., changes in use of ED, physician visit, changes in hospital admissions or length of stay), program costs, employer burden (e.g., absenteeism, productivity)
  3. The collection of data on burdens and economic impacts of treatment options must be appropriate and relevant to the clinical aims of the study. PCORI is demanding stakeholder engagement in part of all their studies. “PCORI requires applicants to engage relevant stakeholders in the formulation of the research question and the development of the study design, as well as the identification of outcomes to measure.” Despite the content described in Principle #1 and #2, PCORI notes that comparative clinical effectiveness is their mandate and PCORI will not fund studies whose primary endpoint is a cost-effectiveness analyses (e.g., cost/QALY).
  4. Beyond the collection of burden and economic impact data, PCORI may support the conduct of certain types of economic analyses as part of a funded research study, to enhance the relevance and value of this information to health care decision-makers. PCORI reiterates that they will not do cost-effectiveness research nor mandate coverage or reimbursement policy.

PCORI is accepting public comments until November 13, 2020 for those who want to weigh in on these changes.

The need for continuous cost-effectiveness analysis

At drug launch, treatments have some benefit and cost. However, drug prices may change over time. Additionally, treatment benefits may rise or fall depending on how well efficacy translates into effectiveness and how well physicians learn to manage adverse events.

Mattingly and Love (2020) provide a case study of how cost-effectiveness analysis can change over time. They examine the case study of hepatitis C virus (HCV) treatments, which is well known since sofosbuvir’s “$1,000 a pill” list price sparked a lot of debate, despite broad evidence that the treatment was cost-effective. New HCV treatments later entered the market including elbasvir/grazoprevir, sofosbuvir/velpatasvir and glecaprevir/pibrentasvir. The authors then examine the cost effectiveness of the best available HCV treatment in 2010, 2012, 2014, 2016 and 2018. They find that:

No HCV treatment resulted in a gain of 11.54 QALYs over a 20-year time horizon at a cost of $42,938. Costs for treated groups were $69,075, $123,267, $125,431, $86,782, and $56,470 for the 2010, 2012, 2014, 2016, and 2018 scenarios, respectively. QALYs gained for treated groups were 12.90, 12.97, 13.34, 13.39, and 13.46 for the 2010, 2012, 2014, 2016, and 2018 scenarios, respectively. The incremental cost-effectiveness ratios in each year compared with no treatment were $19,218 per QALY, $56,104 per QALY, $45,829 per QALY, $23,699 per QALY, and $7,048 per QALY.

In short, treatments became more and more effective over time. Initially, cost rose from 2010-2014 and cost-effectivenes worsened between 2010 and 2012. However, as effiacy continued to rise and cost began to fall, cost-effectiveness improved from 2012 to 2018. Now, treatment costs are less than at launch despite new treatments adding more than 0.5 QALYs per patient.

Mattingly and Love make a compelling case that value assessment is not a one-time endeavor, but needs to be revisited as a treatment landscape evolves.