Value Assessment Tools that take “Precision” out of “Precision Medicine” are Harmful to Patients

BIOtech Now
Dan Durham

The conversation about health care costs is front and center in Washington, and new data on prescription drug spending has helped fuel this important debate. For example, we’ve recently learned national spending on retail drugs increased by only 1.3% in 2016, well below spending growth for hospitals (4.7 percent), physicians and clinical services (5.4 percent) and private health insurance (5.1 percent). We have also learned that changes in prescription drug prices are remarkably stable too. An analysis by a nonpartisan health research organization shows drug prices grew by just 1% — before adjusting for rebates, its lowest reading in four years. Meanwhile, a leading pharmacy benefits manager reports negative growth in net prices for drugs for both government and private payers in the first half this year.

While these numbers are encouraging, more can be done to ensure prescription drugs are delivering value for the patients who need them. With the arrival of a new generation of personalized medicines and cures such as gene therapy, immunotherapy and RNAi interference, the need to adopt a sustainable and responsible approach to health care value and access has never been more important. But value must be accurately assessed.

With this in mind, the Personalized Medicine Coalition (PMC) has released an important new report highlighting the flaws in many frameworks used to measure the value of a given medicine or treatment. As PMC points out:

  • Many assessments typically rely on population averages, and most do not consider the value of diagnostics tests, such as measuring the genetics and protein expression of a patient’s tumor. These are increasingly being used by doctors to inform more targeted treatment decisions. As a result, these assessments don’t always reflect how different subgroups of patients respond and thereby implicitly undermine the specific needs of individual patients.
  • Existing assessments also generally do not reflect how efficiently a treatment is delivered to patients. They do not differentiate between therapies that are available soon after a patient’s diagnosis—saving the patient precious time—and those that are used only after a patient is sent down a winding path of trial and error. Treatment efficiency is all about ensuring patients receive the safest treatments at the best possible time. Additionally, a targeted, efficient treatment plan can reduce unnecessary expenses often associated with less effective treatment options.

Aside from these and other practical concerns, ethical concerns are equally as important. Determining the cost-effectiveness of health care interventions is often based on a calculation of quality-adjusted-life-years (QALYs). A recent Partnership to Improve Patient Care report details several significant concerns with this approach. For example, using an arbitrary QUALY measurement can lead to discrimination against patients with disabilities, leading to less access to the care they need. As the partnership has mentioned, citing a study from the Autistic Self Advocacy Network:

“There is a long history of individuals with intellectual and developmental disabilities being denied organ transplants because of the misguided notion that their lives are somehow less valuable.”

It is important to note that these critical flaws—both practical and ethical—have long been reflected in the value assessments put forward by the Institute for Clinical and Economic Review (ICER), which has suffered from a lack of transparency and the absence of sufficient input from the full breadth of health care stakeholders. As PMC notes in its report, “ICER had no formal policy for updating its evaluations based on the availability of important new data until recently” – a questionable process in itself. As we look to the future of value assessments, the bias approach taken by ICER, and other insurer-backed groups, could have harmful consequences — consequences that will ultimately hinder our efforts to build a sustainable framework based on therapeutic efficiencies, improved outcomes, and patient-centered values. For a better approach, health care stakeholders and policymakers should look to the Innovation and Value Initiative (IVI), which recently introduced its Open-Source Value Project. This transparent and holistic approach for assessing value in health care puts patients first, allows for a broad range of perspectives, incorporates the latest available evidence, and considers the full range of scientifically defensible approaches. This consensus approach will help facilitate a more constructive dialogue between stakeholders with different value perspectives and minimize barriers that stand between medical innovation and the patients who depend on it.

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