SummaryTraditional health technology assessments fall short in accounting for how therapy addresses the total burden of illness experienced by affected individuals.
The systematic evaluation of a health technology and the impact it has on individuals and populations with a given clinical condition is conducted through a process called a health technology assessment (HTA). The World Health Organization (WHO) describes HTAs as “a bridge that connects the world of research to that of policy making,” and explains that they “can be used by decision makers and other stakeholders to support the decision-making process in health care at the policy level by providing evidence about given technologies.” Understanding the value of these health technologies is increasingly important as healthcare costs rise and as both public and private payers across the globe are charged with being good stewards of finite healthcare dollars.
Value frameworks represent the approach used to measure the value of the technology being assessed, outlining the methods, criteria, and evidence types to be used. Historically, HTAs have been conducted using traditional value frameworks that rely on measures of clinical benefit from clinical trial data, health-related quality-of-life estimates, healthcare utilization projections, and efficiency metrics applied to standardized patient populations. While this approach has a significant advantage in feasibility, the recent focus on disparities in access and outcomes indicates a need for a more holistic, patient-centered approach for defining value. For patients living with one of the more than 7,000 known rare diseases— for which indirect, non-medical burdens comprise more than half of the total disease burden— traditional HTAs fall short in accounting for the ways a therapy addresses the total burden of illness experienced by affected individuals.
The last decade has ushered in these new conceptual frameworks, referred to as “patient-centric” frameworks in this white paper, that incorporate additional variables more directly tied to overall societal benefit and the patient’s perspective (e.g., equity, cultural barriers, societal spillovers, value of hope, fear of contagion or disease). Examples of these models include the ISPOR Value Flower, the Generalized Risk-Adjusted Cost-Effectiveness (GRACE) approach, the Patient-Driven Values in Healthcare Evaluation (PAVE) model, the Innovation and Value Initiative (IVI), and the Patient-Centered Core Impact Sets (PC-CIS).
By moving to conduct value assessments using patient-centered models, assessors can overcome many of the challenges in accessing innovative therapies experienced by patients living with a rare disease. Patients, payers, and employers all stand to benefit from a fit-for-purpose, evidence-based approach to value assessment that accelerates appropriate access to life-altering therapies and technology.
In this white paper, we present a case for reconsidering how value is defined for technologies in rare diseases such as generalized myasthenia gravis and for incorporating patient-centered value into HTAs through patient-centered value frameworks.
Funding for this white paper was provided by Alexion Pharmaceuticals. Avalere Health retained full editorial control.
To learn more about patient-centered care, connect with us.
produces measurable results. Let's work together.