Assessing the value of novel therapies has been challenging and controversial using existing methods, pointing to the need for continued exploration of new approaches.
Innovative cell and gene therapies are on the horizon, many with the potential to transform the lives of patients with serious diseases. In a world of finite resources, it is imperative to understand the true value of these therapies to different stakeholders so that their full benefit is realized and patient care is optimized, while costs are also contained. However, assessing the value of these novel therapies has been challenging and controversial using existing methods, pointing to the need for continued exploration of new approaches.
Standard Methods Limit Our Understanding
For the past 30 years, the Quality Adjusted Life Year (QALY) has been the most commonly used measure to assess the incremental value of novel medical interventions. However, limitations of the QALY have been widely documented.1 First, the QALY fails to adequately capture many dimensions of value that may be important to patients, physicians, payers, and greater society, such as productivity or emotional well-being. The QALY cannot take into account contextual differences between diseases that affect stakeholder preferences around value. For example, there may be greater societal willingness to allocate extra resources to treat rare and severe diseases, or diseases affecting children. Furthermore, there are longstanding ethical concerns that the QALY discriminates against older patients (who have shorter remaining lifespans) and individuals with disabilities (who may be awarded fewer QALYs for baseline health).
These limitations become even more pronounced when applied to transformative therapies such as cell and gene therapies. While some researchers have begun to explore ways to use an “augmented” QALY, this presents challenges as well. Meanwhile, cell and gene therapies offer patients newfound hope and pave the way for additional scientific discoveries and truly personalized care—2 factors the QALY is largely unable to incorporate. This means the method risks underestimating the true value of cell and gene therapies.
MCDA: One Promising, Alternative Approach
There is emerging interest in alternative value assessment methodologies, including Multi-Criteria Decision Analysis (MCDA), a group of techniques that explicitly account for multiple factors to inform a decision. Widely used in other fields, the MCDA approach can account for a broader range of factors deemed interesting to decision-makers. While no one method clearly resolves all issues, we believe MCDA is a promising approach that needs further investment of time and resources.
The use of MCDA can potentially allow for a more comprehensive assessment of cell and gene therapies as such methods can take into account patient goals and priorities for care, disease severity, societal preferences, and other novel value elements.
MCDA also better reflects the way complex decisions are made in everyday life and provides a transparent methodology to help stakeholders understand the process and build consensus. Using MCDA and its flexible consideration of a wide variety of value criteria, more stakeholders can contribute to the evaluation process; in turn, decisions about resource allocation can be made more equitable and ethical.
Lastly, MCDA can be particularly helpful when making decisions around transformative cell and gene therapies that have limited or uncertain clinical evidence and real-world evidence at the time of decision-making. These often require more collaborative efforts among different stakeholders. The pivotal clinical trials for such therapies are often single-arm, short-term studies with few study participants. MCDA may allow healthcare decision makers to explicitly consider the uncertainties in available data, and weigh their priorities accordingly.
Formalized MCDA techniques are emerging, and Health Technology Assessment (HTA) agencies have begun to pilot their use, with promising results. For example, in 2010, the German Institute for Quality and Efficiency in Health Care initiated successful workshops to trial 2 potential methods for eliciting stakeholder preferences in the MCDA framework. The trials were a success, with researchers concluding that their techniques could be used to support the value assessment process.
As a nonprofit laboratory for improving methods and resources used for value assessment, the Innovation and Value Initiative (IVI) continues to explore the methods and application of MCDA. Currently, IVI is collaborating with research partners to elicit patient-derived preference and evaluation criteria for treatments in multiple disease areas and to experiment and optimize their use in value assessment. IVI is exploring how to incorporate multiple perspectives into economic models and the use for both MCDA and Comparative Effectiveness Analysis. This ongoing investment in partnership and methods enhancement stems from our core principle—ensuring that value assessment is both scientifically credible and relevant to all stakeholders, especially patients.
As an emerging framework, questions around best practices in MCDA naturally remain. Concerns have been raised around the inherent complexity of MCDA. Adopters of MCDA must be careful that criteria are not overlapping or double counted in the analysis, for example. Criteria that may be important to individual stakeholder groups may not lend themselves to decision contexts. Data may not be sufficiently available or accessible to construct an MCDA perspective. MCDA could create uncertainty or raise more questions since each value assessment model may differ in the criteria it assesses and how the model is constructed. It is important all stakeholders keep these concerns in mind as we continue to explore the promise of using MCDA in real-world decision contexts.
It is equally important that such concerns do not become a barrier (or worse, a justification) to slow the continued exploration of novel approaches, such as MCDA. Cell and gene therapies offer promise for many stakeholders. Patients hold hope that these therapies can transform their lives. Providers and payers are eager for these therapies to produce durable clinical outcomes. Society at large stands to gain from the increased productivity of treated patients and the potential for further innovation and scientific breakthroughs. It is vital that our methods for considering the value of medical interventions can account for these additional dimensions. MCDA has real potential as a more thorough and equitable method for future value assessment.
1. National Council on Disability. Quality-adjusted life years and the devaluation of life with disability: Part of the bioethics and disability series. November 6, 2019. Accessed July 16, 2020. https://ncd.gov/sites/default/files/NCD_Quality_Adjusted_Life_Report_508.pdf