Cell and gene therapies (C>s) are revolutionary advancements that offer potentially life-altering therapies – and in some cases even cures – for patients with rare and severe diseases. Around the globe, various health technology assessment (HTA) and value assessment organizations are attempting to estimate the health and economic value of new C>s to better inform healthcare decision making. These organizations most commonly employ cost-effectiveness analysis (CEA), which seeks to determine whether the costs of a given therapy are justified by its benefits; that is, whether it is a “good buy.”
However, CEA often fails to capture or reconcile key issues of importance to the valuation of C>s. Because C>s utilize unique methods of administration, often treat small patient populations, and often lack long-term data at launch, traditional CEA methods struggle to determine the true value of these therapies. Furthermore, CEA has historically been intended only for a payer audience, while data on the perspectives of other stakeholders, including patients, are often not fully incorporated. Many challenges therefore remain in achieving comprehensive, patient-centered value assessment of C>s, including:
These shortcomings have the potential to lead to very real consequences for patients in the form of restricted and delayed access to novel therapies. It is therefore essential that value assessors incorporate patient experience data more fully into their analyses. A movement to incorporate this information known as patient-focused drug development (PFDD) has already gained traction in other medical research communities. By joining this effort, value assessors can ensure that their evaluations of C>s deliver more accurate and informative evidence for patients and other healthcare decision makers, not just payers.
Click here to read the white paper in its entirety.
- Uncertainties in the available data make it difficult to choose an appropriate duration of effect for clinical benefits.
- Patient heterogeneity and subgroup analyses are often not considered, instead focusing only on the “average” patient.
- CEA relies on Quality-Adjusted Life Year (QALY) gains as a generic measure of health or disease burden, but the QALY cannot capture societal preferences around resource allocation and is widely seen as discriminatory towards patients with disabilities.
- Estimating QALYs gained is also difficult for the rare diseases that many C>s treat; the underlying utility scores may not accurately represent the patient experience.
- Commonly cited cost-effectiveness thresholds may be too low for the rare diseases often treated by C>s.
- Additional elements of value afforded by C>s, such as increased productivity and reduction of caregiver burden, are often omitted in CEA base case analyses.
- Further elements of value important to patients, such as the value of hope and insurance value, are generally not quantified.
- The discount rate used for value assessment may be overly punitive when applied to C>s with durable clinical effects.
These shortcomings have the potential to lead to very real consequences for patients in the form of restricted and delayed access to novel therapies. It is therefore essential that value assessors incorporate patient experience data more fully into their analyses. A movement to incorporate this information known as patient-focused drug development (PFDD) has already gained traction in other medical research communities. By joining this effort, value assessors can ensure that their evaluations of C>s deliver more accurate and informative evidence for patients and other healthcare decision makers, not just payers.
Click here to read the white paper in its entirety.
| health_advances_cgt_value_assessment_paper_final_for_release.pdf |
