Focus on Outcomes: Quality Measurement Recommendations for CMS’ Medicaid Cell and Gene Therapy Access Model

Value-based care models must keep pace with rapid expansion of the cell and gene therapy pipeline, which is expected to result in the approval of 74 therapies in the US market by 2030. Cell and gene therapies can achieve unique, durable outcomes over the course of patients’ lives, such as increased survival; decreased morbidity; halted disease progression; improved quality of life, including higher function, reduced or eliminated pain and suffering, and a psychological sense of well-being; and increased functional capacity and work productivity of recipients and their caregivers. Although cell and gene therapies can bring lifelong value to patients and society, they require high upfront costs that necessitate exploration of innovative reimbursement models.

The Centers for Medicare & Medicaid Services (CMS) has selected a new Cell and Gene Therapy Access Model for testing by the Center for Medicare and Medicaid Innovation (CMMI). The purpose of this model is to allow state Medicaid agencies to assign CMS to coordinate and administer multistate, outcomes-based agreements with manufacturers for certain cell and gene therapies.

As CMMI develops the Cell and Gene Therapy Access Model, policymakers should consider unique characteristics and challenges when measuring the value of therapies. For example, there is a relatively small number of individuals currently being treated with these therapies and long-term follow-up is required to collect data on the durable effects of the therapies. In this post, we explore how CMMI should approach quality measurement for cell and gene therapies, including filling gaps in existing quality reporting program measures, enhancing data availability and integration, incorporating patient-reported measures, and addressing patient portability (ie, collecting long-term data from patients who change health plans).

What Are Outcomes-Based Arrangements?

Outcomes-based arrangements, which are sometimes called value-based arrangements, are used to link payment to a therapy’s performance for an individual patient or patient population. Arrangements may include agreements to modify the price of a therapy based on clinical outcomes or refunds to the payer for ineffective treatment. Outcomes-based arrangements for cell and gene therapies present challenges, including lack of available outcome measures for accountability and data collection barriers, that CMMI will need to consider when designing and implementing the Cell and Gene Therapy Model.

What Quality Measure Challenges Does CMMI Need to Address for the Cell and Gene Therapy Access Model?

Fill Gaps in Existing Quality Measures Relevant to Cell and Gene Therapies

Currently there are no standardized, endorsed measures specific to cell and gene therapies or long-term outcome measures that would capture performance over an extended time. However, some value-based arrangements between manufacturers and payers have used measures to assess performance of other therapies over multiple years. For example, Spark Therapeutics and Harvard Pilgrim have an arrangement to assess performance of Luxturna, a one-time gene therapy for treatment of patients with a form of retinal dystrophy. As part of the arrangement, the level of payment is tied to measured patient outcomes at a 30- to 90-day interval and again at 30 months. The short- and long-term measures designed for the contract are based on change in full-field light sensitivity threshold testing scores over baseline, but these measures have not been developed or endorsed for use in quality-reporting programs more broadly. Similarly, CAR-T outcomes-based arrangements have measured overall remission rates and survival endpoints over five years. However, related endorsed measures are lacking for use in quality-reporting programs. CMMI will need to develop measures to assess performance over multiple years of the durable outcomes associated with cell and gene therapies.

Data Availability and Integration

To fill gaps in quality measures, CMMI will need to address data-collection barriers for cell and gene therapies. Many clinical endpoints for these therapies (eg, survival) are challenging to develop, test, and benchmark as quality measures due to difficulty comparing across settings with variable populations. In addition, there are still relatively few patients receiving cell and gene therapies. For example, Luxturna is indicated for RPE65-associated inherited retinal dystrophy, a condition that is estimated to affect fewer than 2,000 individuals in the US. The relatively low numbers of patients receiving cell and gene therapies make it difficult to collect enough data to measure performance of these therapies at the population level.

Patient-Reported Measures

One way to capture outcomes that are meaningful to patients is through patient-reported data collection. When designing the Cell and Gene Therapy Access Model, CMMI should consider patient-reported measures to assess symptom burden and health-related quality of life with core patient-reported domains such as quality of life, physical functioning, disease symptoms, and goal attainment. In addition, CMMI should engage patients and caregivers to help select measures that are meaningful to individuals receiving, or those caring for someone receiving, a cell or gene therapy. CMMI should also consider innovative solutions, such as mobile apps, to minimize the burden of patient data collection.

Patient Portability Between Health Plans

Patients may frequently switch health plans, shifting between Medicaid, state exchange, and commercial insurance coverage. These frequent changes can create challenges in tracking long-term outcomes. In 2018, 21% of Medicaid and Children’s Health Insurance Program (CHIP) beneficiaries disenrolled in their plan, and 8% reenrolled within 12 months. This churn and fragmented coverage make it difficult for commercial payers to engage in outcomes-based arrangements. CMS is in a unique position to address patient portability by coordinating and administering outcomes-based agreements for Medicaid agencies across multiple states. This coordination could help address data issues as individuals move between states, but CMMI will need a plan for follow-up data collection when individuals transition from Medicaid to other insurers and back. Commercial payers can then adopt successful elements of CMMI’s efforts to collect data as patients move among payers, supporting additional value-based arrangements for cell and gene therapies. 

Conclusion                        

CMMI has a unique opportunity to develop a Cell and Gene Therapy Access Model to increase access to these potentially lifesaving and life-enhancing therapies for Medicaid beneficiaries. Addressing unique barriers to quality measurement in this space by developing new measures to fill gaps, improving data availability, incorporating patient-reported measures, and addressing patient portability between health plans will help assess the true value of cell and gene therapies over a patient’s lifetime. In addition to increasing access to cell and gene therapies among Medicaid beneficiaries, CMMI’s model could have beneficial spillover effects as additional payers adopt successful elements of the Cell and Gene Therapy Access Model.

About the Quality Outlook Commentary Series

Breakthrough treatments in cancer care, including precision therapies tailored to specific patient factors, are driving rapid changes in the definitions of oncology quality and value. Efforts to implement value-based care models in oncology must meet the demands of evolving science, new best care practices, and shifting patient priorities. Quality measures must be up-to-date and relevant. Payment models must recognize the challenges and costs of managing complex patient populations with diverse needs. In this JCP blog series, Quality Outlook, Real Chemistry will explore key issues in oncology quality and value through posts focused on measurement, value-based payment, and quality improvement.

About Jacqlyn Riposo, MBA

Jacqlyn Riposo, MBA, a Director at Real Chemistry’s Market Access team, leads and manages client projects, providing insight and subject matter expertise on quality landscape analyses and measure gap identification. Jacqlyn has led and contributed to projects focused on oncology quality measurement to identify gaps in measures used in accountability programs and opportunities for measure development and quality improvement. Through this experience, Jacqlyn helps navigate quality measurement and value-payments and define strategies for success.

About Tom Valuck, MD, JD
Tom Valuck, MD, JD, leads the Value-based Care Practice of Real Chemistry Market Access. He is a thought leader on health care system transformation and helps lead the firm’s focus on achieving better health and health care outcomes at a lower cost. Tom helps clients develop strategies to achieve success within the value-based marketplace. 

About Real Chemistry
The Real Chemistry Market Access team uses research and strategic advisory services to help our clients improve health and health care through value-based payment and delivery models. These models align performance with incentives by rewarding doctors, hospitals, suppliers, and patients for working together to improve quality while lowering total costs. Real Chemistry is an independent provider of analytics-driven, digital-first research, marketing services, and communications to the healthcare sector.

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