METHODOLOGY

This white paper provides insights, analysis, and recommendations based on two components: (a) a landscape assessment, and (b) interviews with subject matter experts.

The landscape assessment surveyed existing literature, research, and analysis from a wide array of publicly available sources. Such sources included, but were not limited to, the following: 9 9 9 9

Academic, non-profit, and professional society articles, research, and publications

State Medicaid program policies

Media coverage and press reports from national and trade outlets

Policy proposals, guidance, and analysis from the Centers for Medicare and Medicaid Services (CMS), the Food and Drug

Administration (FDA), the Medicaid and CHIP Payment and Access Commission, and other federal sources

Other publicly available sources such as interviews, surveys, and presentations.

Leavitt Partners also conducted interviews with select subject matter experts, including more than 45 experts on cell and gene therapy and Medicaid. These individuals represented a wide range of views representative of the perspectives of: 9

Patient advocacy groups (including advocates for patients with rare diseases)

• Six patient advocacy groups, including four with a rare disease focus and one focused on cancer

Medicaid managed care organizations

• Four large national managed care plans

State Medicaid programs (pharmacy and state program leadership)

• Four former Medicaid directors from different states, a pharmacy director

Drug manufacturers

• Five manufacturers, all involved in the development of current cell and gene therapies or such products under development

Associations and researchers working on cell and gene therapy issues

• Two national associations focused on cell and gene therapy • Researchers focused on value-based payment for cell and gene therapy

Health care providers

• An expert knowledgeable in cell and gene therapy coverage and reimbursement challenges for hospitals

Federal policymakers and researchers

• Congressional staff from both the House and Senate

Leavitt Partners also spoke with a knowledgeable policy staff employed by trade associations representing a number of the organizations and sectors listed above.

Interviews covered a wide range of topics, including challenges observed in access to, coverage of, and payment for cell and gene therapies; insights on potential opportunities for improvement in those areas; and specific recommendations on value-based purchasing arrangements and policy changes to better facilitate access to cell and gene therapies. Through these interviews, Leavitt Partners obtained first-hand accounts of the complexities surrounding Medicaid’s role in ensuring patient access to cell and gene therapies that served as core basis for the content, conclusions, and proposed stakeholder recommendations in the report.

The research in this white paper was financially supported by The Assistance Fund. Leavitt Partners maintained editorial control and independence throughout the writing of this white paper.