High-Investment Medications: Predictability, Affordability, and Accessibility

While the Food & Drug Administration (FDA) is continuing to approve novel therapies to address important medical needs, the costs of certain therapies are raising value and affordability concerns for employers, payers, and patients. AMCP convened key stakeholders in managed care to explore solutions for improving the predictability, affordability, and accessibility of high-investment treatments, with a focus on three objectives:

1. Identifying stakeholder challenges associated with high-investment medications.
2. Exploring the challenges and opportunities related to financial tools to address predictability, affordability, and accessibility for high-investment medications.
3. Determining the challenges and opportunities of potential policy solutions to improve the predictability, affordability, and accessibility of high-investment treatments.

Stakeholders noted that the benefits of these therapies to patients can be significant, and though the financial costs of these high-investment drugs are substantial, these products are used in a targeted and precise manner. However, the uptake of innovative payment strategies to manage them has been slow. To prevent future challenges, payers should implement incremental changes to prepare for the future impact of the growing pipeline of high-investment medications. Opportunities for process improvement include streamlining data collection, standardizing terminology, providing education on new financial tools and policy solutions, and utilizing a variety of financial tools tailored to specific needs. Engaging and partnering multiple stakeholders will be necessary to improve the predictability of outcomes and costs, affordability for patients and the health care system, and accessibility for patients.

Introduction

While the FDA continues to approve novel therapies to address important medical needs, the costs of certain therapies are raising sustainability, value, and affordability concerns for employers, payers, and patients. This
is especially true of innovative medication technologies, such as cell and gene therapies, which offer potentially life-changing therapeutic advances or cures. Many of these high-cost medications are used only once or for a short duration, creating challenges for patients, health care providers, and payers. Examples include the need for complex care coordination, uncertain long-term health benefits, and potential financial losses when patients switch employers or enroll in different health plans. These challenges present opportunities to improve the predictability of outcomes and costs, affordability for patients and the health care system, and accessibility for patients.
 

This market is expected to grow significantly, with hundreds of new product and indication approvals expected in the United States by 2030.1 Included in this category are cell therapy products, such as cellular immunotherapies, cancer vaccines, and other types of both autologous and allogeneic cells for certain therapeutic indications, including hematopoietic stem cells and adult and embryonic stem cells.2 Cell therapy treatments are used for blood cancers, including lymphomas, some forms of leukemia, and, most recently, multiple myeloma. An example of this type of therapy are chimeric antigen receptor T-cell (CAR-T) treatments that involve harvesting patient-specific T-cells and re-engineering them in a lab before infusing them back into the patient.3 Also included in this category are human gene therapies, which work by manipulating the expression of a gene to alter the biological properties of living cells for therapeutic use.2 Examples of this include treatments for hemophilia, bladder cancer, and beta-thalassemia.

Download Report