This brief offers a checklist of key steps that different stakeholders should consider to ensure patients have access to innovative treatments. These therapies present unique challenges for different stakeholders and … Read More
Implementation Brief: Key terms for payment innovation
This brief provides definitions of key terms that are often used in discussions about designing and implementing innovative payment solutions. It offers an overview of solution models, types of risk, … Read More
Implementation Brief: Considerations for developers in applying the Multiple Best Price (MBP) rule to improve access to novel therapies
This brief highlights some key steps and considerations for biopharmaceutical developers of novel, high-value therapies seeking to successfully implement CMS’s Multiple Best Price rule. The rule, which went into effect … Read More
Implementation Brief: Understanding access issues for new therapies
Our first implementation brief outlines several major barriers to patient access for novel cell and gene therapies. It also describes how providers, payers, and developers may overcome these barriers, with … Read More
Emerging market solutions for financing and reimbursement of gene therapies for sickle cell disease: Why do payment innovation?
The FDA has approved two cell and gene therapies for sickle cell disease, marking a significant development for the largest population condition to date. However, the high treatment costs, … Read More
Analyzing 340B and ASP interactions: Do Federal program rules disincentivize the use of VBCs, despite Medicaid Best Price reform?
This paper describes the interactions among Medicaid Best Price rebate calculations and those for 340B Ceiling Prices and Average Sales Prices which can create disincentives for providers and therapy developers … Read More
Are Cell and Gene Therapy programs a better bet?
Compared to traditional drug development programs, do durable cell and gene therapy (CGT) programs experience higher or lower clinical success rates? Estimating the Probability of Technical and Regulatory Success (PTRS) … Read More
Innovating methods for planning, producing, and using real-world evidence
Real-world data (RWD) are a critical element of the evidence needed to help reach this goal, potentially informing a new drug’s development or providing insight for its use after it … Read More
How can self-insured employers prepare for the portfolio impact of highcost gene therapies coming to market?
Self-insured employers (SIEs) currently rely on traditional stop-loss insurance to protect against unexpected high-cost claims, including those for cell and gene therapies. Given that current approvals are in rare diseases, … Read More
Precision for Whom? Partnering with patients to ensure that Precision Medicine targets what matters
Despite decades of growth and adaptation in patient engagement, knowing or being able to find guidance for whichmodel or approach will be most impactful in which healthcare or research context … Read More