Implementation Brief: Considerations for developers in applying the Multiple Best Price (MBP) rule to improve access to novel therapies

Eric NormanFoCUS, Research Briefs

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

Eric NormanFoCUS, Research Briefs

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?

Eric NormanFoCUS, Research Briefs

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?

Eric NormanFoCUS, Research Briefs

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?

Eric NormanFoCUS, Research Briefs

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

Eric NormanLEAPS, Research Briefs

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?

Eric NormanFoCUS, Research Briefs

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

Eric NormanLEAPS, Research Briefs

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

Payer perspectives on outcomes tracking for value-based payment arrangements (VBPs)

Eric NormanFoCUS, Research Briefs

Share the researchWith the new CMS rule changing Medicaid Best Price reporting to encourage VBPs, many options are available for which healthcare system players will collect, protect and adjudicate the … Read More

The resource navigation challenges for patients and caregivers

Eric NormanFoCUS, Research Briefs

Share the researchWhile the problem of finding system-wide, implementable precision financing models for curative and durable therapies involves all stakeholders, patients and caregivers are directly affected by many different elements … Read More