Value frameworks in the United States have highlighted the need for companies to consider value and affordability calculations for their products earlier in the life cycle. Payers are using independent reviews, such as from the Institute for Clinical and Economic (ICER), in price negotiations at market launch. Predicting an ICER review is difficult but evidence generation that flows into value calculations is beneficial regardless of an independent review from ICER or other value frameworks. Understanding key drivers of value for your products can inform evidence generation and market access discussions for your product. Here are three key questions I often receive from companies regarding a potential ICER review and a few tips to address each question:
1. How can value and affordability calculations help us earlier in the life cycle of our product to prepare for a potential review from ICER?
Develop an early phase model to inform value and affordability arguments. Many companies do not involve cost-effectiveness and budget impact calculations until later in the life cycle (i.e., during phase 3). Building a model for your product can inform more than just value and affordability. Models are designed to reveal the relationship between inputs, assumptions, and outputs. For example, over half of clinical trials use a surrogate endpoint that is not linked with long-run survival and quality of life outcomes. Building a model can help you connect the dots between the surrogate outcome and endpoints patients and payers care about. Further, models can reveal data gaps early in the life cycle to inform future evidence generation either through your phase 3 trial program or through using real-world evidence.
Understand the preferences of your end user. Not enough companies collect and submit patient preferences data around additional value criteria to ICER to inform the “Other Benefits and Contextual Considerations” chapter. I know firsthand these other benefits can be very influential for voting panels during deliberations. What evidence do you have around patient-centered outcomes? Can you quantify how your product influences these outcomes? If possible, provide detailed information from focus groups or any other direct interaction you’ve had with patients on additional benefits from your treatment.
2. We found out one of our products is being review by ICER. How can we communicate with ICER and what evidence should we provide – if any?
Develop a communication team and strategy. There are multiple points of communication with ICER where you can influence the outcome – scope, model analysis plan, and draft report. Prioritize your arguments for these three key time points. Dedicate a small cross-disciplinary team to develop a communication strategy, with one point person that solely communicates with ICER. Hire at least one outside advisor not close to your product to provide objective feedback on your communication and dissemination approach. Listen closely to their feedback and adapt quickly to suggestions. The communication strategy should guide the ICER clinical and economic teams. In other words, treat this interaction as a collaboration.
Rely on evidence-based arguments. Communicate only evidence-based arguments with no additional opinion attached (this includes marketing arguments). Understand the decision context (i.e., “what’s a fair price for our product?”) and fit your arguments within that decision context. Is your product targeted to rare disease patients or highly prevalent diseases? The type of population matters for communicating your arguments. Be open to submitting information as academic or commercial in confidence after consulting with your outside advisor.
Leverage existing clinical and economic analyses. Identify any tools your teams have already built for comparative and cost-effectiveness analyses of your product. Do you have a network meta-analysis (assuming that is relevant)? Do you have an early phase model built in house or by consultants? Devote the clinical and economic leads on your cross-disciplinary team to use these existing tools to identify gaps and prioritize information sharing with ICER. Be prepared to describe the key drivers of value for your product.
3. What evidence can we collect and publish post launch to improve the value profile of our product after an ICER review?
Disseminate cost-effectiveness and budget impact analyses. If you have a cost-effectiveness and/or budget impact model analysis, make that a priority to submit to upcoming conferences. Try to get as much information out the door as quickly as possible. ICER will often update past reviews with additional published evidence at one year post-review. This means you have to submit your abstracts and papers just a few months after launch. What upcoming conferences will include presentations prior to this one year post-review timeline?
Real-world evidence generation. By this point your trial program has all but ended but your product is now being used in real-world clinical practice settings. Remember earlier in this article when I said your model may reveal data gaps in your trial program? Well these same models may also be helpful in identifying any additional gaps that were not filled in your trial program. For example, this is a great time to start disseminating sub-group analyses that display the highest potential value from using your product. Or there may have been gaps in the ICER review that you can fill that will improve the value of your product. This is the time to leverage real-world evidence to display the benefits of your product in more populations as compared to patients in your trial program.
About Brett McQueen
I am currently a tenure-track Assistant Professor at the University of Colorado Skaggs School of Pharmacy and Pharmaceutical Sciences and Director, Pharmaceutical Value (pValue).
Since 2012 I have worked directly on value assessment methods and applications in the United States and Europe. My training is in advanced decision-analytic modeling and econometric applications. I provide companies with advice on key evidence gaps and strategies to fill those gaps with existing real-world and trial data sources. My job quite simply is to prepare my clients for the unexpected. More recently, I have advised companies on strategies for value-based contracting. My time in both consulting and academia has forced me to stay active on the most rigorous methods in the field while tailoring those methods for use in practice. I’m active in health economics-related societies such as the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) through contributions to ISPOR short courses, workshops, issue panels, and research presentations. I have personally led or collaborated on modeling submissions for health technology assessments since 2015.
My current focus and interests include:
Advanced decision modeling for value assessment applications
Developing novel methods for value assessment including multi-criteria decision analysis
Strategies and tracking of value-based outcomes agreements
Education and training on advanced methods in the field