Cell and gene therapy: ways to generate evidence to demonstrate value
Although cell and gene therapies (CGTs) are typically expensive products, there are techniques that pharmaceutical companies can execute to underscore why they are worthwhile.
By on Feb 23, 2024It is estimated that at least 50 CGTs are likely to be approved between now and the end of the decade. It is therefore imperative for pharma companies to understand the needs of all stakeholders and learn how to successfully communicate their value.
Actionable Insights:
- Patients can be powerful communicators who can explain to regulators, payers, and policymakers’ areas of unmet need: These stakeholders should be informed of unmet patient needs as early as possible to ease potentially challenging value conversations down the line
- Pharma companies should have a unified internal approach to communicate their therapies’ value: Departments should work together on projects, and not just be in the same meetings, to strengthen collaboration
- Thoughtful data aggregation plans – from the pre-clinical stage to post-approval, real-world stage – will be pivotal in underscoring value: Because payers are data aggregators, they should be key participants in developing outcome-based agreements
To successfully communicate the value of a new CGT, it is important to target and listen to all stakeholders: patients, clinicians, regulators, payers, and policymakers. To understand what they deem valuable, there are formal and informal ways to collect feedback, from market research and advisory boards to casual conversations at a medical congress.
Advocacy groups are critical in learning about specific patient needs, as well as improving a new therapy's market prospects. In fact, patient testimonials can be a powerful way to communicate unmet needs to other stakeholders. Regulators, payers, and policymakers should be informed of unmet need as early as possible. Once a therapy is ready for regulatory or reimbursement review, stakeholders would already understand how the therapy would be valuable to patients.
When crafting the value story of an expensive product, the company should approach it holistically. According to Takeda Pharmaceutical’s Lung-I Cheng, the company does not see product development as a relay race, but as a team sport. Before Takeda starts reaching out to regulators, there is already a clear communications strategy within the company on how to demonstrate value.
To strengthen internal collaboration, pharma company departments should work on projects together and not just be in the same meetings. All departments should work within a common framework; there should be an evidence generation plan with contributions from all departments, from the drug development team to the market access team. Collaboration can help identify gaps and highlight important elements that would underscore value.
There are third-party service providers that can mediate between pharma companies and payers. The advantage of outsourcing is that the pharma company can skip building such a team internally and focus on other strategies. These vendors also have reimbursement expertise. But there should be clear communications between the pharma company and vendor on expectations. Working together is still paramount to successfully communicate a positive value story.
A robust evidence generation strategy is key to successfully getting a new therapeutic approach to market. At present, there are three key areas where data collection misaligns: clinical trial design, clinical trial performance, and real-world evidence (RWE). There are often different perspectives of best study designs such as efficacy measures, or different expectations of what constitutes a successful study. As for RWE, standards of care can change.
The experience of communicating a therapy’s value to payers and regulators can be different between companies that developed the asset from the pre-clinical stage, versus those that acquired a late-phase asset from another company. The advantage of the former scenario is that the company can collect value-driven data as early as pre-clinical research. Nevertheless, all companies will still need to prioritize what data points it should focus on to tell a clear, consistent story of a therapeutic candidate’s clinical value.
To convince payers to reimburse expensive therapies, real-world, long-term data collection will be necessary to keep demonstrating their clinical value. Since payers are data aggregators with large datasets, they should be key participants in developing outcome-based agreements. There should be a pragmatic and targeted approach in collecting real-world data (RWD) as not all data points are relevant to demonstrating value. For example, there are laboratory values that might be more relevant during the early stages of product development but less in practice.
CGTs are one-off treatments designed for long-term benefits. But with relatively short-term data available, it can be a challenge to communicate value to payers. A way to address this information gap is to pinpoint what other healthcare costs the CGT can likely offset. Such an approach can make a therapy’s value more concrete. Metrics underscoring improved quality of life can also be used, such as shorter patient wait times.