Joint Clinical Assessments (JCAs) are now live and will apply to orphan medicines starting in 2028. We’d like to take you through 5 steps to success and share some of what we’ve learned from working on JCA submissions.
Early Planning for JCA
Success in JCA begins with meticulous early planning. First up is deciding if a joint scientific consultation should be conducted to ensure evidence meets JCA requirements and if this should be done in parallel with EMA consultations. Then, evaluating the evidence implications of early PICO simulations and assessing whether patient numbers for all European Economic Area states can be estimated. A commercial assessment of early PICO results is also needed. These steps may then lead us to ask, “How can we determine evidence gaps in key JCA requirements for rare diseases?”
Strategy for Pivotal Trials
Strategy for pivotal trials must include findings from PICO scenario planning and joint scientific consultations, if completed. Strict conflict of interest rules must be considered in the clinical expert and patient advocacy group stakeholder plans. It’s vital to ensure that the clinical trial statistical analysis plan meets JCA requirements and that validity requirements for surrogate endpoints are considered. If a synthetic control arm is required, appropriate evidence must be available and risk assessments conducted to develop rationales for any non-inclusion.
PICO Prediction and Validation
Precisely defining the target population and potential subpopulations is critical. We must make sure there is a robust PICO stakeholder plan in each local affiliate, along with a PICO mitigation strategy. Designing the SLR to meet additional JCA requirements in terms of searches and data extraction is essential. Only surrogate endpoints with established validity, such as a correlation threshold greater than 0.85, should be included.
JCA Submission
Generating epidemiological data for European Economic Area states with limited patient numbers is a challenge we must address. The submission dossier must adhere to JCA requirements and reflect the patient perspective. Keeping SLRs and indirect treatment comparisons updated within the last 3 months before submission is necessary to ensure data validity.
Multi-Country JCA Submissions
National HTA agencies cannot request information already included in the JCA, however, requests for country-specific data analyses should be anticipated. Indirect treatment comparisons completed for the JCA should provide all comparative evidence required for country-specific cost-effectiveness models. It is also critical to assess the pricing and reimbursement implications of the JCA report.
It’s not just knowing what has to be done, it’s having experts that can do it. If you are facing a JCA submission, you want to make sure that you're partnering with a company that has worked extensively in the orphan drug space and can guide you through the entire process.
Reach out to our team at RTI Health Solutions to start a conversation about how we can put our experience and technical expertise to work for you as you begin the journey to JCA for your orphan drug product.
