Elevating Value Dossiers with Patient-Centric Strategies

Patient centricity should be considered before you even start your value dossier, at the evidence generation planning stage, to ensure that you capture the relevant data and can clearly convey the patient-relevant value of your technology to payers.

Patient evidence should ideally convey which aspects of their experience are most important to them, as well as real-world consequences for patients that may not be captured by commonly used clinical endpoints. Patient-centric evidence generation may involve: including additional clinical endpoints in your trial that capture the outcomes most valued by patients (ideally selected in collaboration with patient representatives; these will be specific to the condition in question but may include, for example, specific burdensome symptoms that impact QoL), measuring patient-reported outcomes and patient-reported experiences using quantitative approaches (e.g. patient surveys or robust patient preference studies), or gathering representative patient quotes and qualitative descriptions of the ‘patient journey’ to include in your evidence base. Engagement with clinicians and patients during the evidence generation planning stage can further validate the relevance of your intended trial endpoints and wider evidence generation plan.

The patient perspective can be integrated throughout your value dossier to increase the impact of your technology’s narrative. Complementing conventional clinical and economic data with patient evidence provides a broader perspective on the disease burden, unmet needs and patient value of the treatment, capturing aspects which may have been otherwise overlooked. While including the patient perspective is valuable for any value dossier in any indication, it can be particularly useful in indications with a paucity of other data, such as in rare diseases (see our Data Detectives article for tips on how to tackle the unique challenges associated with value dossiers for rare diseases). Value & Access teams should collaborate with Patient Engagement and Advocacy teams where possible, to leverage any existing patient-centric materials and insights that can enhance your value dossier.

• Understanding disease burden and unmet needs: Your value story should effectively convey the ‘patient journey’ by considering all aspects of the patient experience, including impacts on their QoL, daily activities, social engagement and treatment-related burden. Treatment-related patient experience data could be used to describe challenges/preferences around treatment administration, convenience and adherence, as well as the adverse events that have the greatest impact on their day-to-day lives.  Representative patient quotes can bring QoL data to life, deepening decision-makers’ understanding of the patient’s lived experience. Furthermore, highlighting indirect cost and resource use due to productivity loss, work absences, and need for additional support related to current treatment options, helps to paint a complete picture of the existing economic burden for payers. In many conditions, particularly chronic or severe diseases, the caregiver perspective is just as important as that of the patient, so the value dossier should also capture the shared burden and impact of the condition on the caregiver’s well-being.

• Demonstrating patient and caregiver value of your treatment: Patient and caregiver evidence can supplement clinical data to demonstrate how a new treatment meets unmet needs identified by those directly affected by the condition. For example, incorporating data from discrete choice experiments (DCE) and patient preference studies can highlight the treatment attributes of highest importance to both patients and caregivers, and the trade-offs they are willing to make. This can be very valuable, as the attributes that patients and caregivers value may differ from those valued by clinicians. Including patient-relevant outcomes data from clinical trials can further support the value of a new product for patients, demonstrating the impact of the product on the outcomes which matter most to them.

Patient-friendly writing increases the accessibility and understanding of medical research. While value dossiers generally convey complex information ultimately intended for healthcare professional or payers, including patient-friendly components allows value dossiers to also be leveraged for patient engagement and advocacy activities, ensuring key information is aligned across your materials and consistently communicated with patients. Using clear, concise language and incorporating summary boxes and/or strategic formatting throughout a dossier can help to make it clear to users which information is readily available for sharing with patients.

As patient involvement becomes more standardised in HTA processes, educational materials (plain language summaries [PLSs], videos or patient leaflets, for example) which support collaboration with patients and their caregivers may become increasingly important in the value dossier development process. PLSs of the disease background and treatment under assessment are particularly beneficial for use in activities engaging patients and their caregivers, facilitating meaningful dialogue with the patient community involved in local value assessment and reimbursement processes. Indeed, several HTA agencies (for example, NICE and the Scottish Medicines Consortium [SMC]) require HTA submissions to be accompanied by a summary of information for patients (SIP); including PLSs in your value dossier can therefore streamline the preparation of these essential components during the submission process.

Including patient representatives in the value dossier development process can result in more compelling and relevant value narratives that not only inform readers but also advocate for patient needs, while also empowering patients and caregivers to actively participate in the healthcare dialogue – ultimately driving better health outcomes and fostering trust within the patient community. Patient representatives, or a member of your internal Patient Engagement team, should review any patient-friendly content included in your dossier, ensuring its relevance and accessibility, and could also review the dossier as a whole to provide a patient perspective on your overall narrative and identify any gaps in the value story from the patient perspective. Given the increasing recognition of the importance of the patient perspective by HTA bodies, obtaining patient input early in the value dossier development process can be beneficial not only for your value dossier but for the downstream HTA process as well.