Pharma companies with new drugs headed to market are required to go through the Health Technology Assessment (HTA), and approaching this process in a patient-centric manner is beneficial to market access. Pharma companies play a key role in successfully involving patients in the HTA process, and to learn more, we spoke with Luca Trentin, Senior Consultant, Patient Engagement at Alira Health.

Why is it important to involve patients in HTA activities?

As the end users of healthcare technologies, patients’ experiences, preferences, and needs should represent a central aspect of the decision-making process, giving patients a say in decisions that affect their health and quality of life and ensuring that the HTA process is more equitable and democratic.

Many stakeholders involved in HTA processes may not fully understand the lived experience of those affected by a condition. Therefore, it is crucial for HTA bodies to hear directly from patients to gain a comprehensive understanding of the disease burden, its impact on quality of life, and the potential benefits a treatment may offer.

What role can pharma companies play in patient involvement in HTAs?

Ideally, you should begin to engage with patients early in the drug development process. By involving patients in discussions about clinical trial design, endpoints and outcomes, and their unmet needs, pharma companies can ensure that they understand and include the patient voice throughout the product lifecycle. As a result, the information submitted to the HTA will already contain and reflect the perspectives of the patient community and ideally a patient value proposition.

If we are talking about the direct involvement of the patients in the HTA process, one way to prepare them is to ensure that patients are adequately informed about the products being assessed, including providing data in formats that are accessible to non-experts, for example, plain language summaries. This will help patients to evaluate the potential impact on their lives.

What major challenges do pharma companies face when it comes to patient involvement in HTA activities?

Pharma companies that engage with patients throughout the product lifecycle are positioned for success at the HTA stage. But there are certainly challenges.

For example, the new Implementing Act – JCA Procedure for Medicines for Human Use requires identifying any conflicts of interest when involving patients in the HTA process. Ongoing discussions between the industry, regulators, and patients seek to address this. A key challenge for HTA bodies in this case is the potential conflict arising when the same patients involved throughout a product’s lifecycle also participate in the HTA stage.

Other challenges are out of pharma companies’ control because HTA bodies select the patients who participate in the HTA. These challenges could include a lack of patient understanding of the technical aspects of HTAs which may limit meaningful patient participation. Representation could also present a potential problem, especially when it comes to rare diseases. A specific patient might not always reflect the characteristics of the entire community of people living with that condition. And patients from organized patient groups are so well informed that they might not necessarily represent most people with the disease.

What best practices should pharma companies follow to involve patients in HTA activities?

Early and continuous engagement with patients across the product lifecycle, from the moment of discovery onwards, enables you to understand the patient journey and their real unmet needs. You can collect data directly from patients and create a set of data that complements the dossier you present to the HTA body. That patient data will reflect a more accurate patient experience and add meaning to your clinical data.

On a more tactical level, pharma companies can focus on the creation of clear information and the promotion of already existing HTA patient trainings. Providing technical information in plain language to patients helps them evaluate your product based on the value they perceive. And it is in your best interest to promote the training of patients and patient groups on the HTA process, terminology, and decision-making frameworks. Such training helps patients participate more effectively and ensures that their input is more informed and relevant.

At the recent World Orphan Drug Congress in Barcelona, Alira Health moderated a working group, “Enhancing Patient Involvement in HTAs: Practical Strategies for Stakeholder Collaboration.” Attendees learned more about key issues surrounding patient involvement in HTAs, with a focus on how pharma companies can actively contribute to this critical activity.