Why and how to involve rare disease patients in the evaluation process of a new health technology?

The Health Technology Assessment (HTA) process is a mandatory procedure in development and commercialization for pharmaceutical companies wishing to bring new drugs to market. Following a patient-centered model for the new technology assessment process offers important opportunities for access to innovation, especially important in rare diseases.

In rare diseases, there are so many unmet needs that access to treatments is very difficult. In many cases, patients feel they must take the initiative in the search for treatments, even involving themselves in the clinical process as they desperately seek solutions. For both pharmaceutical companies and evaluating agencies, patient participation in the evaluation process is vital because no one knows their diseases better than they do.

Evaluating agencies and pharmaceutical companies have a responsibility to involve patients, especially in rare diseases that are so poorly understood. These bodies can include patients in their committees and meetings, and pharmaceutical companies should develop a strategy to involve patients that includes data collection for the pricing and reimbursement dossier.

To learn more about why and how to involve patients with rare diseases in the process of evaluating a new health technology, read the interview with Alira Health experts Annabel de Maria, Chief Patient Officer and Ahmad Bechara, Executive Vice President, Global Pharma, "The Importance of Involving Patients in the Health Technology Assessment Process for Rare Diseases."