The relevance of patient perspectives to value: Patient Involvement in Health Technology Assessment

Pharmaceutical manufacturers are increasingly recognizing patients as consumers. Their voices, preferences, and behaviors are crucial to whether a product is able to deliver what the market needs and is willing to invest in.

Understanding patients’ own definitions of an acceptable and valuable treatment enables a product to differentiate its value from the competition. It should also improve the likelihood of patient retention in trials and adherence to treatment.

Patient engagement strengthens the whole product development cycle.

Patients and their families have unequivocal insights into a disease and the treatments for it. These insights situate the benefits and risks of a new product within a real-world context and help decision-makers understand the potential value of a new treatment from patients’ perspectives.

Patients can also articulate the most important outcomes and goals for a new treatment. Asking a patient “what does a successful treatment look like?” uncovers issues around a treatment’s convenience, out-of-pocket costs, and the relative importance of survival and quality of life gains that are not captured in clinical trial data.

Regulators and health technology assessment (HTA) agencies are taking increasing account of patient insights in their assessments of new treatments. This is particularly true where treatments are associated with considerable toxicity, or incremental survival benefit. Patient involvement can also provide insights that justify treatment claims for improving symptoms or quality of life.

Patient experts have traditionally shared their views during committee meetings or in consultation responses. Today, patient preference data from rigorously conducted studies are providing decision-makers with a wider pool of views on the outcomes that patients value. While there is not yet a formal requirement for patient preference data by either regulators or HTA agencies, the data are often taken into account as part of evidence reviews.

As regulators and HTA agencies gain more applied experience of integrating patient perspectives data in their review processes, manufacturers will need to come to market armed with patient insights data to support their product.

>> Read the second piece in this series: “Patient involvement is crucial to product development and assessment of value”