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In the first of her two-part series, Sarah Richard explores the evolution of patient involvement in health technology assessment, and the role of the patient voice in market access.

Sarah Richard is a Consultant with expertise in patient-centric strategy at PRMA Consulting


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-centric product development.

Patient engagement strengthens the whole product development cycle.

  • Patients can improve the design of a study by prioritizing endpoints or highlighting burdens in the protocol associated with monitoring, testing, or attending hospital. They can also guide the selection of relevant patient-reported outcome measures in trials.
  • Patient groups can help review information about a study so that it is understood by patients. They may also publicize studies to patients and generate interest through their own networks.
  • Manufacturers and patient groups can work together after study completion. Results can be disseminated, patients’ experiences on the study can be assessed, and information and education to support patients to make informed decisions about treatments can be developed.

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 HTA agencies are taking increasing account of patient insights in their assessments of new treatments.”

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 insights also are needed to 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.

>> Read: Variability in patient-engagement between European countries

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 engagement is crucial to product development and assessment of value”
PRMA Consulting is an independent consultancy solving some of the most challenging pricing, reimbursement, and market access issues facing international pharmaceutical and biotechnology companies today.

We can offer the following support:

• devising patient engagement strategies and implementing programs
• developing a patient-centric market access strategy for your product or portfolio
• exploring patient engagement opportunities with advocates and representatives
• providing insights and recommendations for or pressure testing engagement projects and programs

For a confidential conversation about incorporating a patient perspective into your market access strategy, please get in touch.

info@prmaconsulting.com
UK (HQ) +44 (0)1252 786284
US +1 (415) 655 6798

Company registration number: 05893400

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