OBJECTIVES: There is a noticeable increase in the value placed on the ‘patient voice’ in the early stages of drug development, through to launch and post-launch activities. However, how is the patient voice collated and what evidence requirements are considered, and do they help improve patient outcomes and healthcare decision‑making?

METHODS: A targeted grey literature search explored the extent of patient involvement in study design, Health Technology Assessment (HTA) (England and France) and regulatory assessment processes (Europe and US). Patient advocacy websites, industry magazines, HTA and regulatory websites were reviewed to assess how evidence from patients is incorporated into decision‑making.

RESULTS: At NICE (England), patient experts sit on the NICE committee; for HAS (France), patients sit on the committee and share information collated via an online portal.

Patients can provide input at various stages of the regulatory process at the EMA, from pre-submission through to post-authorisation. The FDA have established several patient engagement initiatives, including public meetings, listening sessions and committees. Drug manufacturers often work with patient organisations to help improve the drug development process and gain a deeper understanding of a therapy area. Both quantitative and qualitative evidence is considered.

CONCLUSIONS: It cannot be disputed that patient involvement is highly regarded by all stakeholders, patients add significant value in drug development and the decision-making processes. When we consider patient uptake, patients who have actively participated in decision-making about their treatment will surely be more engaged and empowered and have better adherence to medication and treatment plans, leading to improved outcomes. A number of frameworks exist to help implement patient involvement in drug discovery, access and uptake but do we need to harmonise and simplify the process for patients and stakeholders to help increase patient engagement and amplify the patient voice, particularly for patients from hard to reach groups?