What involving patients has changed in real programmes
Patient involvement can change research priorities, the outcomes a programme measures and the information patients receive. Practical examples show patients helping shape development programmes, deciding what treatment plans contain and influencing how their experiences are represented.
A spinal muscular atrophy programme
A spinal muscular atrophy (SMA) collaboration between Roche, PTC Therapeutics and the SMA Foundation began in 2011, with patients, caregivers and advocates involved from discovery through clinical trial planning and design to health-authority submissions and access to treatment. The SMA Foundation, SMA Europe and Cure SMA helped set research priorities.
Patients' and caregivers' emphasis on maintaining independence in everyday life led to co-development of a novel SMA independence scale. The collaboration also prompted a clinical meaningfulness study of the Motor Function Measure 32, the primary endpoint of a pivotal trial. This qualitative interview and survey study was designed with a panel of SMA experts including SMA Europe and Cure SMA.
An advisory board shaping treatment plans
A patient advisory board was involved from beginning to end in developing visual treatment plans for patients in a haematological oncology unit. The plans omitted medication names because the board's former patients said that, at the beginning of treatment, they trusted their doctors and did not want that information, although they might want it if treatment stopped working. Doctors had wanted the names included, but the patients considered them too much information. Patients wanted the option of seeing the plan with or without side effects, particularly to answer questions such as when their hair would fall out.
Patients shaping visual materials
Patients may want to be depicted differently from the way producers imagine them, so they should be asked directly how they want to be drawn in illustrations, comics and videos. Depicting a diverse population allows people to identify with the characters.
By 2024, Boehringer Ingelheim had developed its graphical-abstract template for lay summaries using patient feedback and was regularly testing its lay summaries with patients. Patient experts also gave positive feedback on an example visit schedule: they found it easy to read and understand and thought the icons helped understanding.
Patients represented in a meeting's visual record
At a pharmaceutical-company event bringing together patients, doctors and politicians to incorporate patient perspectives into research, patients initially feared that they lacked the technical language to participate alongside doctors. A graphic recording included drawings of them, and they became the people who shared it most because they felt their contribution had been seen and could recognise themselves in it.