The interface with patients is integral to UK-AILD. As the principal beneficiaries of the research, and key study partners, the proposal has been informed and developed with patient partners. They will play a key role in the delivery and oversight of the programme. We are keen, however, to take the principle of patient and public involvement to a higher level and have developed, in conjunction with our patient partners, a novel UK-AILD Patient Programme. Key elements of this programme are:
- Future Research Priority Setting: AILD is associated with substantial unmet need, PSC representing the most pressing case, lacking as it does any approved therapy. Success in one aspect of our work has the potential, however, to unearth future challenges including symptom and quality of life issues, and the challenge of implementing emerging therapies into practice. A key element of the UK-AILD Patient Programme will be the first-ever comprehensive priority setting exercise in AILD to develop the future programme of research in AILD moving beyond the immediate target of developing and evaluating better therapy.
- The Patient Passport: One specific area of concern for patients, identified in the development of this programme, is access to emerging therapy. There are major challenges around clinician awareness of optimal therapy in complex rare diseases. Whilst there are emerging “top down”” initiatives such as the RARE-LIVER ERN that we lead, our patient partners believe there is a real opportunity for a patient-led initiative around clinical status awareness-raising, with a simple “traffic light” patient passport, based on current guideline-based optimal care, being developed and disseminated by patient group partners. Driven by simple clinical criteria available at all consultations, the passport would increase awareness of areas of clinical need and empower patients to discuss with their clinicians where care could be optimised. It will also deliver tools for patients and clinicians that communicate risk stratification in a way that can be translated into clinical management i.e. real world tools for risk stratification may have greater true impact and uptake directing patients to new therapies or trials, if a compromise on stratification accuracy is adopted, in favour of user acceptability and implementation.
- Patient-Initiated Research (“The Crowd-Sourcing Model”): The ultimate expression of patient involvement in research would be to move from supporting programmes to initiating and eventually delivering research through the structures and systems that the patient groups operate. In addition to the patient passport model we will work with the patient partner groups to identify a priority research question which would be tractable for this model, and explore the feasibility of patient driven resource generation and project delivery. Our patient partners and we believe that this would have the potential to be a uniquely powerful model to challenge public apprehensions around medical research.