1. Patient and public involvement (PPI) in setting the research agenda Dr Noémi Roy Oxford Molecular Haematology

2. What is the research agenda? This is the topics that funding bodies accept as being reasonable, valid and worthy of funding in each field It is partly dictated by researchers’ own interests (intellectual), drug companies’ interests (financial!) and to a certain extent some political guidance (eg Government putting extra funding into dementia research)

3. Why should patients be involved? Ethical reasons: democracy, accountability, empowerment Because patients are the ultimate beneficiaries of the research, involving them in setting the topics increases the RELEVANCE, the VALIDITY and the QUALITY of the research

4. How can patients be involved? This can de done through a PSP (priority setting partnership), which brings together patients/carers and clinicians in a structured way (eg through a JLA partnership- see later) A list of “top 10 topics” is created at the end of the PSP

5. Does this actually make a difference? More and more, funding bodies will look at the topics selected by a PSP and if a research proposal is asking for money and the topic is not one of the “Top 10” chosen by the PSP, it will be very difficult for it to get funded. This ensures the “Top 10” topics really do get money channeled to them

6. What is the JLA? This is the James Lind Alliance. What the JLA does: The JLA facilitates Priority Setting Partnerships. These bring patients, carers and clinicians together to identify and prioritise for research the treatment uncertainties which they agree are the most important. The JLA believes that: – addressing uncertainties about the effects of treatments should become accepted as a much more routine part of clinical practice – patients, carers and clinicians should work together to agree which, among those uncertainties, matter most and thus deserve priority attention

7. (Out of interest, who was James Lind??) Who was James Lind: The James Lind Alliance (JLA) is named after a pioneer of clinical trials, James Lind. Two hundred and fifty years ago, there were many conflicting ideas and unanswered questions about how to treat the deadly disease scurvy. James Lind – a Scottish naval surgeon – decided to confront this uncertainty by treating his patients within a clinical trial comparing six of the proposed remedies. His trial showed that oranges and lemons were dramatically better than the other supposed treatments.

8. How long does it take? The time scale for a Priority Setting Partnership will vary depending on scope and resources. Normally the process will usually take between 12 and 18 months to complete.

9. What about rare anaemias? We in Oxford are setting up a JLA PSP to try to identify the top 10 priorities for patients with rare inherited anaemias, and the clinicians looking after them We will work with clinicians all over the UK, and with as many patient groups as are willing to get on board

10. How does it work? The Priority Setting Partnership is led by a steering group. This has clinicians and patient representatives on it. Using the JLA’s guidance, the groups sets out how it will collect the data- paper surveys, online forms, face-to-face workshops, etc After the surveys have been filled in and the workshops completed, all of the data is looked at by the data handler

11. How does it work? The data handler looks at all the responses and groups similar responses together into different questions Once all the different questions are clearly set out, patient representatives and clinicians together agree on the “Top Ten” questions that they feel research should focus on The steering committee publicises the results to funding bodies and participants

12. What about DBA UK? You are the largest support group of any type of inherited anaemias in the UK You have an excellent track record of interest in research and patient advocacy You are very organised and have regular activities with your members

13. Now what? We would be interested in: – Having someone from your Committee on our PSP Steering Committee – Talking to your members at the Family Weekend about PPI and the JLA partnership we are setting up – Sending the surveys to your members – Holding workshops with some of your members (eg at the adult meeting)

14. Any questions? Please don’t hesitate to contact me: noemi@romahi.com