Rare Inherited Anaemias PSP Terms of Reference


Published: 15 October 2020

Version: 1

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Introduction to the James Lind Alliance and priority setting

The James Lind Alliance (JLA) is non-profit making initiative which was established in 2004 with the aim of enabling groups of patients/carers and clinicians to agree together on priorities in treatment uncertainty research. The JLA thus facilitates Priority Setting Partnerships (PSPs) in particular conditions.

Each PSP consists of patients/carers and their representatives, and clinicians, and is led by a Steering Group. Collaboration of this sort whereby patients and clinicians together set the research agenda is extremely rare, but vital in drawing issues to the attention of research funders which might not otherwise be suggested or prioritised.

The role of the Rare Inherited Anaemias PSP is to identify questions about treatment which have not been answered by research to date, and to then prioritise these. An interim priority setting exercise takes place over email/by post, and a priority setting workshop is then convened where partners debate and finally arrive at a top 10 list of shared uncertainties which are most important to them.
The aim is then to “translate” these uncertainties into research questions and obtain funding for that research.

Further details are at www.jla.nihr.ac.uk

The Rare Inherited Anaemias Priority Setting Partnership

Patients suffering from rare inherited anaemias may take years to arrive at a precise diagnosis, if at all. Lack of specialist knowledge, or poor access to the few centres of specialist expertise in these conditions, means that not all patients with these disorders will have access to the same standard of care. Often, lack of research into these rare disorders means that few treatments are available if at all, and even when there are treatments, there is inconsistent access to these for all patients across the UK and incomplete knowledge of when each treatment should be instituted. A PSP into rare inherited anaemias will provide an opportunity for uncertainties in the diagnosis and management of these conditions to be identified with a view to focusing research into these disorders.

The wider aims and responsibilities of the Rare Inherited Anaemias PSP are set out in the JLA Protocol.

About the Steering Group

The Steering Group is responsible for coordinating and implementing the activity of the PSP. Drawing on members’ expertise and networks, the Steering Group will help encourage membership to the wider PSP and, where members have the capacity and expertise, will carry out the practical work needed to collate the interim and final priority setting exercises. The Steering Group is also responsible for ensuring research funders are made aware of the final top ten uncertainties.

Membership of the Steering Group includes representatives of organisations which can reach and advocate for patients and clinicians, as well as JLA staff. Like the rest of the PSP, Steering Group members are expected to participate in the priority setting exercise.


The Rare Inherited Anaemias PSP Steering Group members are expected to participate in a number of specific tasks during the course of the priority setting process:

  • Taking part in meetings –face to face meetings at key points in the 12-18 month timeline, and monthly telephone conference calls to update on progress, which will last 1-1.5 hours and for which the quorum for decision making is 2 clinical and 2 patient representatives (this may be revisited at any time should the Steering Group wish to do so).
  • Steering group members who are unable to participate to ensure comments are fed back to the co-ordinator before the meeting.
  • Ensuring that allocated actions are completed in the agreed timeframes.
  • Publicising the initiative to potential partners to encourage them to join the PSP. This includes: advising on membership of the PSP (to ensure a wide and representative group of patients and clinicians); emailing contacts to invite them to participate; and keeping all small rare anaemia groups informed.
  • Managing the collection of treatment uncertainties from patients, clinicians and existing literature.
  • At the interim priority setting stage - identifying organisations that may be interested in funding research, and identifying ways of promoting to those organisations once the top 10 has been published.
  • Collating the results of interim priority setting and producing an “interim top 20-30” to take to the final priority setting workshop.
  • Participating in the final priority setting exercise: a one-day workshop which brings partners together (one representative per organisation) to debate, rank and agree a final top ten.
  • Preparing a plan and publicising the final top 10 uncertainties to the sector and to all relevant research funders. This includes working with the National Institute for Health and Care Research Evaluation, Trials and Studies Coordinating Centre (NETSCC) to develop applicable priorities into research questions.

Level of involvement and resources

Steering Group members are asked to contribute, at a minimum, their expertise and their time. Basic involvement, as listed above requires:

  • an interest in the initiative and outcomes being pursued in the project
  • a broad understanding of project management issues and the approach being adopted
  • commitment to working with other members respectfully and constructively
  • advocacy for the project’s outcomes
  • prompt feedback on prioritisation materials
  • participation in the monthly telephone conference calls
  • the sharing of networks and contacts for membership of the PSP
  • participation in the one-day final priority setting workshop
  • taking the time to bring the top ten Rare Inherited Anaemias uncertainties to the attention of funders


The Steering Group will be supported by the NIHR Oxford Biomedical Research Centre James Lind Alliance Project Manager. This includes making arrangements for all meetings and workshops, and ensuring:

  • requests for agenda items are discussed with the group;
  • papers will be available at least a week before meetings;
  • meeting notes will be circulated within two weeks.


See Appendix 1

Key contact

Sandra Regan – Patient Involvement and James Lind Alliance Project Manager
Patient Involvement Working Group | NIHR Oxford Biomedical Research Centre
Churchill Hospital | Joint Research Office |Block 60 | Headington | Oxford OX3 7LE
Tel: (01865) 223298| E-mail: sandra.regan@ouh.nhs.uk |