LifeARC Centre for Acceleration of Rare Disease Trials

This Centre is a consortium between University of Birmingham, Queens University Belfast, and Newcastle University. We aim to boost the capacity and efficiency of rare disease trials across the UK – including facilitating equitable recruitment, streamlining administrative hurdles and offering a ‘one-stop shop’ for trial design and support.

Ultimately, the team hopes to help speed up the time it takes for rare disease treatments to reach those who need them.

What we will do

We will develop a UK 4-nations project to deliver trials of new treatments for Rare Diseases (RD), in ‘one stop’, patient friendly models, to get more treatments to more people, faster.

Why are we doing this

There are too few treatments for PLWRD. Many treatment trials fail due to ‘bottlenecks’ in set-up and delivery, with little evidence that they work. We want to remove these bottlenecks.

How we will do it

We will work with PLWRD to help them be ready to take part in treatment trials with outcomes most important for them. We will recruit teams to deliver new types of trials with ‘nothing about us without us’ trial designs, so that new treatments can be quickly approved for use in the clinic.

How we will involve patients and public

PLWRD are members of our team, and will take part in all decisions for project. We will also work with Genetic Alliance UK and other patient support groups to make sure that all PLWRD with different needs will be fully supported.

Explaining our work to others

As well as engaging with PLWRD, we will engage with diverse RD communities and provide regular updates for the UK RD research community.

Centre work packages

Work Package 1: Facilitating Recruitment

We will:

a) co-develop (with WP A, B, and commissioned external design experts) a secure online RD Recruitment Portal (RDRP) for PLWRD to self-register (Appendix 7). This will include data sharing consents for input by PLWRD, their carers and health care practitioners (HCPs), and an open access Clinical Trial Finder;

b) develop core datasets to facilitate pre-screening and recruitment of patients to trials, partnering with key NHS/NIHR infrastructures and disease specific LifeArc Translational RD Centres (TRDCs);

c) link to NHS digital products, allowing collection of ‘real world’ natural history data to inform Patient-Reported Outcome (PRO) selection (WP2) and synthetic controls for innovative trial designs (WP3);
d) house data in a secure data environment (England), and a federated structure (NI, Scotland, Wales).

Work Package 2: Patient-Reported Outcome Selection

We will:

a) identify PRO tools for a standardised approach to PRO collection in trials, starting with those already supported by FDA (with WP5), for diseases prioritised in WP1;

b) explore barriers to and facilitators of inclusive PRO collection (with WPA,B);

c) Work with WPB to develop consensus guidelines on use of PROs supporting EDI in RD trials;
d) offer PRO design support (with WP3). 

Work Package 3: Trial Design

We will:

a) create RD clinical trial capacity to support: a) delivery of a portfolio of trial proposals (LifeArc Pathfinder projects, other successful TRDCs);

b) incorporation of innovative trial designs and state of the art statistical methods (with MRC RD Challenge node ‘CAPTIVATE’;

c) integration of health economic input into trial design (with WP5), PPIE strategies (with WP A) and approaches to make recruitment to trials more diverse (with WP1, WP B);

d) external grant applicants applying for trial funding,

e) collaborations with industry partners to deliver ‘fit for filing’ trials for marketing authorisation applications (de Wilde B et al 2022)

f) with external funding, develop all trial documents, set up and delivery of trials (with WP4).

Work Package 4 Accelerating Trial Delivery

We will:

a) set up a RD trials site network, through the Clinical Research Facilities (CRF) in Belfast, Birmingham, Cardiff, Edinburgh, London and Newcastle;

b) work with NHS Hospitals to develop a revised NIHR UK RD Research Consortium Agreement, so that one approval is signed up to by multiple recruiting centres (Ref: Musketeer Memorandum);

c) appoint patient logistics facilitators to support participants and their carers participating in trials (with WP A);
d) work with WP3 to de-centralise trials (e.g. home health monitoring).

Work Package 5 Implementing Innovation in the NHS

We will:

a) construct a Value of Information (VoI) framework, building on our lifecycle HTA framework, to set out RD-specific analyses to aid decision making;

b) develop a standardised approach to patient-informed risk benefit trade-offs through the use of Multiple Criteria Decision Analysis (MCDA);

c) identify regulatory barriers to implementation of findings from treatment trials in RD;

d) convene a Citizen’s Jury to address patient-relevant issues around the Early Access to Medicines Scheme; and
e) work with the UK RD-IMAG group to connect to international RD trial developments and activities.

Work Package A: PPIE

We will enable clinicians and scientists to work alongside PLWRD and their families on a truly collaborative and equal basis to inform the ethical and patient-centred development of the RD-TAP’s activities.

Work Package B: Equality Diversity & Inclusion

We will:

a) promote inclusive patient involvement in each WP through representative sampling particularly of underserved groups;

b) incorporate EDI in trial design (WP3), scoping the barriers and facilitators to participation by diverse communities, and formulate strategies to widen participation;
c) promote EDI in the RD-TAP workforce by equitable and inclusive recruitment.

Work Package C: Training & Capacity Building:

We will:

a) establish a translational science/clinical PhD training scheme;

b) create a dedicated professional/post-doctoral training fellowship scheme in RD;
c) Launch a cross-disciplinary training platform to ensure complementary skillsets are developed within the workforce.