CF Patients Help in Defining Top 10 Priorities for Cystic Fibrosis Research

The top 10 priorities for cystic fibrosis research — set by CF patients and specialists alike — range from learning more about treatments that delay or prevent disease progression, to simplifying therapies so they’re less burdensome and getting patients to keep using a prescribed medication, according to a survey exercise led by scientists at The University of Nottingham.

Priorities were set through a three-part exercise managed by the Cystic Fibrosis Priority Setting Partnerships (PSP), an independent group of CF patients and health professionals sponsored by The University of Nottingham, the Cystic Fibrosis Trust and Nottingham Hospitals Charity, and overseen by James Lind Alliance (JLA) specialists.

JLA, a non-profit organization managed by the National Institute for Health Research (NIHR) Evaluation, Trials and Studies Co-ordinating Centre, brings together patients, caregivers and clinicians in these PSPs. The aim here was to identify and prioritize 10 uncertainties, or “unanswered questions,” about treatments and their effects so that future research might be directed to what matters most to both patients and clinicians.

“The James Lind Alliance Priority Setting Partnership in cystic fibrosis (CF) has brought together over 600 people from across the world and includes an even split of medical staff and lay people. Using an innovative online approach, we have collected over 1000 research ideas and have worked with the CF community to choose the 10 research questions which are the top priority to move into clinical studies,” Alan Smyth, a professor of Child Health, and Head of Division of Child Health, Obstetrics & Gynaecology at The University of Nottingham, said in a news release.

In total, 513 people — an even mix of clinicians and patients/caregivers — responded to the first survey, and submitted 1,122 questions of concern, and 675 completed the second survey, choosing their priority questions from a list of 71 uncertainties.

The top 10 CF research priorities were then determined at a later workshop attended by 40 people, representing the CF community, patients and caregivers, physicians and researchers. In a question format, the priorities are:

1. What are the effective ways of simplifying the treatment burden of people with CF?
2. How can we relieve gastro-intestinal (GI) symptoms, such as stomach pain, bloating and nausea in people with CF?
3. What is the best treatment for non-tuberculous mycobacterium (NTM) in people with CF (including when to start and what medication)?
4. Which therapies are effective in delaying or preventing progression of lung disease in early life in people with CF?
5. Is there a way of preventing CF-related diabetes (CFRD) in people with CF?
6. What effective ways of motivation, support and technologies help people with CF improve and sustain adherence to treatment?
7. Can exercise replace chest physiotherapy for people with CF?
8. Which antibiotic combinations and dosing plans should be used for CF exacerbations and should antibiotic combinations be rotated?
9. Is there a way of reducing the negative effects of antibiotics, e.g., resistance risk and adverse symptoms, in people with CF?
10. What is the best way of eradicating Pseudomonas aeruginosa in people with CF?

Treatment uncertainties are defined as questions regarding medications or therapies that cannot be answered by existing research, such as particular treatment options, methods of care, or diagnostic tests.

“This is the first time such a collaborative and representative process has been undertaken in CF. We will now work with NIHR, the UK CF Trust, the US CF Foundation and the UK Research Councils to get these research questions funded and taken forward by experienced and collaborative researchers,” Smyth said.

A CF patient at the workshop, Rachel Taylor, described the exercise as “a very positive and purposeful project,” adding she felt that participants had “really achieved a direction and some focus for cystic fibrosis.”