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Cystic Fibrosis commissioning brief

 

Contents

Summary

NIHR is calling for research proposals involving people with cystic fibrosis, including the evaluation of interventions that could improve the treatment and management of cystic fibrosis and its co-morbidities and complications.  The call aims to support robustly designed studies with the potential to make a step-change in improving health outcomes for patients, the public and social care services.  We are particularly interested in, studies focused around the burden of care and treatment and how they could be simplified, the diagnosis and management of non-tuberculous mycobacteria, the potential benefits of exercise and possible alternative options to physiotherapy. 

Applications should be collaborative and involve investigators spanning different specialties and a range of those delivering care, methodologists and also service users, carers and patients. 

How to apply

Research proposals must be within the remit of at least one participating NIHR Programme. The Programmes involved in this call are:

  • Efficacy and Mechanism Evaluation (EME)  
  • Health Services and Delivery Research (HS&DR)
  • Health Technology Assessment (HTA)
  • Research for Patient Benefit (RfPB)
  • Programme Grants for Applied Research (PGfAR)

Applicants should note that

  • Applicants should justify the importance of their proposed research and its potential impact on patient care.  
  • Applicants should clearly state how their proposed research addresses an explicit evidence gap and how the research adds value to the existing NIHR research portfolio
  • Patient and public involvement should be included within the application and study design. 
  • Applicants should consider contacting their local RDS for support.

Contact information

Applicants who require further guidance may should contact the Programme they feel is the most appropriate for their research idea.  Applicants may also wish to contact their local RDS to discuss their research idea.   

Deadline for proposals

Application forms will be available from participating programmes websites between 1 October 2020 and 18 November 2020.  Please note that submission dates vary and completed forms must be submitted by the date specified on the relevant Programmes website.

Background information

Cystic Fibrosis is a multi-system inherited disorder affecting the lungs, pancreas, liver and intestine which can have a significant impact on life expectancy and quality of life. Advances in screening, diagnosis and management now means life expectancy has increased significantly over recent years.  It also means that individuals are living with a chronic disease and multi-morbidities.

Cystic fibrosis is caused by mutations within the CFTR gene. Over 2000 CTFR mutations have been described.  The UK registry shows that 96% of people with cystic fibrosis have two known mutation. However, this means that in 4% of people at least one mutation has not been identified.

Treatments include antibiotics, physiotherapy, mucolytics, pancreatic enzyme replacements, vitamins and other therapies. As cystic fibrosis is a progressive disease, patients may require either a lung or heart and lung transplant. At present, approximately a third of CF patients on the list die before receiving a donor lung.

Diagnosis is primarily made during newborn screening. The median age at diagnosis is less than 2 months, and 1 in every 2500 babies born in the UK has cystic fibrosis giving a UK CF population of approximately 11,000 people. For the first time more than 60% of people on the UK cystic fibrosis registry are aged over 16 years.

However, recent updates to UK guidelines together with the recommendations from a recent UK CF community consensus priority setting exercise shows that a significant number of uncertainties remain about the management of CF and its complications.