Not seeing the wood for the trees: why we should PROMise to do better

I hate acronyms, but I’m as guilty as the next healthcare professional and use them daily in the NHS without thinking whether people know what they mean. When I mentioned the importance of PROMS recently, I was met with perplexed faces. I explained PROMS are Patient Reported Outcome Measures, but the confused looks didn’t change. I have to admit it wasn’t until the last few years that I really became aware of PROMS and their value.

As Don Berwick – the Administrator for the US Federal Centre for Medicare and Medicaid Services, summed up as long ago as 1997, “The ultimate measure by which to judge the quality of a medical effort is whether it helps patients (and their families) as they see it. Anything done in health care that does not help a patient or family is, by definition, waste, whether or not the professions and their associations traditionally hallow it.”

Our CF healthcare team see this almost daily in clinic. What we measure incessantly might have no importance to the person with CF or their family. Often in healthcare, we do not capture what is important to those with lived experience or measure how that changes with time or therapy modification. Recording and acting on PROMS can help facilitate patient-centred care and can act as a screening tool to identify the presence of a condition, such as depression or anxiety.

It has been shown that reporting on PROMS can improve communication between people with CF (and their families) and the healthcare team, leading to greater satisfaction, improved adherence and better outcomes. PROMS are increasingly being used in clinical trials to measure how effective a therapy is, as well as by health economics teams.

The first recorded PROM was by Florence Nightingale over 100 years ago, when she recorded whether her patients were relieved, unrelieved or dead. Thankfully PROMS have moved on as healthcare has progressed, and the first widespread use of PROMS in the NHS was in 2009, used for surgical procedures like varicose veins and hip operations.

Cystic fibrosis is a complex life-long condition affecting the entire body, causing a wide range of challenging symptoms. This means that designing tools that capture outcomes important to people living with CF can be difficult. There are a range of PROMS that have been used in CF care, and perhaps the most well-recognised is the Cystic Fibrosis Questionnaire-Revised (CFQ-R – another acronym—apologies!). This questionnaire can be completed on paper or online and has been tested to reliably measure the impact of CF on overall health, daily life and perceived wellbeing.

This questionnaire has been used in clinical trials and has 12 themes within it, each looking at a specific component of health, including respiratory health, digestion and emotion. Historically in drug trials, the CF academic and pharmaceutical community have been most interested in the respiratory section, as a change in a person’s respiratory score is deemed important clinically. These scores and changes after a new treatment have been used to help design trials and determine whether a new therapy is effective.

There is now increased interest in looking at the other 11 components of the score, which reflect changes in the body outside of the lungs, which people with CF and their families have said are important.

The UK CF Registry is the envy of many disease conditions, and I believe it has been really important in helping to drive forward research and quality of care in the field of CF. We are grateful that over 99% of people with CF in the UK contribute to this powerful data resource which researchers from across the globe can apply to use in an anonymous or pseudonymous format.

The UK CF Registry now collects this quality of life data using the CFQ-R scores, which people with CF and their families kindly take the time to fill in, and CF centres take time to input into. This means that as a community, we have the ability to look at quality of life data - arguably the most important outcome to measure when looking to see how people with CF are doing and the effect of therapies on wellbeing. To date, the UK CF Registry leads the way on this data.

Five big questions still remain on PROMS, which we need to work hand in hand with people with CF and their families to answer.

We need to make sure that we continue to measure what is important to people with CF. Many people with CF have commented that some PROMS we use were developed many years ago and don’t seem relevant to life with CF right now. But there is excellent work ongoing looking at developing new PROMS such as the CF Tummy Tracker and PRO-CF.

We need to recognise the additional burden measuring PROMS can place on people with CF and their families, and I have witnessed the look of horror at the length of some questionnaires. So knowing who to ask for PROMS, when and where to complete them, and how to collect are also going to be important.

In capturing relevant Patient Reported Outcomes Measures, I feel we can further advance progress in the field of CF, bringing new therapies which improve outcomes that are important to people with CF and their families. Furthermore, for people with CF and their families, owning their PROM data and being able to see trends over time which they can discuss in clinic, could help improve communication, care and, ultimately, health outcomes.

Cystic fibrosis (CF) is a genetic condition which causes sticky mucus to build up in the lungs and digestive system. It affects more than 10,900 people in the UK. One in 25 of us carries the faulty gene that causes it, usually without knowing.

Since 1964, we've supported people with cystic fibrosis to live longer, healthier lives -and we won’t stop until everyone can live without limits imposed by CF.