The European Cystic Fibrosis Society (ECFS) is an international community of scientific and clinical professionals committed to improving survival and quality of life for people with CF by promoting high quality research, education and care. For a rare disease such as CF, it is important that countries work together as much as possible. There is also a need for close cooperation between patients, patient organisations, pharmaceutical industry and academic research institutions.

When trying to standardise new or existing working methods (for example to measure lung function or to perform a sweat test), it is important to have an international dialogue. If everybody works the same way, there is less variation in results, which means that less patients have to participate in a clinical trial to prove the same effect.

To promote such cooperation, the European Cystic Fibrosis Society, took the initiative to setup a “Clinical Trials Network” (CTN). Currently the network combines CF specialists from 57 centres in 17 countries and a coordinating centre in Leuven, Belgium.

The aim of the European Cystic Fibrosis Clinical Trial Network is to intensify clinical research in the area of cystic fibrosis and to bring new medicines to the patients as quickly as possible. This is done by:

• Sharing expertise among dedicated CF researchers
• Involving and cooperating with patient organisations
• Centralising review of clinical trial protocols in cooperation with the pharmaceutical industry
• Supporting the study conduct in the clinical trial sites that are part of the network
• Standardising research procedures and outcome parameters and
• Providing training to the site’s staff.

An important task of the ECFS-CTN, is the review of new study protocols by a team of experts. They look at the scientific quality, feasibility for the patient and the therapeutic importance of the tested drug. Centres that are part of the CTN will only conduct trials that have been approved by this review system. In 2021, the ECFS-CTN reviewed and approved 10 commercial protocols from 4 different companies.

Why is it important that the protocol is reviewed by a family member or person living with CF?

“While a family member/CF patient may not be the perfect medical expert, we are experts when it comes to living with CF. I am convinced that our insights can help to make study designs more practical and easier to integrate. Sometimes researchers are not aware of other CF factors – so we can bring in that perspective and make sure it is not overlooked. Anne, PWCF, Germany”

Apart from study design, motivating patients to take part in research and promoting safety of participants in clinical trials are of great importance. Between November 2020 and November 2021, ECFS-CTN sites enrolled 529 people with CF into clinical trials. Three quarters of these were adults. Twenty-eight studies were undertaken in 2021, mostly assessing CFTR modulators. Studies were mostly phase 3 trials, these are trials that compare new treatments with the best currently available treatment, or standard treatment.

CAR-CF

CAR-CF, the Covid antibody response in CF study, is an investigator-initiated trial supported by ECFS-CTN which collects blood samples from people with CF across Europe to detect whether they have had Covid-19 or not. CAR-CF will also look at how well people with CF develop immunity to COVID-19 after vaccination and how this immunity changes over time. The trial is being conducted across 14 countries in Europe plus Canada and the USA. The first results from this study are expected in 2024.

Vincent, a person with CF explains why he is taking part in CAR-CF:

“Fantastic that a study like the CAR-CF study was initiated! The Covid-19 pandemic affected us all very much – with a special threat to all patients with chronic lung diseases like CF. So gaining scientific knowledge out of this trial is essential. And to hear that this trial is performed as a huge European study is great as we need this unification especially in these currently very difficult times. I am proud to be a part of it”

PRO-CF Patient Reported Outcomes in CF

There are many ways of measuring whether a person’s CF is stable, getting better or getting worse. One deceptively simple way is just to ask the person – after all nobody knows CF better than those living with it. However, turning the question “how are you feeling?” into something that doctors and researcher can track, measure and compare is an altogether bigger task.

And this task is becoming more and more important, since the agencies in charge of approving, licensing and reimbursement of new medicines take into account changes in “quality of life”.

Agencies such as the European Medicines Agency, will only accept quality of life data if it was collected using an agreed questionnaire, also called a patient-reported outcome measure (PROM). These questionnaires are developed and tailored to the condition itself and the age of the patient group taking part in the study.

In CF, the most common quality of life measure, which many PWCF will be familiar with, is a questionnaire called the “Cystic Fibrosis Questionnaire-Revised” or the CFQ-R for short. This questionnaire has been used for many years and has featured in many clinical trials. However, some of the questions are a bit old-fashioned. People with CF have been asking for a questionnaire that’s more adapted to modern day life. In 2019, the ECFS-CTN started working with CF Europe and a group of people with CF to create a new quality of life questionnaire called PRO-CF (patient reported outcomes in CF).  This questionnaire aims to more accurately assess people with CF’s quality of life and allow them to report their symptoms, including areas not previously captured in existing tools.

The questionnaire is now undergoing validation, an important step which will make sure that the PRO-CF questionnaire can be used to support the development and licensing of new medicines

To learn more about the work of the ECFS-CTN, the trials that are ongoing, and to read the experiences of some of the people with CF who participated in trials, please visit https://www.ecfs.eu/ctn You can read the most recent annual report from the ECFS-CTN here: https://www.ecfs.eu/ecfs-ctn-annual-reports