ECFS 2020 - Optimizing pharmaceutical care in cystic fibrosis

212    www.ecfs.eu OPTIMIZING PHARMACEUTICAL CARE IN CYSTIC FIBROSIS CFTR MODULATORS, CFTR DIRECTED THERAPIES AND PRECISION MEDICINE CHAPTER 11 improved. This is despite the fact that we do not even know the full potential of highly effective CFTR modulators e.g. when they are introduced at an early age. Is prevention of male infertility possible? It is imaginable that future children with CF will be treated only with CFTR modulators, from a young age. When patients and families have the oppor- tunity to experience the major health bene- fits from CFTR modulators they are aston- ished and become hopeful for the future. Likewise, physicians and paramedical teams are excited that highly effective ther- apies have finally arrived and are anxious to prescribe these effective treatments as soon as possible to as many patients as possible. But the anticipated high treat- ment costs, along with the lifelong need for modulators are a challenge. How quickly will the triple modulator combination reach the 90% of eligible patients? Introduction of these new drugs will also bring new challenges. We need to monitor the safety of long-term treatment. How can we continue the entire drug develop- ment pipeline? Whilst it is relatively easy to document the benefit from highly effective first-in-class modulators, it will be difficult to evaluate new modulators. How to prove that they are as good as or better than these highly effective compounds? What will be the best comparator: placebo (is this ethically correct?) or the best in class drug (how will this cost be overcome, but by an open-label comparison group)? Will patients still be willing to participate in clinical trials? How should we continue the non-modulator drug pipeline e.g. anti-in- flammatory and anti-infective drugs? How should we progress the alternative strate- gies for CFTR correction that might lead to a one-off correction? We badly need clin- ical trials of how to simplify the treatment burden. But the bottom line is that CFTR modula- tors have brought enormous progress and hope to the CF community. Only a few decades ago, children were dying from CF in their mid-teens. Put in that context, our new challenges can be seen as ‘luxury problems’ and will surely be overcome.

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