ECFS 2020 - Optimizing pharmaceutical care in cystic fibrosis

European Cystic Fibrosis Society   227  OPTIMIZING PHARMACEUTICAL CARE IN CYSTIC FIBROSIS OPTIMIZING PHARMACEUTICAL TREATMENT IN CYSTIC FIBROSIS CHAPTER 12 health care systems faced with multiple competing demands on limited resources. The challenges presented by modulator therapy are replicated in other disease areas where personalized medicine also presents novel effective therapies. These challenges are helping to change how reimbursement decisions are made regarding pharmacolog- ical therapies for CF, and beyond. Patients and their advocates, drug companies, policy makers, pharmaceutical benefit managers, investors, charities and researchers will continue to adapt and shape this changing landscape in ways that, at present, are not wholly predictable. Based on observed reimbursement decisions, it is clear that current approaches to cost effective- ness have provided answers that do not reflect societies’ preferences, suggesting that reimbursement approaches need to adapt. Similarly, beyond pharmacological therapies, decision models that assess outcomes in terms of longevity and quality of life will strain to capture outcomes related to screening tests that might inform choices around pregnancy or carrying a pregnancy to term. These are complex issues that go beyond the discourse of any one discipline; questions that will challenge societies for some time to come. concluded that to sustain such a healthcare market, competing innovators and tech- nology adopters must have incentives to promote long-term dynamic efficiency, and argued that to achieve a socially optimal rate of medical innovation in personalized- medicine, payers must send clear signals to suppliers regarding what they value. This interplay between suppliers and buyers of precision medicines presents a complex marketplace, made more complex to navigate by the availability of additional new medicines. The social media backlash regarding refusal of some reimbursement bodies to pay for ivacaftor was directed at government and not the pricing decisions made by the manufacturing pharmaceu- tical company. Furthermore, coordinated lobbying campaigns have yielded success in many jurisdictions despite concerns over the long-term affordability of personalized- medicine for healthcare systems. Given the increasing R&D spend on precision medi- cines and the potential for patient benefit, it is inevitable that new HTA methodologies and reimbursement systems will emerge. Current research effort is being directed at adaptive clinical trial design, innovative decision modelling approaches, develop- ment of valuation methodologies and real- time clinical data capture. 8 Conclusions The CF landscape is changing. The emer- gence of new pharmacological thera- pies will improve outcomes and improve life expectancy but will also challenge