The last decade has witnessed developments in the CF drug pipeline which are both exciting and unprecedented, bringing with them previously unconsidered challenges. The Task Force group came together to consider these challenges and possible strategies to address them. Over the last 18 months, we have discussed internally and gathered views from a broad range of individuals representing patient organizations, clinical and research teams, the pharmaceutical industry and regulatory agencies. In this and the accompanying article, we discuss two main areas of focus: i) optimising trial design and delivery for speed/efficiency; ii) drug development for patients with rare CFTR mutations. We propose some strategies to tackle the challenges ahead and highlight areas where further thought is needed. We see this as the start of a process rather than the end and hope herewith to engage the wider community in seeking solutions to improved treatments for all patients with CF.

• MeSH
• buněčné kultury metody   MeSH
• cílená molekulární terapie metody   normy   MeSH
• cystická fibróza * farmakoterapie   epidemiologie   genetika   MeSH
• lidé MeSH
• modulátory membránového transportu farmakologie   MeSH
• mutace genetika   MeSH
• protein CFTR genetika   MeSH
• registrace statistika a číselné údaje   MeSH
• schvalování léčiv MeSH
• teranostická nanomedicína metody   MeSH
• vyvíjení léků * metody   normy   MeSH
• zlepšení kvality MeSH
• Check Tag
• lidé MeSH
• Publikační typ
• časopisecké články MeSH
• práce podpořená grantem MeSH
• Geografické názvy
• Evropa epidemiologie   MeSH
• Názvy látek
• modulátory membránového transportu MeSH
• protein CFTR MeSH

The last decade has witnessed developments in the CF drug pipeline which are both exciting and unprecedented, bringing with them previously unconsidered challenges. The Task Force group was brought together to consider these challenges and possible strategies to address them. Over the last 18 months, we have discussed internally and gathered views from a broad range of individuals representing patient organisations, clinical and research teams, the pharmaceutical industry and regulatory agencies. In this and the accompanying article, we discuss two main areas of focus: i) optimising trial design and delivery for speed and efficiency; ii) drug development for patients with rare CFTR mutations. We propose some strategies to tackle the challenges ahead and highlight areas where further thought is needed. We see this as the start of a process rather than the end and hope herewith to engage the wider community in seeking solutions to improved treatments for all patients with CF.

• Klíčová slova
• CFTR modulators, Clinical trial design, Cystic fibrosis,
• MeSH
• cystická fibróza farmakoterapie   genetika   MeSH
• klinické zkoušky jako téma * metody   normy   MeSH
• lidé MeSH
• modulátory membránového transportu farmakologie   MeSH
• mutace MeSH
• objevování léků * metody   trendy   MeSH
• protein CFTR genetika   MeSH
• vyvíjení léků * organizace a řízení   normy   MeSH
• výzkumný projekt normy   MeSH
• zlepšení kvality MeSH
• Check Tag
• lidé MeSH
• Publikační typ
• časopisecké články MeSH
• Názvy látek
• modulátory membránového transportu MeSH
• protein CFTR MeSH