Report of the European Respiratory Society/European Cystic Fibrosis Society task force on the care of adults with cystic fibrosis.

The improved survival in people with cystic fibrosis has led to an increasing number of patients reaching adulthood. This trend is likely to be maintained over the next decades, suggesting a need to increase the number of centres with expertise in the management of adult patients with cystic fibrosis. These centres should be capable of delivering multidisciplinary care addressing the complexity of the disease, in addition to addressing the psychological burden on patients and their families. Further issues that require attention are organ transplantation and end of life management.Lung disease in adults with cystic fibrosis drives most of the clinical care requirements, and major life-threatening complications, such as respiratory infection, respiratory failure, pneumothorax and haemoptysis, and the management of lung transplantation require expertise from trained respiratory physicians. The taskforce therefore strongly reccommends that medical leadership in multidisciplinary adult teams should be attributed to a respiratory physician adequately trained in cystic fibrosis management.The task force suggests the implementation of a core curriculum for trainees in adult respiratory medicine and the selection and accreditation of training centres that deliver postgraduate training to the standards of the HERMES programme.

Developing new products in cystic fibrosis: needs and obstacles for activities of small and middle-sized companies.

Small and middle-sized enterprises (SMEs) can make important contributions to medical progress through the development of new safe and effective drugs that address the greatest unmet needs of patients. Regulatory inconsistencies across agencies in various countries, however, remain major challenges in cystic fibrosis (CF) drug development. Clear and consistent treatment guidelines, well educated clinical trial sites, a patient registry and grant funding for early development programs are important success-factors for an efficient development process. SMEs developing products for CF need partners in the CF community to assist with disease education and awareness for ongoing clinical trials. SMEs should collaborate and communicate with the CF community in a legally compliant way to take a patient-centric approach to drug design, development and administration. Furthermore, they can help to develop educational tools and fund medical education activities to increase the understanding of the underlying defects and mechanisms of CF disease.

Medical needs of cystic fibrosis patients and policies for fair co-operation between small and middle-sized companies and patient organizations.

BACKGROUND AND METHODS: Workpackage 4 of EuroCareCF brought together a group of small and middle-size companies (SMEs) with strong interest in drug development for cystic fibrosis (CF). The common interest of SMEs and patient organizations (PO) in mutually beneficial cooperation was assessed. This was achieved by identifying critical unmet medical needs of CF patients and by analyzing fields of cooperation between SMEs and POs. RESULTS: Over and above all, finding a cure for the disease is considered the most important unmet medical need by POs. However, preventing or slowing down any further deterioration of health and the alleviation of symptoms are also considered valuable objectives. Areas of co-operation with SMEs include the preparation and conduct of clinical trials and co-operation in the post-marketing authorization period. CONCLUSIONS: If a policy of transparency and respect for the independence of POs is applied, SMEs and POs can develop mutually beneficial and sustainable co-operation.