European journal of heart failure, 2017-06, Vol.19 (6), p.718-727
Despite the availability of a number of different classes of therapeutic agents with proven efficacy in heart failure, the clinical course of heart failure patients is characterized by a reduction in life expectancy, a progressive decline in health‐related quality of life and functional status, as well as a high risk of hospitalization. New approaches are needed to address the unmet medical needs of this patient population. The European Medicines Agency (EMA) is undertaking a revision of its Guideline on Clinical Investigation of Medicinal Products for the Treatment of Chronic Heart Failure. The draft version of the Guideline was released for public consultation in January 2016. The Cardiovascular Round Table of the European Society of Cardiology (ESC), in partnership with the Heart Failure Association of the ESC, convened a dedicated two‐day workshop to discuss three main topic areas of major interest in the field and addressed in this draft EMA guideline: (i) assessment of efficacy (i.e. endpoint selection and statistical analysis); (ii) clinical trial design (i.e. issues pertaining to patient population, optimal medical therapy, run‐in period); and (iii) research approaches for testing novel therapeutic principles (i.e. cell therapy). This paper summarizes the key outputs from the workshop, reviews areas of expert consensus, and identifies gaps that require further research or discussion. Collaboration between regulators, industry, clinical trialists, cardiologists, health technology assessment bodies, payers, and patient organizations is critical to address the ongoing challenge of heart failure and to ensure the development and market access of new therapeutics in a scientifically robust, practical and safe way.
Cardiac and Cardiovascular Systems ; Cardiovascular Agents - therapeutic use ; Cardiovascular System & Cardiology ; Clinical trial ; Clinical Trials as Topic ; Consensus ; determine impact ; Drug Approval ; drug-therapy ; ejection ; end-points ; european-society ; follow-up ; fraction ; global mortality ; Heart failure ; Heart Failure - drug therapy ; Humans ; Kardiologi ; Outcome Assessment (Health Care) ; paradigm-hf ; subgroup analyses ; task-force
Academic Search Ultimate
Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals
https://www.ncbi.nlm.nih.gov/pubmed/28345190$$D View this record in MEDLINE/PubMed
Permalink to record
https://gup.ub.gu.se/publication/254998$$DView record from Swedish Publication Index