March 27, 2017ENLIGHTEN (Jurnal Bimbingan dan Konseling Islam)19 citationsOpen Access
New Medicinal Products for Chronic Heart Failure: Advances in Clinical Trial Design and Efficacy Assessment
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Key Result
A joint workshop by the ESC and EMA reviewed clinical trial design, efficacy assessment, and novel therapeutic approaches for chronic heart failure to guide future regulatory guidelines.
Key Points
- To summarize expert consensus and identify research gaps regarding regulatory guidelines, trial design, and efficacy assessment for novel chronic heart failure therapeutics.
- Convened a dedicated two-day workshop by the European Society of Cardiology (ESC) Cardiovascular Round Table in partnership with the Heart Failure Association of the ESC.
- Evaluated the European Medicines Agency (EMA) draft Guideline on Clinical Investigation of Medicinal Products for the Treatment of Chronic Heart Failure.
- Deliberated on three core areas: efficacy assessment and statistical methods, clinical trial design considerations, and approaches for novel therapeutic principles such as cell therapy.
- Identified key areas of consensus on primary endpoint selection, appropriate statistical analyses, defining study populations, and standardizing background optimal medical therapy.
- Highlighted unresolved gaps and special design requirements for emerging treatment modalities, including cell-based regenerative therapies.
- Emphasized multi-stakeholder collaboration among regulators, industry, clinical trialists, and patient groups to streamline safe and effective market access.
Structured PICO
P
Population
Patients with chronic heart failure (in the context of clinical trial design)
I
Intervention
New medicinal products and clinical trial design strategies
This consensus document provides expert guidance on clinical trial design and efficacy assessment for new chronic heart failure therapies, informing the EMA's revised guidelines.
Abstract
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.
Cite This Study
Cowie et al. (2017) conducted a review in Chronic Heart Failure. New Medicinal Products was evaluated. A joint workshop by the ESC and EMA reviewed clinical trial design, efficacy assessment, and novel therapeutic approaches for chronic heart failure to guide future regulatory guidelines.