samedan logo
 
 
spacer
home > ict > winter 2018 > early engagement
PUBLICATIONS
International Clinical Trials

Early Engagement

Patient-reported outcomes (PROs) are any report of the status of a patients’ health condition that come directly from the person, without interpretation of their response by a clinician or anyone else (1). PRO measures (typically surveys) may be generic, measuring, for example, health-related quality of life (HRQoL), or they may be disease-specific, providing information about symptoms experienced by a patient. For generic PROs, another sub-category of preference-based PRO instruments exists, such as the EQ-5D (2). These generate a profile score that summarise patients’ responses to items designed to tap into different domains of HRQoL, for example, ‘mobility’ and ‘self-care’. Therefore, the profile score represents a given health state, which is then converted to an index score using an algorithm that applies weights to the profile score based on population preferences for that specific health state. This is referred to as a health utility value and can be used in cost effectiveness analyses.

Collection of PROs in clinical trials usually serves one of two main purposes. First, generic and disease-specific PRO data are used in labelling claims and product summaries, providing evidence of treatment benefits not easily measured using clinical tools. This can be particularly useful in therapy areas without well-defined physiological markers, such as pain and mental health. Even where robust clinical indicators are available, PROs can demonstrate additional benefits, such as improvements in functioning and alleviation of symptoms. In rare or orphan diseases where treatments are commonly expensive, demonstration of HRQoL or functioning improvements can be particularly important factors for regulatory and reimbursement agencies in the decision-making process. For treatments that aim to alleviate symptoms rather than to cure, PRO evidence may constitute a primary efficacy, whereas for the endpoint of curative or life-extending treatments, PRO evidence may constitute a secondary endpoint, providing evidence of additional treatment benefits. The second way in which PRO trial data are used applies specifically to the use of preference-based PRO instruments. Information from these tools is used to calculate health utilities for cost-effectiveness models. These are one of the cornerstones of health-technology appraisals (HTAs) in the UK and are given prominence in HTA guidelines around the world (3,4). Given that appropriate utility measures are essential for accurate economic modelling, utility collection is a crucial part of regulatory and reimbursement approval.

Read full article from PDF >>

Rate this article You must be a member of the site to make a vote.  
Average rating:
0
     

There are no comments in regards to this article.

spacer
Dr Jenny Retzler is a Senior Research Consultant at York Health Economics Consortium, with a PhD in psychology and an MSc in cognitive neuroscience and neuroimaging, both from the University of Nottingham, UK. As a senior member of the outcomes research team, she works on a variety of projects, specialising in the measurement of treatment benefit. Her work includes the provision of evidence-based strategic advice on the optimal selection, implementation and analysis of outcome measures, and the development and implementation of utility
spacer
Dr Jenny Retzler
spacer
spacer
Print this page
Send to a friend
Privacy statement
News and Press Releases

Prometic Bioseparations are very pleased to introduce their new purpose built ecommerce platform (Webshop)!

Their goal was to build a platform that is easy to navigate, informative and one that makes their products and services easily accessible to researchers and laboratories worldwide. They also wanted to make ordering as quick and easy as possible to meet the needs of the rapidly progressive industry. They have reflected these requirements with an ecommerce store in order to supply high quality products to customers from academia to big pharma.
More info >>

White Papers

Clinical Trials in Russia Orange Paper: Summary of Year 2013

Synergy Research Group

Russia remains a very popular geography for local, regional, and global pharmaceutical companies to conduct clinical trials. Sponsors mention the following reasons for conducting studies in Russia: 1. Fast patient enrollment due to the centralized medical infrastructure. 2. Nearly 100% patient retention 3. GCP trained and certified Investigative Sites generating high quality data 4. Low cost: Average per patient cost is 60% to 70% below US and European prices due to the low cost of Investigators and the high concentration of patients in therapeutically aligned medical centers
More info >>

 
Industry Events

9th Global Drug Delivery & Formulation Summit

12-14 March 2018, Maritim proArte Hotel Berlin

The DDF Summit brings the most interesting studies and exciting technologies to you. This year there will be 4 dedicated streams focusing on Small Molecules, Biologics, Technology & Innovation and for the first-time Device Development.
More info >>

 

 

©2000-2011 Samedan Ltd.
Add to favourites

Print this page

Send to a friend
Privacy statement