Patient and citizen participation at the organizational level in health technology assessment: an exploratory study in five jurisdictions.

Objective: While patient participation in individual health technology assessments (HTAs) has been frequently described in the literature, patient and citizen participation at the organizational level is less described and may be less understood and practiced in HTA bodies. We aimed to better understand its use by describing current practice.

Method: To elicit descriptive case studies and insights we conducted semi-structured interviews and open-ended questionnaires with HTA body staff and patients and citizens participating at the organizational level in Belgium, France, Quebec, Scotland, and Wales.

Inequities in cancer drug development in terms of unmet medical need.

This study measures inequality and inequity in the distribution of clinical trials on cancer drug development between 1996 and 2016, comparing the number of clinical trials with cancer need, proxied by prevalence, incidence, or survival rates for both rare and non-rare cancers. We leverage a unique global database of clinical trials activity and costs between 1996 and 2016, constructed for 227 different cancer types to measure for rare and non-rare cancers: i) inequalities and inequity of clinical trial activity, considering all trials as well as split by R&D stage; ii) inequalities and inequity in R&D investment proxied by trial enrollment and duration; iii) evolution of inequity over time. Inequalities are measured with concentration curves and indices and inequities measured with the health inequity index.

The contribution of French patient and consumer groups to health technology assessments over a 2-year period: an observational retrospective study.

Background: In 2017, The French National Authority for Health (HAS) created an open, online, systematic contribution process to enable patient and consumer groups (PCGs) to contribute to health technology assessment (HTA) carried out to aid public authorities in reimbursement and pricing decision making.

Objectives: This retrospective study analyzes how French PCGs contributed to the HTA process within the HAS for the first 2 years of this new mechanism.

Methods: PCG contributions received between 01 January 2017 and 31 December 2018 and the recording of deliberations leading to reports of the corresponding HTAs were included.

Evaluating the Impact of Intensive Case Management for Severe Vocational Injuries on Work Incapacity and Costs.

Purpose This study investigates the impact of an intensive case management program on sick leave days, permanent work incapacity levels and treatment costs for severe vocational injuries set up by the French National Insurance Fund in five health insurance districts. Methods The method employed relies on a four-step matching procedure combining Coarsened Exact Matching and Propensity Score Matching, based on an original administrative dataset. Average Treatment effects on the Treated were estimated using a parametric model with a large set of covariates.

Effectiveness and efficiency of teleimaging in the transplantation process: a mixed method protocol.

Background: The transplantation process usually takes place without transplant teams being able to use imaging data to assess graft quality. The decision of whether to go get the graft or not is therefore limited and suboptimal. "Cristal images" is a teleimaging project allowing real-time visualization of images of the organs of the donor.

Parents' and children's comprehension and decision in a paediatric early phase oncology trial: a prospective study.

Objective: To analyse parents' and children's understanding of consent information and assess their decision-making process in paediatric oncology.

Design: Prospective observational study.

Settings: Eleven French paediatric oncology units.

COST-EFFECTIVENESS OF INTERVENTIONS BASED ON PHYSICAL ACTIVITY IN THE TREATMENT OF CHRONIC CONDITIONS: A SYSTEMATIC LITERATURE REVIEW.

Objectives: The aim of this study is to review evidence on the cost-effectiveness of exercise-based interventions in the treatment of chronic conditions a decade after the publication of Roine et al. in 2009 (Roine E, Roine RP, Räsänen P, et al. Int J Technol Assess Health Care.

Enrollment in early-phase clinical trials in pediatric oncology: The experience at Institut Curie.

Background: The European Paediatric Regulation was introduced in 2007 to facilitate access to new medicines for children. Our study explored accessibility of early-phase trials in pediatric oncology, in line with the European Paediatric Regulation, to identify the reasons for not inviting patients to participate, parents' refusal, or inclusion failure.

Procedure: We conducted a retrospective chart review at Institut Curie, Paris, for all pediatric patients whose cancer progressed despite known effective treatments between July 2010 and December 2013.

Ethical issues of clinical trials in paediatric oncology from 2003 to 2013: a systematic review.

A state-of-the art approach to the debates on ethical issues is key in order to gain guidance on research practices involving sick children and adolescents, as well as to identify research avenues in which it might be worth cooperating, to generate better or supplementary evidence. Based on a systematic literature search using MEDLINE, we report the main ethical developments in paediatric oncology clinical trials from 2003-13. The present knowledge about normative and empirical ethical demands in this setting is quantified and summarised in a list of 46 issues.