Core Outcome Measures in Effectiveness Trials

Regulatory and Health Technology Assessment Perspectives on Measures of Clinical Benefit in Anti-Leukaemic Therapy

General Information

A robust strategy for medicines developers and clinical researchers of antineoplastic agents, gauging metrics of health benefits that satisfy regulatory and health technology assessment (HTA) requirements, favours drug approvals and enhances patient accessibility. Harmonisation of evidence needs promotes data uniformity along the lifecycle of a medicine, with inferred benefits extending to stakeholders concerned. The purpose of the study is to determine a core outcome set (COS) prioritised by EU regulators and Health Technology Assessment Bodies (HTABs) in the assessment of haematology anticancer agents, specifically those investigated in leukaemia subtypes. Focus of the study is placed on 'what' efficacy indicators should be measured, with additional insights being captured on the tenets of 'when to measure' and 'how to measure'.

Principle investigator: Dylan Said, Department of Pharmacy, University of Malta

Supervisor: Anthony Serracino-Inglott, Department of Pharmacy, University of Malta

Co-supervisor: Maresca Attard Pizzuto, Department of Pharmacy, University of Malta

Advisor: John Joseph Borg, Malta Medicines Authority

Further Study Information

Current Stage:
October 2017 - June 2019
Funding source(s):

Health Area

Disease Category
Blood disorders

Disease Name

Target Population

Age Range
0 - 100


Nature / type of Intervention


Delphi process

Interventional Phase II to Phase IV clinical trials investigating therapies in subtypes of leukaemia for all ages over an eleven-year period (2007-2017) were identified from the EU Clinical Trials Register database. Unique efficacy endpoints were extracted from the selected clinical trials and categorised into four major categories. Clinical trial efficacy data was used to design the Response Evaluation in Leukaemia (ReVALeu) online surveying tool. The tool was validated and subsequently disseminated using a two-round, e-Delphi technique with two independent expert panels having recognised expertise in the therapeutic area of onco-haematology. The first Delphi cycle recruited experts from HTA bodies across EU Member States, while the second Delphi cycle panel was composed of regulatory representatives from the European Medicines Agency (EMA).

Stakeholders Involved

Policy makers
Regulatory agency representatives

Study Type

COS for clinical trials or clinical research

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