Clinical Trials. Introduction + guidance on proposals

Transcription

1 Clinical Trials Introduction + guidance on proposals Open Information Day, Brussels, 29 May 2012 Cornelius Schmaltz Medical Research Unit Directorate Health Research & Innovation DG European Commission

2 Objectives Emphasise importance of clinical trials as key element of Health Research and of the FP7 Health theme Encourage the submission of high-quality clinical trials proposals Provide information from different angles and on different aspects of clinical trials: Information to be provided in proposal Regulatory considerations in clinical trials Assembling a consortium and writing a successful proposal The role of SMEs in clinical trials FP7 Financial and contractual issues in clinical trials projects

3 Agenda Welcome, introduction and guidance for clinical trial applications Cornelius Schmaltz, Health Directorate, Research & Innovation DG Building a consortium and writing an application - report of a successful project Dr. Malcolm Macleod, Centre for Clinical Brain Sciences, University of Edinburgh, UK Performing a clinical trial - the SME perspective Dr. Rebecca Pruss, CSO, Trophos, FR Regulatory considerations in clinical trials Dr. Chantal Belorgey, Head of Clinical Trials and Compassionate Use Department, AFSSAPS, FR Contractual and financial considerations for clinical trials under FP7 rules Cornelius Schmaltz, Health Directorate, Research & Innovation DG

4 Clinical Trials - Proposal General structure and page limitation identical, no special forms Trials vary, so some issues might not apply Expertise and common sense - quality of a proposal also lies in its completeness and correct weighing of different aspects Guidance for (narrative) part B of the proposal developed with external experts will be published (NEW!) in "Guide for Applicants"

5 Clinical Trials stage 1 proposal (1) Background evidence and need for the trial: Scientific rationale and primary hypothesis of the trial Epidemiology of the underlying disease Magnitude of the expected benefit over currently available therapeutic options Preclinical and/or preliminary clinical evidence, systematic review evidence

6 Clinical Trials stage 1 proposal (2) Description and justification of trial design and methodology study type : classification by objective (e.g. human pharmacology, therapeutic exploratory, therapeutic confirmatory, therapeutic use), by phase (I-IV), by methodology: randomised/non-randomised, type of masking (none, single, double, observer blind), type of controls (active, placebo), parallel group/cross-over, prognostic, diagnostic etc proposed setting: number, location, type of centres precise description of intervention(s): experimental, control, duration of intervention, duration of follow-up

7 Clinical Trials stage 1 proposal (3) Description and justification of trial design and methodology (cont'd) key inclusion and exclusion criteria outcome measures/endpoints: primary/secondary, efficacy, safety etc. bias protection: feasability of randomisation, allocation methods, feasability of blinding etc.

8 Clinical Trials stage 1 proposal (4) Description and justification of trial design and methodology (cont'd) statistical justification of proposed sample size/power calculations: # of pts to be assessed for eligibility, to be allocated to trial, to be analysed etc. (including subgroups if applicable) feasability of recruitment: provide evidence that the intended recruitment is achievable trial duration and timing: recruitment period, firstpatient-in to last-patient-out, duration of entire trial

9 Clinical Trials stage 2 proposal (1) Specify all issues mentioned in stage 1 in significantly more detail, provide more indepth justifications, evidence and references In addition address the following issues:

10 Clinical Trials stage 2 proposal (2) trial management: distribution of roles (sponsor, principal coordinating investigator, trial statistician), evidence of trials expertise, specific trials facilities and resources, quality assurance and monitoring strategy trials expertise of individual investigators/sites plans for data and database management, including location, access and regulatory implications

11 Clinical Trials stage 2 proposal (3) statistical analysis: strategy for (multiple) primary outcome(s), interim/subgroup analyses etc. monitoring of recruitment and contingency planning for recruitment problems pharmacy issues: GMP batch production (timeline, facilities, testing, approbal), drug dispensing and accountability management and retention of biological samples, cooperation with existing or creation of new bio-banks

12 Clinical Trials stage 2 proposal (4) reimbursement and contractual involvement of patient recruitment sites and trial management, including CROs if applicable (see related presentation later in this session!)

13 Clinical Trials stage 2 proposal (5) financial plan: trial (data, clinical) management; subject reimbursment/hospitalisation costs; trial drug(s) or device (including GMP batch production); additional diagnostic procedures; co-financing by industry or other third parties, distribution of costs between health insurance, hospital and trial sponsor; statistical analysis, insurance, submission fees for regulatory dossiers, data and safety monitoring boards etc.

14 Clinical Trials stage 2 proposal (6) provisions and timelines for approval by (which?) ethics committess and (which?) national competent authorities: which regulatory requirements have to be fulfilled, which have already been achieved and what is the status of the others, results of prior discussion with authorities overall timeline of the trial, including preparation time

15 Clinical Trials stage 2 proposal (7) independent trial oversight: DSMB, clinical event committee, scientific advisory board, ethical advisory board involvement and specific contribution of patients' organisations ethics: risk/benefits, protection of research participants, informed consent process and forms, participants' compensation, confidentiality, data protection, conflict of interests/commercial interests