EFPIA position on Clinical Trials Regulation trialogue

Transcription

1 EFPIA position on Clinical Trials Regulation trialogue As the revision of the Clinical Trial Directive enters the Trialogue phase, it is critical to remember that the key objective of this legislation process is to boost clinical research in Europe. This can be achieved by simplifying the rules for conducting clinical trials while maintaining the highest standards of patient safety and the robustness and reliability of trial data 1. Ultimately a simple, robust and efficient framework for clinical trials will contribute to faster access to innovative therapies for patients. To achieve these objectives, research-based pharmaceutical industries, represented by the European Federation of Pharmaceutical Industries and Associations (EFPIA) believe that the following elements need to be addressed during the Trialogue: 1. Assessment by Ethics Committees 2. Review timelines and tacit approval 3. Proposal by the sponsor of reporting Member State 4. Definition and requirements for vulnerable patients 5. Transparency 6. Indemnification scheme 7. Review of legislation after 5 years 8. Functionality of EU Portal and Database 1. Assessment by Ethics Committees EFPIA continues to support the specific inclusion of an review by ethics committees in the legal text consistent with the Declaration of Helsinki by the World Medical Association. EFPIA joins the European Commission in respecting that it is for the Member States to determine how they organise ethical reviews and reach a single decision provided that principles of a Part I (co-ordinated) and Part II (national) assessment are respected. Therefore, the proposal for a single decision per Member State should be maintained. EFPIA companies support the ENVI report and Council position, which respect national competence. 2. Review timelines and tacit approval Competitive timelines are the single most important element to strengthen Europe s attractiveness in the field of clinical research. Indeed when sponsors consider where to conduct clinical trials, the timelines for assessment and authorisation are seen as a headline indication of the region s attractiveness. Below is a summarised global comparison: Country Regulator Ethics Regulatory/Ethics Total Approval 1 Fostering EU's attractiveness in clinical research: Commission proposes to revamp rules on trials with Medicines, Press Release, European Commission, July Available at: 1

2 approval time committee review Time approval time relationship Singapore 30 days 30 days In parallel 30 days Australia 50 days days In parallel 50 days South Korea 60 days 8 weeks In parallel 60 days EU (EC s N/A N/A In parallel days proposal) India 3 months 2 months In parallel 90 days Russia 55 days 60 days EC approval first 115 days Canada 30 days 120 days In parallel 120 days South Africa 120 days 45 days HA approval first 165 days Argentina 5 months 30 days EC approval first 180 days Brazil 4 months 2 months EC approval first 180 days China 11 months 60 days HA approval 390 days USA 30 Days * In parallel * In the US, timelines are not defined in legislation - Institutes determine timelines (e.g 13 days at Johns Hopkins Bloomberg School of Public Health, more than one month at Univ of California, 30 days at Duke University) EFPIA companies strongly support the timelines proposed by the Commission (41 without questions, 75 days with questions) since they are overall consistent with the timelines in the current legislation while allowing a fast process in case authorities and ethics committees do not raise further questions. Practically, timelines proposed by the European Commission and defended by the European Parliament are feasible and would not compromise patients safety or the robustness and reliability of trial data as experience with the Voluntary Harmonised Procedure (VHP) shows. On average 56 days are needed to approve a clinical trial through the VHP to which a large majority of Member States participate 2. Tacit approval is inseparable from timelines. This mechanism is the only means to ensuring that timelines for the Part II assessment are respected. Other potential compliance mechanisms (e.g. penalties, infringement procedures or metrics overview) would have little or no enforcement capacity. As proposed by the European Commission and supported by EFPIA companies, tacit approval would only apply to Part II assessment (i.e. site/ investigator qualification, informed consent) and would not impact the robust scientific review mechanism performed under Part I. To create a level playing field between the industry and reporting Member State, tacit approval should be coupled with tacit withdrawal. 3. Proposal by the sponsor of reporting Member State 2 Dr. Hartmut Krafft, Chair CTFG and VHP-Coordinator, The Voluntary Harmonisation Procedure: Where are we now? Presentation, BIA, London, October Available at: 2

3 EFPIA companies reiterate their strong support for the Commission proposal allowing sponsors to propose the reporting Member State (rms) as a starting point for the appointment procedure. This provision would ensure scientific excellence, development continuity and predictability of review process. Ultimately, a Member State has to have a right to refuse to be a rms. 4. Definition and requirements for vulnerable patients One of the priority objectives of this legislative revision is to create more harmonisation on the approval of clinical trials. Unclear definitions of vulnerable patients in the new regulation or multiple national definitions would defeat this objective. EFPIA companies believe that the protection of patients should be consistent across Europe. Therefore the sponsor should apply the highest standards to all patients of a defined vulnerable group as indicated in the World Medical Association Declaration of Helsinki. The creation of individual categories of subjects with specific needs may result in different national interpretations, thus leading to divergence in the level of patient protection across Europe. Special provisions for clearly defined categories of patients (i.e. incapacitated patients and minors) as well as existing national protective measures (i.e. for persons in mandatory military service, deprived of liberty or those who cannot take part in a trial due to a judicial decision or persons in residential care institutions) can be maintained. However, it should be ensured that the above does not represent a basis for a divergent interpretation and hence implementation of the future Regulation. 5. Transparency EFPIA companies support enhancing public health and research through responsible sharing of clinical trial data in a manner that safeguards the privacy of patients, respects the integrity of national regulatory systems and maintains incentives for investment in biomedical research. The global biopharmaceutical industry, represented by EFPIA and its US counterpart PhRMA, believes that it is possible to achieve these goals without legislative interventions by committing to provide qualified scientific and medical researchers with patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials 3. EFPIA companies support a majority of the provisions proposed by the European Parliament and the Council to increase access to clinical data, including: The registration of clinical trials conducted in patients in an international clinical trials registry certified by the World Health Organisation (WHO) 4 ; The publication of a layperson summary (in line with industry data sharing commitment #3 5 ); Conducted in accordance with Directive 2001/20/EC of the European Parliament and of the Council of 4 April 2001 on the approximation of the laws, regulations and administrative provisions of the Member States relating to the implementation of good clinical practice in the conduct of clinical trials on medicinal products for human use 3

4 The exceptions to public availability of the EU database to protect personal data (PPD) and commercially confidential information (CCI) (in line with industry principles and Article 78); and Taking into consideration the authorisation status of the product as to trigger the level and the nature of disclosure of information (i.e. publication of the information after product approval). Regarding provisions related to Clinical Study Reports (CSRs), EFPIA companies believe that these documents are out of the scope of the Regulation since they are technical documents related to a marketing authorisation procedure. However, recognising the need to help patients and healthcare professionals understand the results of clinical trials and the evidence used to approve a new medicine, industry commits to proactively publish at a minimum a synopsis of a CSR after approval of new products or indications. Additionally, when the right conditions are met, researchers can have access to patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients as necessary for conducting legitimate research. EFPIA companies support a harmonised definition of CCI and trade secrets across the Europe Union through a specific legislation and do not believe that the Regulation is the right legislative vehicle to do so. The introduction of a tentative definition of what constitute CCI in the Regulation would be inconsistent with harmonisation efforts currently undertaken by other services at the European Commission 6. The approach to CSRs should also be seen in the context of the Regulatory Data Protection (RDP) framework 7. This framework is of vital importance to incentivise the conduct of research and development, especially in countries or situations where patent protection may not be available. Some highly innovative medicines rely entirely on this form of protection for their market exclusivity, including in the EU. 6. Indemnification scheme National indemnification schemes are welcomed as they provide benefits to noncommercial sponsors, but their use should be optional for all sponsors. Generally commercial sponsors organise their own cover through global contracts with private insurers, which allow costs to be better managed. 7. Review of legislation after 5 years To ensure that the new Regulation remains fit for purpose, a review clause after 5 years should be introduced. This would ensure that the Regulation keeps up with the pace of scientific research and technical advances, such as in the field of personalised medicines. 8. Functionality of EU Portal and Database Article 10, Directive 2001/83/EC of the European Parliament and of the Council of 6 November 2001 on the Community code relating to medicinal products for human use 4

5 As stated by some Member States, the database and portal are key components of the Regulation to ensure the highest level of harmonisation in the process dramatically lacking under the current Clinical Trials Directive. Their full functionality will ensure that the new application processes is workable and efficient by sponsors. Therefore a fully functioning system should be available for use at the time of implementation of the Regulation. To support this, transitional provisions are needed to link implementation of the relevant aspects of the Regulation to the functionality of the database. In addition any launch must be preceded by sufficient user-acceptance testing and training to ensure the functionality fully supports the processes defined in the Regulation. 5