EU Regulation Review: challenges and opportunities for industry

Transcription

1 EU Regulation Review: challenges and opportunities for industry Mia Bengtström, Pharma Industry Finland Nordic Pediatric Conference June 13,2017

2 Paediatric Regulation Consultation: EFPIA s answers general view Regulation has been successful in stimulating paediatric R&D which is now integral to companies development programmes, expanding technical expertise and specialist networks, contributing to the improvement of children s health in Europe Significant progress has been made, and more is needed - the development cycle of new medicines is lengthy and with many PIPs ongoing, more results (products, formulations, information) will be delivered in the coming years Building on experience to date, EFPIA proposes pragmatic measures for optimising the implementation of the Regulation, to better address unmet paediatric needs, improve the efficiency and speed of PIPs and so bring more treatments to children, more quickly

3 Between 2007 and 2015 and 39 new pharmaceutical forms appropriate for children were authorised in the EU Adapted from Irmgard Eichler (EMA Sr. Scientific Officer) EFGCP Better Medicines for Children Conference, Oct EMA 10 year report 3

4 Industry reflections to optimise paediatric development Industry proposals aimed at optimising paediatric research building on the first 10 years of experience with the Paediatric Regulation. They focus on three key areas: Better identification of the paediatric needs Clear and predictable understanding of the references framing discussion on PIPs Improving efficiency of PIPs without changing the timing of the PIP submission through better scientific and regulatory dialogue 4 4

5 1. Better identification of the paediatric needs Comprehensive inventory of unmet, disease-based paediatric needs Based on the existing requirements of Article 43 identifying research priorities Providing information on disease epidemiology and prevalence Will allow an assessment of the feasibility of conducting clinical studies in a particular paediatric population Indicating if there is any research ongoing in the area (including planned or ongoing clinical trials), and if there is, what type of research Involve multiple stakeholders (regulators, epidemiologists, patient groups, paediatric networks, industry) and define the process for maintaining the inventory updated 5

6 Adult condition 1 2. Optimise pediatric development: Better understanding of the references framing discussion on PIPs Does the same condition exist in children? YES NO Is there an indication within this condition listed as an unmet need in the inventory of paediatric needs, taking into account feasibility of trials? 2,3 YES Develop PIP in relevant indication NO For oncology products, considering the mechanism of action of the product and available scientific evidence, is there a paediatric indication with a validated target and an unmet need listed in the inventory of paediatric needs? NO Product specific waiver 3 YES Develop PIP in the most plausible paediatric indication 1 Using a recognised disease classification system e.g. MedDRA, ICD, identify the condition which the adult indication falls within. 2 A comprehensive inventory of paediatric needs, including for oncology needs validated scientific evidence. 3 This doesn t preclude sponsors from investigating other paediatric indications on a voluntary basis. 6

7 3. Better scientific and regulatory dialogue Better efficiency when PIP development fits more naturally within the drug development process: Early interaction with regulators (extended to include additional expert resources from other EMA committees, including the CHMP, and preferably input from patients) to agree on: the types of paediatric studies needed to demonstrate the quality, safety and efficacy of a specific medicine in children, along with the basic features of these studies; the planned date when the PIP will be submitted for agreement with the PDCO. This PIP will then include the full detail on the planned studies Improve PIP and scientific advice procedures. Reducing the need for PIP modifications 7

8 Industry reflections to optimise the implementation of the Paediatric Regulation and better frame paediatric development Proof of principle Proof of concept Phase I Phase II Phase III Marketing authorisation Adult development PIP submission PIP agreement Requests for PIP modification Paediatric development Paediatric label Scope of paediatric development Level of detail in PIP Feasibility 1. Inventory of paediatric needs Validated MOA for oncology 2. Updated policy on PIP scope 3. Early scientific dialogue Current challenges 8

9 What is industry current thinking? More work to be done but changes are already underway Innovative paediatric drug development needs experts at all levels and long term commitment Availability of new treatment options for children with (rare) diseases will only be achieved with concerted effort of all stakeholders Better use of available scientific and regulatory tools and methods Investment in basic research and paediatric disease models Better promote and support collaborative paediatric research, e.g. via IMI Create and strengthen sustainable cross-regional CT networks Involve all stakeholders at the time of PIP development to optimise patients access to innovative medicines Facilitate better convergence between regulatory agencies, eg via ICH A revision of the regulation would cause unnecessary delay, impact negatively all improvements made so far, and jeopardise a promising future 9

10 Pediatric patients in ongoing clinical trials conducted by industry in Finland, excluding vaccines Patients Vaihe I-II Vaihe III-IV PIF 2017

11 Ongoing pediatric clinical vaccine trials conducted by industry in Finland Patients PIF 2017

12 Increasing competitiveness of the Nordic countries in clinical research through collaboration Mia Bengtström, Pharma Industry Finland