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Accessing new drugs: A regulator's perspective

Accessing new drugs: A regulator's perspective . International Society of Paediatric Oncology 2012 congress. Presented by: Ralf Herold Scientific Administrator, Paediatric Medicines Section, Human Medicines Special Areas, Human Medicines Development and Evaluation. "Access to new drugs" .

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Accessing new drugs: A regulator's perspective

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  1. Accessing new drugs: A regulator's perspective International Society of Paediatric Oncology 2012 congress Presented by: Ralf Herold Scientific Administrator, Paediatric Medicines Section, Human Medicines Special Areas, Human Medicines Development and Evaluation

  2. "Access to new drugs" • Access = To study new drug in non-clinical test and in clinical trial • Scientific steps to accumulate evidence • This is an experiment, not medical care • Access = to make sure high-quality medicines are developed and available for authorised use and reimbursement • To advance medical care, which then can be used in clinical trials Accessing new drugs - a regulator's perspective

  3. Regulatory perspective: to safeguard and to improve public health in Europe The choice of the endpoint determines which patient subset is addressed. (after S Hirschfeld) DFS PFS TTF OS • Protect population and ensure safety of medicines • European population in its diversity • Neglected subsets in frequent conditions • Respond to global needs • Neglected diseases Accessing new drugs - a regulator's perspective

  4. General tools of regulators to support the development of medicines • Scientific guidelines • Orphan designation (incentives during and after development) • Paediatric investigation plan (incentives) • Needs lists (Paediatric medicines EMA; WHO Essential medicines) • Continuous exchange with other institutions (e.g., ECDC, FDA) • Collaboration with scientific societies • Consultations in public • High level of transparency Accessing new drugs - a regulator's perspective

  5. Scientific Committees at the EMA • CHMP, PDCO, CAT, COMP, PRAC • Delegates from the 27 EU Member States + NO + IS • Health professional representatives • Patient organisation representatives • Work to achieve scientific consensus Accessing new drugs - a regulator's perspective

  6. Paediatric Regulation (EC) No. 1901/2006 * http://ec.europa.eu/health/human-use/paediatric-medicines/developments/index_en.htm • Relevant for you as medical doctors and investigators • Comprehensive framework of paediatric expertise in EU established: • Paediatric Committee (PeeDeeCeeOh) with EMA secretariat created • European paediatric research network at the EMA (Enpr-EMA) created • Activities and achievements (end 2011*) under Regulation: • PIP / Waiver for all new and (many) authorised medicines mandatory: in total 476 PIP Decisions (33 anti-cancer substances, each 1 or 2 PIPs) • First 43 paediatric indications authorised (1 anti-cancer, more in 2012) • Assessment of completed studies and reports on medicines in children • Difficulties and limitations Accessing new drugs - a regulator's perspective

  7. BevacizumabSmPC: timely update (03/2012), reference to authorised indications, concerns based on results of paediatric trials http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Product_Information/human/000582/WC500029271.pdf Accessing new drugs - a regulator's perspective

  8. Paediatric Investigation Plan agreed by PDCO: the main tool of the Paediatric Regulation • Basis for development and authorisation of a medicine for all paediatric population subsets • PDCO defines paediatric use to be targeted by the study program • Mandatory for new medicinal products under development and for authorised medicines when new indication, route or formulation • Binding on pharmaceutical companies • Completion required once "triggered" in a regulatory submission • Includes studies with details and timing to documentquality, safety and efficacy of the medicine Accessing new drugs - a regulator's perspective

  9. Regulatory activities in medicine development Phase 1 Phase 2 Phase 3 Regulatory Post-approval Development in adults CHMP PDCO PIP initial submission by applicant to PDCO Compliance Validation PIP Modification(s) Non-compliance blocks validation (filing) Orphan medicine designation Scientific Advice Accessing new drugs - a regulator's perspective

  10. Procedure for handling applications for agreement of PIP / waiver (SOP/H/3207) Day 60 = Second discussion in the PDCO Day 90 = Third discussion in the PDCO Day 120 = Possible oral explanation Day 30 = First discussion in the PDCO Clock stop PDCO Opinion 60 days 60 days ~ 3 months Day 1 = After validation, Summary report drafted by EMA Day 120 = Adoption “PDCO Opinion” Agreeing / refusing PIP and / or waiver Day 60 = Adoption “PDCO Request for modification” or “PDCO Opinion” Day 61 = Summary report updated with applicant response Potentially followed by 30-day Re-examination Accessing new drugs - a regulator's perspective

  11. Difficulties and limitations encountered with implementing the Paediatric Regulation (I) • Assessment of old studies and reports inconclusive: Meaningful paediatric studies difficult to define and conduct • General considerations and areas for improvement • Use of new information by health care professionals uncertain • Variable awareness of need for paediatric clinical research • Paediatric care based on poor evidence infrequently challenged • Some paediatric therapeutic areas have been neglected • Wide-spread off label use not abolished by generating paediatric data Accessing new drugs - a regulator's perspective

  12. Activities and responsibilities of regulators and stakeholders Regulating Objectives Investigators Regulators Prescribers Pharma Company Accessing new drugs - a regulator's perspective

  13. Difficulties and limitations encountered with implementing the Paediatric Regulation (II) • Difficulties in paediatric oncology: • Regulatory system previously solely based on histopathology • Data not yet available (e.g., biological principle, xenografts, adult studies) to inform which is paediatric malignancy (if any) that could need medicine • Difficulties to build success on mechanism of action and biomarker(s) • Choosing relevant study designs and strategies difficult for all stakeholders • Inefficiencies of setting up and running studies • Inspections of paediatric trials show criticaland major findings • Many discussions (e.g., COG, NIH, SIOP) not accessible to EMA • Paediatric trials for oncology medicines ongoing, but no PIP Accessing new drugs - a regulator's perspective

  14. Anti-cancer medicines for children: non-clinical and clinical safety findings • Results of first completed juvenile animal studies available • Bone metabolism inhibitor A: effects at all doses, partially reversible, findings have clinical relevance, putative biomarkers found • Tyrosine kinase inhibitor B: no unexpected and no more serious findings compared to clinical profile in adults • Tyrosine kinase inhibitor C: unexpected kidney toxicity (glomerulopathy), generally more serious toxicity compared to clinical profile in adults • Cytotoxic medicine D: no unexpected finding • Pro-apoptotic medicine E: unexpected finding brain hypoplasia Accessing new drugs - a regulator's perspective

  15. Anti-cancer medicines for children: difficult discussions of priorities and paediatric interest • Interesting data … • Should bluebe a priority? • What is missing? • Full transparency necessary • Data update necessary • Medical data survivors? • Review clinical records? Accessing new drugs - a regulator's perspective

  16. Recent activities for paediatric oncology and anti-cancer medicines for children * http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/document_listing/document_listing_000293.jsp&mid=WC0b01ac0580025b91 • “Policy on determination of condition for PIP” (EMA/272931/2011) published: criteria for the PDCO to define the paediatric interest based on unmet needs, mode of action, MedDRA or biological criteria • PDCO discusses revoking class waivers, taking into account the evolution of knowledge and science over the last years • Model oncology PIPs in the works to attract pharmaceutical companies, to flag subsets with high unmet needs, to quote priorities • Paediatric oncology task force with experts from community • “Involvement of children and young people at the PDCO”for public consultation (EMA/PDCO/388684/2012 ) • PDCO involved in assessments (CHMP) of paediatric trial results Accessing new drugs - a regulator's perspective

  17. Summary Regulator's perspective for paediatric oncology: • Oncology PIPs help paediatric oncologists to get medicines for trials and help children with cancer to get safe and efficacious medicines • International scientific exchange collaboration required in view of difficulties in past and present to generate data • Regulators (EMA / PDCO) open to implement collaboration • Need to improve information for high-quality PIPs and priorities Accessing new drugs - a regulator's perspective

  18. Acknowledgements • PDCO: Dobrin Konstantinov, Henk van den Berg, Jacqueline Carleer, Janez Jazbec, Jaroslav Sterba, Koenraad Norga, Paolo Paolucci • EMA: Agnès Saint Raymond, Ralph Bax, Francesco Pignatti, Lynley Marshall, Peter Bauer Accessing new drugs - a regulator's perspective

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