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Regulatory harmonization, a regulatory perspective Hubert Leufkens, Medicines Evaluation Board/Utrecht University.

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Presentation on theme: "Regulatory harmonization, a regulatory perspective Hubert Leufkens, Medicines Evaluation Board/Utrecht University."— Presentation transcript:

1 Regulatory harmonization, a regulatory perspective Hubert Leufkens, Medicines Evaluation Board/Utrecht University

2 College ter Beoordeling van Geneesmiddelen Leufkens Regul Harmonization Professor of Pharmacoepidemiology, Utrecht Institute of Pharmaceutical Sciences, 0.4 FTE. Chairman of the Dutch Medicines Evaluation Board (MEB), since mid Co-opted member of CHMP PhVWP, ; since 2009 co-opted member of CHMP. Director WHO-Utrecht Collaborating Centre on Pharmaceutical Policy and Regulation, since This talk reflects my personal views; I am being inspired and challenged on a daily basis by many colleagues from these ‘ environments ’. Declaration of interests

3 College ter Beoordeling van Geneesmiddelen Leufkens Regul Harmonization Non-harmonized world of cellphone adaptors Feira da Ladra, Lisbon's Flea Market, Summer 2012

4 College ter Beoordeling van Geneesmiddelen There are many reasons to harmonize drug development and regulation, i.e. to ensure equal access to patients. improve work-sharing and efficient use of scarce regulatory resources. avoid redundant use of animals in toxicity testing. limit number of study subjects in regulatory BE testing and pivotal clinical trials. facilitate international trade and commerce. contribute to a sustainable/affordable model of drug development. promote (global) trust in medicines. Leufkens Regul Harmonization

5 College ter Beoordeling van Geneesmiddelen The stakes are high.... Novo Nordisk shares fell 14% in the afternoon of February FDA requests additional cardiovascular outcomes data from a dedicated cardiovascular outcoms trial before the review of the NDA (Tresiba) can be completed. Authorities in the EU and Japan have already approved the drug. Novo Nordisk CEO adds ‘the FDA is more risk-averse than its counterpart in the EU which looks more at the benefits’. Leufkens Regul Harmonization WSJ 2013 February 12: 20.

6 College ter Beoordeling van Geneesmiddelen Leufkens Regul Harmonization Science 2011 Apr 8; 332(6026): Regulatory systems Patient safety Public health Innovation

7 College ter Beoordeling van Geneesmiddelen Leufkens Regul Harmonization QuestionToday's challenges Robust definition and diagnosis of disease? Psychiatric morbidities, sepsis, somatic functional disorders Clinically relevant endpoints to evaluate drug effects? PFS and OS in oncology, HbA1C or outcomes data in diabetes Identifiable target population (indication) that may benefit? Biomarkers to identify responders, to evaluate response What kind of comparison is useful, needed and feasible? Placebo, active controls and dynamics in treatment options Key questions in the lifecycle of a medicine

8 College ter Beoordeling van Geneesmiddelen Five archetypes of problematic dossier crunching We don’t like this product at all. Lost in details. We struggle, asking for more data makes us feel good. Reluctant to cross borders, risk avoidance. Why are we so positive? Leufkens Regul Harmonization

9 College ter Beoordeling van Geneesmiddelen Leufkens Regul Harmonization … Football, like most American sports, is heavily rule-bound. …Soccer is a more principles-based game. There are fewer rules, and the referee is given far more authority than officials in most American sports to interpret them and to shape game play and outcomes… A bad or biased ref can ruin a soccer match in the way that no bad ref can ruin a football match, the lesson being that regulator independence is key in a principles system... Principles-based versus rule-based regulation Economist. Metaphor of the day, April 21, 2008

10 College ter Beoordeling van Geneesmiddelen Leufkens Regul Harmonization Nat Rev Drug Discov 2012; 11:

11 College ter Beoordeling van Geneesmiddelen Leufkens Regul Harmonization Elgazayerly AN, de Graeff PA, Leufkens HG. N Engl J Med 2012 Mar 22 ;366 :

12 College ter Beoordeling van Geneesmiddelen Leufkens Regul Harmonization 2011 Jun 1;29(16):

13 College ter Beoordeling van Geneesmiddelen Leufkens Regul Harmonization Scrip 2011 Nov 25:18.

14 College ter Beoordeling van Geneesmiddelen Leufkens Regul Harmonization JAMA 2008; 300:

15 College ter Beoordeling van Geneesmiddelen Leufkens Regul Harmonization placebostandard treatmentalternative scenario societal effects HTA clinical outcome biomarker Outcomes and making comparisons

16 College ter Beoordeling van Geneesmiddelen Leufkens Regul Harmonization Rethinking the regulatory system: The Escher-project


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