Regulatory Science «A Ferring perspective» Sukhwinder S. Jossan, PhD Associate Vice President, Global Regulatory Affairs

Transcription

1 Regulatory Science «A Ferring perspective» Sukhwinder S. Jossan, PhD Associate Vice President, Global Regulatory Affairs

2 Regulatory Science: Introduction Literature search in1985 did not find the phrase regulatory science In 2014 search for regulatory science resulted in 200,000 hits Insignificant role of science in decision process before 19th century Religious leaders and other authorities established laws and policy Science was insufficiently developed and intermingled with beliefs Advancement of industries resulted in need to regulate for instance manufacturing or air pollution 1

3 Regulatory Science: Introduction Regulation required relevant scientific information - regulatory science The term regulatory was first used by in 1970 Regulatory science described as the science used to develop regulations Institute for Regulatory Science was established in 1985 Regulatory science refers broadly to the scientific and technical foundations upon which regulations are based in various industries particularly those involving health or safety Regulatory science is increasingly recognized as an evolving science serving regulatory and other policy decision making 2

4 Regulatory Science and Regulatory Affairs Regulatory Science focuses on the scientific underpinnings and concerns Regulatory affairs and regulatory law refer to the administrative and legal aspects including implementation, compliance, or enforcement 3

5 Regulatory Science: Definition FDA Regulatory Science is the science of developing new tools, standards, and approaches to assess the safety, efficacy, quality, and performance of all FDA-regulated products CORS Regulatory Science "The science of developing new tools, standards and approaches to evaluate the efficacy, safety, quality and performance of medical products in order to assess benefit-risk and facilitate a sound and transparent regulatory decision making Regulatory science a scientific discipline consisting of the development and application of scientific methods, tools, approaches, and other relevant processes derived from various scientific disciplines used to support regulatory and other policy objectives. An abbreviated definition regulatory science consists of the application of science to support policy notably regulatory objectives 4

6 Regulatory Science: Future FDA s Strategic Plan for Regulatory Science is designed to allow the Agency both to meet today s public and animal health needs and to be fully prepared for the challenges and opportunities of tomorrow to help harness revolutions in science that can be translated into products that help make and keep our nation both safe and healthy 5

7 Regulatory Science: Future The need for transparency in regulatory science by identifying assumptions, judgments, and related processes during the regulatory science process. Those involved in development of regulations, typically this group is employed by regulatory agencies Those who must comply with regulations, typically this group consists of employees or contractors of regulated community The scientific community who performs research and development in relevant areas 6

8 Dose finding and dose selection Workshop held in London in Dec 2014 Academia, Regulatory bodies and Industry participated Misconception: D-E-R and dose selection are solely at Company s risk Debate to update dose finding regulatory guidance FDA and EMA s experience phase 2 is often abbreviated The selection of dose is frequently empirical and rarely scientifically sound Poor dose selection may result in delayed rejection, changes in dose post approval Poor dose selection is one of the main causes for increasing cost of drug development 7

9 Dose finding and dose selection Conclusions Model-based dose finding methods are more efficient than pairwise comparisons approaches Longitudinal data analysis, time vs drug effect, a powerful tool Implementation of innovative approach requires support of senior research and development industry leaders Improved regulatory guidance from HA can drive engagement from senior leadership 8

10 Thorough QT study-early QT Assessment ICH, E14 requires most NCEs to be tested in a specifically designed TQT study Collaboration to design Phase 1 study to generate data to replace a thorough QT study Six marketed drugs with QTc effects were identified Randomized placebo-controlled study in 20 healthy subjects Two doses of each drug on separate, consecutive days Study designed in collaboration with the FDA 9

11 Thorough QT study-early QT Assessment Successful outcome to provide evidence supporting replacement of the TQT with ECG in standard early development studies for a new chemical entity The group fulfilled its goal of conducting a prospective study in early Phase1 with 5 known QTc positive drugs and one QTc negative drug On the bases of these results FDA has begun considering to grant waiver for TQT studies 10

12 Complex biological medicinal products 11

13 Regulatory Science: Introduction regulatory science is both interdisciplinary and multidisciplinary and relies upon a large number of basic and applied scientific disciplines Regulatory science is an emerging area of interest within pharmaceutical medicine as the shaping and implementation of legislation and guidelines 12