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Please give a briefing of your own observations and outcomes of the congress: : |
Some 250 professionals from five continents are attending the International Conference on Advances in Pharmaceutical Drug Development, Quality Control and Regulatory Sciences (DDRS 2021). The participants discussed drug development, quality control and regulatory challenges. The conference had to be postponed twice due to the coronavirus pandemic. There were so famous scientists argued What is regulatory science? “Regulatory science is the science of developing new tools, standards and approaches to assess the safety, efficacy, quality and performance of (US Food and Drug Administration, FDA) regulated products.” It involves scientific tools, information gathering and analytical systems to study data, people, health systems and communities. To be most effective, advances in regulatory science must be fully integrated into the entire product development process. The regulatory science can be utilized to speed innovation and improve regulatory decision-making process Regulatory Science is a broad umbrella term for all different kinds of science that impact drug development or device development. This oversight is based on the sound science, called regulatory science, and is the foundation of FDA’s day-today decisions. Scientists throughout the agency research the development of new ways to evaluate FDA-regulated products. FDA utilizes varieties of tools to communicate policies and to invite scientific discourse - advisory committee meetings, publications, sponsor research, conferences / symposia / workshops to discuss scientific issues / develop consensus / develop reports (White papers), publication of guidance – product specific guidance. The aim is to have ‘science-based regulations ‘Pharmacokinetics of the drug – Absorption, distribution, metabolism and Excretion (ADME), bioavailability (BA) and bioequivalence (BE) concepts has been the focus for evaluation of different formulations of new drugs and also generic drugs. These concepts have provided strong scientific basis for bioequivalence-based approval of all generic drugs Principle of dissolution has been utilized as a product quality and performance tool. The scientific basis of Biopharmaceutics Classification System (BCS) provides biowaiver for certain classes of generic drug products, thus reducing regulatory burden without sacrificing drug product quality Generic Drugs User Fee Act (GDUFA): - Regulatory science priorities of GDUFA included post market evaluation of generic drugs and determining equivalence of complex dug products. The scientific research supports the development of guidance and policy that clarifies the Abbreviated New Drug Application (ANDA) pathway for complex products. The research in regulatory science has been used in innovative statistical approaches to provide the most reliable treatment outcome information to patients and clinicians, in supporting drug development through Physiologically based pharmacokinetic (PBPK) modeling and in developing tools to evaluate complex drug products, e g , for complex Active pharmaceutical ingredient (API) like Peptides, for complex mixtures of APIs like iron sucrose, for complex formulations like Liposomes and Colloids, for complex dosage forms like Transdermal Drug Delivery System (TDS), Long acting injectables, for complex route of delivery like Topicals, Inhalation products, and for complex drug device combinations like Auto-injector, Metered Dose Inhalers (MDI). The scientific research supports the development of guidance and policy that clarifies the ANDA pathway for complex products. Regulatory science is also utilized in strengthening US drug product manufacturing FDA/Center for Drug Evaluation and Research (CDER) publishes a quarterly Regulatory Science newsletter featuring new developments, opportunities, and initiatives in drug development regulatory science, with the goal of advancing medical product development. Needs for regulatory guidance and standards for gene, cell-based immunotherapies, and complex therapies to meet unmet medical needs and for rare diseases have prompted novel regulatory science for speeding regulatory approval Regulatory encouragements for application of model-informed approaches including PKPD, PBPK, and systems modelling and simulation; adaptive clinical trials; real-world evidence; and Bayesian statistics are among the latest targets for research and application in regulatory science. Currently, advanced regulatory science research by Agency and academic scientists is leading to greater speed, economy and informativeness of medical product development and regulation. There is all reason to expect continued expansion and intensification of research and application of novel regulatory science to meet public health needs of the future. Vaccine Development in Global Pandemic Time presented by TIBOR FABÓ. Pfizer-BioNTech collaboration started in 2018 in order to develop mRNA flu vaccine. Because of the covid19 pandemic the two companies started to focus on mRNA vaccine development for the prevention of covid19 infection In March they signed the Letters of Intent. Initially there were four vaccine candidates including unmodified mRNA, nucleoside-modified mRNA and self-amplifying mRNA for further development the nucleoside-modified mRNA was chosen. In April Phase 1/2 study was completed in Germany and in May in the USA Two 30 µg doses 3 weeks apart induced neutralizing antibody titers comparable to natural infection and strong CD4+ and CD8+ Tcell responses were observed Phase 2b/3 clinical trial started in July involving more than 43 000 participants in 153 sites. The result showed 95% efficacy with mild and moderate local and systemic events For safety reason all participants will be followed for 2 years after the second dose Based on rolling review regulatory agencies were able to approve within a short period of time in December 2020, first MHRA in UK, then FDA authorized for Emergency Use and EMA granted Conditional Marketing Authorization on 21 December 2021 for 16 years old and older. |