CV Drug Development PDF

Drug Development in Cardiovascular (CV) Diseases Wenda Ramma, PhD MBA Bristol Myers Squibb Medical Affairs November 5, 2024 DDPM 201 Pharmacotherapy and Diseases  Learning objectives Understand the landscape of CV drugs Overview of Clinical Development Ove...

Drug Development in Cardiovascular (CV) Diseases Wenda Ramma, PhD MBA Bristol Myers Squibb Medical Affairs November 5, 2024 DDPM 201 Pharmacotherapy and Diseases  Learning objectives Understand the landscape of CV drugs Overview of Clinical Development Overview of US regulatory pathways Challenges in CV drug development Future of CV drug development  Introduction Globally, CV disease is the leading cause of death There has been a lot of progress made in the CV field, yet….. In the US, – > 18 million people have CV diseases I – > 850,000 death due to CVD in the U.S. each year can ted Eliities Biomedical Innovation In CV Care Is Important Given The Significant Burden Of CVD  Examples of CV Conditions and Treatments Common CV Drugs Hypertension HF Statins Antiplatelets Beta-blockers ACE Inhibitors Ezetimibe (Clopidogrel, ACE Inhibitors ARBs PCSK9 inhibitors Aspirin ) Calcium Channel Beta-Blockers Omega-3 fatty Warfarin Blockers Diuretics acids DOACs Diuretics SGLT2 inhibitors LDL Stages of Drug Discovery  Phases of Clinical Trials Q It takes an average of 10 years for a CV drug to be approved Focused on drug safety and typically enroll up to a few dozen healthy volunteers Dose-ranging studies to demonstrate safety in humans and to assess the pharmacokinetic and Phase 1 pharmacodynamic properties of the drug such as half-life, metabolism, and excretion Months Few dozen to 1000 patients First safety data in patients who have the disease or condition for which the drug is indicated, Phase 2 and may provide preliminary insight into the efficacy of the drug that can be used to plan ~3.5 years subsequent trials Comparative testing of drug efficacy and safety and may enroll 100s to 1000s of patients with the disease or condition of interest. Phase 3 Blinded and randomized, use a comparator, and test clinical end points (e.g, mortality, ~5 years hospitalization, relief of symptoms), as opposed to surrogate markers of disease activity Regulatory approval (FDA) takes about 1 year  US Regulatory Approval smart prologies Once a NDA or a BLA is submitted, the FDA takes about 60 days to accept the application For regular review, the review time is 10 months. Orphan Drug Designation (1983) – Orphan disease defined as