Challenges in Rare Disease Clinical Trials Symposium Sandeep Menon November 6, 2019
Rare Diseases: A High Unmet Need Rare Diseases – High Unmet Need • Approx. 7,000 diseases; affects ~30M in USA EUROPE NORTH AMERICA • Therapies available for 52M ~5% of orphan diseases 40M 220M ASIA • Half of people affected globally are children 77M AFRICA • 30% of children affected by 28M 0.3M a rare disease will not live beyond their 5 th birthday SOUTH AMERICA AUSTRALIA & ANARCTICA Source: GlobalGenes.org 2
Pfizer Rare Disease: At-a-Glance Pfizer Rare Disease – At-a-Glance ~$2B 7 >1,000 2017 Revenue Inline Brands RD Colleagues Worldwide 4 ~100 ~95K Pivotal Trials Countries Patients where Pfizer RD Medicines receiving Pfizer RD Medicines with substantial combined peak are available sales potential Numbers as of mid-2018 3 3
Regulatory Landscape • Since the Orphan Drug Act of 1983 (ODA) was enacted, over 600 drugs and biologics have been approved for rare diseases , compared to roughly 1 drug per year being approved prior to the ODA • Other regulatory constructs to facilitate and encourage rare disease drug development: fast track designation, breakthrough therapy designation, accelerated approval, and RMAT designation and the Complex and Innovative Design Program 4
• Small sample sizes, limited data availability and • Use of external (or natural history) control data poor understanding of disease course instead of concurrent placebo data • Use of placebo controls in clinical studies is often • Innovative clinical trial design methods such as N of unethical 1, adaptive/seamless designs, use of master protocols, and Bayesian methods • Large amount of variability on clinical outcomes 5
Next Up… KANNAN NATARAJAN, PH.D SVP, GLOBAL HEAD OF BIOMETRICS AND DATA MANAGEMENT, PFIZER GIANLUCA NUCCI, PH.D HEAD OF EARLY CLINICAL DEVELOPMENT CLINICAL PHARMACOLOGY, PFIZER 6
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