Trials Symposium Sandeep Menon November 6, 2019 Rare Diseases: A - - PowerPoint PPT Presentation

Challenges in Rare Disease Clinical Trials Symposium

Sandeep Menon November 6, 2019

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Rare Diseases: A High Unmet Need

Rare Diseases – High Unmet Need

• Approx. 7,000 diseases;

affects ~30M in USA

• Therapies available for

~5% of orphan diseases

• Half of people affected

globally are children

• 30% of children affected by

a rare disease will not live beyond their 5th birthday

40M 52M 220M 77M 28M 0.3M

NORTH AMERICA EUROPE AFRICA SOUTH AMERICA ASIA AUSTRALIA & ANARCTICA

Source: GlobalGenes.org

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Pfizer Rare Disease: At-a-Glance

Pfizer Rare Disease – At-a-Glance

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~$2B 7 >1,000 4 ~100 ~95K

2017 Revenue Inline Brands RD Colleagues Worldwide Pivotal Trials with substantial combined peak sales potential Countries where Pfizer RD Medicines are available Patients receiving Pfizer RD Medicines

Numbers as of mid-2018

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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

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• Small sample sizes, limited data availability and

poor understanding of disease course

• Use of placebo controls in clinical studies is often

unethical

• Large amount of variability on clinical outcomes
• Use of external (or natural history) control data

instead of concurrent placebo data

• Innovative clinical trial design methods such as N of

1, adaptive/seamless designs, use of master protocols, and Bayesian methods

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Next Up…

GIANLUCA NUCCI, PH.D

HEAD OF EARLY CLINICAL DEVELOPMENT CLINICAL PHARMACOLOGY, PFIZER

KANNAN NATARAJAN, PH.D

SVP, GLOBAL HEAD OF BIOMETRICS AND DATA MANAGEMENT, PFIZER