Balancing Patient-Centric and Model-Informed Drug Development (MIDD) Approaches to Optimize Dose Selection in Rare Diseases: Are We There Yet?

Session Description

This session addresses a topic of high relevance for patient-centric clinical pharmacology of therapeutics in the rare disease space. Clinical development programs for rare diseases are vastly diverse in terms of their strategy to address dose-selection and justification. Some programs have conducted formal dose-finding studies, some incorporate dose-finding aspects in a pivotal study, and others directly utilize the dosage approved in another indication. While it is natural that dose-selection strategies are inherently disease (and drug)-specific, the goal of identifying an optimal dose to ensure patient-centricity should essentially be disease-agnostic.

Additionally, in the setting of rare diseases, patients can be hard to find, and many have life-threatening conditions with no available therapy, thus necessitating innovative ways of thinking to select a therapeutic dose efficiently and quickly.

The regulatory institution is best set up to balance the patient centricity aspect with public health needs and so their perspectives on leveraging MIDD approaches augmented with appropriately validated biomarkers at different stages of drug development paradigm is also a key aspect.

Learning Objectives

1. Appreciate the delicate balance between enabling robust dose-selection in rare diseases and ensuring patient-centricity
2. Critically assess if MIDD is being optimally used to inform dose-selection for pivotal clinical studies in rare diseases and what more can be done
3. Understand patient-centric themes and regulatory perspectives for dose-decisions in drug development programs

Session Schedule

Introductory Remarks
Noha Rayad, PhD and Sathej Gopalakrishnan, PhD

Patient-Centricity in Rare Diseases
Roberta Smith, CNMT

Point vs Counterpoint

Discussion: Progress Made in MIDD Enabling a Robust Dose-Selection
Rajesh Krishna, PhD, MBA

Point vs Counterpoint

Discussion: Challenges and Limitations in Dose Selection for Rare Diseases: A Realistic Perspective
Robert Schuck, PhD

Panel Discussion