DIA 2016 Annual Meeting

Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-16-590-L01-P; CME 1.00; IACET 1.00; RN 1.00

Early consideration of the issues unique to rare disease development allows sponsors to proactively and adequately identify/address them during development and to have productive interactions with Health Authorities (HA). Unique program elements will be compared and contrasted, focusing on regulatory aspects of rare/orphan development and early HA consult to create successful programs that address the particular challenges. We will also discuss how to strategically take advantage of the various US and EU incentives such as market exclusivity, tax reduction, and expedited review process, as well as grants to facilitate the development of orphan drugs, to enhance orphan drug development.

Learning Objectives

Discuss the regulatory requirements unique to the global development of rare disease and orphan drugs, including the regulatory implications; Explain the regulatory considerations to create efficient, feasible, and sufficient rare disease and orphan drug programs; Summarize regulations regarding orphan drug designations in the US and EU; Discuss and demonstrate how drug developers can take advantage of the regulators' incentives to optimize their investment in orphan drug development.

Chair

Maureen Smith

Speaker

Maximizing the Advantages of US and EU Orphan Drug Designation Incentives
Irene Pan

Global Regulatory Considerations in Rare Disease and Orphan Drug Development: When Patients Are Waiting and Everyday Counts
Lauren Peterson Tornetta

Component Type: Session
Level: Basic
CE: ACPE 1.00 Knowledge UAN: 0286-0000-16-591-L01-P; CME 1.00; IACET 1.00; RN 1.00

Although much can be learned from interventional-type clinical trials, a fuller picture of disease course and experiential journey can only be understood by observing patients longitudinally in real-world settings where co-morbid conditions, medication noncompliance, economic considerations, and a host of other factors come into play. However, capturing real-world data in rare diseases poses distinctive demands: fewer patients, patient inability to report on disease experience due to youth or infirmity, and need for longitudinal tracking. This panel seeks to present conventional and novel approaches to capturing real-world data in rare diseases.

Learning Objectives

Discuss the benefit of real-world data; Summarize the distinctive demands for real-world data and data collection in rare diseases; Describe ways to address real-world data demands in rare diseases.

Chair

Badri Rengarajan, MD

Speaker

Unique Paradigms to Rare Diseases Research
Donny Chen, MBA

Real-World Evidence and Rare Diseases
Derenda Nichols

Component Type: Session
Level: Intermediate
CE: CME 1.00; IACET 1.00; RN 1.00

A well-designed natural history study can help in designing an efficient drug development program and support marketing approval. This session will discuss the utility of natural history studies and strategies for designing them.

Learning Objectives

Recognize the ways in which a natural history study can guide each stage of drug development; Identify features of natural history registries that will support drug development activities; Summarize the use of natural history controls to support important FDA drug approvals.

Chair

James E. Valentine, JD

Speaker

FDA Draft Guidance and the Utility of Natural History Studies in the Development of Drugs for Rare Diseases
Jonathan C. Goldsmith, MD, FACP

Considerations in the Use of National History Studies: Development and Registration Perspective
Camilla V. Simpson, MS

Use of Historical Controls to Support Drug Approvals
James E. Valentine, JD

Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-16-593-L01-P; CME 1.25; IACET 1.25; RN 1.25

This session will focus on the critical need for developing medicine for orphan diseases impacting the pediatric population, review global pediatric orphan drug development challenges, and provide information on various strategies designed to overcome these challenges.

Learning Objectives

Specify updates in regulatory policy and industry activities in pediatric orphan drug development; Discuss key clinical development challenges in demonstrating substantial evidence of effectiveness for orphan diseases in pediatrics; Review initiatives to address clinical development and regulatory approval challenges.

Chair

Kinnari Patel, PharmD, MBA, RPh

Speaker

FDA Perspective
Mary Dianne Murphy, MD

CDER Perspective
Lynne P. Yao, MD

Industry Perspective
Kinnari Patel, PharmD, MBA, RPh