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Trk 17: Rare-Orphan Diseases [clear filter]
Tuesday, June 28
 

10:30am

#227: Unique Global Regulatory Considerations and Drug Development Incentives in Rare Disease and Orphan Drug Development
Limited Capacity seats available

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



Chair
avatar for Maureen Smith

Maureen Smith

Patient Advocate / Secretary, Canadian Organization For Rare Disorders (CORD)
Maureen Smith’s M.Ed. (Educational Psychology) interest in patient advocacy stems from a rare disease diagnosis at the age of 8. She is the Secretary of the Canadian Organization for Rare Disorders (CORD), co-chair of the Rare/Orphan Disease program track for the Drug Information... Read More →

Speakers
IP

Irene Pan

Senior Research Scientist, UBC: An Express Scripts Company
Irene Pan, MSc, is a Senior Research Scientist with UBC: An Express Scripts Company. With over 12 years of clinical research experience, Ms. Pan has conducted numerous studies in a wide range of therapeutic indications including ophthalmology, neurology, cardiology, oncology and respiratory... Read More →
avatar for Lauren Tornetta

Lauren Tornetta

Director, Global Regulatory Affairs, Pfizer Inc
15 years of experience across public and government (FDA) sectors with an MBA in biotechnology and an MS in biochemistry. Global experience leading reg strategy with drugs (NCE, line extensions, drug: drug codev, biologic products, and combination products [drug: device]), in all... Read More →


Tuesday June 28, 2016 10:30am - 11:30am
103C Pennsylvania Convention Center 1101 Arch Street, Philadelphia, PA 19107 USA

2:00pm

#252: Capturing Real-World Data in Rare Diseases
Limited Capacity seats available

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



Chair
avatar for Badri Rengarajan

Badri Rengarajan

Medical Affairs Lead, ASPIRE Unit, Actelion
15 years’ experience across medical affairs, product development strategy and new product planning, regulatory affairs, business development, strategy: Former Board President, International Pemphigus and Pemphigoid Foundation (rare disease foundation); Senior Medical Director, Archimedes... Read More →

Speakers
avatar for Donny Chen

Donny Chen

Senior Director, Medical Affairs Research Operations, PPD
Donny Chen, senior director of medical affairs research operations at PPD, has spent the last 18 years designing and spearheading observational research studies in a variety of therapeutic areas, with particular expertise in strategy, analysis and project management. Mr. Chen graduated... Read More →
avatar for Derenda Nichols

Derenda Nichols

Senior Director, Clinical Trial Management, Medpace
Derenda Nichols has more than 25 years experience in the CRO industry. Her diverse background includes monitoring and site management, regulatory affairs, medical writing, global project management, operations and most recently, executive management. She has successfully participated... Read More →


Tuesday June 28, 2016 2:00pm - 3:00pm
103C Pennsylvania Convention Center 1101 Arch Street, Philadelphia, PA 19107 USA
 
Wednesday, June 29
 

2:00pm

#353: The Utility of Natural History Studies in Drug Development and Approval
Limited Capacity seats available

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



Chair
avatar for James Valentine

James Valentine

Attorney, Hyman, Phelps & McNamara, PC
James Valentine, JD, MHS is an attorney at Hyman, Phelps & McNamara where he assists medical product industry and patient advocacy organization clients in a wide range of regulatory matters, including new drug and biologic development and approval issues. Mr. Valentine has been central... Read More →

Speakers
avatar for Jonathan Goldsmith

Jonathan Goldsmith

Associate Director for Rare Diseases, Office of New Drugs, CDER, FDA
Dr. Goldsmith is Associate Director for Rare Diseases in the Office of New Drugs/CDER/FDA. Prior to federal service, he was a tenured professor in academia and focused on clinical drug development in regulated industry, at NHLBI/NIH and at orphan disease foundations. He earned his... Read More →
avatar for Camilla Simpson

Camilla Simpson

Group Vice President Regulatory Affairs, BioMarin Pharmaceutical Inc.
Camilla Simpson is a Global Regulatory Affairs executive with 20+ years in the Biopharmaceutical Industry. She is currently working for BioMarin, where she leads a team of 150 professionals. Prior to this she was with Shire Pharmaceuticals, and Abbott Laboratories. She is experienced... Read More →


Wednesday June 29, 2016 2:00pm - 3:00pm
103C Pennsylvania Convention Center 1101 Arch Street, Philadelphia, PA 19107 USA

4:00pm

#376: Pediatric Rare Disease Drug Development
Limited Capacity seats available

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



Chair
avatar for Kinnari Patel

Kinnari Patel

Vice President, Head of Regulatory, Pharmacovigilance, and Compliance, Rocket Pharmaceuticals

Speakers
MM

Mary Murphy

Director, Office of Pediatric Therapeutics, Office of Special Medical Programs, FDA
Dianne Murphy, MD, FAAP, is Director of the Office of Pediatric Therapeutics (OPT) Office of the Commissioner at FDA. Dr. Murphy was a medical officer at Bethesda Naval; Dir. of the virology laboratory at the U. of Tenn., Knoxville & tenured Professor at the U. of Florida, Jacksonville... Read More →
avatar for Lynne Yao

Lynne Yao

Director, Division of Pediatric and Maternal Health, Office of New Drugs, CDER, FDA
Lynne Yao, MD, is the Director, Office of New Drugs, Division of Pediatric and Maternal Health in the Center for Drug Evaluation and Research, US Food and Drug Administration. The Division of Pediatric and Maternal Health oversees quality initiatives within the Office of New Drugs... Read More →


Wednesday June 29, 2016 4:00pm - 5:15pm
103C Pennsylvania Convention Center 1101 Arch Street, Philadelphia, PA 19107 USA