Rare Disease, Orphan Conditions, Patient Advocacy sessions at Philly June DIA2012

DIA 2012 Collaborate to Innovate

48th Annual Meeting
June 24-28, 2012
Philadelphia, PA
Pennsylvania Convention Center

•Orphan Drug Development: Global Regulatory Challenges and Initiatives
This impactful session will focus on the critical need for developing orphan drugs, provide a review of global orphan drug development challenges, and offer information on various strategies designed to overcome these challenges.

•Challenges of Orphan Drugs in the US, EU, and Japan
This session addresses the current status and forthcoming activities related to orphan drugs in the US, EU, and Japan. Orphan drug development is obviously essential in these regions, and the provisions in place on drugs including rare diseases are often passed over. We will also address the unique market situation of orphan drugs in the US and development and commercialization strategies of orphan drugs in each region.

•Natural History Studies for Rare Diseases and Orphan Conditions
This session presents an overview of the design, conduct, and benefits of natural history studies of rare diseases and orphan conditions from varied perspectives of researchers, patients and a regulatory agency.

•Social Media 2.0: The Power of Online Rare Disease Communities to Connect and Engage ePatients
FORUM

•Drug Development Strategies for Integrating Academia, Non-governmental Organizations (NGOs), and Industry Based on Experience in Neglected and Infectious Diseases
This session will describe drug development strategies for integrating academia, non-governmental organizations (NGOs), and industry based on experience for the development of drugs for neglected and infectious diseases in the global health arena.

•Patient Advocacy in Medical Product Development: The Evolving Relationship Between FDA and Its Patient Stakeholders
This session will explore the role of patient advocates in FDA decision-making. FDA will describe its patient advocacy programs and the value they have added. A patient representative will discuss their interactions and experience with the agency.

•Rare Disease Clinical Research Consortia: Immediate and Rich Sources of Translational Research Data, Partnering Opportunities
Rare disease clinical research consortia offer a rich data source for translational research. Investigating diseases from porphyria to pediatric cholestasis, they present an array of collaborative opportunities for the pharmaceutical industry.

•Patient Advocacy and Your Next Generation of Research:  How Nonprofit Organizations Can Accelerate Product Development
This session provides insights into the rapidly emerging world of collaborations between pharma and patient advocacy organizations to accelerate basic and clinical research by leveraging the data and participation from patients.

•Understanding the Challenges of Conducting Studies for Orphan Indications and Rare Diseases
Conducting a research study for an orphan indication and/or rare disease involves unique considerations in addition to the more common challenges of clinical research. Key logistical and operational considerations will be discussed including sharing of specific, relevant examples from different therapeutic areas and clinical settings as well as strategies and techniques to navigate the challenges. The session will also address specific examples of materials, procedures and techniques developed and utilized for protocol/study design development, site identification and support, IRB/EC approval, subject recruitment and retention, subject support, and program timeline and budget management.
 
•Emerging Role of the Patient Voice on Drug Policy in Japan
Patients and lay people in Japan now form one of the major groups that support current drug development, safety, supply, and regulatory affairs. This session will discuss recent change and the significant effect of the patient voice on drug policy and the progress of the amendment of the Pharmaceutical Affairs Law.