Meet to Focus on Rare Diseases & Orphan Product Research, Development, and Access

Meet to Focus on Rare Diseases & Orphan Product Research, Development, and Access Mobile App Now AvailableClick to download today. US Conference on Rare Diseases & Orphan Products: The New Era in Health Care October 7-9 | North Bethesda, MD Co-sponsored with National Organization for Rare Disorders Overview: At this annual conference, all stakeholders in the rare disease/orphan product community - patients, patient organizations, researchers, drug and device companies, investors, thought leaders and government – meet to focus on rare diseases and orphan product research, development, and access. Search the Online Program. Keynote Speaker: Bill Corr Deputy Secretary U.S. Department of Health and Human Services Luncheon Speaker: FDA Initiatives on Orphan Products Monday, October 7 | 11:45AM-1:00PM Janet Woodcock, MD Director, Center for Drug Evaluation and Research (CDER) FDA Plenary Sessions: • The Affordable Care Act and the Rare Diseases Community • Plenary Session 2: - NINDS NeuroNext Program - NORD Initiative on Natural History Studies • Health Care System of the Future • Research Frontiers in Rare Diseases: The Next Opportunities • The Next 30 Years Session Topics: • Patients and Industry: Partnership and Collaboration in Research Funding and FDA Review • The Investment Environment for Orphan Drugs/Devices • FDA's Orphan Grants Program • FDASIA Provision Highlights • Collaboration from Bench to Bedside: How Industry and Patients Can Partner in Rare Diseases • Hearing the Voice of the Patient • The International Perspective on Orphan Drug/Devices • Assuring Patient Access to Treatments • Paying for Orphan Therapies • Repurposing: FDA and NIH Perspectives • Managing Orphan Drug Recalls and Shortages • NIH Clinical Center-CDER Clinical Trials and Regulatory Training Collaborative • Plus More Just Added: New Tutorial | October 6: Overview of The Regulatory Frameworks and Opportunities for Orphan Medicinal Products (OMPs) The tutorial will describe regulatory frameworks from FDA and EMA and incentives for development of Orphan Medicinal Products (OMPs). It will cover details regarding incentives for developments of OMPs, opportunities and challenges. Additionally, this tutorial will give an overview of the different options to gather control data in rare/ultra rare disease settings. Case studies for requests for ODDs and marketing authorizations for OMPs will be presented and discussed. Overview | Agenda | Register Now Move Toward Better Communication and Collaboration Among Patients, Payers, and Product Developers A Model of Patient, Payer, and Product Developer Collaboration to Support Innovating for ValueOctober 30-31 | Washington, DC DIA and the Brookings Institution are collaborating to strengthen communication between health care stakeholder communities. This conference will focus on highlighting the perspectives and outcomes most relevant to patient communities, reimbursement requirements and innovative payment models of most interest for payer groups, and how both can help inform product developers as they drive toward novel value-based innovation. Register by October 9 to Save $100! Keynote Speaker: James Heywood Co-Founder, Chairman PatientsLikeMe Session Topics: • Aligning Perspectives on Value and Outcomes • Identifying Stakeholder Needs and Challenges to Strategic Communication • Exploring Opportunities for Efficient Engagement Throughout Discovery, Clinical Development, Regulatory Review, and Product Use • Case Studies and Current Initiatives • Next Steps: Charting a Course Forward • And More Overview | Register Online

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Clinical Trail Endpoints: Last Chance to Save on a One-Day Interactive Workshop with Special Emphasis on Rare Diseases - Oct 25 WDC

Last Chance to Save on a One-Day Interactive Workshop with Special Emphasis on Rare Diseases

Clinical Trial Endpoints: Methods and Practice in Developing Measurements
October 25
University of California | Washington, DC

This one-day interactive workshop with special emphasis on rare diseases will provide a detailed examination of the process for developing, validating, and implementing patient-focused clinical trial outcome measures that will meet regulatory requirements for adequate and well-controlled clinical trials, product approval, and labeling.

Session Topics:
     • Attention to Measurement in Clinical Trials: Why it Matters
     • Preparing the Groundwork for Clinically Meaningful Measurement: Getting the Content Right
     • Generating an Instrument with an Interpretable Score
     • Incorporating a Well-Defined and Reliable Measure into  and Adequate and Well-Controlled Study

Program Overview | Register Online

Patients and Patient Organizations are invited to attend the conference, at a reduced registration fee of $400, to network with other stakeholders in the Rare Diseases Community. Submit a registration form by fax to +1.215.442.6199.

Related Events:

US Conference on Rare Diseases & Orphan Products: Shaping the Future Now
October 22-24 | Washington, DC
Co-sponsored by NORD

DIA Adaptive Designs in Clinical Trials: Overcoming Persistent Barriers
November 29-30 | Washington, DC

Data Monitoring Committees: Best Practices and Future Directions
November 29-30 | Washington, DC

Featured Media Partner for DIA/NORD’s US Conference on Rare Diseases & Orphan Products:

BioCentury Publications, Inc. is recognized as the leading provider of value-added information, analysis and data essential to the development and sustainability of life science ventures.

www.biocentury.com

US Conference on Rare Diseases and Orphan Products - Special Challenges in Rare Diseases

October 22-24, 2012
Washington, DC

Special Challenges in Rare Diseases

• Learn how the government and private sector are addressing the special challenges faced by patients and companies in the new health care environment
• Meet individuals who face the same challenges that you do
• Gain a better understanding of how the FDA and NIH work and how they interact with the drug and device development processes
• Learn ways to de-risk your investments in orphan products and understand better the timelines for research and regulatory reviews

Featured Speakers Include
Keynote:

• John J. Castellani: President & CEO Pharmaceutical Research and Manufacturers of America (PhRMA)

The US Conference on Rare Diseases and Orphan Products is Going Mobile! Download the Mobile App Today. The US Conference on Rare Diseases and Orphan Products app is FREE and available for all registered attendees on Android, iPhone, iPad, and Mobile Web version. Download the app to access a wide range of Rare Diseases and Orphan Products information as well as the ability to:      • Create and Manage your Agenda and Appointments      • Receive News and Announcements      • Network with Fellow Attendees      • Receive Event Information in Real-time      • View a List of Exhibiting Companies Download from the Android Market | Download from the Apple Store | Download Mobile Web Version To download, go to your mobile device’s app store and search for DIA/NORD RARE DISEASES. Table Top Exhibits View Exhibiting Companies Eager to Meet with You. Maximize Your Learning in Washington, DC Register for the post-conference workshop,Clinical Trial Endpoints: Methods and Practice in Developing Measurements which has a special emphasis on rare diseases - and save $200 off your registration.* Contact Customer Service if you would like to attend this one-day workshop. Clinical Trial Endpoints: Methods and Practice in Developing Measurements October 25 | 8:30 AM – 5:00 PM *Discounts taken from your Clinical Trial Endpoints: Methods and Practice in Developing Measurements registration fee. Follow #DIARAREDISEASES on Twitter for real-time updates.

Meeting Program