Life Science Items of Interest: Your Tailored Agenda Covering Rare/Orphan Diseases for DIA 2013 49th Annual Meeting

Navigate Your Way to Success at the
Largest Life Sciences Meeting in the Region

We want to make your DIA 2013 49th Annual Meeting experience a breeze. Navigating through more than 250 educational offerings across 22 tracks can be overwhelming to select the sessions that best fit your needs. We have provided a tailored schedule for professionals involved with Rare/Orphan Diseases. We did the work for you!

What’s more, bring your colleagues or your whole team - divide and conquer - with so many exceptional sessions, split the sessions and benefit from more at DIA 2013 49th Annual Meeting. Contact our Group Discount Representative for details!

Don't miss out on this opportunity to meet with people from around the world, share your views and knowledge, network, and build new relationships with the greatest educational offerings under one roof.

Monday, June 24

European Town Hall: Implementation of New Safety Legislation and Other Hot Topics
11:00AM-12:30PM ET
The focus of this forum will be on the first experiences with the new PRAC and how the PRAC interacts with the different EU committees (such as the CHMP and CMDh).

Roundtable on Personalized Therapy Innovation in Rare Disease: Focus on Public Policy
2:30-4:00PM ET
Orphan product developers face unique challenges that multiply when combined with the need for co-registered diagnostic devices. This forum will discuss the latest policy trends impacting personalized orphan products and patient access.

Tuesday, June 25

Research Advances for Rare Diseases and Orphan Products
8:00-9:30AM ET
In 2012, a third of the drugs approved by the FDA had orphan designation. This session will examine the current research efforts and discuss the role of the patient community, the FDA, the NIH and the drug industry in research priorities and challenges.

Is There a Recipe for Successful Implementation of Registries for Rare Diseases?
1:45-3:15PM ET
This session will describe how registries can support development of new drug for rare diseases, how they can contribute to regulatory approval, and decisions on access to orphan medicinal products.

Development for Rare Disease Treatments
4:00-5:30PM ET
This symposium will review various aspects of therapy development for rare and orphan diseases. The difficulties and trade-offs in developing a comprehensive patient registry to assess natural disease progression and treatment effects will be highlighted using pemphigoid as a case example.

Wednesday, June 26

Models for Genomic Research Success: How a Patient-Researcher Relationship Led to the
Discovery of a Norepinephrine Transporter Deficiency and the Emerging Role of Crowd Sourcing
in Rare Disease Research
8:00-9:30AM ET
Get a review of successful genomic studies that have been driven by strong
patient-investigator relationships, and the recent role of social media and crowd sourcing in rare
disease studies.

Using Legislation to Advance Regulatory Science: "I'm Just a Bill..."
8:00-9:30AM ET
Several legislations have been introduced for regulatory reform of the drug approval process. This session will discuss impacts on the pharmaceutical industry, investment community, and patient community.

Rising to the Challenge of Developing Novel Orphan Medicines for the Global Market
1:45-3:15PM ET
One in ten people could be affected by a rare disease; for many there is no treatment, yet modern technology could resolve this. This session investigates the opportunities available to facilitate successful global development of novel orphan medicines.

Rescuing and Repurposing Drugs: Challenges and Opportunities
4:00-5:30PM ET
Drug rescue and repurposing presents an opportunity to spur the drug development process by building upon previous research and development efforts. However, this strategy also has challenges, particularly related to treatments for rare and orphan diseases.

Thursday, June 27

The Not So Rare Challenge that Faces Rare Disease Development: Demonstrate Value
9:00-10:30AM ET
Increased development of orphan products has changed how payer regulators view rare diseases. Rare disorders can now have several products FDA indicated for use; this requires that developers demonstrate the value of these rare disease products.

Orphan Drug Development Strategy by Big and Medium/Small Pharmaceutical Industries
9:00-10:30AM ET
This session will address considerations for planning the development and commercialization strategy of orphan drugs by large as well as small companies.

Sunday, June 23 Preconference Tutorials

Kick off the week by extending your education prior to the start of the Annual Meeting. Register for two half-day tutorials or one full-day tutorial and SAVE $100 on of your Annual Meeting registration.

View Preconference Tutorials

‘Class of 2013’ Patient Advocate Fellowship Program

     • African-American Community Health Group
       of the Central Coast
     • AIM at Melanoma
     • American Cancer Society Cancer Action
       Network
     • Angioma Alliance
     • Ann's Place: the Home of I CAN
     • Coalition for Pulmonary Fibrosis
     • Dystonia Medical Research Foundation
     • Hepatitis Education Project
     • Hermansky-Pudlak Syndrome Network
     • Leukemia & Lymphoma Society - Mass Chapter

• Lymphatic Research Foundation
• MitoAction
• National Foundation for Celiac Awareness
• NephCure Foundation
• PMP Pals
• Program in Personalized Medicine & Targeted
Therapeutics
• Recurrent Respiratory Papillomatosis
Foundation
• Spinal Muscular Atrophy Foundation
• The Michael J Fox Foundation for Parkinson's
Research

Keynote Speaker	Hotel & Travel	Exhibiting

Daniel Kraft, MD Executive Director, FutureMed	Book within the DIA Room Block, get special perks!	90% of the hall is SOLD. Reserve your booth today!