Rare/Orphan Diseases Track at DIA's 49th Annual Meeting

	Is there a recipe for successful implementation of Registries for RareDiseases?
	What is the emerging role of Crowdsourcing in Rare Disease research?
	What incentives are available in US, EU and Japan to develop orphan medicines?
	How can my organization engage with rare disease patient communities to develop treatment?
	What are the challenges inherent in drug rescue and repurposing for rare disease and orphan products?
	How do developers demonstrate the value of rare disease products to payer regulators?
	What are the challenges facing development of new orphan drugs?

Project/Portfolio Management Track at DIA's 49th Annual Meeting

 

• Effective Diverse Team Collaboration and Management for drug development: East meets West  
• So you want to be a pharma Project Manager?
• What are the requirements for submissions to HTA organizations that affect the drug development team?
• What is the importance of proactively leveraging country selection to support marketing plan objectives?
• How do large and small pharma companies develop orphan drug commercialization strategies?
• Interactive Workshop ~ What are the process and decisions made during Phase 2 drug development?
• Interactive Workshop ~ What are the factors influencing a decision to file for an NDA?What are the key aspects in developing and managing budgets for global drug development projects?
• Where will your project/portfolio management role lead you?
• How do you navigate potential Regulatory land mines in Due Diligence?
• What PM practices are most critical to project success in  biopharma industries?
• How can you influence your organization to create and adopt project management competencies?
• What are financial, resource and planning challenges of Incorporation of Mandatory Language into protocols?
• How can adopting an Adaptive Design Strategy help you Manage Portfolio Risk and Value? Using Innovation to Bridge the Gap Between Strategy and Execution
• How does application of pharmacometrics affect range of drug  development and decision-making activities?
• How are drug development approaches being adopted by companies that develop biosimilars?
• How will Clinical Trials Transformation Initiative’s potential methods apply to QbD?
• What techniques and tools can be used to apply QbD in designing and running clinical development programs?

 

DIA and the Rare Diseases Patient Community

Rare Disorders Without Borders Today marks the sixth international Rare Disease Day when hundreds of patient organizations from more than 60 countries participate in activities to raise awareness about the 6,000 rare diseases affecting more than 60 million people in Europe and the US alone. View the official Rare Disease Day video. View the official Rare Disease Day video. DIA and the Rare Diseases Patient Community DIA is working to ensure that the "voice of the patient' is heard globally in every facet of the discovery, development, and life cycle management of pharmaceuticals, biotechnology, medical devices, and related products. Visit the DIA Rare Disease Day site for upcoming events, educational materials, videos, and more. Important Dates: RESOURCES: DIA 2012: Orphan Drug Development How is DIA Raising Awareness? In 2012, DIA and NORD hosted the 2nd Annual US Conference on Rare Diseases and Orphan Products which brought together more than 300 stakeholders - patients, patient organizations, researchers, drug and device companies, investors, thought leaders and government – that met to discuss rare diseases and orphan product research, development and access to address the needs of the millions that suffer with rare diseases. Save the dates for 2013: October 7-9 in Bethesda, MD. How is DIA Helping? "My daughter suffers from a rare disease called Mastocytosis. We just found out last summer that I also have another version of the same disease called Mast Cell Activation Disorder. There is a medication that is available for us to take it is called Zaditen and it is only available in the UK and the DIA is actually helping us right now to get this medication into the United States so we can continue to use it.” - Kelli Foster, Patient Advocate, The Mastocytosis Society “And for the people that have one of these conditions, they can be life-altering, and in some cases, life-ending, and having that voice for those people and having a group that is willing to listen to us and wants to learn more about it, it’s so meaningful, it’s very hard to describe.” - Daniel Smith, National Dysautonomia Research Foundation Read More In the News As Rare Disease Day Approaches, Patient Advocates Celebrate 30 Years of Collaboration But Worry About Looming Budget Cuts Now THAT'S a Great Friend: Seven-Year-Old Boy Raises Over $300,000 to Help Find a Cure for His Classmate's Rare Genetic Condition Man with a Plan: Emil Kakkis Aims to Lure Big-Market Drugs Toward Rare Diseases Orphan Drugs – An Alternative to the declining Blockbuster Model? Join the Conversations on Rare Disease Day’s Social Media facebook.com/rarediseaseday ‘Like’ the RDD Facebook page, view our timeline and participate in the conversation on Rare Disease Day twitter.com/rarediseaseday Follow Rare Disease Day on Twitter @rarediseaseday and use the hashtag #raredisease youtube.com/rarediseaseday Watch the videos, comment and link to the many videos and upload your video to the Rare Disease Day channel via rarediseaseday.org flickr.com/photos/rarediseaseday Upload your photo on to the Rare Disease Day Photo Wall

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