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

Email Preferences | My DIA | Forward to a Friend |

Waxman Commemorates Orphan Drug Act's Anniversary.

 

The Hill (1/7, Beard) reported in its "Healthwatch" blog that Rep. Henry Waxman (D-CA) commemorated the 30th anniversary of the Orphan Drug Act's passage, saying, "By definition, the market for a drug for rare disease was too small to attract drug companies to do the research and go through [the US Food and Drug Administration's] approval process." The Act, "signed into law by President Reagan on Jan. 4, 1983,...created financial incentives for products that treated those afflicted with rare diseases." According to the National Organization for Rare Disorders, "more than 400 treatments have been approved by the FDA" in the thirty years since the Act was passed.

DIA Daily is a digest of the most important news selected from thousands of sources by the editors of BulletinHealthcare. The presence of content or advertising does not endorse, nor imply endorsement of, any products or services by the Drug Information Association. Neither BulletinHealthcare nor the Drug Information Association is liable for the use of or reliance on any information contained in this briefing.

If you would like more information about, or have a question pertaining to, DIA Membership and its benefits, please email membership@diahome.org.Click here to check your membership status.

Drug Information Association | 800 Enterprise Road, Suite 200 | Horsham, PA 19044

Copyright © 2012 by BulletinHealthcare| 11190 Sunrise Valley Drive, Suite 130 | Reston, VA 20191

 

 

National Health Service to Map DNA of Up to 100,000 Patients

Effort to focus on cancer in hopes of accelerating diagnosis, improving treatment, and sparking innovation.

 

SHERYL P. DENKER

The Burrill Report

“Understanding and harnessing genetic information offers huge potential to target effective treatments and develop new treatments and cures.”

 

The United Kingdom’s National Health Service says it will map the DNA of up to 100,000 patients with cancer and other rare diseases over the next three to five years at a cost of $162 million (£100 million).

“Britain has often led the world in scientific breakthroughs and medical innovations, from the first CT scan and test-tube baby through to decoding DNA,” says Prime Minister David Cameron, “and Britain should become the first country to introduce genetic sequencing to its mainstream health service.”

DIA 2013 49th Annual Meeting - Professional Posters Deadline: February 28, 2013

Call for Professional Posters Submit Your Original Research! Professional Posters Deadline: February 28, 2013 Interested in Presenting at the DIA 2013 49th Annual Meeting?  We are now accepting abstracts for the DIA 2013 49th Annual Meeting Professional Poster Session. Professional posters will be presented on:      • Tuesday, June 24 from 11:45AM-4:00PM      • Wednesday, June 26 from 11:45AM-4:00PM Submission Guidelines | Submit Abstract Online Please Note: DIA username and password is needed to submit an abstract. Did you land on the My DIA Page? If so, please select “My Abstract Submissions” on the left side to submit an abstract. Poster submitters are encouraged to submit topics related to one of the DIA 2013 49TH ANNUAL     MEETING TRACKS noted below:      Track 1: Clinical Operations      Track 2: Project/Portfolio Management and Strategic Planning      Track 3: Innovative Partnering Models and Outsourcing Strategies      Track 4: Nonclinical and Translational Development/Early Phase Clinical Development      Track 5: Regulation of Product Advertising and Marketing in an Ever-changing World      Track 6: Medical Communications, Medical Writing and Medical Liaison      Track 7: Processes and Technologies for Clinical Research      Track 8: Regulatory Affairs and Submissions      Track 9: Medical Devices, In Vitro Diagnostics, and Combination Products      Track 10: Public Policy/Health Care Compliance/Law      Track 11: Innovative Approaches to Ensuring Compliance with Good Clinical Practice,                        and Quality Assurance      Track 12: Pharmaceutical Quality      Track 13: Health Economics and Outcomes (HEO )/Comparative Effectiveness Research                        (CER)/Health Technology Assessment (HTA)      Track 14: Clinical Safety and Pharmacovigilance      Track 15: Statistical Science and Quantitative Thinking      Track 16: Professional Development      Track 17: Rare/Orphan Diseases While the theme for 2013 is “Advancing Therapeutic Innovation and Regulatory Science” the following are suggested hot topics for this year’s Professional Poster Program:      • Neuroscience      • Oncology      • Cardiovascular/Metabolic      • Infectious Diseases      • Rare Diseases      • Women’s Health      • Geriatrics • Video Game Treatment for Therapeutic Intervention • Vaccines and Biotech products • Genomics and Personalized Medicine • Interdisciplinary Clinical Research Activities • Clinical Research, Regulatory and Site Operations • Informatics and Biotechnology • Global Clinical Trials           Benefits:      • Designated poster area and poster board to display poster and network with attendees        at the DIA Annual Meeting in Boston      • Accepted abstracts will be posted on the DIA 2013 49th Annual Meeting website      • Electronic version of the poster to be displayed in the DIA 2013 49th Annual Meeting Live        Learning Center      • Abstract title and lead author will be printed in the final program that is distributed to        the Annual Meeting attendees      • NEW THIS YEAR!         Accepted abstracts will be published in an upcoming edition of the Global Forum,         DIA’s digital association news magazine Selected poster presenters are required to pay the applicable meeting registration fee and all related expenses. Questions? Joanne.Wallace@diahome.org

BIO Investor Forum – Raring to Go: The Race to Treat Ultra-Rare Diseases

from BIOtechNow by biotechnow@bio.org (Biotechnology Industry Organization)

 

The signing of FDASIA by President Obama in July reinforces the country’s commitment to fostering innovation for rare diseases. With a record 460 medicines in late-stage clinical trials, biopharmaceutical companies have embraced rare disease medicine and are rapidly developing the pipeline. In anticipation of an even more compelling opportunity, many companies are taking a step beyond and focusing on ultra rare diseases.

Last week’s 11th Annual BIO Investor Forum in San Francisco hosted a panel, Raring to Go: The Race to Treat Ultra-Rare Diseases, which provided an in-depth analysis of this thriving market opportunity by featuring companies specializing in the ultra rare disease market alongside clinical and policy / regulatory experts in the field.

Moderated by Ellen Licking, Senior Writer & Analyst, Real Endpoints, the panel opened with a focus on the legislation and the regulatory environment. Licking pointed out that there has been a recent uptick in interest on rare diseases which has led to an increase in deal-making. With the recent passage of FDASIA, there have been new opportunities identified for expediting review and approval of rare disease drugs, including accelerated approval, expanded reviewer training and priority review voucher programs to include pediatric rare diseases.

for complete article: BIO Investor Forum – Raring to Go: The Race to Treat Ultra-Rare Diseases

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

 

US Conference on Rare Diseases & Orphan Products: Shaping the Future Now

Register by October 1 to Save US Conference on Rare Diseases & Orphan Products: Shaping the Future Now October 22-24 | Washington, DC Co-sponsored by NORD Register by October 1 to Save! The US Conference on Rare Diseases and Orphan Products features keynote presentations, high-level plenary and smaller group sessions, including comprehensive coverage of today's hottest topics in sessions intended to promote advancements in science, care, and other considerations that will address the needs of the 30 million Americans with rare diseases.  Day 2 Keynote Speaker: Stephen P. Spielberg, MD, PhD Deputy Commissioner, Medical Products and Tobacco Office of the Commissioner Food and Drug Administration Featured Session Topics:      • Access and Reimbursement      • Challenges of Reimbursement for the Rare Disease Patient      • Comparative Effectiveness Research and Health Technology Assessments (HTA)      • Facing the Crisis in Biomedical Innovation: A Venture Investor’s Perspective      • Impact of FDASIA on Orphan Product Development      • Investing in Orphan Products: Is the Environment getting Better Or Worse      • Natural History Studies      • New Relationship with the Patient Community – FDA and the Patient      • New Relationship with the Patient Community – Industry and the Patient      • NIH Updates      • Postmarketing Period-Opportunities for Continued Learning      • Shaping the Future of Health Policy Now      • Working with the FDA   Program Overview Table Top Exhibit Opportunities: Showcase your company's products and services. Submit your application today to Shannon Lewis. Special Offer: Register for both US Conference on Rare Diseases & Orphan Products and Clinical Trial Endpoints: Methods and Practice in Developing Measurements – and Save Up to $500!*  Stay Connected with DIA One-Day Interactive Workshop with a Special Emphasis on Rare Diseases Clinical Trial Endpoints: Methods and Practice in Developing Measurements October 25 | 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. Register by October 4 to Save!   Program Overview | Register Now *Offer expires October 1 and only applicable to the Industry rate. You must register for both events at the same time. Already registered for one of these events? Contact Customer Service

DIA Worldwide Headquarters
800 Enterprise Road, Suite 200
Horsham, Pennsylvania 19044-3595
T. +1.215.442.6100
F. +1.215.442.6199