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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A Big Year for Novel Drugs Approvals

What was your top Biopharma highlight for 2012? If it wasn’t the stellar move in the AMEX Biotech Index (up 42%), or the reauthorization of PDUFA, then perhaps it was the decade high for innovative drugs approvals. We would need to go back to the 1990s to find a year with as many FDA approvals for novel drugs as we had in 2012. Adding to the positive trend in numbers, up 29% from the robust 31 approvals in 2011, a number of last year’s approvals came earlier than expected. Vertex’s Kalydeco and Ariad’s Iclusig, for example, both came three months ahead of their respective PDUFA dates. Over 50% of the approvals had FDA Priority review or Orphan designations.



http://www.biotech-now.org/business-and-investments/inside-bio-ia/2013/01/a-big-year-for-novel-drugs-approvals?utm_source=Feedburner&utm_medium=feed&utm_campaign=Feed%3A+BiotechNow+%28BIOtech+Now%29&utm_term=RSS+Subscription

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.

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FDA & Compounders: More Oversight Needed?

FDA & Compounders: More Oversight Needed?

from Pharmalot by Ed Silverman

Over the past two years, compounded medications have figured in two highly controversial episodes. In one instance, Roche attempted to prevent ophthalmologists from using its older Avastin med for treating wet macular degeneration when its newer and more expensive Lucentis is the only approved treatment. The drugmaker has claimed that rejiggered vials of Avastin pose a safety risk (read here and here).

In another, KV Pharmaceutical caused a huge ruckus by initially charging $1,500 for its Makena premature birth drug and trying to use its FDA approval under the Orphan Drug Act to prevent compounding pharmacies, which charged a fraction of the price, from competing. The move outraged physicians, among others, who maintained the compounded versions were safe and kept a lid on rising healthcare costs.

Now, an outbreak of meningitis that has been traced to a compounding pharmacy in Massachusetts has, once again, placed compounding in the spotlight, and renewed debate about the extent to which oversight is sufficient. The outbreak, which has left five people dead and another 30 in various stages of illness across six states, appears to have been caused by a compounded steroid drug contaminated by a fungus.

for full article:
FDA & Compounders: More Oversight Needed?

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

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