- published: 03 Feb 2012
- views: 2245
An orphan drug is a pharmaceutical agent that has been developed specifically to treat a rare medical condition, the condition itself being referred to as an orphan disease.
In the US and EU it is easier to gain marketing approval for an orphan drug, and there may be other financial incentives, such as extended exclusivity periods, all intended to encourage the development of drugs which might otherwise lack a sufficient profit motive. The assignment of orphan status to a disease and to any drugs developed to treat it is a matter of public policy in many countries, and has resulted in medical breakthroughs that may not have otherwise been achieved due to the economics of drug research and development.
According to Thomson Reuters in their 2012 publication "The Economic Power of Orphan Drugs", there has been increased investing in orphan drug Research and Development partly due to the U. S. Orphan Drug Act of 1983 (ODA) and similar Acts in other regions of the world and also driven by "high-profile philanthropic funding." The period between 2001 to 2011 was the "most productive period in the history of orphan drug development, in terms of average annual orphan drug designations and orphan drug approvals." For the same decade the compound annual growth rate (CAGR) of the orphan drugs was an "impressive 25.8 percent, compared to only 20.1 percent for a matched control group of non-orphan drugs." By 2012 the market for orphan drugs was worth USD$637 million compared to the USD$638 million matched control group of non-orphan drugs.
Why an Orphan market should be approached differently What is done differently in Orphan markets compared to larger markets How to leverage Orphan market dynamics to drive value for your product
12 Hour MBA in Orphan Drugs The 12 Hour MBA in Orphan Drugs is an introductory-level online training course designed to bridge knowledge gaps. It is most useful to new entrants, senior managers needing a big picture refresher and professional advisors and suppliers to the field. Explore real-world lessons they don't teach you on the job, from the comfort of your desk. In just 12 hours you will: - Gain a comprehensive overview of the orphan drugs industry and market - Look at worldwide orphan drugs legislation - Discover market access procedures for orphan drugs - Understand some of the challenges that patients with rare diseases have to face - Learn about the role of advocacy groups and rare disease networks - Find out how rare disease research is funded in Europe and the US - Discuss th...
12 Hour MBA in Orphan Drugs The 12 Hour MBA in Orphan Drugs is an introductory-level online training course designed to bridge knowledge gaps. It is most useful to new entrants, senior managers needing a big picture refresher and professional advisors and suppliers to the field. Explore real-world lessons they don't teach you on the job, from the comfort of your desk. In just 12 hours you will: - Gain a comprehensive overview of the orphan drugs industry and market - Look at worldwide orphan drugs legislation - Discover market access procedures for orphan drugs - Understand some of the challenges that patients with rare diseases have to face - Learn about the role of advocacy groups and rare disease networks - Find out how rare disease research is funded in Europe and the US - Discuss th...
Chris Garabedian, Chief Executive Officer of Sarepta Therapeutics, presented at World Orphan Drug Congress USA 2013 on the topic, 'Leveraging a personalized medicine platform: an approach to treat all patient genetic sub-groups in a rare disease.' • Establishing a foundation of evidence • Standardizing the preclinical and clinical approach • The regulatory environment World Orphan Drug Congress USA is the largest international, commercially-focused event for the advancement of rare disease research and orphan drug development. For more information, go to www.terrapinn.com/orphandrug. Or, check out our blog at blogs.terrapinn.com/total-biopharma for up to date information on the orphan drugs and rare disease.
In this exclusive interview with Rare Disease Report, Ms Divino discusses the study’s methods, results, and conclusions. For complete details of the study, click here. https://ash.confex.com/ash/2014/webprogram/Paper68994.html Reference Divino V, Dekoven M, Wang W, et al. Budget impact of orphan drugs in U.S.: A 2007-2013 MIDAS data sales analysis. Presented at: American Society of Hematology Annual Meeting; December 6-9, 2014; San Francisco. Abstract #736.
A presentation for BTEC 2320, describing the events before and the circumstances surrounding the Orphan Drug Act of 1983, and the Rare Disease Industry.
Speaker: James H. Reese (FDA) First plenary session: orphan designation around the World Worldwide Orphan Medicinal Designation Workshop 10 March 2014
Orphan products grants program supports the clinical development of products for use in rare diseases or conditions where no current therapy exists or where the proposed product will be superior to the existing therapy. FDA OOPD provides grants for clinical studies on safety and/or effectiveness that will either result in, or substantially contribute to, market approval of these products.
“US Orphan Drug Market Outlook 2018” research report by KuicK Research comprehensive insight on following developments related to US orphan drug market: US Orphan Drug Market Overview Orphan Drug Designation Criteria Market Specific Reimbursement Policy & Regulatory Framework US Orphan Drug Pipeline by Phase, Indication & Originator Marketed Orphan drug List by Indication & Brand Name Key Issue to be Resolved Competitive Landscape
“Global Orphan Drug Market Outlook 2018” research report by KuicK Research comprehensive insight on following developments related to global orphan drug market: Global & Regional Orphan Drug Market Overview Orphan Drug Designation Criteria Across Key Markets Market Specific Reimbursement Policy & Regulatory Framework Orphan Drug Pipeline by Phase, Orphan Designated Disease & Country Competitive Landscape
"Global Orphan Drug Market Future Outlook 2020" report highlights: Global Orphan Drug Market Overview Global Orphan Drug Market Segment Analysis FDA & EMA Regulation for Clinical Trials Orphan Designated Drugs Orphan Drug Designation Criteria & Reimbursement Policy by Region Comprehensive Insight on Global Orphan Drugs Clinical Pipeline & Patent Analysis by Company, Country, Indication & Phase Global Orphan Drugs Clinical Pipeline: 697 Drugs Majority Orphan Drugs in Phase-II Trials: 249 Drugs Marketed Orphan Drugs: 274 Drugs
“Global Orphan Cancer Drug Market & Clinical Pipeline Insight” Report Highlights: Orphan Cancer Drug Market Outlook Orphan Drug Designation Criteria: US, Europe & Asia Reimbursement Policy: US, Europe & Asia Regulatory Framework: FDA & EMA Guidelines Orphan Cancer Drugs Clinical Pipeline by Phase, Country & Target Indications Orphan Cancer Drug Patent Analysis Orphan Cancer Drug in Development Phase: 235 Majority of Orphan Cancer Drugs in Phase-II: 81 Marketed Orphan Cancer Drugs: 70
“Europe Orphan Drug Market Outlook 2018” research report by KuicK Research comprehensive insight on following developments related to Europe orphan drug market: Europe Orphan Drug Market Overview Orphan Drug Designation Criteria Market Specific Reimbursement Policy & Regulatory Framework Europe Orphan Drug Pipeline by Phase, Indication & Originator Marketed Orphan drug List by Indication & Brand Name Key Issue to be Addressed Competitive Landscape
Brad Crittendale, president of the Canadian Association of Pompe talks about the current regional systems that are set up to dispense orphan drugs to Canadians and why this fragmented system is one of the reasons why the country needs an Orphan Drug Framework at the national level.
Conferenceseries LLC invites all the participants from all over the world to attend ‘Annual Congress on Rare Diseases and Orphan Drugs’ during Oct 26-27, 2016 in Chicago, USA which includes prompt keynote presentations, Oral talks, Poster presentations and Exhibitions. This scientific gathering guarantees that offering the thoughts and ideas will enable and secure you the theme “The voice of rare diseases - research and treatment”. Rare Diseases-2016 is a global platform to discuss and learn about Rare Diseases, rare cancer, morgellons, renal cell carcinoma, malignant glioma, Turner syndrome, Multiple myeloma, Hepatocellular carcinoma, Diabetes in the young, Systemic Amyloidosis, Autoimmune diseases, Disorders of genetic origin, idiopathic disorders, rare genetic diseases, Orphan Drugs, Al...
Get your free audiobook or ebook: http://zaxo.space/mabk/30/en/B00LBXYKVU/book Rare Diseases and Orphan Drugs shows that much of what we now know about common diseases has been achieved by studying rare diseases. It proposes that future advances in the prevention, diagnosis, and treatment of common diseases will come as a consequence of our accelerating progress in the field of rare diseases. Understanding the complex steps in the development of common diseases, such as cancer, cardiovascular disease, and metabolic diseases, has proven a difficult problem. Rare diseases, however, are often caused by aberrations of a single gene. In rare diseases, we may study how specific genetic defects can trigger a series of events that lead to the expression of a particular disease. Often, the disease ...
Listen to the full audiobook, or read it's ebook version: http://downloadapp.us/mabk/30/en/B00H1YWV3C/book This authoritative and comprehensive book makes the reader familiar with the processes of bringing orphan drugs to the global market. There are between 5,000 and 7,000 rare diseases and the number of patients suffering from them is estimated to be more than 50 million in the Us and Europe. Before the orphan drug legislation enacted in the Us in 1983, there was a limited interest from industry to develop treatment for very small patient groups. One of the difficulties is, of course, that similar levels of investment are needed from a pharmaceutical company to bring a drug to the market for both small and large patient groups.the journey from application of an orphan drug designation to...
Martine Zimmermann, Executive Director Global Regulatory Affairs at Alexion Pharmaceuticals, presented at World Orphan Drug Congress USA 2013 on the topic, 'Where industry stumbles in Europe's orphan drug regulatory process.' World Orphan Drug Congress USA is the largest international, commercially-focused event for the advancement of rare disease research and orphan drug development. For more information, go to www.terrapinn.com/orphandrug. Or, check out our blog at blogs.terrapinn.com/total-biopharma for up to date information on the orphan drugs and rare disease.
From the 15th Annual BIO CEO & Investor Conference at the Waldorf Astoria Hotel in New York on February 12th, 2013 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 with hopes of achieving the ever elusive blockbuster drug. Some companies have even taken a step beyond and focused primarily on ultra rare disease, potentially providing an even more compelling opportunity. This panel will provide an in-depth analysis of the thriving market opportunity that is rare disease by featuring industry, clinical and regulatory experts specializing in this exciting...
Early access to orphan drugs – the rationale, challenges and options available • Why consider early access? • What are the challenges for early access? • Introduction to the mechanisms, regulatory environment and options for early access. • Current trends in early access • Insight into the potential benefits and risks of early access Mark Corbett, Senior Vice President, Global Access Programs, Clinigen Pharma World Orphan Drug Congress USA 2014
On April 10, 2013, Freda Lewis-Hall, MD, Chief Medical Officer of Pfizer, Inc delivered the keynote speech at Terrappin's World Orphan Drug Congress USA 2013 in Washington, D.C. To learn about Pfizer's commitment to rare diseases, go to http://www.pfizer.com/partnering/areas_of_interest/rare_diseases.jsp
MYTHS AND REALITIES OF THE CANADIAN ORPHAN DRUG MARKET, presented by Éric Gervais at the World Orphan Drug Congress Europe, Geneva, November 2013
John Maddox, Managing Director of Infusion Pharma Consulting, presented at World Orphan Drug Congress USA 2013 on the topic, 'The impact of payers financial reality in shifting the orphan drugs pricing environment.' • What happens if the pricing latitude for orphan drugs is discontinued? • How difficult it is to get pricing in a potential low value market? • Competitiveness in the orphan drug space to benefit payers. What are payers' ability to pay? World Orphan Drug Congress USA is the largest international, commercially-focused event for the advancement of rare disease research and orphan drug development. For more information, go to www.terrapinn.com/orphandrug. Or, check out our blog at blogs.terrapinn.com/total-biopharma for up to date information on the orphan drugs and rare dise...