Personalized Medicine & Orphan Drugs Review and Outlook 2010
UBM Canon, August 2010, Pages: 233
The $232 billion personalized medicine market in the U.S. is projected to grow 11% annually over the next several years. Experts believe that eventually personalized medicine will become the standard of care - simply called "medicine." Advancements in science and diagnostic technology are required to reach this standard of care. In addition, health agencies need to establish clear regulatory frameworks for this complicated field, insurers must allow for adequate reimbursement, and medical providers will need to adopt new methods and procedures. Once the groundwork is laid, the benefits will be many, including reduced costs of healthcare by eliminating ineffective treatments, faster approvals due to predictable safety and efficacy, lower rates of adverse reactions, earlier detection and prevention.
Personalized medicine will help developers and marketers identify patient populations most likely to respond to their medications. In addition, increased safety and efficacy profiles provide value and justify higher price points. Strategic alliances, such as the sanofi-aventis alliance with Scripps Genomic Medicine, are being formed with academic and private research institutions as well as small biotech companies as major pharma companies increasingly look for alternative ways to strengthen their pipelines. Roche's Herceptin, Novartis' Gleevec, Agendia's MammaPrint, and Genomic Health Inc.'s Oncotype Dx are all examples of early success stories in personalized medicine. According to FDA, MammaPrint was the first approved product that profiles genetic activity. The genetic test determines the likelihood of breast cancer returning within five to 10 years after a woman's initial cancer. Groundwork Laid for Orphan Drug Success Industry experts estimate that orphan drugs bring in about $40 billion in annual sales. With drug companies facing devastating patent expirations and new drug opportunities drying up in existing markets, orphan drug development has become an enticing solution. FDA and Congress have further lured pharma companies to this niche sector with various incentives.
The Orphan Drug Act of 1983 provides seven-year market exclusivity, tax breaks, and regulatory help for companies developing new drugs for orphan diseases. Since its introduction, more than 2,100 compounds have been submitted for orphan status in the United States and more than 350 have received designation. FDA is urging large drug makers, such as Roche and Johnson & Johnson, to determine if existing medicines may be able to help neglected disorders. Drugs with orphan drug designation are likely to receive fast-track status. Despite the small patient pools they are being developed for, the most successful orphan drugs have more than recouped R&D investment for their developers. Merck's brain cancer drug Temodar was originally approved in 1998 for treating the orphan indication anaplastic astrocytoma, has since been approved for other brain cancer forms, and generated sales of more than $1 billion in 2009. Genzyme Corp., the leader in the orphan drug sector, realized $793 million in sales in 2009 with its Gaucher disease drug Cerezyme. Genzyme's success with Cerezyme and other rare disease treatments is likely the driver behind sanofi-aventis' potential takeover of the company. More large companies are entering the orphan drug sector. Earlier this year, both GlaxoSmithKline and Pfizer Inc. opened research units dedicated to developing new biologics to treat rare diseases.
The most transformational devices to be developed in recent years are drug-eluting stents. These devices improved procedural quantities in two major cardiology markets. Another device success, Vitrasert, is an intraocular sustained delivery system for AIDS-related cytomegalovirus retinitis. This device has been very effective for this indication and, more importantly, has lead to commonplace use of intraocular drug delivery for other indications, including Lucentis for age-related macular degeneration.
This special report focuses on the market leaders for orphan drugs as well as the products in development anticipated to head this area in the years to come. This compilation’s prescription-product pipeline details the following information for each medicine: chemical or substance composition, intended indication, class of drug, clinical status, region of development, and the product developer/intended marketer. Pipeline status details preclinical development, Phase I, Phase I/II, Phase II, Phase II/III, Phase III, and awaiting approval. Regions of development include the United States, Europe, and Japan. A directory of orphan drugs prescription-drug developers and marketers is included. Additionally provided in this report is a listing of personalized medicine and orphan drug medical-device entries, sorted by device class, as well as a directory of medical-device companies associated with the personalized medicine and orphan drug field.
Personalized Medicine Overview
Orphan Drug Overview
Orphan Drugs and Biologics: U.S. FDA Designations
Prescription Orphan Drug and Biologic Pipeline
Awaiting approval
Phase III
Phase II/III
Phase II
Phase I/II
Phase I
Preclinical
Prescription Orphan Drug and Biologic Developers and Marketers
Personalized Medicine and Orphan Drug Devices
Class III
Personalized Medicine and Orphan Drug Device Companies
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