Rare Diseases: Solutions for Orphan Drugs
Orphan diseases affect fewer than 200,000 people nationwide. These diseases include: Cystic fibrosis, Lou Gehrig’s disease, and Tourette’s syndrome. Rare disease are discovered almost every year, and will be either be inherited, by defective genes (Human body has over 30,000 genes, and if one of those genes is defective or mutated, prevents the DNA that carries information needed to make proteins – building blocks of the body. Sometimes may cause serious disease), which happens one out of every 200 births (There are more than 6,000 known single – gene disorders) or environmental toxic conditions. Patients that have rare diseases are difficult to diagnose, since their disease, not well known to many physicians. National Institute of Health, does research for treatments. Gene Therapy provides means of curing genetic diseases, either by replacing, manipulating or supplementing nonfunctional genes, with healthy genes.
In 1983, the United States Congress passed the Orphan Drug Act (ODA). The ODA created incentives for drug companies, and manufactures to receive tax credits, for cost of clinical research, government grant funding, and seven-year period to receive exclusive marketing – rights, when an orphan drug is produced and marketed, after approval from the Food and Drug Administration. From 1983 – 2003, 250 orphan drugs have been developed. In 2004, Food and Drug Administration approved 150 drugs and treatments, for orphan designation. Two of these drugs include: Yondelis manufactured by Zeltia, (partnership with Johnson & Johnson) for treating soft tissue sarcomas (cancer effects body tissue such as fat and muscle), and Aplidin manufactured by Zeltia, for treating multiple myeloma, a bone narrow cancer. In 2005, Interleukin 21 (IL-21) manufactured by ZymoGenetics, was approved for Orphan Drug designation, treating melanoma patients with advance or aggressive disease. In 1993, Japan developed an orphan drug plan. The Japanese Ministry of Health, Labor and Welfare, approves drugs for the treatment of rare diseases. According to their standard, a rare disease has less than, 50,000 reported cases. In 2000, the European Union implemented a centralized policy, regarding orphan drugs. European United Medicinal Products Regulation grants sponsors, ten years of marketing exclusivity for products, with orphan designation. Their recognition of orphan drug status is prevalent upon, fifty cases of a disease per 100,000 people. Success of the United States Orphan Drug Act, total worldwide market of orphan drugs, will exceed $28 billion in 2003, and $43.6 billion by 2008. Some orphan drugs have sales exceeding one billion dollars annually.
Rare Disease Act of 2002 establishes an official Office of Rare Diseases, in National Institutes of Health: Bill sponsored in the House of Representatives, by Congressmen John Shimks (R-IL), and Henry Waxman (D-CA). New office would promote, and coordinate research on rare disorders, and create academic centers for research. Funding for the office would receive initially $24 million, and $25 million would be allocated to Food and Drug Administration Orphan Products Research Grant Program. This program provides funding for clinical trials, for new orphan drugs, diagnostics, medical devices and medical foods. Senator Edward Kennedy (D-MA) introduced to the senate, a previous legislation for Rare Disease Act of 2001.
In 2003, Senator Christopher Cox (R-CA) introduced the Medicare Patient Access Drugs for Rare Diseases Act of 2003 (Cox Act). The new legislation ensures Medicare beneficiaries will continue, access to orphan drugs, and renewing incentives for pharmaceutical companies to develop orphan drugs. Most importantly, Cox Act overrides the 2003, Medicare and Medicaid Services limitation, only providing four orphan drugs, which created a financial hardship for those elderly patients, taking other orphan drugs not listed.
Scientists believe through the process of stem cell research, would be possible to cure, many common and rare diseases. Process extracts stem cells from human embryos, which are treated in the laboratory, and then inserted into the patient. The treated cells will regenerate tissues of damaged and malfunction organs. Controversy arises from this method, because stem cells are removed from human embryos, that are destroyed, instead of possibly implanted, into a womb, of a woman subject to in-vitro-fertilization. In 2004, California legislatures approved three billion dollars, to fund stem cell research.
The European Rare Diseases Therapeutic Initiative (ERDIT – www.erditi.org), Coordinates drug companies and academic researchers, together to find new treatments for rare disease. The organization realizes, thousands of compounds have been developed, by pharmaceutical companies for common diseases, often get abandoned or fail to continue research for other diseases, because of possible toxicity, lack of efficacy or strategic reasons, known to the pharmaceutical company. These compounds are brought to the attention, of this organization to continue further research, which hopefully, will lead to treating rare, and common diseases. Also, may become profitable to the pharmaceutical industry, if these compounds cure diseases. Currently, the participating drug companies include: GlaxoSmithKline, Roche, Sanofi-Aventis, and Servier.
The National Organization for Rare Disorders (NORD – 55 Kenosia Avenue, PO Box 1968, Danbury, CT 06813-1968 – E-mail: orphan@rarediseases.org), volunteers that help people with orphan diseases. Goal of the organization: Identity treatments, and cures, through programs of education, advocacy, research, and services (referrals support groups, and other assistance). Almost twenty years, NORD provided a clearinghouse for information regarding rare disorders. Also, helps patients obtain certain drugs, which would have been impossible to afford. Primary source of funding: Contributions, membership fees, and sale of products (books and Rare Disease Database reports). National Human Genome Research Institute Center (PO Box 8126 Gaithersburg, MD 20898-8126, E-mail: gardinfo@nih.gov – Telephone Number: 888 – 205 – 2311) provides information regarding genetic, and rare disease information. Center has staff specialists to answer questions.
