"Biologics are genetically-engineered proteins derived from human genes" so essentially they have the means to fully develop a cure, but depending on how much money they can make from it they can drag out as long as needed?

Development of New Therapeutic Drugs and Biologics for Rare Diseases

This is sickening they sound so proud of their profits

"small market is generally viewed as a disincentive for the development of drugs. Many of the costs of developing a new drug are incurred regardless of the size of the potential market. If, however, a company can set a price that is high enough to recover its costs and generate profits because enough public and private health insurance plans and patients and families will pay that price, then a manufacturer may not be deterred by a small target market. Some orphan drugs are among the most expensive drugs in the world, costing as much as $400,000 per year."

Orphan drugs can be very profitable. Wellman-Labadie and Zhou (2009) reported that 43 brand-name drugs with global sales of more than $1 billion had orphan drug designations, and 18 of these had been approved solely as orphan drugs. Most had been approved for the relatively more prevalent rare conditions such as multiple myeloma, but one, imiglucerase (Cerezyme), was approved for Gaucher’s disease, which has a U.S. patient population estimated at 3,000 to 6,000.1 In 2008, about 1,500 patients in the United States were taking the drug, which was priced at more than $300,000 per year (Pollock, 2008).

This site has a lot of good info from the chapter before too. This shows how they are only worried about the bottom dollar money, money, money....

Next step is stem cells where everyone already has their own personal cure inside them. Unless big companies get to it first and get patents on every cell before treatment is ever given back.

wow, 10% more effective than placebo and only a puny 2.2 million payout lol whats that 1 week of government incentives of the many years it was selling for.

The US Orphan Drug Act: Rare disease research stimulator or commercial opportunity?

Results

Currently, 2002 products have obtained orphan drug designation with 352 drugs obtaining FDA approval. Approximately 33% of orphan drugs are oncology products. On average, products obtain 1.7 orphan designations with approximately 70% obtaining a single designation. At least 9% of orphan drugs have reached blockbuster status with two-thirds having two or more designations. An additional 25 orphan drugs had sales exceeding US$ 100 million in 2008 alone. Since 1983, at least 14 previously discontinued products have been recycled as orphan drugs .

Conclusions

The United States Orphan Drug Act has created issues which, in some cases, have led to commercial and ethical abuses . Orphan Drug Act reform is necessary but current incentives, including 7 year market exclusivity, should be maintained in order to favour patients as well as economic prosperity. Suggested reforms include price regulation, subsidy paybacks for profitable drugs and the establishment of an International Orphan Drug Office.