Orphan Act was passed for good intentions. Pharm has abused those good intentions.
It was passed to encourage pharmaceutical manufacturers to develop treatments for rare diseases affecting fewer than 200,000 individuals in the United States. Pharm is given taxpayer money to research and develop drugs in this category, provided tax breaks and given extended ownership of the drug or patent blocking other companies from producing it or a cheaper generic version.
These drugs even though they service less people are the top selling drugs. They have the most profit. Of the drugs that have the most profit Orphan drugs are the top 7 or 8 or 10 year after year.
Congress passes bills but does not revisit them to ensure abuse is not occuring. It is good to keep the original intent of the bill. That includes how it is being used. Pharm was not given a blank check.
If Pharm is being given taxpayer money to pay for the research and development, given tax breaks, and extended ownership why do these drugs cost so much money. Pharm needs to be audited by DOGE to determine if the expense of manufacturing the drug justifies the cost to the patient. It is not likely that 2 million dollars or 200,000 dollars per patient is required.
Do these drugs require independent manufacturing lines? If so how many since it is a rare disease there are not as many to manufacture? Are they made at the same plant as other drugs? What are the costs exactly to make the product? Is Pharm intentionally price gouging individuals who have a rare disease because they are the only supplier that can help the person? Is it possible for small compounding pharmacies to make any of these drugs?
Also, have citizens been paid back the taxpayer money Pharm was given to make the product with interest? If not, this amendment needs to be added to the bill.
Remaining:
How can the cost of these drugs be contained without treading on freedom? If we dictate how much companies can charge without the market setting the limit we run the risk of losing our liberties. However, since these drugs are funded by the taxpayers they are already part of the government system and not being made in a true free market. How can we encourage these drugs to continue to be made and sold at a reasonable price? The Orphan Act was originally passed because Pharm did not want to make and sell drugs for rare diseases that would not bring them as much profit as a drug that is used by the masses for many years. Solutions to stop the abuse?