Everyone knows what an orphan. But what are “orphan drugs” and why should you care?
The Orphan Drug Act got its start in 1983, when Congress recognized the need to encourage pharmaceutical companies to develop drugs for rare diseases. The FDA originally called such drugs “important drugs of limited commercial value.”
That was because everyone assumes that companies would not be able to get a drug benefit that would be required for relatively few patients. In those days medicines at an affordable price.
The idea that a company can charge thousands of dollars for treatment was absurd. To make a lot of money on a drug, a pharmaceutical company had to develop a product that would be taken by millions of people, as a treatment for heartburn, high cholesterol or hypertension.
An executive of the pharmaceutical industry once confided that if a company discovered a cure for cancer would almost have to give the medicine away. He and his colleagues within large pharmaceutical companies believe that if they pay what lead the industry market would become a pariah and be punished for such misconduct. Unfortunately, times have changed!
orphan drug incentives have been subverted:
People in Congress and the FDA who wanted to encourage pharmaceutical companies to develop drugs for rare diseases knew they would have to offer a lot of incentives. Regulators assumed that if pharmaceutical companies could not make huge profits to develop such drugs, then there should be other rewards.
Congress offers great tax advantages to companies working in orphan drugs. In addition, these pharmaceutical manufacturers were offered the patent and market exclusivity. In some cases, the clinical research was funded in part. The FDA waived certain fees and received valuable coupons companies drug test. The assumption was always that any drug that evolved from this program would be kept affordable.
Now, however, orphan drugs that could treat only a few thousand patients a year can generate incredible amounts of revenue for manufacturers. Consider the recent approval of orphan drug, eteplirsen ( Exondys 51 ). The FDA just approved this drug for the treatment of a subgroup of patients with a rare disease, Duchenne muscular dystrophy. There are perhaps 2,000 children in the US with this genetic disease linked to chromosome X.
The manufacturer plans to sell the drug at $ 300,000 per year after discounts. Hopefully all those young people in wheelchairs have very good insurance coverage, because otherwise might have little access to a drug that could help them have a better quality of life.
Greed and Orphan Drug Act
decades ago, when this legislation pharmaceutical companies still had a sense of shame was approved. The pharmaceutical industry was highly profitable despite drug prices were reasonable. That has completely changed. No controls drug prices in the United States. Neither the FDA nor Congress has any interest or ability to alter the price of medicines that can save lives.
In recent years, a pharmaceutical company that developed an orphan drug could make so much money on him as a prescription drug. This is due to the ability to set the price of orphan drugs in the stratosphere. An example is the sofosbuvir hepatitis drugs ( Sovaldi ), with a starting price of $ 1000 per day. anticancer drugs like ipilimumab ( Yervoy ) or pembrolizumab ( Keytruda ) run more than $ 100,000 per course of treatment.
Another rare condition, Gaucher disease is treated with an orphan drug called eliglustat ( Cerdelga ), which can cost more than $ 300,000. It is not surprising that the manufacturer Exondys 51 put its price at $ 300,000.
Exondys 51 is worth $ 300,000 a year?
You could also argue that enlarging or save the life of a child with Duchenne muscular dystrophy is worth $ 300,000 a year. And you may be right. The question is, however, if Exondys 51 really do that. The drug was approved based on surprisingly little data. The study included 12 patients and had no control arm. No one knows how well the drug actually works.
Advisory Committee of the FDA did not recommend approval of the drug, but the director of the Center of the Agency for Drug Evaluation and Research, Janet Woodcock, annulled the committee. She said, “We look forward to learning more about the effectiveness of this drug through a clinical trial confirmation.” We hope the FDA also learn more about security.
Proponents of patients with Duchenne muscular dystrophy probably tipped the balance for approval when presented at the hearing of the FDA with emotional appeals for their children in wheelchairs. We hope that they will not end up cruelly disappointed a drug that will be so expensive.
What patients do?
recent testimony by the CEO of Mylan (make EpiPen) has revealed that the company played fast and loose with their earnings reports. Here is a link to last in this sordid story .
Visitors to this website reveal the depth of his anger:
Sylvia in Michigan indicates that insulin prices are also skyrocketing:
“The Epi-Pen is not the only drug that has been an impressive increase in the price. There are several others. Insulin Lantus my husband has existed for years, but recently has become so expensive that we are considering replacing a lower insulin because we can not afford the Lantus. I hope that will not adversely affect their health to change, but we have no choice.
“The pharmaceutical industry sees the writing on the wall as a result of their greed and spend a lot of money on advertising to make people believe that these increases are necessary for survival. What about patients who can not afford the price? I think I just lay down and die in silence. “
Chris in California are sick and tired
“Why do Americans have to pay more for drugs than Canadians, Europeans and anyone else? Because lobbyists and deafness of Congress to the voices of anger of the American people. the only solution to the greed and corruption of pharmaceutical corporations are the laws governing price increases.
“Apart from the big pharmaceutical companies, which want these high drug prices surprisingly Something must be done
Penelope in Florida says that competition is essential.
“Big Pharma needs a good dose of competition, we should be allowing the importation of drugs from Canada (which does much better detection US)”.
drug costs be cut? US
For more information on Canadian pharmacies and pricing policies found our Guide to save money on drugs practical value. Not just a series of online pharmacies are reviewed, but we provide tips on how Americans can reduce their pharmacy bills. If Congress and the FDA will not protect us we have to protect ourselves from the greed of the pharmaceutical industry.