What exactly is an orphan drug, and why are they so expensive? According to the FDA, orphan drug status “is given to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug.”
Some orphan drugs are “ultra-orphan” meaning the patient population is less than 6,000 in some disease states, and as low as 600 in others!! When you begin considering the time and costs for bringing new drugs to market, you can get an idea of why they are so expensive once they are approved for use by the FDA.
The FDA Office of Orphan Products Development (OOPD)’s mission is to encourage the development of treatments for rare disease states and provides incentives for drug developers to explore treatments for diseases with unmet needs. For example, “the program has successfully enabled the development and marketing of more than 400 drugs and biologic products for rare diseases since 1983. In contrast, fewer than 10 such products supported by industry came to market between 1973 and 1983.”
One example of an ultra-orphan drug is Soliris used to treat paroxysymal nocturnal hemoglobinuria, a rare life-threatening blood disease. It costs more than $410,000 per patient per year. Alexion was able to generate $541 million in sales for Soliris in 2010, which is pretty amazing when you think that only 6,000 people suffer from the disease, and it carries an increased risk of meningococcal infection and is therefore subject to a Risk Evaluation and Mitigation Strategy (REMS) program. But what about the ultra, ultra-orphan drugs that have a patient population of less than 1,000?
Companies developing products in uber rare disease states have to be able to recover their costs of bringing a product to market too; the lower the number of patients, the harder it is to recoup the costs. Part of this difficulty is in the drug development process itself; the “typical clinical trial model may not be best suited for generating evidence that a drug is safe and effective against diseases where patient enrollment can be difficult and diseases progress slowly and unevenly over a long period of time”, according to Steven Grossman, who worked on the original Orphan Drug Act in 1983.
In 2012, more than one in four drugs approved in the United States targeted rare or “orphan” disease. According to Reuters, two trends are driving drug makers into this new market:
“First, there’s the lure of astronomical list prices. For example, Europe recently approved the Western world’s first drug to fix faulty genes. Glybera treats an uncommon condition called lipoprotein lipase deficiency, which can cause lethal pancreatic inflammation in patients. The drug costs $1 million per patient. Meanwhile, one drug maker expects to generate $2.6 billion in revenue by 2017 for a drug that costs $440,000 per patient per year.”
But how would you feel if your child suffered from a rare disease state that no drug company would consider finding a therapy for because its just too expensive to bring a drug to market for such a small population? You would be devastated. Your child would suffer and quite possibly die just because it didn’t make financial sense for anyone to invest in therapies for that disease state.
Procysbi is a new drug that was approved by the FDA on April 30th of 2013 and it’s indicated for the treatment of nephropathic cystinosis in adults and children 6 years of age and older. Nephropathic cystinosis is the most common form of cystinosis in which toxic levels of cystine, a naturally occurring amino acid, build up in the body’s cells and organs. The disease, which affects only about 500 patients in the United States, is usually fatal by the age of 10.
Raptor Pharmaceutical Corp developed Procysbi as an alternative to the current therapy which is “the only delayed-release product approved by FDA to treat nephropathic cystinosis, offering patients with this rare disease an important new treatment option,” Andrew E. Mulberg, M.D., deputy director of the Division of Gastroenterology and Inborn Errors Products in the FDA’s Center for Drug Evaluation and Research, said in a statement.
The only available treatment for nephropathic cystinosis prior to Procysbi was Cystagon with eventual renal transplantation. Cystagon requires dosing every six (6) hours, whereas the new delayed release Procysbi allows for dosing every 12 hours. As a parent, I can imagine all of the difficulties with dosing every six hours for young children; it means waking them in the middle of the night, and ensuring they get dosed while in school!
Patient adherence under the best of circumstances isn’t great – add in the additional complexity of a dosing regimen of every six (6) hours for children, and you can almost guarantee an adherence disaster. Every missed dose means disease progression, so a simpler dosing regimen can make all of the difference in the world for young patients suffering from nephropathic cystinosis.
Finally, Raptor Pharmaceutical Corp has only one (1) product: Procysbi – yet it must have much of the same infrastructure as a large pharmaceutical company. Once you start to factor in all of the pieces of the puzzle, it’s not surprising to see why products like Procysbi are premium priced; but if you are the parent of a child with nephropathic cystinosis, I’m sure you are extremely happy that Raptor Pharmaceutical Corp took the time, risk, and expense of bringing Procysbi to market. Patients are anxiously awaiting the first shipments of Procysbi, which are expected in June.
Sources:
FDA website: http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/default.htm
Global ‘orphan’ drug market estimated at $50 billion
The Daily Briefing
FDA approves Raptor’s drug for nephropathic cystinosis
Reuters
FDA approves Procysbi for nephropathic cystinosis
medicalexpress.com
Filed under: Biomedical Ethics, Healthcare, Healthcare Reform, Medical Benefit, PBM Payer, Pharmaceutical Products, Pharmacy, Social Tagged: Biomedical Ethics, Cystagon, Cystinosis, healthcare, Orphan drugs, pharma, Pharmacy, Procysbi, Raptor Pharmaceutical, Social
