The HAE community cheered in July of 2015 when another new therapy was approved by the FDA to treat acute HAE attacks.
Now, the pharma company has another reason to cheer.
So, what does that mean? It means no biosimilars (read: generic versions) of this drug can come to the market before July 16, 2026, by referencing the research done on Runconest. Ie – “ Dear FDA- you know ours is good because see the research on theirs.” Market exclusivity is the highest prize for new drugs.
Pharma companies love this because it means they don’t have to worry about competition for the years they have exclusive rights to the drug. And that means big bucks in their pockets. Data exclusivity is a little different. Competitors can still apply for approval, but they must conduct their own research, and show it. The first company’s research shines the light, but does not allow others to jump on their train.
Pharma claims exclusivity is necessary for them to recoup all the money they have spent in researching and developing a drug, Pharming Group NV (EURONEXT: PHARM) and Salix Pharmaceuticals did come up with the first C1 esterase inhibitor that doesn’t come from human blood. And HAE is a small patient population. Do most new drugs come from free market countries, because there is a financial incentive?
A PUB MED Article: stated: “ Market exclusivity is a strong instrument for fostering orphan drug development and drug availability.”
What does that mean for, you know, the patients?
On the one hand, it means you get a new treatment for HAE attacks, and one that doesn’t use human blood as its base.
On the other hand, it means that if you use this therapy, you’re going to be stuck paying whatever price the pharma company decides for the next 12 years, or until another company comes up with their own version, doing their own research.
Patients, and insurance companies, charitable organizations and all tax payers foot the bill. An HAE patient I spoke with said she often thinks about the value of her life. She says
“ My meds cost significantly more than $650,000 per month . I’m constantly fearful of being denied access to the meds I need to stay alive. If my identifying information were attached to the actual cost, I fear I would become a target for insurance companies, the employer who currently provides this insurance, and politicians seeking to find ways to highlight high cost of orphan drugs. And what about next year…..? The black humor about this disease is, ‘what would you do without these medications?’ Answer, ‘Die.’
These and other orphan drugs are unconscionably expensive, and many of us believe the more effective next generation meds due on the market in a couple of years will be as expensive if not more so.
I personally believe that research for more accurate diagnostic tools for all forms of non-allergic angioedema will eventually lead to a cure for this disease, which would likely be at least $1,000,000.00 if not more, but still significantly less money than the pharmaceutical companies can make marketing meds that are not close to 100% effective for all people with this disease.
Many of us with this disease are demanding research into more accurate diagnostic tools because men, women, and children are dying for the lack of accurate diagnostic testing for all forms of nonallergic angioedema, and more accurate prophylactic medications, and a cure.”
What do you think? Is it right and fair for pharma to charge such high prices to get their “money back”?
Do you think we get more new drugs because of the opportunity to “make a killing?”Or, to protect the patient, should there be limits to how much pharma can charge for their drugs? And what happens when one company controls all, or most, the available treatments for an orphan disease because they buy out their competitors ( think Shire here)?
The Laura and John Arnold Foundation announced in February that they have given 7.2 million dollars in grants ” aimed at addressing the rising cost of pharmaceutical drugs and reducing the financial barriers that can make it difficult to obtain life-saving treatments. The research projects will focus on analyzing how regulatory policies and programs impact drug pricing, drug development, and patients’ access to medication.”
A much needed start.
And Congress has started as well with The Rare Disease Fund Act
A previously published version of this article stated there was market exclusivity rather than data exclusivity. This was amended 5/20/2016