Our First $1M Drug! Who’s Buying?

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As the world turns, so do advancements in gene therapy.

Technology is changing our world so drastically that soon gene therapy will be simplified into a one-shot treatment. With it, we could see blindness reversed, restoring of blood-clot functions, but most importantly, the official cure of rare diseases that are crippling our humanity.

Sounds amazing right? It also sounds expensive, which begs the question: Who will pay for such a shot?

Spark Therapeutics won over a Food and Drug Advisory panel for its brand new gene therapy drug called Luxturna. The next step is for it to win over the FDA, which it most likely will. Of course something as powerful enough to cure blindness in children can’t be cheap and is estimated to come in at $1 million per patient.

It’s possible that private insurers or taxpayers could pay through Medicare but what if after all that, the drug doesn’t work? It’s a huge risk considering that the human body is unpredictable.

For example, it’s possible that the drug might work a month into treatment which will make the insurer more inclined to pay the full amount, only to find their disease coming back in full force the following week. They would have dropped $1 million on a “what if?” There would have to be a system in place where the insurer would get back some of their money depending on the results.

“If we were to propose a model like this to a single commercial insurer, the financial penalties would be significant,” said Marrazzo, the CEO of SPARK “The government’s existing price reporting mechanisms are too rigid — so we can’t financially make sense of doing it.”

Experts weighed in on the issue of charging this insurmountable price: ‘There’s simply no mechanism now for a company to spool out a million-dollar bill over several years while assessing the patient’s health and response to therapy at regular intervals.’

Of course we still don’t know how much it will actually cost as we are still waiting for that FDA approval. We also don’t know what kind of payment options will be available to the public. Analysts predict that it might come in the form of a one-time payment for a one-time treatment.

Whatever happens, it will take the unity and team work among those that make the policies, the drug companies themselves and insurers to devise a reasonable payment method. If it means curing blindness and other rare diseases, it’s worth the work.

“we’re confident we can figure it out,” Nick Leschly said, CEO of Bluebird Bio. “Because if someone has a very serious disease, and we can cure it, the system will find a way to reward that.”


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