Gene-therapy treatment
Drugmakers argue that high price reflects high value of a cure for rare diseases |
The first gene therapy treatment in the US was approved recently by the Food and Drug Administration, heralding a new era in medicine that is coming faster than most realise — and that perhaps few can afford.
The treatment, Kymriah, made by Novartis, is spectacularly effective against a rare form of leukaemia, bringing remissions when all conventional options have failed. It will cost $475,000 (Dh1.74 million).
With gene therapy, scientists seek to treat or prevent disease by modifying cellular DNA. Many such treatments are in the wings: There are 34 in the final stages of testing necessary for FDA approval, and another 470 in initial clinical trials, according to the Alliance for Regenerative Medicine, an advocacy group.
The therapies are aimed at extremely rare diseases with few patients; most are meant to cure with a single injection or procedure. But the costs, like that of Kymriah, are expected to be astronomical, alarming medical researchers and economists.
One drug, to prevent blindness in those with a rare genetic disease, for example, is expected to cost between $700,000 and $900,000 per patient on average, noted Dr Aaron Kesselheim, director of the program on regulation, therapeutics and law at Brigham and Women’s Hospital.
Drugmakers argue that the prices ought to reflect the value of a curative treatment to the patient. Kesselheim and other experts are far from convinced.
“We don’t pay the fire department that way,” he said. “When the fire department shows up at a burning house, they don’t ask, ‘How much is it worth to you to put out the fire?’”
Executives at drug companies declined to say what they plan to charge for the gene therapies they are developing. But they said a variety of factors justified setting unprecedented prices.
Funds for research
By definition, there are very few patients with the rare diseases that the treatments target. Companies thus will have comparatively fewer opportunities to make enough money to pay for their investment, to turn a profit and to fund future research.
“The reason why it’s a peculiar situation is not only because a lot of these gene therapy products are targeting small populations,” said Matt Kapusta, chief executive of UniQure, which is developing haemophilia treatments. “It is a one-time administration with potentially curative impact for the patient.”
Kapusta also noted that one injection of UniQure’s drug could be expected to replace regular infusions of blood products that can cost $5 million over 10 years.
“When you are spending a lot of money to develop therapies for a rare disease, you need to enable a larger price umbrella,” Kapusta said. “If you are saving $5 million per patient, that gives you a sense of value to the payer.”
Jeffrey D. Marrazzo, chief executive of Spark Therapeutics, which is developing the drug to prevent a form of blindness, said it should be worth a lot to keep your eyesight. “We should be compensated for generating that value,” he said.
Elizabeth Pingpank, a spokeswoman for Bluebird Bio, which is developing several gene therapies, said the company realises its prices will be a challenge.
Insures to pay
Bluebird and several other companies have set up a consortium with academics to try to figure out novel ways to enable insurers to pay the expected high prices.
“We recognise that most payers in the US are not currently set up to support onetime therapies that generate long-term transformative benefits,” Pingpank said.
Indeed, health care executives already are rushing to develop new payment models.
When Kymriah was approved, officials at Novartis said they would take the unusual step of taking into account how well it worked in a particular patient.
The company said it was collaborating with the federal Centers for Medicare and Medicaid Services on an approach in which, for children and young adults, there would be no charge if the patient did not respond to the treatment within a month.
If, as expected, Kymriah is approved for other blood cancers, its price may vary depending on how effective it is for those diseases.