At last month’s Veeva Summit in Philadelphia, Spark Therapeutics CEO Jeff Marrazzo said one of the next phases for gene therapies is broader application to disorders other than the rare, highly specialized ones on which their development has lately been focused. Yet, an expert at a subsequent conference in the same city expressed reservations about whether the healthcare system can absorb the plethora of new gene therapies expected to hit the market in the near future, while another had some ideas for bringing prices down.
Currently, only two gene therapies are approved in the U.S., both for rare disorders. Spark’s Luxturna (voretigene neparvovec-rzyl) won Food and Drug Administration approval for blindness associated with mutations of the gene RPE65 in December 2017, while Novartis’ Zolgensma (onasemnogene abeparvovec-xioi) won approval last month for spinal muscular atrophy in infants.
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Both therapies have drawn public scrutiny with their high price points, with Luxturna carrying a list price of $850,000 for both eyes, and Zolgensma’s price set at $2.1 million. However, those price points have been deemed cost-effective because both drugs are meant as one-time treatments given with curative intent for very small populations. Still, with ever increasing numbers of such therapies expected to hit the market, including for disease states with larger populations, how to make them cost-effective as well becomes a question.
“There’s two ways to make this happen: It’s either for plans to increase the premiums they’re charging to cover revenues or pay it off over time,” said pharmaceutical industry consultant Ambrose Carrejo, in an interview at the Biotechnology Innovation Organization’s annual meeting last week. On the one hand, he said, there is not much room for payers to increase premiums, given the amounts families are already paying. At the same time, thin margins also do not leave a lot of room for paying over time when a large number of gene therapies come onto the market.
According to a report released by trade group the Pharmaceutical Research and Manufacturers of America, as of November there were 289 cell and gene therapies in clinical development. The largest share of these, 111, were for cancers. Eye disorders, cardiovascular disease, neurological disorders and blood disorders each had more than 20 therapies in development.
“When we’re talking about SMA, the populations are small,” he said. “But now we’re talking about hemophilia, and those populations are very large.”
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Spark has three gene therapies in development for hemophilia, including one each in Phase III development for hemophilia A and hemophilia B. The latter, SPK-9001 (fidanacogene elaparvovec) is partnered with Pfizer. BioMarin also has a Phase III hemophilia A gene therapy, BMN 270 (valoctocogene roxaparvovec). According to the Centers for Disease Control and Prevention, the exact number of patients living with hemophilia is unknown, but estimated at around 20,000 in the U.S.
One way to help manage the costs of gene therapies would be to devise ways to make them come down.
To start with, there are two main factors driving cost, explained Mo Heidaran, an executive at the contract research organization Parexel specializing in cell and gene therapies, in an interview at BIO. “There’s the cost of manufacturing of the product and what the market bears as far as being able to pay for it,” he said.
The latter factor is based on a competitive analysis by manufacturers of what the market will pay. But the former comes from the cost of raw materials that go into actually making gene therapies.
All of the gene therapies on the market today – Luxturna, Zolgensma and bluebird bio’s Zynteglo, which the European Medicines Agency approved last week for the blood disorder beta-thalassemia – use specially engineered viral vectors to get into cells and transfer corrected genes to their nuclei. But manufacturing those still requires a lot in the way of materials – serums, reagents, culture media and growth factors – and these can all be very expensive. Heidaran said that given those raw materials’ high cost and labor intensiveness, governments should get involved and partner with private industry to make their production more profitable.
Some efforts are underway, Heidaran said, such as by the National Institute for Innovation in Manufacturing Biopharmaceuticals, or NIIMBL, which is an industry-government consortium designed to develop new technologies for manufacturing.
How much bringing down the cost of raw materials would have an effect on the cost of gene therapies themselves remains uncertain, Heidaran said. However, automation of production could play a significant role. “Currently, for developing reagents, automation is not part of the scenario,” he said. “You can lower the labor costs by developing better platform technology.”
Photo: iLexx, Getty Images
