Gene Therapy Advances, But Hemophilia Is No Easy Target

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non-gene therapies, some of which are updates to old standards. Their emergence could change the dynamics of the market, give insurance companies more reasons to restrict access to certain drugs, and force gene therapy developers to recalculate their asking prices if their products reach the market.

A number of companies are either working on better clotting factors, or have already brought them to market. New hemophilia drugs that don’t rely on gene modification—and don’t promise long-lasting effects—might still come with six-figure annual price tags. Now that value is an industry watchword, companies are eyeing current annual costs as a comparison. The cost to a severe adult patient who receives regular treatment several times a week and doesn’t suffer any bleeding episodes is “quite easily” $250,000 to $400,000 worth of factor each year, says Kimberly Haugstad, president and CEO of the nonprofit Hemophilia Federation of America. The bills run higher for those who have inhibitors—immune responses that require the use of additional, expensive drugs.

Roche’s Genentech division is pricing its antibody drug emicizumab (Hemlibra) accordingly. The FDA approved emicizumab in November for the 20 percent of hemophilia A patients with inhibitors. It could soon be approved for the entire hemophilia A population.

Emicizumab is taken once a week via a subcutaneous injection, not an intravenous injection or infusion like other hemophilia treatments. And it recently showed it might work with only one dose a month.

Roche’s price: $482,000 for the first year and $448,000 each year afterwards—a discount to the list price of Shire’s FEIBA, the type of drug that emicizumab aims to replace. Because it’s a discount, Genentech spokeswoman Elizabeth Walmsley says the price was set “with the intent to reduce payer barriers to patient access.”

That strategy is unusual in a market that, despite more and more competition, has seen its drug prices increase year after year, says Haugstad. Out-of-pocket costs are climbing as well, and patients are concerned that payers may start implementing restrictive formularies, she says.

Yet another drug making its way through the pipeline could also become part of a complicated pricing puzzle. An RNA interference treatment from Alnylam Pharmaceuticals (NASDSAQ: ALNY), fitusiran, for both hemophilia A and B patients—subcutaneous, taken monthly, and cheaper to produce than biologic drugs—is in late-stage testing, though recent safety concerns have delayed the drug’s progress.

Former Biogen executive Pierce, who is a boardmember or advisor for a few hemophilia foundations and biotechs, says the eventual pricing of emicizumab for the full hemophilia A population could be a bellwether for the entire sector. For example, if it prices at a discount to clotting factor therapy, it could create a ripple effect of lower prices. “It seems like that would be a reasonable thing to do,” Pierce says. Factor VIII and Factor IX can be made “very, very inexpensively. The profit margin is huge.”

The first gene therapy ever approved had a $1 million price tag. How high could a hemophilia gene therapy go?

It’s complicated. Right now, the costs of standard factor replacement therapy are well known to insurers. Eliminating them long-term would definitely save money. In a “dream” scenario, Haugstad says, a gene therapy returns a patient’s clotting factor levels approaching 50 percent of normal, at which point the patient should no longer need other drugs or emergency room visits.

Unfortunately, clotting factor levels don’t tell the whole story and couldn’t be used solely to predict someone’s future health—or set a fair price for a gene therapy. As Haugstad notes, a therapy could raise two severe patients to a 15 percent factor level and each could have different outcomes. One patient may never have a spontaneous bleed again, while the other does and still requires more than $100,000 worth of factor each year. Because not all gene therapy recipients will have the same results, the financials will be hard to map out, Haugstad says.

BioMarin could be the first to confront these issues, given that val-rox should begin the first of two pivotal trials this year—the first hemophilia gene therapy to get this far. Jeff Ajer, BioMarin’s executive vice president and chief commercial officer, says payers have been encouraged by the effect val-rox has had on factor VIII expression, bleeds per year, and replacement therapy usage. But they’re also skeptical because of the uncertainties. “They’ve seen products not pan out before,” Ajer says.

The longest-term data BioMarin has at this point, unveiled at the American Society of Hematology’s annual meeting this week, was proof that val-rox’s effects held up for 1.5 years in a handful of patients. BioMarin will continue following these patients, and they’ll be a critical part of its case to payers should val-rox eventually get to market. Will the effects prove to last five years? 10? For life?

There’s one more consideration that often gets lost in the discussion about technology and policy and pricing: The personal decision. Would a parent decide to put a young child on treatment, not knowing whether it will work in the long term? What about others in the patient community who, Haugstad says, believe their disease is part of who they are? “I think that’s crazy, but it’s real,” she says. “Change is hard and it’s different and it’s new.”

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