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Notes From the JPM ’18 Vortex: Gene Therapy’s Reckoning and Lonely Cabs

Xconomy National — 

The annual J.P. Morgan Healthcare conference in San Francisco winds down today, meaning the remaining stragglers are either presenting to half-empty rooms or headed back to colder climes.

Compared to a 2017 full of scientific milestones—FDA approvals of gene and cell therapies, a clinical victory for RNA interference, and more—the meeting itself was rather dull. But the action always takes place in the surrounding hotels, streets, and cramped restaurants, where attendees vie for the spotlight, cut deals, and ponder the burning biopharma questions lying ahead.

Xconomy will dive into many of those issues in the weeks and months to come. But as with previous years, we’ve wrapped up the conference with a peek into our notebooks. We hope it gives you a little taste of the four-day frenzy.

Keeping a Close Eye on Spark

In December the FDA quickly approved the first U.S. gene therapy, a one-time treatment for a rare form of vision loss from Spark Therapeutics (NASDAQ: ONCE). Spark last week unveiled an $850,000 price tag for its voretigene neparvovec (Luxturna), with promises of modest rebates, money-saving contracts with health plans, and pledges to push the federal government for more pricing flexibility. (More details about Spark’s plan, beyond what CEO Jeff Mazzarro told Xconomy last week, are coming soon.)

It’s only one product, and the field remains concerned about regulators’ attitudes and the pricing climate. Bluebird Bio CEO Nick Leschly, whose experimental treatment for beta-thalassemia is amongst those next in line, liked what he saw from the FDA. “There wasn’t some weird or overly conservative stance,” he says. “There’s this positive energy in the system, and that counts for a lot.”

Approval is one thing. Insurance payments are another. The two gene therapies approved in Europe have struggled mightily. One of them, from UniQure (NASDAQ: QURE), was pulled from the market last year.

Spark CEO Jeff Marrazzo feels a “responsibility” to make sure that history doesn’t repeat with voretigene neparpovec.

Leschly says Spark’s payment plans are “bold for a product that is so small” and hopes it paves the way for future gene therapies.

But now comes the hard part, carrying out these new payment models that will require all kinds of logistical nuances and new tricks. For example, says Leschly, what to do about “churn”—when an insurer pays for a big gene therapy bill only to see the patient switch to a new insurer. (Spark’s deal with Harvard Pilgrim calls for Harvard Pilgrim to receive rebates for failed therapies even when patients have moved on.) Or how about this: Should insurers mandate that patients show up for periodic tests to make sure their gene therapy is working as promised?

AveXis (NASDAQ: AVXS) CEO Sean Nolan, whose gene therapy AVXS-101 is in  late-stage testing for spinal muscular atrophy (SMA), is curious about Spark’s distribution plan. (Marrazzo says Spark will spend 2018 getting “six to eight” major eye centers up and running in 2018.)

Nolan is also concerned about speed of reimbursement. Unlike the gradual vision loss Spark aims to treat, every month could be critical for patients with SMA and other diseases that gene therapy developers are targeting.

“You don’t want the usual ‘drag our feet for six months and make it difficult for you,’” Nolan says. “Kids can die.”

Get Off Your Phone and Into My Cab

A big topic of conversation in San Francisco this week was the integration of data science into healthcare. Those of you who muttered at least once, “If I hear ‘AI’ again I’m going to barf,” raise your hand.

Now put your hand down. You were probably one of those … Next Page »

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