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Gene Therapy Lurches Ahead, Sees Thorny Future Questions on Price

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that kind of stratospheric price, at least in my mind.

Its treatment is for lipoprotein lipase deficiency, a potentially deadly metabolic disease that can cause flare-ups of pancreatitis. The drug has been tested in three clinical trials with a total of 27 patients, according to uniQure’s prospectus. The gene therapy failed to meet its primary endpoint in a key clinical trial, as it was unable to show it could reduce excessive triglycerides in the blood beyond 12 weeks of observation.

The company, after an initial regulatory rejection in the European Union, was able to convince European officials that a secondary measurement from the trial—a reduced presence of fat-carrying particles called chylomicrons in the blood after mealtime—was a valid sign of clinical benefit. The company met with the FDA twice in 2013, and U.S. regulators said the data aren’t enough, and that uniQure should design another “adequate and well-controlled trial” to prove a clinical benefit.

Even so, uniQure is now becoming a real business in position to bargain with payers across the pond and start generating real cash flow. Here’s what the company said about those talks in its IPO prospectus:

Gene therapy represents a potential shift in the paradigm of medical care, with the commercialization challenges that often accompany a new approach. With our collaborator Chiesi, we are the first to initiate the market roll-out of an approved gene therapy in the European Union, including designing new models for product pricing and reimbursement based on a one-time intervention, expanding key opinion leader relationships, identifying centers of excellence, and developing physician and patient education and patient access programs. We believe our experience with Glybera in the European Union will facilitate our future efforts, subject to obtaining marketing approval, to commercialize Glybera and additional gene therapies in the United States and elsewhere.

And then the kicker:

We and Chiesi are developing a gene therapy pricing and business model for Glybera that is designed to capture the significant value we believe Glybera delivers to patients.

If uniQure opts for a long-term payment installment plan, you can imagine some complex questions. If a patient and his or her insurance company agree to a five-year payment plan, will the payments be contingent on periodic checkups to verify that the drug is still working? What measurements must be taken at each checkup to verify effectiveness? How often should the checkups be done, especially if invasive measurements like biopsies must be taken? What happens if the drug fails after three years, and the patient/payer are on a five-year payment plan? Will there be an escape clause, or potential for partial refunds? Or, conversely, should the drugmaker continue to get paid for years after an initial contract period if the drug exceeds expectations? Especially in the U.S., what happens if a patient on a long-term payment plan switches jobs, and health insurers?

The one-and-done payment model is the most attractive for gene therapy developers, Patterson says, because it offers a chance to recoup R&D investment quickly, it’s predictable, and it’s simple. An annuity-type system could also offer a predictable, recurring future revenue stream for drugmakers, Carter says.

Payers and patient advocates have gotten savvier about drug development in recent years, and they should be at the table with industry now to work out the fairest system for everyone. If companies don’t include the other parties, then you can expect a series of high-priced drug roll-outs followed by intense backlash.

Carter, a former president of the American Society of Gene & Cell Therapy, says the insiders in gene therapy are beginning to realize that it’s better to get in front of this issue now, before it could blow up in a few years. The gene therapy society is planning some formal meetings about commercial challenges at its annual meeting in May, he says.

“Everyone in the society realizes we have to start thinking about this,” Carter says. “It’s coming to the fore now. We’ve been successful in driving the technology, and now we need to drive thought on how we pay for all this.”

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  • Here’s a comment I just got from Vertex Pharmaceuticals spokesman Zach Barber, about what Vertex is doing to show its CF drug is still working over a number of years, as spending adds up over a patient’s lifetime.

    “We recognize that Kalydeco is a medicine that people with CF may take for their entire lives and believe that the price of Kalydeco reflects the significant benefits it provides, the time and cost to develop it and – importantly – our commitment to reinvest to create additional CF medicines. With that in mind, we are currently gathering long-term data for KALYDECO to evaluate its impact over many years, as we believe this type of long-term data will be important for many people involved in the treatment of CF — patients and their doctors and families as well as payers and regulators. These data were presented last year and showed that the safety profile was consistent with earlier studies and that the benefits from Kalydeco in lung function, weight gain and certain quality of life measures were maintained through nearly three years of treatment.”

  • Timos Papagatsias

    Nice post. I think it is unlikely anyone would pay upfront for GT Tx, particularly when you just don’t know how long the vectors will be active (the CMV promoter in the Glybera AAV vector is known to be a bit “funny”); annual installments following appropriate testing is probably adequate at this stage. I am watching Glybera’s commercialization in EU very closely but I see it very difficult making into the US market anytime soon…in fact the EU situation is probably not very good either. Nevertheless, it is indeed a test case for GT and will affect following GT products

  • Guest

    So much of what makes these therapies possible is paid for by society before the drug matures.
    The scientist’s and physician’s years of training are subsidized by government grants to Universities and hospitals.
    The early research is covered by NIH grants.
    The patient treatments are covered by their health plans and the hospitals double bill R&D companies for taking blood pressure and temperature readings from patients enrolled in trials.
    For rare diseases, disease-specific charities pay for much of the clinical work through grants.
    There are many scientists and doctors out there that will be very happy to devote their lives to finding cures even if it will not make them millionaires.

    What is needed is a structuring of rare disease payment model in a way that the NIH will cover the small scale clinical trials required and the patients will get the treatment at cost – the bill is already paid for.

    The only down side is that no one gets to be a millionaire off of someone else’s problem.