After a career spent in pharmaceutical commercial strategy and operations, I thought I had a handle on what was required to launch a new therapy. But I’ve learned many lessons — and faced a few curveballs — in the 12 months since the FDA approved Luxturna (voretigene neparvovec), a one-time gene therapy for the treatment of patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells.

To bring this first-of-its-kind therapy to U.S. patients, Spark Therapeutics, where I serve as chief commercial officer, had to create a new pathway for distribution and commercialization that I hope can offer valuable lessons and help other biopharma companies get innovative therapies to their patients.

The traditional pharmaceutical commercial model is built on moving large quantities of drugs on fixed schedules throughout the year. From the perspective of this model, patients are essentially outputs to the system, receivers of medications for which the demand can be reliably forecasted and budgeted.

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That model just doesn’t work for a one-time gene therapy for an ultra-rare disease.

To begin with, fewer than 2,000 people spread across the entire U.S. have been diagnosed with the condition that our therapy was approved to treat. Each dose is a single vial of the drug that must often be delivered to the hospital as the patient is on his or her way there. The patient, his or her physician and family, the hospital, the payer, and our team must stay in lockstep throughout the entire process. Oh, and we also need to track patients for years after treatment.

Embracing this level of detail and engagement with every player in a patient’s care journey is unusual — even for rare diseases. Manufacturers, payers, and distributors typically set up highly repeatable processes for the delivery of medicines. With our therapy, however, we learned that every patient had varied and individual needs, which our team constantly had to anticipate.

To prepare for delivering the therapy, we identified and designated 10 geographically dispersed treatment centers throughout the U.S. Balancing the interest of surgeons to deliver this treatment with the expected volume of patients who need it presented a challenge: We wanted to ensure that the treating surgeons and their teams, as well as the pharmacy staff on site who prepare the treatment immediately before surgery, would become highly experienced in delivering this novel therapy. We addressed this by putting in place comprehensive education programs for the centers’ pharmacists and surgeons on the preparation and administration of the therapy. In the 12 months since approval, surgeries have been performed in nine of the 10 centers.

One-time gene therapy for an inherited disease — a completely new paradigm — required Spark to determine a one-time upfront price to match the therapy’s value over many years. We needed to devise new strategies across reimbursement, market access, and treatment, all of which challenged our teams’ assumptions of best practice commercial management.

When insurers agree to cover the therapy under an outcomes-based model, Spark commits to years of monitoring, relationship management, and compliance with long-term outcome assessments, including an efficacy assessment in the months immediately after treatment, followed by a durability assessment around 30 months after the initial procedure. This long tail of case management is needed to validate the outcomes-based arrangements that assess whether the treatment continues to work.

This may seem mundane, but the logistics were surprisingly challenging. Gene therapies aren’t conducive to being delivered to a standard hospital receiving dock. We had to build capacity for just-in-time delivery because payers and treatment centers are reluctant to store the gene therapy dose for significant periods. This required creating a flexible and tightly integrated specialty pharmacy partnership as part of the supply chain.

In just nine months, Spark was able to establish 85 percent coverage across the major insurers and 50 percent coverage across public payers. To do this, we had to address two major challenges: first, working with multiple stakeholders in the U.S. health care system to help them understand and accept the new modality that gene therapy provides, and second, delivering the first-ever pharmacologic treatment for an inherited retinal disease. There was no road map for either challenge, so we had to create one. I attribute our early success to:

  • Early, transparent engagement and education with all stakeholders
  • A clearly stated mission to create a path for a new therapy, one that insurers understood they couldn’t fit into existing payment and reimbursement models
  • Creating innovative payment and distribution arrangements that offered benefits to key stakeholders

None of this could have been accomplished without close collaboration with patients and their advocates. They have informed every step of Spark’s journey, from early development of the therapy to the development of distribution and commercialization models.

Someone recently asked me, “What were the key milestones through launch?” To be honest, it felt like every day brought another milestone. From the time of FDA approval, the firsts didn’t stop coming: onboarding the first treatment center; the first positive coverage determination; the first patients treated; the first Medicaid coverage; the first outcomes assessments. And I’m sure there are still more to come — we are still learning how best to get this therapy to patients.

The delivery and payment models we built are unique to this therapy at this time. But I hope that they will provide a model that other gene therapy companies can build on.

Ron Philip is the chief commercial officer at Spark Therapeutics.

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