Executive Summary

Institute for Clinical and Economic Review launches value assessment of Spark's blindness treatment vortigene neparvovec, the first gene therapy likely to reach the US market, in an effort to facilitate development of coverage policies.

The public discussion over how insurance should pay for gene therapies is moving to center stage in the US as Spark Therapeutics Inc.'s vortigene neparvovec, a one-time treatment for inherited blindness, heads toward a possible approval by early 2018.

Spark's treatment became the first gene therapy to be submitted for approval to the US FDA with the completion of a rolling BLA on May 18. The company is seeking approval in patients with vision loss due to confirmed bi-allelic RPE65-mediated inherited retinal disorder IRD.

To help payers in developing policies for covering the drug, the Institute for Clinical and Economic Review plans to develop a report assessing the comparative clinical effectiveness and value of voretigene neparvovec by January 2018, coinciding with a likely FDA decision on the treatment and preparations for a possible launch.

Schedule For ICER's Review Of Voretigene Neparvovec

Stakeholder open input period ends July 6

Draft scoping document posted: July 10

Public comment deadline on scoping document: July 31

Revised scoping document posted: Aug. 7

Draft evidence report posted: Nov. 14

Comment deadline on draft evidence report: Dec. 13

Evidence report posted: Jan. 11, 2018

Public expert panel meeting on topic: Jan. 25, 2018

To begin the process, ICER is accepting public comment during an "open input" period ending July 6 to "allow stakeholders to share key information relevant to the development of the evidence report," the group announced June 15. A draft evidence report is scheduled to be released in November 2017, followed by a final report in January 2018. ICER will seek public comment at several points in the process. (See box.)

Spark has not indicated how it will price the therapy but analysts have projected the cost could reach $1 mil. when both eyes are treated.

Expectations are based in part on the prices for gene therapies approved in Europe. uniQure NV's Glybera, a treatment for lipoprotein lipase deficiency, was approved for use in the European Union in 2012. It is priced at $1.4m and so far, has been paid for use in only one patient in Europe. 

GlaxoSmithKline PLC's Strimvelis for severe combined immune deficiency in children, was approved in Europe in 2016. Currently, patients can be treated only at a facility in Italy, due to the treatment’s extremely short shelf life. Its price in that country is approximately $664,000.

The prospect of gene therapies coming to the US has heightened concerns about their affordability under existing paradigms of pricing and payment, ICER notes in a March 2017 white paper on gene therapy. Payors have also been speaking up about their concerns. (See sidebar for related story.)

Gene Therapy Reimbursement: Is Blindness A Bad First Test?

By Laura Helbling29 Jun 2017

The first gene therapy approval could be coming soon – followed shortly by the first ever gene therapy coverage decision. An Express Scripts executive argues that the tone may be affected by the initial indication.

Read the full article here 

"Based on the initial pricing experience with gene therapy in Europe, should a growing number of gene therapies come into use at costs of $1-$2m, the cumulative budget impact would be substantial, and perhaps unsustainable," according to the paper, which was developed with input from policy leaders at a meeting convened by ICER in December 2016.

"Even if gene therapies are developed to treat only one in 10 patients with a genetic condition – approximately 1% of the total population – the cumulative budget impact at that price could rise to $3tn, as much as is currently spent in a year on all healthcare in the US."

The prospect of a one-time curative gene therapy regimen has driven expectations for extremely high upfront payments, ICER notes. "Like organ transplantation, gene therapy replaces a defective function, which may offer a curative or long-lasting benefit, but some payers and policymakers feel that this scenario does not create an a priori justification for per patient prices of $1m or more."

"If private insurers [are] going to consider some kind of payment through amortization, the proposal and the vehicle for doing so would have to come from the manufacturer."

The types of studies conducted for gene therapy will present challenges in assessing value based on existing methods, ICER acknowledges. For example, "short-term trials mean that we have significant uncertainty around estimates of clinical effect, which makes it difficult to produce robust estimates of health and economic impact."

Other issues include "what extra value, if any, should be attached to the potential that these therapies may cure severe and life-threatening disease – compared to the value attached for more incremental benefits."

Outcomes-Based Agreements, Amortized Payments

Promising approaches to paying for gene therapies include outcomes-based arrangements and various types of payment amortization, ICER says. Outcomes-based agreements "can serve a critical function in addressing the inherent uncertainty in longer-term outcomes of gene therapy by linking payment levels to the real-world outcomes achieved by patients."

Amortization, or breaking the price into smaller payments over time, "figures prominently in many discussions of payment strategies for gene therapies," the paper notes. Certain characteristics would make some gene therapies better candidates for amortization then others. They include:

One-time or very short-term treatment regimens with curative clinical impact, meaning that benefits accrue over time.

Durability of clinical benefit that is well-established or can be monitored through an outcomes-based mechanism.

A population size that is big enough to create concerns that upfront payment would not be easily managed.

A method by which the regimen price is fairly set to reflect the added value for patients and underlying development costs.

Sources for financed pricing might include the federal government or manufacturers, according to the paper. "The risk and high costs of gene therapies may prove very difficult to manage within the fragmented private insurance, and some stakeholders believe that the federal government will need to intervene and take over coverage, much as it did with renal dialysis," it says.

On the other hand, "it became clear through the discussion of the policy summit that if private insurers were going to consider some kind of payment through amortization, the proposal and the vehicle for doing so would have to come from the manufacturer," ICER reports.

Some manufacturers might have the size and financial resources to provide the financing themselves, and offer the payer some form of installment payment plan, the paper notes. Others "may need to work with third-parties to come up with some kind of financial instrument that they could then offer to payers."