The unprecedented speed at which COVID-19 vaccines progressed from research labs into real-world interventions during 2020 was a potent example of the great promise of biotechnology.
Another example of this promise is the long-awaited arrival of gene therapies. While the definition of what is a gene therapy varies, for this article we will focus on one-time (potentially curative), directly administered therapies that add a new copy of the gene in order to replace a defective copy.
So far, two such gene therapies have come to market to treat rare diseases. In December 2017, the FDA approved Luxturna® (voretigene neparvovec-rzyl) to treat patients with an inherited form of vision loss that may result in blindness.1 In May 2019, the FDA approved Zolgensma® (onasemnogene abeparvovec-xioi) for the treatment of certain patients with spinal muscular atrophy, a progressive disease attacking the motor neurons of babies and young children.2
The availability of these gene therapies is noteworthy for several reasons. One reason is that therapies such as Luxturna and Zolgensma are meant to be persistent, meaning these therapies are single-dose treatments that insert a new gene to replace a malfunctioning or missing gene. The inserted gene is expected to provide lasting functionality in the targeted cells. Thus, these gene therapies have the potential to cure or provide long term symptom alleviation of rare and often fatal monogenetic diseases, many of which have no known cure or have expensive and/or inconvenient existing treatment regimens.
An unavoidable consideration for gene therapies is cost. Luxturna launched with a cost of $425,000 per eye or $850,000.3 Zolgensma launched with a $2.125 million price tag.4 Moreover, while only Luxturna and Zolgensma are FDA-approved today, the market is expected to grow rapidly over the next five years with as many 10-20 gene therapies expected by 2025.5 Some of the disease states with gene therapies currently in clinical trials include hemophilia, sickle cell disease, Duchenne muscular dystrophy and Parkinson’s disease.6
A risk management approach
The price tag on gene therapies means we need to fundamentally rethink long-standing assumptions of how treatment costs are managed. As a pharmacy care services provider with unmatched resources, capabilities and clinical expertise, Optum is uniquely positioned to help manage these innovative but high-cost therapies.
Optum Gene Therapy Risk Protection is a risk-bearing product designed to provide patients appropriate access to ultra-high-cost gene therapies while helping plan sponsors manage the financial risk and volatility these therapies pose. Beginning Jan 1, 2022, the product will cover Luxturna and Zolgensma as well as two other gene therapies expected to enter the market in the next year. Instead of facing the prospect of unexpected claims for gene therapies, the plan sponsor will instead pay a flat PMPM fee that provides coverage for the cost of clinically appropriate gene therapies. This risk management approach removes uncertainty around the potential financial impact of gene therapies by spreading the cost into a manageable, and predictable fee.
In addition, to further help mitigate financial volatility surrounding gene therapies, the risk protection plan includes quality management services such as utilization management/prior authorization and appeals to ensure clinically appropriate use of these therapies for administering and coverage. To further manage costs when expected clinical outcomes are not met, Optum is negotiating outcomes-based contracts with manufacturers.
New hope, future challenges
While a focus on cost is essential, it is vital to remember that gene therapies represent the result of decades of work by scientists as well as a new source of hope for people with genetic diseases and their loved ones. Nonetheless, gene therapies come with new logistical, procedural and financial complexities for patients and caregivers.
Given this, we need to think beyond the bounds of traditional “pharmacy” to help solve complexities and remove the barriers patients with genetic disease and their families face across the health care ecosystem. With the unique needs of these patients in mind, Optum has developed embedded care management services dedicated to helping them manage their care and navigate the process.
As the market for gene therapies grows, additional challenges related to therapy administration and complex supply chain management will also need to be addressed. Since these therapies have been approved based on clinical trials with very small population sizes and without long-term data, plan sponsors will need help ensuring the right patient receives the right treatment and that the benefits will persist over a significant period of time.
We will keep you informed of the most important new developments regarding gene therapies going forward and will be sharing more detail around our solution in the weeks to come.
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- U.S. Food and Drug Administration. “FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss.” Accessed at: https://www.fda.gov/news-events/press-announcements/fda-approves-novel-gene-therapy-treat-patients-rare-form-inherited-vision-loss
- Novartis. “AveXis receives FDA approval for Zolgensma®, the first and only gene therapy for pediatric patients with spinal muscular atrophy.” Accessed at: https://www.novartis.com/news/media-releases/avexis-receives-fda-approval-zolgensma-first-and-only-gene-therapy-pediatric-patients-spinal-muscular-atrophy-sma
- Reuters. “Spark's price for Luxturna blindness gene therapy too high: ICER.” Accessed at: https://www.reuters.com/article/us-spark-icer/sparks-price-for-luxturna-blindness-gene-therapy-too-high-icer-idUSKBN1F1298
- BioPharmaDive. “First oral drug for spinal muscular atrophy approved by FDA.” Accessed at: https://www.biopharmadive.com/news/roche-ptc-risdiplam-approval-spinal-muscular-atrophy/583167/
- FDA. “Statement from FDA Commissioner Scott Gottlieb, M.D. and Peter Marks, M.D., Ph.D., Director of the Center for Biologics Evaluation and Research on new policies to advance development of safe and effective cell and gene therapies.” Accessed at: https://www.fda.gov/news-events/press-announcements/statement-fda-commissioner-scott-gottlieb-md-and-peter-marks-md-phd-director-center-biologics
- Clinicaltrials.gov. Accessed at: https://clinicaltrials.gov/ct2/results?cond=gene+therapy&age_v=&gndr=&type=&rslt=&phase=2&Search=Apply
STATEMENT REGARDING FINANCIAL INFLUENCE:
This article is directed solely to its intended audience about important developments affecting the pharmacy benefits business. It is not intended to promote the use of any drug mentioned in the article and neither the author nor Optum Rx has accepted any form of compensation for the preparation or distribution of this article.