New Cell and Gene Therapy Coverage: How to Interpret the Evidence
In the coming years, insurers and employers will be forced to make high-stakes decisions about coverage for the most transformative therapies—getting those decisions right will take expert guidance and flexible policies.
Purchasers - including self-insured employers, commercial insurers, Medicare, and state Medicaid programs - face a new and very high-stakes set of coverage decisions. Breakthrough cell and gene therapies promise life-changing treatments that were barely imaginable a few decades ago. In some cases, these therapies require only a single dosage. But the costs can be astronomical, sometimes exceeding $4 million per patient, and many therapies are so new or relatively untested that their long-term efficacy remains unclear.
How can insurers and self-insured employers balance their fiscal responsibility to provide coverage for many against potentially life-saving treatments for the few? These decisions would be challenging even with high-quality evidence around safety and effectiveness. But the science of CGTs is evolving so rapidly that purchasers need help staying informed and recalibrating their level of certainty as new data emerges.
The Data and Uncertainty Challenge
For most drugs, FDA approval comes after years of research and testing, including multiple phases of clinical trials involving thousands of patients, followed by lengthy review. Once in the market, those drugs accrue more years of safety and efficacy data with clear-dosing protocols, known side effects, and existing care standards for comparison.
In contrast, CGTs are often approved by the FDA after testing on dozens to hundreds of patients, often without randomized control groups, and with limited follow-up periods. That approval is often fast-tracked or accelerated, based on potential rather than measured clinical impact. For new drugs, real-world evidence is collected in real time often across a very small population. Side effects may be unclear and long-term efficacy and durability unknown.
As an example, Lipitor, a statin used to lower cholesterol, was approved after clinical trials on tens of thousands of patients. Insurers understood the mechanisms and side-effects and had considerable data on clinical efficacy and outcomes when making coverage decisions.
In contrast, Zolgensma, a one-time therapy for children with spinal muscular atrophy, was approved based on 36 patients with around 14 months of follow-up study. While the evidence is very compelling, we still don’t know its long-term durability or side effects and it comes with a $2.1 million price tag.
Similarly, Roctavian, a one-time gene therapy for severe hemophilia A, was approved based on 134 patients followed for approximately 3 years. Patients with severe hemophilia A generally require infusions that replace missing clotting factor, costing approximately $800,000 annually, compared to a one-time, $2.9 million cost for treatment with Roctavian. However, durability of the gene therapy remains uncertain - raising questions about how frequently and when patients may need to resume routine clotting factor replacement. Such uncertainty has been an important driver in low utilization of Roctavian, and the ultimate decision to discontinue marketing the drug.
A Flexible Stance in an Evolving Landscape
For an individual with a life-threatening condition or disease, those uncertainties may be worth the clinical risk. But, in our fragmented healthcare system, the brunt of the financial risk falls on purchasers even as they may also be targeted for criticism should they withhold coverage for an individual in need.
Aradigm was formed to solve that predicament. We pool risk across multiple payers to make breakthrough treatments financially sustainable. We provide expert clinical evaluation so coverage decisions are based on the best possible evidence. We develop outcomes-based contracts with manufacturers that tie payment to results. And we connect the patients who will benefit most with providers who have the infrastructure and expertise to deliver these complex therapies safely.
We know the data and evidence for these therapies is constantly evolving. So, we survey the available literature for new evidence and regularly update our clinical policies as that information becomes available.
We recommend flexible coverage policies that allow purchasers to continually update their decisions as new evidence emerges. Over time, there will be increasing clarity on many outstanding questions such as long-term impact, side effects, durability, and which populations respond best to the treatment.
It’s important that purchasers can adjust their policies and support for members accordingly. It’s also important that we encourage and support development of these treatments as a society. The science of CGTs is remarkable. We owe it to patients to make the best decisions about those therapies that the evidence allows and ensure that coverage and access is responsible, equitable, and sustainable.
