From “lightning strike” to “lightning storm”: Navigating the new frontier of gene and cell therapy
Once a rarity, the promise of gene and cell therapies for broader patient populations brings new challenges in access, affordability and sustainability.
It’s not hyperbole to say that the emergence of gene and cell therapies has revolutionized the treatment of rare diseases, offering potential cures for conditions that previously had limited, if any, treatment options. The FDA approved the first gene therapy in 2017; less than a decade later, there are 27 approved gene and cell therapies with more than 2.000 in the pipeline.
Initially aimed at small patient populations with incredibly rare diseases, these therapies now target larger groups, such as those with hemophilia or sickle cell disease. A class of therapy that could initially cure a few hundred patients nationwide, can now help populations in the tens of thousands.
While gene and cell therapies offer life-changing outcomes, they also present manufacturers, payers, prescribers and patients with unprecedented challenges. As the health care system adapts, specialty pharmacies are critical partners in ensuring that these treatments reach those who need them in a sustainable way.
Economic implications of gene and cell therapies for payers and patients
Because early gene therapies targeted very small patient populations, they posed a limited financial risk for payers. Payers generally viewed these treatments as isolated events, like a lightning strike—rare and unlikely to affect them. But as gene and cell therapies have expanded to treat more prevalent conditions, the cost implications have grown exponentially. Whereas payers used to be able to assume they wouldn’t be affected, there’s now a much greater chance therapies with price tags in the millions will impact their plan. What was once seen as a “lightning strike” has become a “lightning storm” that requires a long-term, strategic approach to coverage.
How specialty pharmacies support gene and cell therapy manufacturers
As the demand for gene and cell therapies grows, manufacturers face a range of challenges in bringing their products to market. Specialty pharmacies play a pivotal role in managing the complex process of delivering these therapies, from before the launch through to ongoing patient support.
Early engagement: Manufacturers, especially smaller biotech firms new to this space, benefit significantly from early collaboration with specialty pharmacies. These partnerships offer a roadmap for addressing payer requirements, managing complex authorizations and preparing for logistical challenges. By engaging well before launch, manufacturers can align their commercialization strategies with the realities of the health care landscape, reducing the risk of delays and disruptions.
Simplifying access: For manufacturers, ensuring that patients and providers can navigate the approval process is critical. Programs like GeneAXS™ bridge this gap by addressing key hurdles such as prior authorization, benefits navigation and payer approval. This targeted support not only reduces administrative friction but also provides manufacturers with valuable insights into payer expectations and patient pathways, creating a smoother and more predictable launch experience.
Partnering with Qualified Treatment Centers (QTCs): Patients with extremely rare diseases often receive treatment at a Center of Excellence, where the clinicians are the foremost experts in their complex conditions. However, for larger patient populations like those with hemophilia, their prescribers may be unfamiliar with the ins and outs of gene and cell therapies. Manufacturers rely on specialty pharmacies to establish and manage relationships with QTCs—facilities equipped to handle the complex administration and monitoring requirements of these therapies—to ensure patients are receiving the appropriate care at every step.
Customized care models: No two gene or cell therapies are alike. Each requires tailored solutions for distribution, administration and cost management. Specialty pharmacies work closely with manufacturers to design custom care models that address the unique demands of each therapy’s distribution needs, payer criteria and more. This specialized support, such as that offered through Accredo’s fifteen Therapeutic Resource Centers, helps manufacturers optimize their delivery processes and simplifies care for these complex conditions.
Overcoming challenges in gene and cell therapy access and affordability
Given the high costs and risks associated with gene and cell therapies, specialty pharmacies, payers and manufacturers are exploring innovative solutions that balance access and affordability. Payment-over-time agreements allow the cost of treatment to be spread out over several years, easing the upfront financial burden. Portable payment models allow payment responsibilities to transfer with patients if they switch insurers, distributing costs equitably across multiple payers. These collaborative approaches are essential to ensuring that these high-cost therapies are both accessible and sustainable.
Looking ahead
Gene and cell therapy continue to redefine the possibilities in health care, providing curative options for conditions that previously had few or no treatment options. Making these groundbreaking therapies widely accessible requires thoughtful planning, cost-sharing innovations and strong partnerships with specialty pharmacies. By working together to develop sustainable coverage models, the health care industry can help gene and cell therapy fulfill their promise, offering life-changing solutions to more patients and setting a new standard for the future of treatment.
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GeneAXS
Accredo’s GeneAXS team problem-solves and creates a smoother experience, exclusively for gene therapy patients. This team leverages any of the Accredo TRCs clinical expertise when it comes to condition- or drug-specific clinical matters, but they are particularly focused on, and are intimately familiar with the nuances associated with gene therapy access, such as payer contracting, clearance, and customized patient services.
