Cell and gene therapies are revolutionizing the field of medicine by offering life-changing benefits to people with rare diseases and cancers. These therapies have been gaining considerable attention recently, both for addressing long-standing unmet needs and for being exceptionally pricey.

As of October 2019, five cell and gene therapies have been launched in the United States:

  • Spark Therapeutics’ Luxturna ($425,000), a gene therapy for Leber congenital amaurosis, a rare eye disease
  • Gilead’s CAR-T drug, Yescarta ($373,000), a cancer therapy for adults with relapsed or refractory large B-cell lymphoma
  • Novartis’ CAR-T product, Kymriah ($475,000), a cancer therapy for patients up to age 25 with cancer B-cell precursor acute lymphoblastic leukemia and adults with relapsed or refractory large B-cell lymphoma
  • Biogen’s Spinraza ($375,000 - $750,000), a therapy for spinal muscular atrophy
  • Novartis’s Zolgensma ($2.1 million), a gene therapy for spinal muscular atrophy

The manufacturing and research and development costs associated with gene therapies are significantly higher than conventional biologics and range between $500,000 and $1 million. Moreover, these therapies are administered as one-time only injections and are applicable to only a very small patient pool, which contributes to their higher price tags. The reimbursement environment in the U.S. is still under-equipped to absorb the financial implications associated with these drugs, so it is imperative for pharma companies to consider new business models and explore partnerships with a range of stakeholders, including private payers and the government, to ensure that these innovative treatments are easily accessible and commercially viable.

Decision Resources Group has been closely tracking the innovative approaches that pharma companies are exploring in the U.S. to ease access to, and reimbursement of, cell and gene therapies. The following are some key strategies currently employed by pharma:

Establish cost-effectiveness:

Given that payers and policymakers have been scrutinizing drug prices more, it is vital for pharma companies to demonstrate the cost-effectiveness of new therapies. Novartis, for example, determined through its health-economic models that Zolgensma costs half as much as competitor Spinraza over a 10-year period. Zolgensma is a one-time therapy, while Spinraza is administered throughout a patients’ lifetime with an initial annual cost of $750,000 for the first year, then $375,000 every year thereafter. As of October 2019, Novartis has secured formulary coverage for Zolgensma with four large national plans— Aetna, UnitedHealthcare, Cigna, and Anthem—that cumulatively account for 23 percent of covered lives, according to Decision Resources Group data.

The Institute for Clinical and Economic Review has emerged as a quasi-governmental health technology assessment body in the U.S. The organization has been publishing reports and commentaries about cell and gene therapies, including a CAR-T cell therapy evidence report from March 2018 in which the institute concluded that CAR-T agents are cost-effective since they provide small to substantial net health benefits with moderate certainty relative to common chemotherapies or no therapy at all. In another report assessing Novartis’s Zolgensma, ICER stated that the drug’s $2.1 million price tag “falls within the upper bound of ICER’s value-based price benchmark range.” These positive affirmations from the institute are expected to aid Zolgensma’s uptake and commercial performance.

Explore innovative payment plans with payers:

Staggered payment plans and pay-for-performance deals are a common strategy between pharma and private payers. Staggered payment plans allow insurers to amortize the cost of therapies over multiple years, easing the financial burden associated with cell and gene therapies. Most recently, Novartis has partnered with Accredo—a specialty pharmacy owned by Cigna-Express Scripts—to offer a five-year payment plan for Zolgensma.

In pay-for-performance models, insurers can negotiate higher discounts or rebates if therapies do not show positive health outcomes for patients. Essentially, these models partially shift financial risk from payers to pharma, which may prompt better coverage on payer formularies. Spark Therapeutics and Harvard Pilgrim, for example, have inked a pay-for-performance deal for Luxturna. In the deal, Harvard Pilgrim receives refunds or rebates if the treatment loses its effectiveness after a certain period of time.

Engage providers and coordinate care:

Providers play a pivotal role in patient identification for, and administration of, new therapies. The processing of cell and gene therapies is a highly complex task and involves additional infrastructure costs. Therefore, establishing and maintaining a robust network of providers with the equipment and personnel to administer cell and gene therapies is imperative for pharma companies. Novartis and Gilead have formed partnerships with key oncology centers across the country and have provided extensive support to train personnel administrating life-saving CAR-T therapies, which are associated with adverse events such as cytokine release syndrome and neurologic toxicity. Managing these events requires seamless coordination between providers and pharma.

Most pharma companies active in the cell and gene therapy space are leveraging sophisticated technology platforms to help providers coordinate patient care. These platforms help with visit scheduling, processing of therapies, and product delivery, and also have web-based portals that lead patients to treatment sites. Pharma is also investing in electronic health record integration to enable optimal patient care and help providers better understand the factors impacting individual clinical outcomes.

Offer patient assistance programs to address unique circumstances:

Treatment with cell and gene therapies can require high out-of-pocket spending by patients if payers do not cover most or all of the therapies’ costs. Patient assistance programs for cell and gene therapies have some unique provisions compared to those in place for other high-cost medications. For example, patient assistance programs for CAR-T therapies include travel and lodging costs. Due to the limited number of centers offering CAR-T therapy, patients may be required to travel long distances to access treatment.

Utilize alternative distribution channels

The traditional buy-and-bill model of drug distribution—wherein a provider purchases a product from a pharmacy, administers the product to a patient, and then submits a claim for reimbursement to the patient’s insurer—does not suit the distribution requirements for cell and gene therapies. Manufacturers, therefore, are remodeling their specialty distribution capabilities to address the unique challenges presented by these agents.

Spark Therapeutics, for example, was the first drug company to explore an alternative distribution arrangement with Express Scripts. In the arrangement, instead of having providers purchase Luxturna from the manufacturer, Express Scripts ships the drug through Accredo Specialty Pharmacy or CuraScript Specialty Distribution to the provider after the patient receives health plan authorization. Biogen and Novartis have similar arrangements for Spinraza and Zolgensma, respectively.

Conclusion

There are currently more than 300 cell and gene therapies at various stages of development, of which around 30 are in late-stage development. By 2030, it is expected that more than 50 agents will reach the market and bring new hope to patients with rare conditions that have limited efficacious treatment options. As additional cell and gene therapies go to market, it will be fascinating to witness the novel pricing and market access strategies pharma will employ to increase patient access.

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