DNA matrix image

[Image: Adobe]

Cell and gene therapies are upending the treatment of a growing number of diseases by addressing the underlying causes of genetic disorders. Yet the high costs associated with these therapies, sometimes costing multiple millions of dollars for a single treatment, pose significant challenges for patients, payers and healthcare systems. To address this matter, a growing number of companies are pioneering novel pricing and reimbursement strategies such as outcomes-based agreements and risk-sharing models to help ensure patient access while mitigating financial risks.

Spotlight on Beqvez: A promising hemophilia therapy

In that vein, Pfizer recently announced the FDA approval of Beqvez, a one-time gene therapy for adults with moderate to severe hemophilia B. In tandem with the launch, Pfizer revealed a novel warranty program “based on durability of patient response to treatment.” The firm noted that the program is aimed at providing “greater certainty to payers,” while also bolstering access for eligible patients while providing financial protection in the event of efficacy failure.

Pfizer’s warranty program for Beqvez is designed to align the financial interests of healthcare providers and payers with the long-term efficacy and safety of the gene therapy. By offering refunds or financial offsets if the therapy does not meet certain efficacy thresholds over a specified period, Pfizer is sharing the risk of efficacy failure.

Promising clinical trial results for Beqvez

In the pivotal BENEGENE-2 study, Beqvez the therapy, whose scientific name is idanacogene elaparvovec-dzkt, demonstrated several significant benefits. Recipients of the therapy experienced a reduction in annualized bleeding rate (ABR) from a mean of 4.43 total bleeds per year during the (factor IX) FIX prophylaxis period to 1.30 bleeds per year post-infusion. For treated bleeds, the ABR fell from 3.35 to 0.73 bleeds per year. In addition, the annualized infusion rate (AIR) of exogenous FIX fell significantly from 58.83 infusions per year during prophylaxis to 4.46 infusions per year post-infusion.

The BENEGENE-2 study also found that steady state circulating FIX (FIX:C) levels increased, with geometric mean percentages of normal FIX:C levels ranging from 12.62% to 25.90% across different assays. The annual amount of FIX patients needed fell dramatically from 3170.74 IU/kg per year during prophylaxis to just 235.04 IU/kg per year after receiving the therapy.

A look at other novel reimbursement strategies in cell and gene therapies

Before the approval of Beqvez, several pharmaceutical companies have pioneered novel pricing and reimbursement strategies to improve patient access to their transformative gene and cell therapies while mitigating financial risks for payers and healthcare systems. For instance, Novartis, with its outcomes-based agreements (OBAs) for Zolgensma, a gene therapy for spinal muscular atrophy, and Kymriah, a CAR-T cell therapy for certain blood cancers. For Zolgensma, Novartis offers a pay-over-time option of up to five years, with a portion of the cost at risk based on the therapy’s continued performance. Similarly, for Kymriah, payment is contingent upon the patient’s response at 30 days post-treatment, ensuring that the healthcare system only pays for successful outcomes.

Bluebird Bio has also embraced OBAs for its gene therapy Zynteglo, which treats beta-thalassemia. Under these agreements with certain state Medicaid agencies, payers are reimbursed up to 80% of the therapy cost for patients who fail to achieve and maintain transfusion independence over a specified period, up to two years.

Spark Therapeutics, maker of Luxturna for biallelic RPE65 mutation-associated retinal dystrophy, has introduced a multi-faceted approach to improve access and financial sustainability. Their initiatives include outcomes-based rebates tied to efficacy thresholds over time, an novel contracting model that shifts the purchasing responsibility to payers or specialty pharmacies, and extended payment options that allow costs to be spread over several years.

The table below summarizes the novel pricing and reimbursement strategies covering a variety of prominent cell and gene therapies:

Company Product Indication Innovative Pricing/Reimbursement Strategy
Novartis Zolgensma (onasemnogene abeparvovec) Spinal muscular atrophy
  • Pay-over-time option up to 5 years
  • A portion of the cost at risk based on the therapy’s performance over that period
Kymriah (tisagenlecleucel) Certain blood cancers
  • Payment is conditional on the patient’s response at 30 days
  • No payment required if a positive outcome is not achieved within that timeframe
Bluebird Bio Zynteglo Beta-thalassemia
  • Outcomes-based agreements (OBAs) with certain state Medicaid agencies
  • Payers are reimbursed up to 80% for patients who do not achieve and maintain transfusion independence, up to 2 years
Spark Therapeutics Luxturna (voretigene neparvovec-rzyl) Biallelic RPE65 mutation-associated retinal dystrophy
  • Outcomes-based rebates: Provides rebates if the therapy does not meet certain efficacy thresholds over specific periods, up to 30 months
  • Novel contracting model: Changes the traditional buy and bill model by having the payer or their specialty pharmacy purchase the therapy directly
  • Extended payment options: Allows payers to spread payments over several years to manage the high upfront costs, with the potential for larger rebates based on the therapy’s long-term success
Pfizer Beqvez (fidanacogene elaparvovec-dzkt) Moderate to severe hemophilia B
  • Novel warranty program: Offers refunds or financial offsets if the therapy does not meet certain efficacy thresholds over a specified period
  • Aims to reduce financial risks associated with the therapy’s performance and ensure broader access and acceptance