Patient Support Design is Critical for Cell and Gene Therapies

Cell and gene therapies (CGTs) afford a radical shift in our approach to disease treatment: Scientists have determined that repairing abnormal cells or modifying the expression of a patient’s genes often addresses the root cause of diseases. CGTs offer the promise of enduring and profound treatment outcomes that can effectively cure a disease or condition. Nonetheless, patient access is a significant barrier.

To secure patients’ access to CGTs, most of the focus is on establishing reimbursement mechanisms. Manufacturers attempt to do so by developing payer and healthcare system engagement plans that communicate the therapy’s clinical and overall economic values. Gathering data necessary for value analyses starts in early-phase clinical trials, but some manufacturers do not adequately integrate patient assistance program requirements into the overall clinical development plan. Simply pricing a product equal to the value of what a patient receives for treatment does not guarantee access to therapy. Value-based pricing doesn’t account for the direct and indirect out-of-pocket costs associated with the therapy.

For example, the current estimated lifetime cost for a person with sickle cell disease (SCD) reaching age 50 ranges from $1.7 million¹ to $8.7 million.² Multiple gene therapies are under clinical development in which the SCD patient’s stem cells are modified through the use of a viral vector to produce normal, disc-shaped red blood cells. Presuming the gene therapy leads to a cure, the value to a patient is significant; however, the total cost of care for the therapy is overwhelming for a patient population with an average annual household income of $45,000.³ The final treatment protocol may require the patient to take multiple pharmaceutical products before and after the gene therapy treatment, travel to a facility authorized to perform the procedure, take time off work, and continue testing and follow-up for years. These additional treatment requirements constitute a significant cost, above copayments and coinsurance, to the patient and family.

Manufacturers’ patient assistance programs can reduce the cost burden. These programs provide drug copayment assistance to commercially insured patients or give free drugs to low-income patients. While these programs are ubiquitous to pharmaceutical products, they must be modified to support the needs and characteristics of the patient population that may benefit from curative CGTs.

When designing patient support programs, manufacturers must understand the expected patient population’s payer mix and the opportunities and limitations each payer presents. A gene therapy indicated to treat a pediatric population like SCD would need a program built around the needs of the Medicaid population; innovative therapies that treat older patients should cater to the requirements of Medicare and the senior demographic. All offerings must adhere to the restrictions imposed by the Office of Inspector General.

Assessing whether to support the costs of care for services outside of actual drug costs—such as testing and diagnostics—should also be considered. If drugs manufactured by another company are needed posttreatment to combat cytokine release syndrome, a common side effect of CGTs, should access to those drugs be supported financially? Should the indirect costs, such as loss of income for caregivers and transportation and lodging costs to receive care, be factored into the development of a complete program? Further, if manufacturers seek annuity payment approaches, how should they modify their program to provide continued copayment or coinsurance assistance—possibly for years and across payers?

Developing the solutions for patient affordability should start at the inception of the clinical development plan. The time has come to focus on patient assistance design and support long before launch preparations begin.

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1. Hulihan M, Hassell KL, Raphael JL, Smith-Whitley K, Thorpe P. CDC Grand Rounds: Improving the Lives of Persons with Sickle Cell Disease. Morb Mortal Wkly Rep. November 24, 2017.

2. Lanzkron S, Carroll CP, Haywood C, Jr. Mortality rates and age at death from sickle cell disease: U.S., 1979-2005. Public Health Reports. March-April 2013.

3. American FactFinder. Selected Population Profile in the United States 2017 American Community Survey 1-year Estimates – Black or African American alone. United States Census Bureau. https://factfinder.census.gov/faces/tableservices/jsf/pages/productview.xhtml?src=bkmk#. Updated 2017.