Patient views and challenges

Understanding patient challenges and experiences is a critical part of developing standards for access to cell and gene therapy (CGT). The shared CGT journey in our integrated view of stakeholder perspectives and challenges has many nuanced implications for patients.

The journey begins with the decision by the patient to seek out and receive a CGT treatment, which is strongly influenced by the disease severity and treatment options. For diseases with severe consequences, such as impacting life expectancy, the decision to seek CGT treatment is anticipated to be high. For diseases with less severe or more long-term consequences, only a small percentage of eligible patients may seek to be treated. How greatly available non-CGT treatments impact quality of life will also influence a decision for treatment.

Knowledge of clinical trial results of the cell or gene therapy will also likely factor into a patient’s decision making. CGTs are primarily targeted towards rare diseases, limiting the potential size of any clinical trials. At the time of initial regulatory approval and market launch for CGTs, real-world efficacy and durability are uncertain. Patients may need to consider performance uncertainty characterized as two types of risks:

  1. Uncertainty as to the expected benefits of treatments in a broader, real-world population, given that regulatory approval will be based on limited clinical trial data and real-world results are sometimes different from clinical trial results; and
  2. Uncertainty as to whether the benefits will be durable and if not, how long they will last.

Once a decision is made to seek treatment, the patient faces financial and implementation challenges. To understand the patient and caregiver financial and implementation challenges, FoCUS undertook a systematic literature review focusing on patient views about the impact of economic or financial burden, stress, or distress on patients, their caregivers, or family.

The results highlighted 27 themes spanning finances, mental health, demographics, and socioeconomics, and a range of challenges encompassing how patients and caregivers actually navigate the healthcare system to access treatment. FoCUS stakeholders have further examined the patient impact of these new therapies, and the financial and operational implications for their treatment.

Individual products in the broad category of CGTs have different profiles and characteristics that affect patient access and the various Precision Financing Solutions outlined by FoCUS stakeholders. For example, new infrastructure may be needed for treatment delivery and follow-up care. New information technology (IT) and data collection requirements may be required for product performance measures. These concepts are illustrated in the research briefs Precision Financing challenges for solid tumor adoptive T-cell therapies and Payer perspectives on outcomes tracking for value-based payment arrangements (VBPs). For these product profiles, patients share concerns with other stakeholders regarding risks, limitations, and unknowns.

Patient and caregiver experience will vary by payer and therapy, but there are several common issues:

1. Determining status for access to treatments: Patient access to therapies depends on a variety of factors, including geography, ability to navigate institutional environments, insurance status, and, when insured, the coverage and formulary decisions of payers. Resource navigation was the fourth-most common theme established (after payments, socio-economic challenges, and medical care reduction) in the FoCUS literature review. Patients need support that includes the management of financial challenges. This includes navigating the fragmented landscape of potential monies available and locating advisors who could help with this task.

2. Accessible provider networks: To control costs, some plans may restrict their provider networks, establishing ‘narrow networks.’ Moreover, for CGTs, there may be limited providers (Centers of Excellence) who are authorized by either a developer or a payer to deliver a particular therapy to ensure quality administration. Out-of-state access to providers and treatment centers may be needed.

Most CGTs today are administered by physicians certified to do so in Centers of Excellence (COE). These COEs may not exist in every state. Patients may need to travel out-of-state for treatment. (Read more here regarding COEs and here regarding out-of-state networks.) Payers may need to establish specific processes for patients to access providers who are not typically ‘in network.’ This is particularly relevant for Medicaid payers, HMOs, and regional plans who may traditionally work only with in-state providers.

3. Potential lost income: The patient’s age will determine whether and how the income-earning members of their household are affected. The lost income may result from the patient or caregiver’s limited availability to work due to health status and the time required for medical appointments. In some cases, there may be limited providers who are authorized to deliver a particular therapy within the patient’s regional area. As a result, patients will need to seek out new providers for which travel is required, causing the patient and/or caregiver to lose more time from work.

Some states have enacted paid family medical leave programs, which can help the income earners in these situations. Also, disability benefits provided by employers can protect a portion of lost income for patients who are employees. While helpful, these programs do not fully replace previous earnings.

4. Monitoring over time: As CGT is a new area of science, patient monitoring over time will be required from a regulatory perspective. Patients will need to provide input on methods for the least burdensome and most beneficial tracking mechanisms developed by payers and developers. The Food and Drug Administration (FDA) requires developers to obtain long-term follow-up data for these novel therapies. In addition, FoCUS has recommended financial solutions incorporating payments or rebates that would require patient data tracking over multiple years. This can be particularly challenging if patients switch health plans or providers. Patients may be asked to commit to a follow-up care schedule and to allow their data to be shared with the organizations administering performance contracts before they start treatment. Read more here regarding patient mobility and here regarding outcomes tracking.

5. Financial burden: Generally, for CGT, the patient will experience a high financial burden due to out-of-pocket costs from co-insurance, copays, deductibles, and in some cases, travel to care sites. The specific impact will vary by patient, but this burden is likely to be experienced all at once under the current payment structure. As a one-off cost, new treatments can affect out-of-pocket costs much more at the start of the year than at the end. For some patients, the treatments may represent new costs. Other patients receiving existing high-cost treatments may already be hitting their deductibles and out-of-pocket maximums. Still other patients may see the costs of CGT treatment offset by savings from traditional treatment regimes.

Note: Patient copay costs primarily serve to encourage patients and their clinicians to make more appropriate healthcare choices when effective, lower-cost options are available. FoCUS participants, including several payers, suggested that copays, deductibles, and coinsurance should be waived for CGT products provided it could be done without inducing adverse selection. That said, such a change in benefit design can require state insurance approval and may take up to 18 months. You can read more patient cost sharing in the patient financial implications section. This FoCUS research brief describes State Insurance Regulations Regarding Benefit Design (Deductible and Co-Pay Waivers).

6. Education: To help patients self-advocate for access to CGTs and outcome monitor requirements patients can support for innovative CGT financing, patient and caregiver education on financing options and financial literacy is also needed to enable effective planning.

Potential elements to alleviate patient financial burden

Stakeholders and FoCUS participants have suggested potential solutions, such as pharmaceutical assistance programs, patient access support programs (not available for patients covered by government payers), provider discounts and charitable write-offs, waived co-pays, deductibles and coinsurance benefit designs for these products, provided it could be waved without inducing adverse selection and continued innovation in financial service offerings.

  • Pharmaceutical assistance programs: Manufacturers of chronic, rare disease medications (e.g., intravenous immunoglobulin or enzyme replacement therapies) often offer copay support programs for patients with commercial insurance as well as donations to 501(c)(3) organizations that offer financial support for patients with public insurance. This solution could also apply to CGTs.
  • Access support programs: Resources for help with insurance coverage and connections to financial assistance are frequently provided by manufacturers and non-profit organizations as ‘patient support programs.‘ In the gene therapy space, for example, Spark Therapeutics provides a support team through their program, Generation Patient Services to find, among other services, resources to support out-of-pocket costs. Another example, The Assistance Fund, is an independent charitable organization that provides funds to support access to treatment of various diseases for patients including those with Medicare or Medicaid plans. These types of programs are helpful, but likely inadequate. Further limiting the patient benefit of assistance programs, some Pharmacy Benefit Managers (PBMs) have instituted Accumulator Adjustment Programs that can have a negative financial impact on patients, by no longer allowing copay assistance amounts to count towards a member’s accumulator (the dollar amount applied to the patient’s deductible or out-of-pocket costs). If adjustments are applied to CGTs, then any positive effects of a manufacturer’s assistance program would be negated.
  • Charitable care and financial assistance: Providers may also provide discounts and charity care, particularly non-profit providers that have both mission and legal rationale to do so. Such capacity, however, is limited.
  • Financial services solutions: The financial services industry already provides patients with second mortgages, consumer loans, and credit card debt, among other services to aid in financing healthcare costs. However, accessibility to these is low and very unevenly distributed in society.

FoCUS stakeholders have suggested additional financial instruments, such as personal healthcare loans. Continued innovation in financial services, particularly for patients with poor current credit scores but improved prospects after therapy, is needed.