Connecting Employees to Cell and Gene Therapies Outside the United States: Opportunities and Challenges

As innovative high-cost therapies enter the market, employers should consider and evaluate their role in connecting employees to cell and gene therapies around the world.

July 12, 2024

Introduction

Cell and gene therapies (CGTs) target serious and sometimes fatal conditions, and if successful, can save lives. As research and development of these treatments rapidly progresses, employers have an opportunity to explore cost-effective approaches for making these therapies available to employees who need them.

Employers both in and outside the United States (OUS) face many issues when considering CGTs. These therapies, though potentially curative, are medically complex to administer subjecting patients to a multiyear care journey. Further, the price of these therapies and the associated cost of hospitalization and care are staggering. The overall impact on health plan costs is poised to continue to rise as more therapies enter the pipeline and receive approval for use. According to the 2024 Large Employer Health Care Strategy Survey, 79% of employer respondents are very concerned or concerned about the patient and plan affordability of CGTs in the pipeline.

These problems are even more complex for employers OUS. Appropriate access to CGTs is hindered by inconsistent or nonexistent coverage in different countries, as well as limited clinical capacity, expertise and evidence. Given these realities, employers are faced with a crucial question: What can they do to bring CGTs to all employees/covered members who need them, including those OUS?

Perhaps the best way for employers to address this question is by considering the appropriate use of these high-cost treatments and then determining how to mitigate the risks and volatility associated with paying for them. This approach will enable global consistency in coverage and promote a more equitable health system regardless of location. As such, employers have an opportunity to consider the role they can play in ensuring employees around the world have access to these cutting-edge therapies.

Gene Therapy, Cell Therapy, Immunotherapy, CAR-T: What’s the Difference?

Gene therapy is the introduction, removal or change in the content of a person’s genetic code with the goal of treating or curing a disease.

Cell therapy is the transfer of intact, live cells into a patient to help lessen or cure a disease.

Immunotherapy or biological therapy is the treatment of disease by activating or suppressing the immune system.

CAR-T therapies are a type of immunotherapy that makes use of genetically modified T cells. In other words, it uses CGT as a means to immunotherapy.


Overcoming Challenges: Considerations and Opportunities for Employers to Seek Cost-Effective Access to Cell and Gene Therapies Outside the U.S.

Challenge #1: Limited Access and Inconsistent Coverage

Employers face a challenge in ensuring cost-effective access to CGTs for employees located OUS in part due to country-by-country variations, including:

  • Fewer accredited CGT administration sites;
  • Limited clinical expertise and clinical trial access;
  • Varying reimbursement structures;
  • Gaps in government-provided health care across countries; and
  • Resource-intensive storage (i.e., ultra-cold temperature requirements).

Consideration: With coverage levels varying widely across the world, employers can address the gaps in access by topping off government-funded health plans. Moreover, such an approach can align well with employers’ global consistency strategy. Multinational employers will need to carefully identify how they wish to support government-funded health plans, and their choice may depend on their footprint. For example, if a government does not provide coverage for impactful CGTs, will the employer supplement coverage, and if so, is it fully or partially covered? In either case, it will be crucial for employers to carefully curate a message to explain the coverage to employees who may be eligible for CGTs. Employers will also need to consider travel-related costs associated with the treatment.

Opportunity: Having proactive conversations to raise awareness and determine what internal processes are needed for managing CGTs is important given the fact that access to CGTs remains limited in many low- and middle-income countries.


Challenge #2: High Costs of Cell and Gene Therapies

CGTs usually come with extremely high price tags, sometimes reaching hundreds of thousands or even millions of dollars per therapy, excluding the cost of hospitalization and other care delivery services. For example, Lenmeldy, a gene therapy used to treat a rare genetic condition known as metachromatic leukodystrophy (MLD), a pediatric neurological disease, has a wholesale price of $4.25 million in the U.S., an average cost of $2.4 million in Germany and $3.1 million in the United Kingdom (U.K.).1,2 These high costs may pose a challenge for government-funded programs considering coverage, which in turn may further limit access to CGTs globally. Still, it is important not to disregard these therapies when considering the quality-of-life improvement they can bring to the patients who receive them.  

Consideration: Employers can consider location-based approaches to advance access in countries where the infrastructure/market would allow it. India’s public health insurance system is largely decentralized and is mixed between public and private payers, resulting in many coverage gaps across different areas/states.3 From a population health perspective, India has a relatively high burden of “rare” genetic diseases compared to other countries.4,5 Given the significant disease burden, the Indian government aims to increase self-reliance for widespread treatment access and is investing in becoming a cost-effective manufacturing hub for gene therapies6,7 Additionally, India has a significant generic pharmaceutical industry, which could potentially lead to more affordable options for gene therapies in the future, once patents expire.8

Opportunity: Having a captive provides employers with the opportunity to get better, more timely insight into their claims data. Many multinational employers are already considering implementing a captive strategy to better manage costs, drive global consistency and/or promote health equity. A captive may similarly prove useful in ensuring access to treatment for specific genetic conditions, enabling employers to make more informed, data-driven decisions for employee populations.


Challenge #3: Extensive Wraparound Services Are Required

CGTs often require specialized treatment centers, and completing a course can take several months or sometimes even years, resulting in many logistical hurdles for the employee needing care. As such, significant wraparound services are often needed to adequately support employees throughout the care continuum (e.g., leave and time away accommodations, flexible work schedules, childcare support/subsidies, etc.) For example, CAR-T cell therapy can take up to 3 months to complete and is associated with some side effects and complications that require close clinical monitoring and management for up to 30 days, particularly in the immediate posttreatment period.9 While the cost of administering CGTs can vary widely, one analysis estimates that the list price of Kymriah, a CAR-T therapy for treating children with leukemia is $361,000 in the U.K.10 This challenge is further exacerbated for multinational employers providing wraparound support to geographically dispersed employee populations living in countries with different regulations and health care coverage. Employers also have to contend with the needs of the caregiver who is supporting the patient throughout their treatment journey.

Consideration: Supporting care continuity requires consideration of travel needs and wraparound services to ensure positive treatment outcomes. Programs designed to support patients in accessing CGTs must consider the caregiver’s needs, especially if the patient has multiple follow-ups and needs ongoing monitoring. It is also important for employers to keep in mind any potential legal implications when considering a travel-based approach. When a patient is eligible for a CGT and recommended clinically, if no local options exist for treatment, employers can consider supporting travel to locations where treatment is available. Though logistically difficult, offering travel support for employees to receive care outside their home country is an option that can enable access to CGT for employees located OUS who otherwise would be unable to receive treatment. As the infrastructure and capacity to support CGT care delivery OUS continue to grow, employers can consider opportunities to bolster regional travel options where clinically appropriate. It is equally important that employers also factor in the travel needs of caregivers throughout treatment and follow up.

Opportunity: Employers have an opportunity to effectively manage wraparound support by proactively taking steps to ensure expensive gene therapies are only administered in highly specialized settings, where employees can receive adequate follow up and/or where travel accommodations are logistically feasible. In addition, employers should be aware that different CGTs may require several types/levels of care support. For example, CAR-T cell therapy follows rigid timelines to optimize treatment outcomes; the ability to travel back and forth between accredited treatment centers and home may be a challenge.


Challenge #4: Insufficient Evidence

The lack of standardized approaches for assessing CGTs by Health Technology Assessment (HTA) agencies OUS poses a significant challenge. HTA agencies typically rely on evidence from high-quality studies such as randomized controlled trials, yet evidence for these therapies is often less robust. Moreover, in low- to middle-income countries where more prevalent diseases (e.g., HIV, TB, malaria) persist, CGTs may not get the funding and resource allocation necessary for HTA entities to build a credible evidence base. Consequently, differences in HTA methodologies across countries can lead to variations in coverage decisions for CGTs.11,12 A noteworthy example of this is playing out in the U.K. where contrary to guidance from the U.S. Food and Drug Administration (FDA), the National Institute for Health and Care Excellence (NICE) is not recommending a sickle cell gene therapy known as Casgevy in its current draft guidance.11,12 While many countries have established HTA entities to evaluate the cost-effectiveness of new health technologies, the specific impact on coverage for CGT remains unclear due to the dearth of evidence on this matter.13-15  With a new HTA process ready to be rolled out in January 2025 in Europe, the hope is that more evidence will be available to more countries, resulting in greater alignment of approvals (see callout box).

The European Commission announced a new continent-wide HTA process that is slated to roll out starting in January 2025.16 This effort aims to provide countries in the European Union (EU) greater access to the evidence needed to appropriately price and determine coverage for CGTs once approved by the European Medicine Agency (EMA). Aside from providing greater evidence to countries, it is uncertain how this effort will directly impact the role and decision-making processes currently used in countries that already possess more sophisticated HTA entities, such as the U.K. (i.e., NICE). Ultimately, it may allow for greater evidence sharing among individual countries, which can be especially beneficial for smaller countries with a less robust HTA decision-making process in place.17


Consideration: Inconsistency in assessment frameworks underscores the need for greater specificity and harmonization in evaluating these innovative treatments on a country-by-country basis. Balancing resources to address both immediate health crises and long-term innovations remains a critical challenge for multinational employers.

Opportunity: Proactive employers can leverage guidance from countries OUS that already have well-established HTAs that are shaping the CGT coverage landscape, such as the U.K. and France.12

  • United Kingdom: National Institute for Health and Care Excellence (NICE): NICE conducts thorough assessments of the clinical and cost-effectiveness of CGTs. The assessment considers factors such as the cost of the therapy compared to its health benefits and the impact on the overall health care budget. NICE plays a significant role in determining the coverage and accessibility of CGTs within the U.K.’s National Health Service (NHS) because if it recommends a therapy as cost-effective and clinically sound, it is more likely to be funded and accessible to patients in the U.K.11,12
  • France: Haute Autorité de Santé (HAS): HAS is the French national health authority responsible for evaluating the clinical efficacy, safety and cost-effectiveness of new health care technologies, including CGTs. Similar to NICE in the U.K., HAS plays a crucial role in determining the coverage and reimbursement of these therapies within the national health care system.18,19

Challenge #5: Rapidly Evolving Pipeline

The CGT pipeline is robust for the near future, resulting in a field that is rapidly expanding, characterized by new product launches with associated protocols and the emergence of different targeted patient populations for each CGT. These changes can be difficult to keep up with. Moreover, given the inherent newness of CGTs, it will likely take time for health care providers to attain the clinical expertise needed to support administration of the medications in different settings around the world. Though clinical expertise, accredited facilities and supporting staff are globally limited, multinational employers are well-positioned to proactively leverage their diverse landscape of vendor partners to stay abreast of CGT product developments and coverage decisions made around the world.

Consideration: In 2024 there were 76 CGTs available worldwide, which is nearly double the number launched in 2013.20 Some of the upcoming launches in the pipeline include new treatment approaches for specialty conditions like hemophilia B, sickle cell disease and certain types of blood cancer.21 Recently approved CGTs in the U.S. include Casgevy, with a list price of $2.2 million.22 Another CAR-T cell therapy, Breyanzi, was approved in March 2024 by the FDA for the treatment of chronic lymphocytic leukemia and small lymphocytic leukemia; the list price is $410,300.23

Opportunity: Each country has a different system and approach in place to assess the emerging pipeline of CGTs and their impact. Employers may want to leverage local and regional expertise where possible to better grasp when/if these innovative treatments may be available. For example, in recent years, there have been efforts among various stakeholder groups in India, including clinicians, researchers, industry partners, patient advocates and others, to raise awareness about genetic diseases and promote CGT development in India.6 Though access is currently limited, clinical trials are increasingly being considered and taking place in India; for example, in February 2024 India launched its first clinical trial of a treatment for hemophilia A.24 In addition, CAR-T cell trials within academic centers are supported by the Indian Council of Medical Research (ICMR), which has a gene therapy task force responsible for  developing the National Guidelines for Gene Therapy Product Development and Clinical Trials in India.6


Employer Recommendations

  • 1 | Assess the CGT needs in your population: Where legally possible, look at population-level health data to identify how many employees and dependents may have a condition that can be treated by a CGT. Employers will want to keep in mind existing data and privacy laws that may make this effort a challenge, such as the General Data Protection Regulation (GDPR), applicable in the European Union.
  • 2 | Monitor the gene therapy pipeline in the U.S. and other countries: Multinational employers seeking to monitor the fast-growing CGT pipeline around the world can rely on their consultants to monitor and inform them on pipeline advancements. Additionally, staying abreast of regulatory changes and guidelines issued by governmental agencies such as the FDA, EMA and equivalent regulatory bodies in other regions is crucial.
  • 3 | Keep up with emerging vendor solutions/offerings: Work with the health plan, as well as vendor and broker partners, to help keep an eye on emerging evidence and innovative solutions that can help inform your efforts to effectively manage and mitigate long-term cost challenges and ensure appropriate utilization of CGTs. For more information on the various financing mechanism available for employers to manage costs of CGT risk, check out our resource here.
  • 4 | Make sure employees and caregivers have access to wraparound care: Work with local HR teams and regional partners where possible to ensure that employees/covered members and their caregivers have the necessary wraparound support needed during and after treatment. To learn more about what the CGT patient experience entails, click here.

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