Unlocking the potential of cell and gene therapy

Navigating pricing and access for a healthier future

By Rina Chotai, Head of IPG Health Global Market Access and Eunmi Ha, Senior Analyst, IPG Health Global Market Access

Updated May 14, 2024

In the evolving landscape of healthcare, few innovations hold as much promise, or command such large price tags, as cell and gene therapy (C&GT). These groundbreaking treatments have the potential to revolutionise the way diseases are treated, offering a one-time functional cure, but hold multi-million-dollar prices that create a barrier for patients who may previously have had no meaningful treatment. The potential held by C&GT comes with a complex challenge for manufacturers, payers, prescribers and patients, and one that manufacturers and payers are beginning to navigate together. We explore some of these innovative arrangements that ensure access for patients in need.

Navigating pricing pressures
 

The most expensive pharmaceutical products in the world are C&GTs, and the list of approved therapies is growing. Currently there are several therapies that surpass $3M per dose in the US.^1,2

The ultra-high prices of these treatments are at the forefront of public discourse. Additionally, payers have shown resistance to multi-million-dollar price tags, with concerns regarding the high upfront costs and the long-term efficacy of novel C&GT. 

Inability to reach a consensus on fair pricing can result in manufacturers pulling out of the market completely - leaving patients without a functional cure.

Innovative finance agreements
 

With the rise of the C&GT era, payers have increasingly begun to enter into nontraditional finance models to better provide care for those being underserved by existing models. Two creative reimbursement models are payment-over-time and pay-for-performance. ^3,4,5 Similar to a mortgage, the first better reflects the value provided by C&GT as a long-term investment and may help reimbursement systems configured to long-term, progressive costs cope with the high upfront costs of C&GT. In the pay-for-performance or outcomes-based model, risk is shared between the payer and the manufacturer, reducing the risk associated with large upfront payments and helping to reassure the payer. This method helps to offset the perceived risks of these novel treatments with limited long-term evidence.

Spinal muscular atrophy case study
 

A national access model (US)
 

A new, outcomes-based C&GT access model has been announced by the Centers for Medicare & Medicaid Services (CMS) in the US, with roll-out estimated for January 2025. With the aim of improving the lives of people with Medicaid living with rare and severe diseases, it targets some of the US’s most vulnerable populations.¹⁰ The initial focus for this model will be sickle cell disease (SCD) - estimated to affect 1 out of every (SCD) – estimated to affect 1 out of every 365 Black or African American births and about 1 out of every 16,300 Hispanic American births¹⁶. This comes as positive news for an indication where several manufacturers have discontinued investment and development of genetic treatments for the blood disease.^12,13 The US currently has two approved SCD C&GTs.^13,14,15 In the UK and EU, only one option is available.

Cell and Gene Therapy (C&GT) Access Model¹³
 

A voluntary model for states and manufacturers with two dual goals:

To improve health outcomes for people with Medicaid who could benefit from C&GTs by supporting outcomes-based agreements  (OBAs) between states and manufacturers that will provide for treatments within a framework that lowers prices for states and ties payment to outcomes.

To increase access to transformative therapies, and potentially help address the historic disparities, poor health outcomes, and low life expectancy associated with SCD.

Market access considerations
 

Several analyses by the Institute for  Clinical and Economic Review (ICER) have indicated cost-savings: the high price of these drugs balanced by the long-term cost-savings to the healthcare system.^16,17 By framing the clinical benefits in a manner familiar to payers, the long-term savings of C&GT can be assessed more fairly. Discussions with payers and patients should be conducted early to identify the payer reimbursement framework and insights built into evidence generation and launch strategy to directly target concerns when negotiating reimbursement agreements.

The number of products in the C&GT pipeline are growing, with the rate of approvals likely to increase significantly in the coming years. Patient assistance programs, reimbursement strategies, and innovative insurance models can all play a role in widening access and reducing financial barriers. C&GT hold immense promise for patients. However, realising this potential requires a delicate balancing act between innovation, pricing, and access. By adopting a value-based pricing approach, fostering collaboration among stakeholders, and ensuring equitable access, a way can be paved to a healthcare landscape where life-changing cures are within reach for all.

Value based pricing
 

Frames the potential healthcare benefits of new treatments in a language that resonates with payers

Ties the price of a therapy to its demonstrated clinical value, taking into account factors such as improved quality of life, reduced hospitalisations, and increased patient productivity.
 

What companies can do and where we can help to gain optimal access:

1. Discover

Discover the reimbursement potential of your product space with landscape analysis and set up your product for success with early payer engagement. We can facilitate early dialogue with payers, clinicians and patient advocacy groups to provide insights into the perceived value of your product and target product profile, understand evidence requirements and test the willingness to pay. Early payer research aligns regulatory and reimbursement activities to strengthen product positioning and strategy. For innovative treatments, we recommend a collaborative approach to designing payment models – co-creation with payers through facilitated workshops and discussions.

2. Define

Define meaningful value in a language that resonates with payers. We build cost-effectiveness and budget impact models to highlight the benefits of new products within the paradigm familiar to each market. Assessments into burden of illness, unmet need and patient access can further support payer discussions and negotiations. We recommend evidence gap analysis and early mitigation plans to help define product strategy, strengths and positioning.

3. Develop

Develop product value narratives tailored for targeted payer profile.  We craft value propositions, global value dossiers, models, healthcare utilisation calculators and omnichannel stakeholder communication tools, so the right asset reaches the right payer, at the right time. Our extended network of payers and healthcare professionals can be leveraged to validate the treatment pathway and model inputs.

4. Deliver

Deliver the value of the product with external value communications. We can, plan and facilitate stakeholder advisory boards, and Delphi panels to provide actionable insights to tight timelines, including the development of expert consensus statements, for peer reviewed publication. We recommend an omnichannel strategy of globally aligned talking points and objection handlers to ensure the right messages reach, the right stakeholder, at the right time to support successful market access.

5. Differentiate

Differentiate your product with an optimized market access strategy, reflecting the developing competitor, pricing, and reimbursement landscape. We recommend localisation of external value communications and strategies to ensure your product always stands out, based on local market dynamics.

If you are interested in embarking on this journey together please reach out to …