Unlocking the Potential of Cell and Gene Therapies: A Path to Affordable Healthcare

By Melike Belenli Gümüş

May 21, 2024

Introduction

In healthcare, we are witnessing a global shift with the advent of cell and gene therapies (CGTs). Advancements in CGTs open the door for innovative treatments that hold the key to addressing complex medical conditions. These innovative therapies, often referred to as “living drugs,” offer a ray of optimism for individuals struggling with rare diseases like blood disorders, cancer, and neurological diseases. However, despite their immense potential, challenges regarding access and affordability cast a significant shadow over the healthcare landscape.

Evolution of Cell and Gene Therapies

CGTs mark an important advancement in healthcare strategies, introducing innovative methods that deviate from conventional treatment practices. Gene therapy utilises genetic material to combat or prevent diseases, while cell therapy involves administering live cells to alleviate or potentially cure medical conditions. The spectrum of CGTs is vast, with over a thousand gene therapy variants and thousands of ongoing clinical trials exploring diverse cell therapy applications.

Navigating the Cost Conundrum

The cost implications of CGTs present a multifaceted challenge, encompassing the high price tags associated with these therapies, upfront financial burdens, and uncertainties regarding long-term benefits. The significant costs associated with CGTs arise from complex Research and Development (R&D) procedures, clinical trial expenditures, and the limited prevalence of target conditions. All of these reasons amplify the financial strain on patients, payers, and healthcare systems.

For instance, treatments like ABECMA (the first cell-based gene therapy for adult patients with multiple myeloma), HEMGENIX (the first gene therapy for hemophilia), and Lantidra (the first cellular therapy to treat patients with type 1 diabetes) come with substantial price points, underscoring the financial difficulties that patients and insurers face. While efforts to introduce value-based payment models aim to balance accessibility and affordability, the fragmented nature of healthcare systems poses obstacles in implementing such mechanisms effectively.

Proposing a Path to Sustainability

Amidst the complexities of financing CGTs, an alternative model emerges as a potential solution to streamline access and affordability. Introducing a publicly funded special plan with outcomes-based contracts for CGTs could offer a cohesive framework to address the challenges encountered by disjointed insurance structures. This model could enhance the sustainability of CGT payments by pooling resources from public entities and private insurers. As a result, a continued access for patients can be ensured while mitigating financial risks for insurers.

Can Cell and Gene Therapies Transform the Healthcare?

In conclusion, as the advancements in healthcare continues to evolve, embracing innovative payment models is necessary to unlock the full potential of CGTs. By bridging gaps in insurance coverage, aligning payment with outcomes, and fostering collaboration among stakeholders, we can get one step closer to a more efficient and equitable healthcare ecosystem. Through collective efforts and forward-thinking strategies, we can exploit the power of CGTs to enhance the well-being of individuals and drive positive change in healthcare delivery.

Reference url

Recent Posts

Assessment of Glofitamab With Gemcitabine in DLBCL Reveals No Additional Benefit

By João L. Carapinha

August 19, 2025

A recent G-BA decision provides a systematic evaluation of Glofitamab Gemcitabine DLBCL, combined with Gemcitabine and Oxaliplatin (GemOx), for treating adults with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) who are ineligible for autologous stem cell transplantation. The review...
Key Decisions on Health Technology Assessment at ACP124: Implications for Rare Disease Therapies
Health technology assessment (HTA) plays a crucial role in determining whether new therapies are reimbursed within healthcare systems. At the July 18, 2025 meeting of the Adviescommissie Pakket (ACP), two high-profile orphan drugs—omaveloxolone (Skyclarys®) for Friedreich’s ataxia and exagamgloge...
Eli Lilly’s Innovation Statement: Advocating Drug Pricing Reforms

By Staff Writer

August 18, 2025

Eli Lilly has issued a statement advocating for US drug pricing reforms. It emphasizes maintaining America's leadership in biopharmaceutical innovation. The Eli Lilly innovation statement highlights a $50 billion US manufacturing investment since 2020. This includes four new sites and opposes tar...