Ensuring Equity in Access to Advanced Therapies: Hematologist Highlights Challenges Across Spain

Advanced Therapies Offer Hope for Blood Disease Patients, Equity of Access Remains a Key Challenge

Madrid, Spain – September 8, 2025 – Significant strides in treatment options for blood diseases are emerging, but a critical challenge regarding equitable access persists. This was the key message delivered during the recent II National Meeting of Oncohematological Diseases Associations, organized by the Spanish group of entities dedicated to fighting leukemia and blood disorders.

Unprecedented Advances in Treatment

Fermín Sánchez-Guijo, Head of the Hematology Service at the University Hospital of Salamanca, emphasized the transformative potential of advanced therapies.”Car-T cell therapies and gene therapies have unlocked possibilities that were unimaginable a decade ago,” he stated. “these treatments are delivering enduring responses in cases with previously poor prognoses.”

Experts predict expansion of these advanced therapies in the coming years, perhaps including treatments for hemoglobinopathies, hemophilia, and a range of other genetic disorders. This integration into standard clinical practice promises a new era of medical intervention.

National Initiatives Fueling Progress

The creation of a robust network of reference centers, facilitated by the national Plan for Advanced Therapies and the Research Network in Advanced Therapies (TERAV), is instrumental in driving this progress. These centers operate under consistent standards, with public funding and dedicated research programs allowing for increased patient treatment capacity. According to data released by the Spanish Ministry of Health in july 2025, funding for advanced therapies has increased by 35% in the last two years.

The Challenge of Equitable Access

Despite these advancements, Sánchez-Guijo cautioned that ensuring equitable access to these therapies remains a primary concern. It is crucial to translate these innovations into tangible benefits for all patients, regardless of location or socioeconomic status. Streamlining implementation processes and addressing the projected rise in the number of eligible candidates are vital steps.

Patient advocacy groups are being called upon to play a more significant role, actively participating in decision-making, testing, and evaluating treatment results. Such collaboration, experts argue, is integral to ensuring these advancements meaningfully improve patient quality of life.

Precision Medicine: Tailoring Treatment to the individual

Dr. Fernando Martín, a hematologist at the Ramón y Cajal University Hospital in Madrid, highlighted the increasing sophistication of precision medicine in hematology. Recent breakthroughs in understanding the biological underpinnings of hematological tumors enable more accurate diagnoses and personalized treatment plans.

“We’ve moved beyond a one-size-fits-all approach to offering treatment tailored to each patient’s unique biological profile,” Martín explained. However, he also noted that delays in diagnosis and logistical hurdles in administering new treatments continue to impede access to precision medicine for many.

To overcome these barriers, Martín advocated for stronger coordination among treatment centers and enhanced communication to ensure patients and families are fully informed about all available options, including participation in clinical trials.

Patient-Centric Care: Adapting to Individual Needs

ascension Hernández,President of Aelclés,underscored the importance of adapting treatments to the individual circumstances of each patient. she argued that individuals with diseases are not merely recipients of clinical protocols, but people with complex lives, responsibilities, and personal challenges.

Patient associations strongly support this emphasis on personalized care, believing that it enhances treatment effectiveness and promotes true patient-centered attention.

Therapy Type	Key Benefits	Challenges
CAR-T Cell Therapy	Long-lasting responses in severe blood cancers	high cost, logistical complexity
Gene Therapy	Potential cure for genetic blood disorders	Long-term effects still under investigation, accessibility
Precision Medicine	Tailored treatments based on individual biology	Diagnostic delays, unequal access to advanced testing

Understanding Advanced Therapies

Advanced therapies represent a paradigm shift in medical treatment. These therapies frequently enough involve modifying a patient’s own cells or introducing new genetic material to combat disease. While promising,they require specialized infrastructure,expertise,and significant investment. Ongoing research is focused on improving treatment efficacy, reducing side effects, and lowering costs to make these therapies more accessible.

Did You Know? CAR-T cell therapy involves engineering a patient’s own immune cells to target and destroy cancer cells.

Pro Tip: If you or a loved one is considering an advanced therapy, it’s crucial to discuss the potential benefits and risks with a qualified medical professional.

Frequently Asked Questions

What role do you believe patient advocacy groups should play in shaping the future of advanced therapy access? Share your thoughts in the comments below.

Do you think enough is being done to address the logistical challenges of delivering these life-saving treatments to all who need them?

## Summary of Barriers to Accessing Advanced Therapies in the Spanish Healthcare System

As a hematologist practicing in Spain, I’ve witnessed firsthand the revolutionary potential of advanced therapies – including CAR-T cell therapy, gene therapy for blood disorders, and hematopoietic stem cell transplantation (HSCT) – in treating previously intractable blood cancers and genetic conditions like sickle cell disease and thalassemia. Though, this progress is shadowed by notable disparities in access across our country. This article details the challenges hindering equitable distribution of these life-changing treatments, focusing on regional variations, economic barriers, and infrastructural limitations within the Spanish healthcare system.

Regional Disparities in Advanced Therapy Availability

The Spanish healthcare system is decentralized, granting significant autonomy to its 17 autonomous communities. While this allows for tailored healthcare approaches, it also creates a fragmented landscape for advanced therapeutic medicinal products (ATMPs).

Concentration of Expertise: Highly specialized centers capable of delivering CAR-T cell therapy and complex gene therapies are largely concentrated in major cities like Barcelona, Madrid, Valencia, and Seville. This geographical limitation forces patients from smaller provinces to travel long distances, incurring substantial costs and logistical difficulties.

Funding Allocation: Funding for novel cancer treatments, including ATMPs, varies considerably between regions. Communities with stronger economies frequently enough allocate more resources to these therapies, creating a postcode lottery for patients.A patient diagnosed with acute lymphoblastic leukemia (ALL) in Catalonia may have a substantially higher chance of accessing CAR-T therapy than a patient with the same diagnosis in Extremadura.

Varied Implementation of National Guidelines: While national guidelines exist for certain hematological malignancies, their implementation differs across regions. This inconsistency impacts patient eligibility criteria and the speed at which treatments are approved and administered. Chronic myeloid leukemia (CML) patients, such as, may experience differing access to newer tyrosine kinase inhibitors (TKIs) depending on their region.

Economic Barriers to Accessing Innovative Treatments

The high cost of advanced therapies presents a major obstacle to equitable access. These treatments often carry price tags exceeding €300,000 per patient, placing a significant strain on healthcare budgets.

1. Reimbursement Challenges: Securing reimbursement for ATMPs from regional health authorities is a complex and frequently enough protracted process.The lack of standardized health technology assessment (HTA) procedures across Spain further complicates matters. Health economics plays a crucial role, and demonstrating cost-effectiveness is paramount.
2. Patient Co-pays: While the Spanish healthcare system is largely publicly funded, some patients may be required to pay a co-pay for certain medications or procedures. for expensive therapies like gene therapy, even a small co-pay can be prohibitive for low-income families.
3. Indirect Costs: Beyond the treatment cost itself, patients face significant indirect costs associated with travel, accomodation, and lost income during treatment. these costs disproportionately affect patients from rural areas or those with limited financial resources. Financial toxicity is a growing concern.

infrastructural and Logistical Hurdles

Delivering advanced therapies requires specialized infrastructure and a highly trained workforce. Spain faces challenges in both areas.

Qualified Personnel Shortage: There is a shortage of hematologists, oncologists, and specialized nurses with expertise in administering and managing patients receiving ATMPs.This shortage limits the capacity of existing centers and hinders the expansion of these therapies to new regions. Continuing medical education (CME) is vital.

Logistics of Cell Processing & Transport: CAR-T cell therapy involves collecting a patient’s T cells, genetically modifying them, and then re-infusing them back into the patient. This process requires specialized facilities and a robust logistical network to ensure the cells remain viable during transport. Maintaining the cold chain is critical.

Limited Apheresis Capacity: Apheresis, the process of collecting blood cells for therapies like HSCT and CAR-T, is not universally available across Spain. This limits the number of patients who can be considered for these treatments.

Data Collection & Registries: A national registry for patients receiving advanced therapies is crucial for monitoring outcomes, identifying areas for improvement, and informing future funding decisions. Currently, data collection is fragmented and inconsistent. Real-world evidence (RWE) is increasingly important.

Case Study: Access to Gene Therapy for Beta-thalassemia

The recent approval of gene therapy for severe beta-thalassemia highlights the challenges of equitable access. While a few centers in Spain are now offering this treatment, the high cost and complex eligibility criteria mean that only a small fraction of eligible patients have been able to benefit. The process of identifying suitable candidates, navigating the reimbursement process, and coordinating the treatment has proven to be lengthy and arduous. This underscores the need for streamlined pathways and increased funding.

benefits of Improved Equity in Access

Addressing these challenges is not merely a matter of fairness; it’s a matter of improving patient outcomes and maximizing the value of these groundbreaking therapies.

Improved Survival Rates: Equitable access to advanced therapies will lead to improved survival rates for patients with previously incurable blood cancers and genetic disorders.

Reduced Healthcare Costs: While the initial cost of these therapies is high, they can possibly reduce long-term healthcare costs by eliminating the need for chronic treatments and hospitalizations.

Enhanced Quality of life: Advanced therapies can significantly improve the quality of life for patients, allowing them to return to work, school, and other activities.

Strengthened Research & Innovation: investing in advanced therapies will stimulate research and innovation in the Spanish biotechnology sector.

Practical Tips for Patients and Advocates

Navigating the system can be daunting. Here are some practical tips:

Seek Expert Consultation: Consult with a hematologist specializing in advanced therapies to determine if you are a suitable candidate.

Understand Your Regional Healthcare System: Familiarize yourself with the specific procedures and requirements for accessing ATMPs in your autonomous community.

Advocate for Your Rights: Don’t hesitate to advocate for your rights and seek assistance from patient advocacy groups. Patient empowerment is key.

Explore Financial Assistance Programs: Investigate whether you are eligible for any financial assistance programs that can help cover the cost of treatment.

Participate in Clinical Trials: Consider participating in clinical trials, which may provide access to cutting-edge therapies.

Keywords: Advanced Therapies, CAR-T cell therapy, Gene Therapy, Hematopoietic Stem Cell Transplantation (HSCT), Spain, healthcare Equity, Blood Cancer, Leukemia, Lymphoma, sickle Cell Disease, Thalassemia, access to Treatment, Reimbursement, Health Technology Assessment (HTA), Regional Disparities, Financial Toxicity, Patient Advocacy, Beta-Thalassemia, Chronic Myeloid Leukemia (CML), Novel Cancer Treatments, health Economics, Continuing Medical Education (CME), cold Chain, Real-world Evidence (RWE), Patient Empowerment.