20 Custom Therapy Eligibility Statistics: Critical Data for Rare Disease Patients and Providers in 2025

Comprehensive data on personalized therapeutic access, eligibility barriers, and the operational challenges facing rare disease populations globally

Key Takeaways

• Rare disease populations face massive treatment gaps – About 300 million people live with rare diseases globally, and roughly 95% of rare diseases have no FDA-approved treatment, creating urgent need for personalized therapeutic platforms that can scale beyond traditional pharmaceutical economics

• Eligibility barriers block potential candidates – Studies of CAR-T cellular therapy show substantial attrition, with many patients initially referred never receiving treatment due to disease progression, declining health status, or failure to meet complex eligibility criteria during lengthy evaluation processes

• Manufacturing and operational delays determine access – The referral-to-infusion timeline for CAR-T therapy can span weeks to months, creating critical windows where patients deteriorate, highlighting need for platforms that accelerate feasibility assessment and manufacturing coordination

• Geographic and infrastructure limitations concentrate access – Access to experimental custom therapies remains concentrated at specialized academic centers with FACT accreditation and specific capabilities, leaving patients in underserved regions without viable pathways to personalized treatment options

• Early intervention dramatically improves eligibility rates – Patients referred for custom therapy evaluation at earlier disease stages show significantly better eligibility qualification and treatment outcomes, yet most families only learn about options after exhausting multiple conventional approaches

• Evidence standards create accessibility challenges – Custom therapy development requires synthesis of peer-reviewed studies, case reports, mechanistic data, and trial information specific to individual genetic mutations, creating research barriers that modern platforms now address at scale

The Treatment Access Gap in Personalized Medicine

1. About 300 million people live with rare diseases globally, and roughly 95% of rare diseases have no FDA-approved treatment

The global rare disease burden affects approximately 300 million people worldwide, yet the vast majority of these conditions lack approved therapies. While supportive and symptomatic care may exist, roughly 95% of rare diseases have no FDA-approved treatment specifically targeting the underlying condition. This massive gap stems from traditional pharmaceutical economics that cannot support development for ultra-rare populations, forcing families to navigate fragmented research, manufacturing, and regulatory landscapes independently. Modern platforms addressing these operational challenges through coordinated support can dramatically expand access to personalized therapeutic approaches.

 Source: World Health Organization

2. Over 50,000 patients have received CAR-T therapies globally since 2017

Since initial regulatory approvals for CAR-T cellular therapies in 2017, more than 50,000 patients globally have accessed these personalized treatments, establishing proof of concept for scaled custom medicine delivery. This growing patient population demonstrates that personalized therapy development can transition from experimental one-offs to systematic programs when proper infrastructure exists. However, this number remains small compared to rare disease populations needing personalized approaches, revealing the urgent need for platforms that can coordinate geneticists, research labs, manufacturers, and regulators at scale. Source: PR Newswire

3. The referral-to-infusion timeline for CAR-T therapy creates critical eligibility windows

The referral-to-infusion timeline for CAR-T cellular therapy—from patient identification to actual therapeutic delivery—can span multiple weeks depending on manufacturing complexity and healthcare system infrastructure. For progressive rare diseases where patients deteriorate rapidly, this multi-week window creates critical risks where initially eligible candidates become ineligible due to declining health status. Traditional manual processes for literature review, feasibility assessment, manufacturer identification, and regulatory coordination consume substantial time within this timeline. Platforms that significantly accelerate these processes can preserve eligibility for patients whose disease progression would otherwise eliminate treatment options. Source: Experimental Hematology

Eligibility Assessment and Process Barriers

4. Studies show substantial attrition from referral to CAR-T infusion

Research on CAR-T cellular therapy indicates that a meaningful proportion of patients initially referred do not proceed to actual treatment delivery. This attrition occurs through multiple mechanisms: disease progression during evaluation and manufacturing periods, deteriorating organ function below safety thresholds, death before treatment availability, or discovery during detailed assessment that therapeutic mechanisms won't address the specific condition. The exact rate varies by product, indication, and center. Early referral when patients maintain better health status improves completion rates, yet most families only discover custom therapy possibilities after exhausting conventional options and experiencing significant disease progression. Source: JCO Oncology Practice

5. Manufacturing success rates for CAR-T exceed 95% once production begins

When CAR-T cellular therapy manufacturing actually commences, success rates in producing viable therapeutic products exceed 95%, meaning very few patients fail to receive therapy due to production issues once the process starts. This high manufacturing reliability indicates that production challenges represent minimal barriers compared to upstream eligibility determination and operational coordination obstacles. The real bottlenecks exist in identifying appropriate patients, completing eligibility assessments, coordinating multiple stakeholders, and securing regulatory approvals before manufacturing begins. Source: Blood

Clinical and Functional Eligibility Criteria

6. Performance status significantly impacts CAR-T eligibility and outcomes

Patients maintaining good functional performance status demonstrate measurably better response rates to CAR-T cellular therapies compared to those with diminished functional capacity. This correlation makes early referral critical, as progressive diseases systematically degrade performance status over time. Families waiting until conventional options are completely exhausted often discover their loved ones no longer meet functional eligibility thresholds for experimental approaches. Clear guidance on when to seek custom therapy evaluation—at diagnosis or first progression rather than after multiple treatment failures—can preserve eligibility windows that close as disease advances. Source: Blood Advances

7. In aggressive lymphoma CAR-T cohorts, bridging therapy is frequently required during manufacturing

Among patients qualifying for CAR-T therapy in aggressive lymphoma settings, bridging therapy during manufacturing periods is commonly needed to control disease progression while awaiting treatment production, though rates vary by product and disease kinetics. This creates delicate clinical management challenges where controlling advancing disease while preserving eligibility criteria demands sophisticated medical coordination. Platforms accelerating manufacturing timelines through optimized contract manufacturer matching and automated coordination reduce the duration patients must successfully navigate this precarious bridging period. Source: Journal for ImmunoTherapy of Cancer

8. Eligibility thresholds vary by product and center

Organ function requirements create important safety boundaries for personalized therapeutic eligibility. Many CAR-T programs require cardiac function with left ventricular ejection fraction ≥45-50% and adequate pulmonary reserve, though specific thresholds are product- and protocol-specific. These requirements exist for legitimate safety reasons, as experimental treatments carry toxicity risks requiring physiologic reserve. However, progressive rare diseases systematically degrade organ function over time, creating urgency for early evaluation before patients cross irreversible eligibility thresholds. Nome's AI-powered assessment platform helps families identify treatment windows while organ function still permits experimental therapy access. Source: Blood

Geographic and Infrastructure Access Barriers

9. Specialized center requirements limit geographic accessibility

Access to CAR-T cellular therapies remains concentrated at specialized centers with FACT (Foundation for the Accreditation of Cellular Therapy) certification, experienced multidisciplinary teams, and advanced toxicity management capabilities. These centers must demonstrate ICU capabilities, 24/7 expert availability, and robust monitoring protocols. Geographic concentration of these resources creates access barriers for patients in rural or underserved regions who must travel substantial distances for evaluation, treatment, and monitoring. Centers with higher volumes treating these complex therapies generally demonstrate better outcomes, yet this concentrates expertise at a limited number of institutions. Source: ISCT 

10. Treatment center volume correlates with patient outcomes in cellular therapies

Centers performing higher volumes of cellular therapy procedures demonstrate measurably superior patient outcomes compared to low-volume facilities, as documented in multi-center registry analyses. This volume-outcome relationship creates concentration of expertise at specialized institutions and highlights the importance of provider experience in managing complex eligibility assessments, manufacturing coordination, and toxicity protocols. However, this creates access bottlenecks where wait times at experienced centers can extend substantially while disease progresses. Source: Cytotherapy

Evidence Requirements and Clinical Decision-Making

11. Custom therapy eligibility determination requires synthesizing multiple evidence types

Personalized therapeutic feasibility assessment requires synthesizing peer-reviewed studies, patient registries, case reports, mechanistic data, and current trial information specific to individual genetic mutations. This evidence synthesis traditionally demands extensive manual literature review by research teams with expertise spanning genetics, drug development, and rare disease biology. The research burden creates substantial barriers for community providers lacking dedicated research staff and for families attempting independent investigation. Modern systems analyzing scientific literature and databases can fundamentally transform who can access comprehensive eligibility assessments based on timeline constraints. Source: ResearchGate

12. Adult CAR-T recipients show a median age in the sixth to seventh decade

Current data for adult CAR-T cellular therapy recipients shows median ages commonly in the 60-65 year range, with a meaningful proportion of patients 70 years or older successfully treated. This age distribution reflects both the diseases currently addressed by approved personalized approaches and evolving evidence about age-related eligibility considerations. However, rare genetic diseases disproportionately affect pediatric populations, with half of the 300 million rare disease patients being children. Expanding personalized therapeutics into rare genetic disease populations requires adapting eligibility frameworks, manufacturing processes, and delivery systems for pediatric patients whose needs differ substantially from adult oncology populations currently served. Source: Journal for ImmunoTherapy of Cancer

Economic and Coverage Considerations

13. CAR-T therapy costs range from approximately $373,000 to $475,000 per patient

Treatment costs for approved CAR-T cellular therapies vary by product and indication, with list prices ranging from approximately $373,000 to $475,000 per patient. These substantial costs create economic barriers even when scientific eligibility exists. Insurance coverage varies dramatically across payors, with some providing comprehensive coverage while others categorize experimental approaches as investigational and deny payment. Many families pursuing custom therapies for ultra-rare conditions must self-fund development entirely, as no existing coverage frameworks address genetic mutation-specific treatment creation. Nome's mission to drive costs down dramatically while maintaining quality aims to reach economic viability thresholds where payors will cover personalized approaches as standard care. Source: Healthline

14. The global CAR-T therapeutics market is projected to reach $30.25 billion by 2033

Market analysts project the CAR-T cellular therapy sector will grow from approximately $3.71 billion in 2023 to $30.25 billion by 2033. This growth trajectory indicates expanding commercial viability as operational efficiencies reduce costs and clinical evidence supports broader applications. However, market growth concentrated in commercially attractive indications may bypass ultra-rare populations unless platforms specifically addressing operational barriers to small patient numbers emerge. Scalable approaches treating thousands of individual rare diseases simultaneously represent the only economically sustainable path to universal personalized medicine access. Source: Vision Search Reports

15. Poor data quality generates substantial organizational costs annually

Organizations report that poor data quality creates significant annual costs through targeting incorrect information, wasted resources, and systematic opportunity losses. In rare disease contexts, inaccurate genetic databases, outdated literature, and fragmented patient registries compound these costs while delaying eligibility determinations for patients whose timelines cannot afford delays. Real-time data validation, comprehensive literature integration, and automated database reconciliation become essential for platforms serving progressive rare diseases where delays from poor data quality can eliminate treatment windows entirely. Source: Gartner

Technology Acceleration of Eligibility Processes

16. Modern platforms significantly accelerate feasibility assessment timelines

Traditional custom therapy feasibility assessment requiring extensive manual literature review, manufacturer identification, and regulatory pathway analysis can now be significantly accelerated through advanced technology platforms. This acceleration directly expands eligible populations by preserving treatment windows for progressive diseases where patients deteriorate during lengthy manual evaluation periods. Nome's platform analyzes scientific papers and databases on genetic mutations, delivering Summary Reports that score feasibility and outline next steps before disease progression eliminates options that existed at referral. Source: Nome

17. Hundreds of active clinical trials expand eligibility beyond approved indications

Hundreds of active clinical trials currently investigate personalized therapeutic approaches across diverse diseases, expanding eligibility substantially beyond approved indications. These trials provide access to investigational therapies for patients with conditions not yet covered by regulatory approvals or seeking intervention at earlier disease stages. However, identifying relevant trials matching specific genetic mutations and eligibility criteria requires sophisticated matching capabilities processing thousands of inclusion/exclusion criteria across multiple databases. Platforms automating trial matching and continuously monitoring new opportunities ensure patients access all possible eligibility pathways. Source: Telemedicine Technologies

18. Five-year ZUMA-1 data shows 42% overall survival in large B-cell lymphoma

Long-term follow-up data from the pivotal ZUMA-1 study of axicabtagene ciloleucel (axi-cel) CAR-T therapy in relapsed/refractory large B-cell lymphoma demonstrates 42% overall survival at five years, with many patients achieving durable remission. These outcomes substantially exceed historical expectations with conventional approaches and validate the personalized medicine paradigm when appropriate patient-therapy matching occurs. However, achieving optimal matching requires comprehensive genetic analysis, mechanistic understanding, and evidence synthesis beyond most community providers' research capacity. Platforms democratizing access to this matching expertise expand the population achieving these superior outcomes. Source: Blood

Coordination and Operational Success Factors

19. Systematic coordination approaches improve rare disease treatment access

Custom therapy development inherently requires coordinating geneticists, research labs, contract manufacturers, regulatory bodies, and healthcare providers—a complexity that defeats most families attempting independent navigation. Systematic approaches that address operational barriers through coordinated support can substantially improve outcomes compared to fragmented efforts. Nome's Operating System for Personalized Therapeutic automatically coordinates these complex pieces, removing operational barriers that prevent scientifically eligible patients from accessing treatments and transforming custom therapy from exceptional family-driven efforts into scalable programs. Source: Nome

20. Evidence-based eligibility scoring enables informed decision-making

Comprehensive eligibility assessment goes beyond simple literature searches to provide mechanism-level rationale with citations, feasibility scoring, regulatory pathway guidance, manufacturing partner identification, and actionable next steps. This complete assessment enables families and providers to make informed decisions about pursuing personalized therapeutic development with realistic understanding of timelines, requirements, and success probabilities. Nome's HIPAA-compliant platform accepts genetic test results and delivers expert-reviewed evaluations scoring whether personalized therapy could change disease trajectory for specific mutations. Source: Nome

Frequently Asked Questions

What percentage of rare disease patients currently have treatment options?

Approximately 95% of rare diseases lack FDA-approved treatments. About 300 million people live with rare diseases globally, and while supportive or symptomatic care may be available for some conditions, the vast majority have no approved therapy specifically targeting the underlying disease. This massive treatment gap creates an urgent need for personalized therapeutic platforms that can address ultra-rare conditions outside traditional pharmaceutical economics.

Do I need a confirmed genetic diagnosis to qualify for custom therapy evaluation?

Yes, a confirmed genetic diagnosis through comprehensive sequencing is essential for custom therapy eligibility assessment. The specific genetic variant determines therapeutic mechanism selection, evidence synthesis, and feasibility scoring. Nome's HIPAA-compliant platform accepts genetic test results and delivers expert-reviewed evaluations scoring whether personalized therapy could change disease trajectory for that specific mutation, providing families with clear guidance on potential next steps.

How does performance status affect eligibility for personalized therapies?

Functional performance status significantly impacts both eligibility and treatment outcomes. Patients maintaining better functional capacity at the time of evaluation demonstrate superior response rates compared to those with advanced disease and diminished function. This makes early referral critical, as progressive rare diseases systematically degrade performance over time. Families seeking evaluation at diagnosis or first progression—rather than after exhausting all conventional options—preserve eligibility windows that may close as disease advances.

Are personalized rare disease therapies covered by insurance?

Coverage varies substantially by payor, therapeutic approach, and specific circumstances. Some experimental personalized therapies may qualify for compassionate use or clinical trial coverage, while others require family funding of development costs that can range from $373,000 to $475,000 or more. Nome's mission includes driving costs down dramatically to reach economic viability thresholds where payors will cover personalized approaches as standard care, but current coverage remains inconsistent and often requires extensive appeals processes.

Why is geographic location important for accessing custom therapies?

Access to advanced personalized therapies like CAR-T is concentrated at specialized centers with specific accreditation (such as FACT certification), experienced multidisciplinary teams, ICU capabilities, and proven toxicity management protocols. These requirements ensure patient safety but create geographic barriers for patients in rural or underserved regions who must travel substantial distances for evaluation, treatment, and monitoring. Platforms that extend expertise through coordinated support can help bridge these geographic gaps.