Genetic Diseases in Children of U.S. Military Service Members

Data Strategies Current Therapies, and Leveraging Federal Resources for Global Impact

By MAJ Justin T. Albrecht

July 29, 2026 | Pulse of Army Medicine, 2026 E-Edition

Read Time: < 14 mins

INTRODUCTION

Genetic diseases, encompassing monogenic disorders such as cystic fibrosis, neurodevelopmental conditions like autism spectrum disorder (ASD), and complex multifactorial conditions, affect approximately 1 in 10 births in the United States. For millions of active-duty service members and their dependents, the demands of military life—frequent permanent change of station (PCS) moves, prolonged deployments, and potential exposures to environmental hazards at military installations—exacerbate challenges in diagnosing and managing genetic conditions. Relocations disrupt continuity of care, especially for rare diseases that require specialized providers. In addition, exposure to toxins such as PFAS (perand polyfluoroalkyl substances) at bases may compound risks for multifactorial disorders. The DoW’s Exceptional Family Member Program (EFMP) aims to support families with special needs but lacks emphasis on genetic diseases, resulting in gaps in data collection and access to therapy.

With 9.6 million beneficiaries, DoW’s TRICARE presents a unique opportunity to tackle these diagnostic challenges through centralized data aggregation and care coordination. By examining genetic diseases within this population, the DoW can enhance military readiness by alleviating family stressors that impact service member retention and performance. Moreover, insights from military families could benefit the 30 million Americans and 300 million people globally with rare diseases, as defined by the National Institutes of Health (NIH) and World Health Organization (WHO).

This paper proposes a three-pronged strategy: (1) establishing a TRICARE-based databank for natural history studies, (2) assessing and enhancing access to cutting-edge therapies, and (3) leveraging federal resources through a genetic health initiative to promote innovation. Addressing untreated genetic diseases not only preserves DoW resources but also positions the United States as a leader in global health equity.

SECTION 1: TRICARE-BASED DATABANK TO SUPPORT ALL NATURAL HISTORY STUDIES

Natural history studies track disease progression over time. These studies are essential for developing therapies and securing regulatory approval. For example, longitudinal data on spinal muscular atrophy (SMA) underpinned the development of Zolgensma, a $2.1 million gene therapy. While similar studies for SYNGAP1-related disorders characterized by neurodevelopmental disability, intellectual disability and epilepsy—could unlock targeted trials. Currently, no DoW-specific database captures genetic disease prevalence or progression among military dependents. TRICARE relies on International Classification of Diseases (ICD-10) codes, such as Q99.9 for congenital anomalies, which provide diagnostic snapshots. However, the ICD-10 lacks granular data on symptoms, treatment outcomes, or military-specific stressors like relocations. Civilian registries, such as those maintained by the National Organization for Rare Disorders (NORD), exclude military contexts. Therefore, these registries cannot capture how unique military stressors affect disease progression in military families.

A TRICARE-based, de-identified genetic disease databank, accessible via secure application programming interfaces (APIs), could revolutionize research by providing longitudinal data to biotech firms, academic institutions, and regulators. This databank would streamline clinical trial designs, validate biomarkers, and support FDA orphan drug designations. The databank could also reduce private-sector R&D costs by an estimated 20 to 30 percent by minimizing duplicative studies (based on industry benchmarks). For example, natural history data could accelerate approvals for therapies targeting ultra-rare conditions like SYNGAP1, which affects fewer than 1 in 100,000 children. Governance would involve a DoW-led oversight committee, industry representatives, and patient advocates, ensuring HIPAA compliance and public trust through transparent protocols.

PROPOSED METHODS FOR IMPLEMENTATION:

The DoW must leverage its internal health infrastructure and external medical partnerships to establish a genetic disease databank. The following proposed methods methodology outlines a roadmap for implementation:

• TRICARE claims analysis

• Family surveys

• Biobanking expansion

• Public private collaborations

• Digital tools with artificial intelligence (AI) integration

TRICARE claims analysis utilize machine learning to analyze claims data, identifying diagnoses such as cystic fibrosis (F508del mutations) or SYNGAP1 (haploinsufficiency) and correlating them with outcomes like hospitalization rates. The estimated cost for initial analytics infrastructure is approximately $500,000.

Implementing anonymous family surveys (per Title 10, Section 1782) will collect family-reported data on symptoms, care barriers, and the impacts of relocation. This effort involves collaboration with the Defense Health Agency to distribute the surveys, aiming to reach 10,000 families each year. The estimated cost for survey design and analysis is approximately $300,000.

Biobanking expansion involves leveraging the Million Veteran Program of the Department of Veterans Affairs. This program has sequenced 900,000 genomes by incorporate pediatric samples from military dependents with parental consent. Biobanking expansion entails partnering with DoW medical centers to gather saliva or blood samples, aiming for a repository of 5,000 samples during the first year of implementation. The estimated cost for sample processing and storage is $1 million.

Public-private collaborations require partnering with the NIH’s Undiagnosed Diseases Network and nonprofits like the SynGAP Research Fund (SRF). SRF’s digital registry, powered by Citizen Health, tracks SYNGAP1 data from over 1,000 global patients and offers a model for military integration. SRF could co-fund app development (approximately $500,000) and align TRICARE data with global standards. This alignment ensures the data is immediately usable for biotech firms developing targeted gene therapies, such as antisense oligonucleotides, for SYNGAP1. This partnership could reduce pilot costs by 25 percent, freeing up funds for additional pipeline therapies.

Implementing digital tools with AI integration involves developing a HIPAA-compliant mobile app that allows families to log symptoms, medications, and environmental exposures in real time. This effort will incorporate AI-driven analytics to recognize disease patterns and forecast progression, thereby enhancing trial recruitment. The estimated cost for app development and AI modeling is approximately $700,000.

PILOT PROGRAM

A pilot program aimed at reaching 1,000 families across five major bases (e.g., Fort Bragg, Naval Station Norfolk, Joint Base San Antonio, Camp Pendleton, and Eglin Air Force Base) could validate the implementation methods. The estimated cost for this pilot program is approximately $2.5 million during the first 18 months. By pursuing SRF cost-sharing initiatives, DoW can reduce the cost burden to $1.8 million.

This pilot program would prioritize conditions such as SMA, SYNGAP1, and cystic fibrosis, leveraging existing TRICARE data to establish baselines. The program will adhere to ethical safeguards to protect privacy, including Institutional Review Board (IRB) oversight and compliance with DoW Instruction 3216.02 (Protection of Human Subjects and Adherence to Ethical Standards in DoW-Conducted and -Supported Research).

Military families face unique obstacles due to TRICARE policies and geographic limitations. Approved therapies, such as Zolgensma (costing $2.1 million) for SMA and Luxturna (costing $850,000) for retinal dystrophy, have revolutionized patient outcomes but necessitate complex administration, which is often unavailable at rural or overseas bases. The limited access to specialists outside the continental United States is another geographic limitation.

TRICARE’s preapproval process for high-cost therapies can take 60 to 90 days. These authorization delays disrupt treatment for progressive diseases such as SMA.

CHALLENGES AND MITIGATIONS

Executing a large-scale, cross-agency genetic databank involves inherent challenges such as data integration, family participation, and cost overruns. Therefore, the DoW must adopt mitigation strategies to ensure initiatives remain sustainable (Table 1).

This databank has the potential to generate 10,000 longitudinal records over five years, facilitating trials for more than 50 rare diseases. This databank initiative can also establish the DoW as a data hub for global research.

Families stationed abroad are frequently ineligible for experimental trials in the United States because of travel expenses, limiting their access to cutting-edge options. Emerging CRISPR-based trials for sickle cell disease present promising advancements. However, the trials remain inaccessible under TRICARE, as it excludes coverage for experimental treatments (Table 2). For conditions like SYNGAP1-related disorders, no targeted therapies are available, resulting in families relying on noncurative interventions, including behavioral therapy or anti-seizure medications. These interventions also may be disrupted by PCS moves.

SECTION 2: ENHANCING ACCESS TO NOVEL THERAPIES

Access to therapies for genetic diseases varies significantly, ranging from transformative gene therapies to supportive care. However, military families encounter the following barriers to access:

• Geographic disparities

• Authorization delays

• Trial exclusion

• Information gaps

The absence of centralized resources creates information gaps. This barrier leaves families unaware of therapies such as Trikafta or opportunities for clinical trials.

EMERGING THERAPIES AND DOW OPPORTUNITIES

Beyond gene therapies, small-molecule drugs such as Trikafta for cystic fibrosis and antisense oligonucleotides for conditions like SYNGAP1 are making progress. NIH’s Bespoke Gene Therapy Consortium aims to develop treatments for ten rare diseases by 2026. However, small patient populations and high costs (ranging from $1 to $ 3 million per trial) continue to pose challenges. The DoW’s Congressionally Directed Medical Research Programs (CDMRP), which had $1.5 billion allocated in fiscal year 2024, could focus on pediatric genetics to complement civilian efforts. Globally, initiatives like the United Kingdom’s Genomics England, which has sequenced 100,000 genomes, illustrate how centralized data can expedite therapy development and provide a model for TRICARE.

The following recommendations will enhance access to novel therapies:

• Telehealth expansion: Allocating $5 million for virtual consultations with geneticists to address geographic disparities.

• Trial access pilot: Allocating $3 million to subsidize travel for families to trial sites in the United States, prioritizing ultra-rare conditions.

• Centralized portal: Developing a TRICARE portal (approximately $1 million) to inform families about therapies and trials, thereby reducing delays.

• CDMRP investment: Advocating for $10 million in CDMRP funds (in the second year of implementation) for SYNGAP1 and SMA trials by leveraging military data.

By tackling access barriers, the DoW could ensure equitable access for millions of military dependents and establish a global standard that surpasses disjointed civilian systems.

SECTION 3: CREATION OF A GENETIC HEALTH INITIATIVE TO PROMOTE INNOVATION

Military families wield significant advocacy power, as evidenced by the $6 billion Camp Lejeune Justice Act settlement for victims of toxic exposure. With the DoW’s $50 billion health budget for fiscal year 2025 and TRICARE’s $20 billion allocation, there are untapped resources available to address genetic diseases. A genetic health initiative could harness this potential, framing genetic diseases as a readiness issue that affects recruitment, retention, and operational effectiveness. Ensuring coordinated, high-quality care for military dependents minimizes early service separations due to family hardships, preserving the DoW’s retention and recruitment initiatives.

PROPOSED STRATEGY

The following milestones detail a proposed genetic health initiative strategy:

1. Advocacy campaign ($2.5 million, Year 1).

   • Collaborate with the National Military Family Association (NMFA) and SRF to engage families, highlighting the experiences of children with SMA or SYNGAP1.

   • Present genetic diseases as a priority issue for the Senate Armed Services Committee (SASC), focusing on senate advocates.

   • Organize a DoW Family Health Summit to align stakeholders, including NIH and biotech companies.

   • Initiate claim analysis.

2. DoW-NIH partnership ($10 million pilot program, Year 1-2).

   • Establish a collaborative pilot program with the NIH’s National Center for Advancing Translational Sciences (NCATS) to integrate TRICARE data with civilian registries.

   • Utilize the pilot program to showcase cost savings, forecasting a $50 million decrease in TRICARE expenses over a decade by preventing disease progression.

   • Secure $3 million in NIH co-funding to offset DoW costs.

3. Legislative push ($50 million, Year 2-3).

   • Advocate for a $50 million line item in the National Defense Authorization Act (NDAA), modeled after CDMRP’s autism funding of $15 million in 2024.

   • Engage with the U.S. Congressional Rare Disease Caucus to foster bipartisan support.

   • Propose the establishment of a genetic health task force within the Defense Health Agency to manage funds.

   • Expand the pilot program to include biobanking.

4. International collaboration ($5 million, Year 3).

   • Collaborate with NATO allies (e.g., United Kingdom, Canada) to exchange genetic data, utilizing TRICARE’s databank for multinational trials.

   • Align with the WHO’s rare disease framework to extend therapies to 300 million patients worldwide, enhancing U.S. soft power.

   • Scale the databank to 10,000 records and finance the first DoW-led trial.

BUDGET JUSTIFICATION

A $2.1 million dose of Zolgensma for one child surpasses the cost of a $10 million pilot benefiting thousands. Scaling up to $50 million could support trials for 20 rare diseases, potentially saving $500 million in long-term TRICARE costs by reducing hospitalizations. Competition with weapons programs may challenge funding, but framing for readiness and bipartisan support helps mitigate risks.

THOUGHTS ON MOVING FORWARD

By combining a centralized TRICARE databank with enhanced access to therapy and a strategic funding model, this proposal for a genetic health initiative addresses critical gaps in managing genetic diseases among military dependents. By providing longitudinal data, the databank empowers biotech firms to design efficient trials, secure FDA orphan designations, and reduce R&D costs by $100 million to $200 million annually across the rare disease sector. Partnerships with organizations like SRF enhance expertise, while TRICARE’s portal ensures families have access to therapies like Zolgensma or Trikafta without delay. Economically, healthy children reduce TRICARE’s $2 billion annual pediatric budget and bolster retention, saving $1 billion in recruitment costs over a decade.

Ethical safeguards are built directly into the program’s design. The initiative protects family privacy using HIPAA-compliant APIs for secure data sharing. In addition, IRB oversight ensures all research complies with federal standards to prevent data misuse. Challenges include securing initial funding within DoW’s $850 billion budget and maintaining family engagement during relocations. These challenges can be mitigated through phased pilots and telehealth incentives. In the long term, the databank could integrate AI to predict disease trajectories, thereby supporting precision medicine for over 50 rare diseases.

Globally, the DoW’s leadership could inspire systems like the EU’s Orphanet, benefiting over 300 million patients. NATO collaboration could standardize data-sharing, accelerating therapies such as antisense oligonucleotides for SYNGAP1. This initiative transforms challenges into opportunities, enhancing readiness and delivering hope. The DoW, Congress, and biotech stakeholders must act swiftly. Fund the $10 million pilot advocate for NDAA support and establish the databank to lead globally. Military families deserve solutions, and the world awaits innovation.

REFERENCES:

Statement on the Use of Generative AI
The author used Grammarly, a large language model, to assist writing this article throughout the drafting and iterative revision process. All actions taken by the AI were under the author’s direct guidance and editorial control. The final text, including all arguments, structural decisions, and conclusions, represents the author’s own work and intellectual property. The AI served as a tool to accelerate research, test arguments, and enhance the clarity and depth of the author’s original ideas