What Is FDA Expanded Access?
FDA expanded access — also referred to as "compassionate use" — is a regulatory pathway that allows patients with serious or life-threatening conditions to receive investigational medical products (drugs, biologics, or devices) that have not yet been approved by the FDA. The program exists for patients who have no comparable or satisfactory alternative treatment options and who are unable to participate in clinical trials.
The legal basis for expanded access is found in 21 CFR Part 312, Subpart I (Sections 312.300–312.320), and Section 561 of the Federal Food, Drug, and Cosmetic Act. The FDA has maintained this pathway for decades, and it is one of the most permissive access programs of any regulatory agency worldwide.
Three Types of Expanded Access
| Type | Patients | Relevance to Cancer Vaccines |
|---|---|---|
| Individual Patient | 1 patient at a time | Most relevant. Each personalized vaccine is manufactured for a single patient. This is the pathway currently used by the Jaime Leandro Foundation and academic centers. |
| Intermediate-Size | Smaller groups | May become relevant as programs scale. Allows a coordinated protocol for multiple patients using similar (but individually manufactured) products. |
| Treatment IND | Widespread use | Typically used when a drug is further along in the approval process. Not yet applicable to personalized cancer vaccines. |
The FDA approves the vast majority of expanded access requests. According to FDA submission data, more than 99% of individual patient expanded access applications are authorized, typically within 30 days. Emergency requests can be processed within 24 hours.
Expanded Access vs. Right to Try
The federal Right to Try Act (2018) is a separate pathway that allows terminally ill patients to access investigational drugs without FDA pre-authorization. However, Right to Try has a critical structural limitation for personalized cancer vaccines: it requires the drug to have completed a Phase I clinical trial. Because each personalized neoantigen vaccine is manufactured uniquely for a single patient, "the drug" has never been tested on anyone before. This makes Right to Try inapplicable in most cases.
Some states are working to address this gap. New Hampshire (HB 1292, HB 1735) and Texas (SB 984) have introduced legislation in 2026 to expand right-to-try laws to explicitly cover "individualized investigational treatments" including neoantigen vaccines. Until such laws pass, expanded access remains the only established federal pathway.
How Expanded Access Works for Personalized Cancer Vaccines
The process of obtaining a personalized cancer vaccine through expanded access involves six major steps, spanning approximately 4 to 5 months from biopsy to first dose. Here is what each step involves, who performs it, the typical timeline, and what can go wrong.
Tumor Biopsy and Tissue Preservation
What happens: A surgeon removes tumor tissue (either a dedicated biopsy or tissue from a prior surgery). Both tumor tissue and a healthy tissue sample (typically blood) are needed for comparison.
Who does it: The patient's surgical oncologist or interventional radiologist. The tissue must be handled according to specific protocols — flash-frozen or preserved in a validated medium to maintain DNA and RNA integrity.
Timeline: Depends on the patient's surgical schedule. Tissue procurement itself takes hours; preparation for shipping may take 1–3 days.
Cost: Varies widely. If tissue is available from a prior surgery, there may be minimal additional cost. A new biopsy procedure can range from $1,000 to $5,000+ depending on the location and method.
What can go wrong: Insufficient tumor material (the sample is too small for reliable sequencing), degraded tissue (improper preservation), or the tumor biopsy site is inaccessible. If archived tissue from a prior surgery is used, it must be evaluated for DNA quality, which occasionally fails.
Whole Exome Sequencing (WES)
What happens: A specialized sequencing laboratory performs whole exome sequencing on both the tumor DNA and the patient's normal (germline) DNA. This comparison identifies somatic mutations — genetic changes unique to the tumor. Many labs also perform RNA transcriptome sequencing to determine which mutated genes are actually being expressed by the tumor.
Who does it: Sequencing laboratories such as Tempus, Foundation Medicine, or CeGaT (Germany). CeGaT's CancerNeo product is notable because it bundles sequencing, HLA typing, and neoantigen prediction into a single package.
Timeline: Approximately 2–4 weeks for standard turnaround, though some labs offer expedited processing.
Cost: $1,100–$2,800 for WES alone (self-pay). CeGaT's CancerNeo package, which includes vaccine design, costs approximately $11,000. Foundation Medicine's FoundationOne CDx may be covered by Medicare for solid tumors but sequences only 324 genes (a targeted panel, not a full exome), which may be insufficient for comprehensive vaccine design.
What can go wrong: Low tumor purity in the sample (too much normal tissue mixed in), low sequencing depth, or tumors with very few somatic mutations (low tumor mutational burden), which may not yield enough neoantigen targets for a vaccine.
Neoantigen Identification and Vaccine Design
What happens: Bioinformatics algorithms analyze the sequencing data to identify which somatic mutations produce "neoantigens" — abnormal proteins that can be recognized by the patient's immune system. The algorithms consider factors including HLA type (which determines which peptides can be presented to T-cells), binding affinity, and expression level. The output is a ranked list of the most immunogenic targets, from which 10–20 are typically selected for inclusion in the vaccine.
Who does it: Bioinformatics companies such as NoRD Bio (Cambridge, MA) using pipelines like pVAC-Seq, or CeGaT as part of their CancerNeo package. Academic centers like Washington University (St. Louis) and Mount Sinai also have neoantigen prediction capabilities.
Timeline: Approximately 4–6 weeks.
Cost: Typically included in the overall vaccine design fee. As part of the JLF protocol, neoantigen identification and vaccine design costs approximately $23,000.
What can go wrong: The tumor has too few somatic mutations for effective targeting (common in some pediatric cancers and certain tumor types). The predicted neoantigens may not provoke a strong immune response in practice, despite algorithmic predictions. HLA typing errors can lead to the selection of peptides the patient's immune system cannot present effectively.
Vaccine Manufacturing (GMP Synthesis)
What happens: A contract manufacturing organization (CMO) synthesizes the personalized vaccine under Good Manufacturing Practice (GMP) conditions. For peptide vaccines, this involves synthesizing 10–20 long peptides (typically 20–30 amino acids each), performing purity and identity testing, vialing the product under sterile conditions, and conducting release testing (including a 14-day sterility test that is itself a major bottleneck). For mRNA vaccines (as used by Moderna and BioNTech), the process involves encoding the selected neoantigens into a single mRNA construct, encapsulating it in lipid nanoparticles, and performing analogous quality testing.
Who does it: GMP-certified peptide CMOs. The Jaime Leandro Foundation currently uses CSBio (San Jose, CA). Other capable manufacturers include GenScript, AnaSpec (Fremont, CA), Biosynth (Netherlands), and CPC Scientific (Sunnyvale, CA).
Timeline: Approximately 12–14 weeks. This is typically the longest step in the process and the primary bottleneck.
Cost: Manufacturing and vialing costs approximately $53,500 under the current JLF protocol, plus approximately $17,210 for shipping and immune monitoring materials.
What can go wrong: Manufacturing delays (batch failures, contamination, equipment issues). Certain peptide sequences are difficult to synthesize and may require multiple attempts. Each patient's vaccine is a unique batch, so there is no economy of scale. Critically, 15% of early JLF patients (4 of 26) died before their vaccine was ready, according to STAT News (2023). The 4–5 month timeline can be too slow for patients with rapidly progressing cancers.
FDA Filing and Vaccine Administration
What happens: The treating physician files an individual patient IND application (FDA Form 3926) and obtains Institutional Review Board (IRB) approval for the single-patient protocol. Once the FDA authorizes the expanded access request (typically within 30 days), the vaccine is administered as a series of approximately 7 injections over several months. The vaccine may be administered alone or in combination with a checkpoint inhibitor such as pembrolizumab (Keytruda) or nivolumab (Opdivo), depending on the clinical protocol.
Who does it: The treating physician (typically an oncologist experienced in immunotherapy) files the IND and administers the vaccine. Current JLF administration sites include San Francisco, Los Angeles, St. Louis, Miami, and Dallas. Academic medical centers may also serve as administration sites.
Timeline: FDA authorization typically takes up to 30 days. The injection series itself spans several months.
Cost: Physician administration fees range from $7,000 to $25,000, depending on the physician, facility, and geographic location.
What can go wrong: FDA authorization delays (rare, given the 99%+ approval rate). IRB review may take additional time at some institutions. The patient's health may deteriorate during the administration period, requiring dose modifications or treatment suspension. Immune-related adverse events may occur, particularly when the vaccine is combined with checkpoint inhibitors.
Immune Monitoring and Follow-Up
What happens: After vaccination, the patient undergoes regular monitoring to assess whether the immune system is responding. This includes CT scans or other imaging to track tumor size, blood tests to measure T-cell receptor (TCR) activity against the vaccine targets, and standard oncology follow-up. The physician reports adverse events to the FDA as required under the expanded access IND.
Who does it: The treating oncologist and their clinical team. Some specialized monitoring (such as TCR sequencing) may be performed by external laboratories.
Timeline: Ongoing, typically for at least 12–24 months following the final vaccine dose.
Cost: Standard oncology follow-up costs, typically covered by insurance. Specialized immune monitoring may incur additional out-of-pocket costs.
What can go wrong: The vaccine may fail to produce a detectable immune response. Even when immune responses are measurable in blood tests, the tumor may not regress clinically. The disease may progress despite vaccination, requiring the patient to transition to alternative treatments.
It is important to emphasize that neoantigen vaccine technology is not experimental in the sense that it is purely theoretical. Multiple Phase 2 and Phase 3 clinical trials have generated clinical evidence, and the underlying science of tumor-specific immune targeting has been extensively validated. What remains investigational is the use of this technology for individual patients outside of controlled clinical trial settings.
Who Qualifies?
Eligibility for a personalized cancer vaccine through expanded access is determined on a case-by-case basis. However, existing programs and the FDA's expanded access framework establish general criteria that most patients will need to meet.
General Clinical Criteria
- Diagnosis with no curative therapies available — The patient has exhausted, is ineligible for, or is unresponsive to standard-of-care treatments.
- Life expectancy of 12+ months — The patient must be expected to survive long enough to complete the vaccine manufacturing and administration process (4–5 months minimum).
- 5-year overall survival less than 50% — The severity threshold that qualifies the condition as "serious or life-threatening" under FDA guidelines.
- Adequate organ function — Kidney, liver, and bone marrow function sufficient to tolerate immunotherapy.
- ECOG performance status 0–2 — The patient is able to carry out self-care and at least light daily activities. Patients who are bedridden (ECOG 3–4) generally do not qualify.
- No active autoimmune conditions requiring immunosuppressive therapy — Immunosuppressants would directly counteract the vaccine's mechanism of action.
Tumor Types with Strongest Evidence
While personalized neoantigen vaccines are theoretically applicable to any solid tumor with sufficient somatic mutations, the strongest clinical evidence as of March 2026 exists for:
| Tumor Type | Evidence Level | Key Data |
|---|---|---|
| Melanoma | Phase 3 (ongoing) | Moderna/Merck V940 + pembrolizumab: 49% reduced recurrence (Phase 2b). Phase 3 enrolling 1,089 patients. |
| Non-Small Cell Lung Cancer (NSCLC) | Phase 2 (enrolling) | Multiple programs. BioNTech iNeST has active studies. Moderna/Merck expanding beyond melanoma. |
| Head & Neck Squamous Cell Carcinoma | Phase 2 | Transgene TG4050 and BioNTech programs active. |
| Colorectal (MSI-H) | Phase 1/2 | Microsatellite instability-high tumors have high mutational burden, making them strong candidates for neoantigen targeting. |
| Pancreatic Cancer | Phase 1 | BioNTech: 8/16 patients showed T-cell response, 50% recurrence-free at 18 months. |
Who Does NOT Typically Qualify
- Patients with rapidly progressing disease and very short life expectancy — The 4–5 month manufacturing timeline may not be compatible with aggressive cancers.
- Patients on high-dose immunosuppressive therapy — The vaccine requires an active immune system to work.
- Tumors with very low mutational burden — Some cancers (certain pediatric tumors, some prostate cancers) produce very few somatic mutations, yielding insufficient neoantigen targets.
- Patients who are bedridden or unable to care for themselves — ECOG performance status 3–4 generally disqualifies patients.
If you do not qualify for expanded access, alternatives may include enrolling in a clinical trial, exploring other immunotherapy options (checkpoint inhibitors, CAR-T, TIL therapy), or discussing off-label use of approved immunotherapies with your oncologist.
The Key Players
Organizations Facilitating Expanded Access
Jaime Leandro Foundation (JLF)
The largest dedicated facilitator of personalized neoantigen vaccines through expanded access in the United States. JLF is a nonprofit organization that coordinates the entire process from sequencing through vaccine administration. As of August 2025, JLF had completed protocols for approximately 48 patients with an additional 24 in queue. By 2024, JLF reported $4.76 million in revenue, indicating rapid growth.
Administration sites: San Francisco, Los Angeles, St. Louis, Miami, Dallas
Website: jaimeleandrofoundation.org
Academic Medical Centers
Several academic institutions run their own neoantigen vaccine programs, primarily through clinical trials but occasionally through expanded access:
- Mount Sinai — Developed PGV001, a multi-peptide personalized vaccine. Phase 1 results published in Cancer Discovery.
- Dana-Farber Cancer Institute — Operates the Center for Cancer Vaccines with multiple active programs.
- MD Anderson Cancer Center — Involved in various neoantigen vaccine clinical trials.
- Washington University (St. Louis) — Provides vaccine design support in collaboration with JLF.
Sequencing and Manufacturing Partners
| Company | Role | Key Detail |
|---|---|---|
| Tempus | Sequencing | Full exome + RNA transcriptome. Physician orders via Tempus Hub. |
| CeGaT (Germany) | Sequencing + Vaccine Design | CancerNeo: end-to-end WES + HLA typing + neoantigen prediction. ~$11,000. Patients can order directly. |
| Foundation Medicine | Sequencing (targeted panel) | FoundationOne CDx: 324 genes. Medicare-covered for solid tumors. Not full exome. |
| CSBio | Vaccine Manufacturing | JLF's current GMP manufacturing partner. San Jose, CA. |
| GenScript | Vaccine Manufacturing | End-to-end: design through GMP production. China + US operations. |
| AnaSpec | Vaccine Manufacturing | Expedited turnaround. ISO 7 cleanrooms. Fremont, CA. |
Active Clinical Trials (Not Expanded Access, but Related)
While expanded access operates outside of clinical trials, the trial landscape is important context for understanding the maturity of the science:
| Trial | Phase | Indication | Key Detail |
|---|---|---|---|
| Moderna/Merck V940 + Keytruda (INTerpath-001) | Phase 3 | Adjuvant melanoma | 1,089 patients. Primary completion Oct 2029. NCT05933577 |
| BioNTech BNT122 (iNeST) | Phase 2 | Multiple solid tumors | Individualized mRNA vaccine. Active at multiple sites globally. |
| Mount Sinai PGV001 | Phase 1 | Various solid tumors | 6/13 patients survived 5 years, 3 tumor-free. Published 2025. |
| Moderna/Merck KEYNOTE-942 | Phase 2b | Adjuvant melanoma | Completed. 49% reduced recurrence. Led to Phase 3. NCT03897881 |
How Much Does It Cost?
The total cost of a personalized neoantigen cancer vaccine through expanded access is typically $100,000 to $119,000. This figure is based on current pricing from the Jaime Leandro Foundation, the largest existing program.
| Component | Estimated Cost |
|---|---|
| Neoantigen identification & vaccine design | $23,000 |
| GMP vaccine manufacturing & vialing | $53,500 |
| Shipping & immune monitoring materials | $17,210 |
| Physician administration fees (7 doses) | $7,000 – $25,000 |
| Total | $100,710 – $118,710 |
Source: Jaime Leandro Foundation FAQ. Costs are subject to change and may vary by program.
Insurance Coverage
As of 2026, no insurance companies cover personalized neoantigen cancer vaccines. Because these vaccines have not received FDA approval, there are no billing codes (CPT codes) for reimbursement. Some components of the process may be partially covered — for example, a tumor biopsy performed for diagnostic purposes, or genomic sequencing through Foundation Medicine (which has Medicare coverage for solid tumors) — but the vaccine manufacturing and administration costs are entirely out-of-pocket.
Cost Recovery Is Legal
Under 21 CFR 312.8, the sponsor of an expanded access IND may charge patients for the direct costs of providing the investigational drug, with prior FDA written authorization. This is how JLF and similar programs are able to charge patients — they are recovering the actual costs of manufacturing, not generating profit on the drug itself.
Financial Assistance Options
- Home equity loans or HELOCs — For homeowners, tapping home equity may provide the lowest interest rate for large medical expenses.
- Retirement account withdrawals — IRS allows penalty-free early withdrawal from 401(k)/IRA for qualified medical expenses exceeding 7.5% of AGI.
- Crowdfunding — Platforms like GoFundMe are increasingly used for experimental cancer treatment costs.
- Cancer-specific financial assistance programs — Organizations like the Patient Advocate Foundation and CancerCare may provide grants for treatment-related expenses (though typically not for investigational treatments directly).
- Clinical trial enrollment — If eligible, clinical trials provide the vaccine at no cost to the patient. The Moderna/Merck Phase 3 trial (NCT05933577) and BioNTech trials are actively recruiting.
The Legal Framework
The legal and regulatory infrastructure for accessing personalized cancer vaccines through expanded access is well-established, though complex. Here are the key laws and regulations governing this space.
Federal Laws and Regulations
Section 561 of the FD&C Act
Establishes the statutory authority for expanded access to investigational drugs for individual patients. This is the foundational law that enables the entire expanded access pathway.
21 CFR 312.310 — Individual Patient IND
The specific regulation governing individual patient expanded access. Requires the treating physician to determine that the patient has a serious or life-threatening condition, that no comparable or satisfactory alternative therapy is available, and that the potential benefit justifies the potential risks. The physician files FDA Form 3926.
21 CFR 312.8 — Charging for Investigational Drugs
Permits sponsors to charge patients for the direct costs of providing investigational drugs under expanded access, with prior FDA written authorization. For individual patient access, only direct costs (manufacturing, materials, labor, shipping) may be recovered. The FDA's February 2024 finalized guidance on charging provides detailed clarification. Notably, the regulations make no distinction between nonprofit and for-profit sponsors — any entity that qualifies as a "sponsor" under 21 CFR 312.3 may seek cost-recovery authorization.
Right to Try Act (2018) — 21 USC 360bbb-0a
An alternative pathway that bypasses FDA pre-authorization but requires the drug to have completed a Phase I trial. As discussed above, this is generally not applicable to personalized neoantigen vaccines due to their one-of-a-kind nature. Some state-level legislation (New Hampshire, Texas) is working to address this gap.
State-Level Considerations
State laws add additional complexity. The most relevant consideration is the definition of "practice of medicine," which varies by state. For example, New York's Education Law Article 131 defines the practice of medicine broadly, which means that any service that could be construed as medical practice (such as interpreting sequencing results for patients or recommending specific treatment protocols) requires a medical license.
Entities that facilitate expanded access as administrative coordinators — handling logistics, paperwork, and scheduling without making medical decisions — generally operate outside the scope of medical practice requirements. However, this distinction should be reviewed with a healthcare attorney for each state of operation.
HIPAA and Privacy
Any entity that handles protected health information (PHI) — including tumor sequencing results, pathology reports, and medical records for FDA filings — must comply with HIPAA as a business associate. This requires Business Associate Agreements (BAAs) with all physician and laboratory partners, HIPAA-compliant data storage and communication systems, breach notification procedures, and workforce training on PHI handling.
What the Research Shows
Clinical evidence for personalized neoantigen cancer vaccines is growing rapidly. However, it is important to understand the context of the data: most results come from the adjuvant (post-surgical) setting, meaning the vaccines were given to patients after their tumors were surgically removed, to prevent recurrence. Data on treating active metastatic disease with neoantigen vaccines is more limited.
Moderna/Merck: V940 (Intismeran Autogene) + Pembrolizumab
Phase 2b Results (KEYNOTE-942)
In the completed Phase 2b trial (KEYNOTE-942), 157 patients with resected high-risk melanoma (Stage III/IV) received either V940 + pembrolizumab (Keytruda) or pembrolizumab alone. At approximately 3-year follow-up, the combination demonstrated:
- 49% reduction in recurrence or death (hazard ratio 0.51)
- 62% reduction in distant metastasis or death
These results, published in The Lancet (2024;403:632-644), led to the FDA granting Breakthrough Therapy Designation. However, the FDA denied a request for accelerated approval based on Phase 2b data in September 2024, requiring Phase 3 confirmation.
Sources: Lancet 2024;403:632-644 (3-year data); Merck press release (Jan 20, 2026) (5-year update)
Phase 3 (INTerpath-001) — Ongoing
The Phase 3 trial (NCT05933577) is enrolling approximately 1,089 patients with resected high-risk melanoma. Primary completion is estimated for October 2029. This trial has not yet met its primary endpoint — it is ongoing and no interim results have been publicly reported as of March 2026. If positive, this could support a Biologics License Application (BLA) filing, with potential FDA approval in the 2029–2030 timeframe.
Mount Sinai: PGV001
Mount Sinai's Phase 1 trial of PGV001, a personalized multi-peptide vaccine, treated 13 patients with various solid tumors (lung, bladder, head and neck, and others). At 5-year follow-up:
- 6 of 13 patients survived at 5 years
- 3 patients remained tumor-free
- All patients showed measurable neoantigen-specific T-cell responses
Results were published in Cancer Discovery (Vol. 15, Issue 5) and reported by Mount Sinai. This was a very small Phase 1 trial (safety and feasibility), so the efficacy data should be interpreted cautiously.
BioNTech: Pancreatic Cancer
In a small study of patients with resected pancreatic ductal adenocarcinoma, BioNTech's individualized neoantigen mRNA vaccine demonstrated:
- 8 of 16 patients showed strong T-cell responses to the vaccine
- Responders had a median recurrence-free survival of 27.1 months
- 50% of responders remained recurrence-free at 18 months
- Non-responders had a median recurrence-free survival of only 8.6 months
Reported by the National Cancer Institute. Pancreatic cancer is one of the most difficult cancers to treat, making these results particularly notable despite the small sample size.
Important Caveats
- Most data is from the adjuvant setting — Patients had their tumors surgically removed before vaccination. Results may differ significantly for patients with active, measurable disease.
- Sample sizes are small — The largest completed trial (Phase 2b) had 157 patients. The Phase 1 studies had 13-16 patients. Larger Phase 3 trials are needed to confirm efficacy.
- Combination therapy confounds results — Most studies combined the vaccine with checkpoint inhibitors. It is difficult to isolate the vaccine's contribution from the checkpoint inhibitor's contribution.
- Expanded access outcomes are not published — JLF and other expanded access programs have not published peer-reviewed survival data for their patients. Individual outcomes through expanded access may differ from clinical trial results.
- Patient selection bias — Patients who qualify for and can afford expanded access tend to be healthier and wealthier than the general cancer patient population, which may inflate apparent outcomes.
Frequently Asked Questions
How long does the expanded access process take for a personalized cancer vaccine?
The entire process typically takes 4 to 5 months from initial biopsy to first vaccine dose. This includes approximately 4 weeks for whole exome sequencing, 4–6 weeks for neoantigen identification and vaccine design, and 12–14 weeks for GMP manufacturing. The FDA typically responds to individual patient expanded access requests within 30 days, though emergency requests may be processed in as little as 24 hours.
Does insurance cover personalized cancer vaccines under expanded access?
As of 2026, no insurance companies cover personalized neoantigen cancer vaccines obtained through expanded access. Because these vaccines have not yet received FDA approval, there are no billing codes for reimbursement. The total out-of-pocket cost is typically $100,000 to $119,000. Some patients explore options like home equity loans, retirement account withdrawals, or crowdfunding platforms to cover costs.
What are the side effects of personalized neoantigen cancer vaccines?
In clinical trials, the most commonly reported side effects include injection site reactions (pain, redness, swelling), fatigue, flu-like symptoms (fever, chills, muscle aches), and nausea. When combined with checkpoint inhibitors like pembrolizumab (Keytruda), the side effect profile may include immune-related adverse events such as colitis, hepatitis, or thyroid dysfunction. Serious adverse events have been reported but are relatively uncommon in published trial data. Your oncologist will monitor for and manage side effects throughout the treatment course.
What is the success rate of personalized cancer vaccines?
Published data is encouraging but limited. In the Moderna/Merck Phase 2b trial, the combination of V940 + pembrolizumab showed a 49% reduction in recurrence or death compared to pembrolizumab alone in adjuvant melanoma. Mount Sinai's Phase 1 trial reported that 6 of 13 patients survived at 5 years, with 3 remaining tumor-free. It is critical to note that "success rate" varies significantly depending on cancer type, stage, patient health, and whether the vaccine is used in the adjuvant (post-surgical) setting versus treating active disease. Individual outcomes vary and there are no guarantees.
How do I start the process of getting a personalized cancer vaccine?
The first step is to discuss personalized neoantigen vaccines with your oncologist. Your oncologist will need to determine whether you meet the clinical eligibility criteria and be willing to serve as the treating physician who files the individual patient IND application with the FDA. You will also need to arrange whole exome sequencing of your tumor tissue and coordinate with a vaccine manufacturer. Organizations like the Jaime Leandro Foundation can help navigate these logistics.
What does an oncologist need to do to prescribe a personalized cancer vaccine?
The treating physician must file an individual patient IND application (FDA Form 3926) with the FDA. This form requires documentation of the patient's diagnosis, prior treatments, rationale for expanded access, and an assessment that the potential benefit justifies the risks. The physician must also obtain Institutional Review Board (IRB) approval (or use the expedited single-patient review process), report adverse events to the FDA, and supervise the treatment protocol. The FDA approves more than 99% of individual patient expanded access requests.
Can I access a personalized cancer vaccine outside the United States?
Yes, in some cases. Germany-based CeGaT offers an end-to-end sequencing and vaccine design service (CancerNeo) that patients can access internationally through their myCeGaT portal. Clinical trials by BioNTech (Germany), Moderna/Merck (global), and academic centers in Europe and Asia may accept international patients. However, regulatory frameworks differ by country, and cross-border logistics for tumor specimens and custom-manufactured biologics add complexity and cost. Consult with a physician familiar with international medical logistics.
What if I am not eligible for expanded access for a personalized cancer vaccine?
Several alternatives may be available. Clinical trials for neoantigen vaccines are actively recruiting at sites run by Moderna/Merck (Phase 3, melanoma), BioNTech (Phase 2, multiple tumors), and academic medical centers. You can search for active trials at ClinicalTrials.gov. Other immunotherapy options such as checkpoint inhibitors (if not yet tried), CAR-T cell therapy, or tumor-infiltrating lymphocyte (TIL) therapy (FDA-approved as Amtagvi for melanoma) may be appropriate depending on your cancer type and stage. Discuss all available options with your oncologist.
Next Steps
If you or a loved one is considering a personalized cancer vaccine through expanded access, here are the concrete next steps.
Check Your Eligibility
Answer key questions about your diagnosis, treatment history, and overall health to understand whether you may qualify for expanded access.
Check EligibilityEstimate Your Costs
Get a personalized cost breakdown based on your location, cancer type, and preferred sequencing and manufacturing partners.
Estimate CostsGet Weekly Research Updates
New developments in neoantigen science emerge monthly. Subscribe to receive clinical trial updates, regulatory changes, and program availability.
Subscribe FreeTalk to Your Oncologist
Your oncologist is the critical first step. Here is a brief script to start the conversation:
"I've been reading about personalized neoantigen vaccines available through FDA expanded access. Given my diagnosis, I'd like to discuss whether I might be a candidate. Would you be willing to explore this with me, or refer me to an oncologist experienced in this area?"
Medical Disclaimer: This guide is for educational purposes only and does not constitute medical advice, diagnosis, or treatment. The information provided is based on publicly available sources, published clinical trial data, and regulatory documents. Individual medical decisions should be made in consultation with a qualified oncologist or treating physician.
ImmunaPath does not diagnose, prescribe, or provide medical treatment. We provide information and navigational support to help patients and their families understand available options.
Sources and Further Reading
- 21 CFR 312, Subpart I — Expanded Access to Investigational Drugs
- 21 CFR 312.8 — Charging for Investigational Drugs
- FDA Guidance: Charging for Investigational Drugs Under an IND (Feb 2024)
- FDA Expanded Access Submission Data
- Weber JS et al. — Individualized neoantigen therapy mRNA-4157 (V940) plus pembrolizumab in resected melanoma. Lancet 2024;403:632-644
- Mount Sinai PGV001 Phase 1 Results — Cancer Discovery Vol 15, Issue 5
- ClinicalTrials.gov — INTerpath-001 (NCT05933577)
- ClinicalTrials.gov — KEYNOTE-942 (NCT03897881)
- Jaime Leandro Foundation FAQ
- Triage Cancer — Expanded Access vs. Right to Try
- American Cancer Society — Cancer Facts & Figures 2025
- Harvard Medical School — When Will Patients See Personalized Cancer Vaccines?