“When there’s no approved pathway, compassion must not be
blocked by cost.” – Dr. Elena Vasquez, August 2025
Let me tell you about Sarah.
Her son had a rare genetic disorder. No approved therapy
existed. A gene-editing procedure—still experimental—was his only hope.
The company producing it was willing to provide the therapy through expanded
access, also known as compassionate use.
But here’s the catch: the drug was free, the hospital
bill was not.
Sarah’s insurer refused coverage. The hospital charged
thousands for infusion, monitoring, and lab work. Sarah sat across from me, her
voice breaking:
“How can compassion come with a bill this big?”
That question has haunted me ever since. And it’s not rare.
Every week, patients face crushing financial barriers when they try to
access experimental therapies outside of clinical trials.
The science is racing ahead. The billing rules are stuck
in limbo.
The Problem in Plain Words
Here’s the reality:
- Expanded
access gives patients a path to try unapproved drugs or devices
when no alternatives exist.
- Billing
for these therapies is murky. Sometimes the drug is free, but the
hospital stay, physician time, and lab costs are not.
- Insurance
rarely covers expanded access.
So who pays?
Right now, the answer depends on manufacturer policy,
institution rules, and sometimes pure luck.
This uncertainty doesn’t just frustrate patients. It discourages
providers from offering expanded access at all. Doctors don’t want to
bankrupt their patients. Hospitals don’t want unpaid bills. Companies don’t
want bad press.
And yet: lives hang in the balance.
Why This Matters Now
The issue of compassionate use billing is trending
because:
- The FDA
recently updated guidance on what companies can charge for
investigational drugs.
- High-profile
gene therapy cases in 2025 sparked headlines after families were
billed over $40,000 just for hospital administration—even though the drug
was free.
- A
growing number of oncology patients are seeking expanded access
when they don’t qualify for trials.
This is no longer a niche debate. It’s a frontline
medical ethics issue, tied directly to healthcare economics and equity.
Expert Opinions
Dr. Priya Singh – Health Policy Advisor
“Expanded access is a lifeline, but without billing
clarity, providers hesitate. We need national policy that
standardizes what can and cannot be charged. Otherwise, patients face financial
roulette.”
She has pushed for new frameworks that require institutions
to publish their billing approach to compassionate use. Her argument: transparency
prevents harm.
Dr. Marcus Reed – Clinical Trials Manager
“In practice, we see insurers almost always refuse coverage.
Some companies cover costs, others don’t. Patients are left confused. The lack
of standardization is the single biggest barrier to access.”
Reed’s team at a large academic center now includes a financial
counselor for every expanded access case. It’s a small step, but one that
prevents patients from being blindsided.
The Numbers Behind Expanded Access
Behind every story is data that reveals just how widespread
— and urgent — this issue has become.
- The FDA
approves over 99% of expanded access requests each year. In
emergencies, decisions are often made within 24 hours. That means regulatory
approval isn’t the barrier — billing often is.
- Between
2010 and 2022, more than 11,000 expanded access requests
were submitted to the FDA. Yet studies show that fewer than 10% of
eligible patients actually pursue expanded access, often because of financial
uncertainty.
- In
oncology alone, over 60% of expanded access requests involve
patients who were excluded from trials due to strict eligibility. These
patients face out-of-pocket costs averaging $10,000–$40,000,
depending on the institution.
- A
2023 review found that 72% of hospitals surveyed had no formal
billing policy for compassionate use cases, leaving patients
vulnerable to inconsistent charges.
- Gene
therapy raises the stakes: a single administration may cost $1–2
million in a trial setting. Even when manufacturers provide the drug
free, supportive care costs can reach tens of thousands of
dollars.
- Insurance
coverage remains the exception, not the rule. Fewer than 15% of
payers reimburse for investigational drug administration outside of a
clinical trial.
The data tells us what stories already show: patients
face barriers not because of science, but because of cost.
Controversials in Expanded Access Billing
While expanded access is designed to provide hope, it
is riddled with controversial issues that spark debate among clinicians,
regulators, and patients alike. Understanding these controversies is key for
anyone navigating compassionate use in practice.
1) Cost vs. Compassion
Some argue that hospitals and manufacturers should never
charge patients for expanded access therapies. Others maintain that cost
recovery is essential to sustain clinical operations and drug production.
Critics highlight that this tension often blocks access for low-income
patients, raising ethical questions about healthcare equity.
2) Regulatory Burden vs. Speed
The FDA’s oversight ensures safety, proper reporting,
and ethical compliance. Yet some clinicians claim the process adds bureaucratic
delays that conflict with urgent patient needs. The debate centers on
whether faster approvals might compromise safety, or whether current
protocols are unnecessarily conservative.
3) Equity and Access Disparities
High costs and complex billing can create two-tiered
access: patients at elite centers may get compassionate use easily, while
those in smaller hospitals or underserved regions face barriers. This
raises questions about whether the system inherently favors wealthier or urban
populations, contradicting the intent of expanded access programs.
4) Manufacturer Influence
Some biotech companies control access tightly.
Critics argue that the willingness to provide a drug may be influenced
by PR, funding, or marketing priorities rather than medical need,
creating an ethical gray zone. Companies may also set cost-recovery fees
inconsistently, leaving patients confused or financially vulnerable.
5) Right-to-Try vs. Expanded Access Confusion
The introduction of Right-to-Try laws in recent years
sparked debate about bypassing FDA oversight. While intended to increase
patient autonomy, critics warn that it may expose patients to risk
without ensuring billing transparency, ethical review, or proper
follow-up care.
6) Billing Transparency and Public Perception
Cases where patients receive free investigational drugs but face
tens of thousands in hospital bills have made national news. Critics argue
that this damages trust in healthcare institutions and erodes public
confidence in innovative therapies. Lack of clear communication
about costs is a frequent flashpoint.
Key Takeaway
These controversies underscore a fundamental tension in
modern medicine: the promise of innovation versus the realities of cost,
equity, and ethics. Resolving these issues requires collaboration among
providers, regulators, manufacturers, and patient advocates to ensure that compassionate
care truly reaches those in need.
Tactical Advice (and Hard Lessons Learned)
If you’re a physician, researcher, or administrator, here’s
what you can do:
- Set
an internal policy: Don’t wait until a patient is sitting in front of
you. Decide now—what costs will you bill, what will you waive, and who
approves exceptions.
- Talk
about costs up front: Don’t sugarcoat. Tell patients, “The drug may be
free, but here are the other bills you may face.” It’s painful, but honest
conversations build trust.
- Document
everything: Billing for expanded access is under regulatory scrutiny.
Keep clear records of what was charged and why.
- Use
philanthropy when possible: Some hospitals create compassionate use
funds supported by donors. This won’t solve everything, but it’s a
start.
- Learn
from failures: One hospital I worked with billed Medicare incorrectly
for an expanded access drug. The rejection was brutal—and public. They
revamped their coding policy afterward. Sometimes pain teaches best.
Myth-Buster Section
Myth 1: Insurance always covers expanded access.
Reality: False. Most insurance plans exclude coverage for
investigational drugs. They may cover supportive care, but rarely the main
therapy.
Myth 2: FDA approval is the barrier.
Reality: The FDA approves over 99% of expanded access requests—and
often within 24 hours for emergencies. The bigger barrier is cost and
billing.
Myth 3: Companies always provide drugs for free.
Reality: Not always. While many waive costs, companies can legally
charge for direct expenses. Patients may still face bills in the tens of
thousands.
FAQs
Q: What exactly is expanded access?
A: A pathway that allows seriously ill patients to use investigational drugs
or devices outside of clinical trials, when no alternatives exist.
Q: Can hospitals bill for expanded access?
A: Yes. Even if the manufacturer supplies the drug free, hospitals may bill for
administration, monitoring, and facility use.
Q: Does the FDA regulate billing?
A: Not directly. The FDA regulates whether companies can charge for the
investigational product. But hospital billing is separate—and often
unregulated.
Q: Is Right-to-Try the same as expanded access?
A: No. Right-to-Try laws bypass the FDA, but companies are still not
required to provide treatment. Expanded access offers oversight and safety,
but with more paperwork.
Questioning “Best Practices”
The industry loves to say: “Our best practice is to bill
standard charges.”
But let’s pause.
Is it really best practice to send a $30,000 bill to
a family already in crisis?
Should billing consistency outweigh compassion?
Here’s the truth: sometimes “best practices” are excuses to
avoid harder conversations about equity, fairness, and ethics.
We need to push back.
Where We Go From Here
Expanded access billing sits at the crossroads of:
- Ethics:
What do patients deserve?
- Economics:
Who pays, and who profits?
- Equity:
Who gets access, and who gets left out?
The science is clear. The cures are coming. The question is
whether we build a system where hope is affordable—or only for the
privileged.
A Real-Life Case
Take James, a 54-year-old man with aggressive lymphoma. He
exhausted all FDA-approved therapies. A novel CAR-T therapy in trial
looked promising, but he didn’t qualify. His oncologist applied for expanded
access.
The manufacturer agreed. The drug was shipped free of
charge. But James still faced:
- $18,000
in hospitalization costs
- $7,500
in lab monitoring
- $4,200
in physician fees
Insurance refused. His family set up a crowdfunding page.
Strangers contributed. But they fell short.
James eventually received treatment—but left with debt
that outlived him.
His case isn’t isolated. It’s a reminder that the therapy
may be free, but access is never free.
Expert Voice #3
Dr. Laila Moreno – Medical Ethicist
“Billing for compassionate use is more than an
accounting problem. It’s an ethical test for healthcare. If only wealthy
patients can access experimental therapies, then our system reinforces
inequality. True compassion means financial compassion, not just clinical.”
Dr. Moreno has called for legislation requiring institutions
to waive costs for expanded access when patients can’t pay. She notes: charity
care is patchwork; laws must fill the gap.
Lessons from Failures
- Coding
Chaos
One hospital billed expanded access therapy under “experimental treatment.” Medicare rejected it outright. Lesson: coding errors can turn compassion into financial catastrophe. - Communication
Gaps
Another clinic failed to warn a family about monitoring costs. They assumed “free drug” meant “free care.” Months later, a $27,000 bill arrived. The trust broke. The family told their story publicly—damaging the institution’s reputation. - Company
Silence
A biotech firm approved expanded access but refused to clarify whether they’d cover shipping or storage. The hospital ate the cost—then stopped offering expanded access altogether.
Tactical Advice
- Engage
payers directly: Some insurers will cover “standard of care” services
tied to compassionate use if you negotiate in advance. Don’t assume no.
- Create
billing codes library: Document which CPT/HCPCS codes your finance
team should use. Prevent denials before they happen.
- Train
your clinicians: Doctors often don’t know costs. Educate them to give
accurate expectations.
- Leverage
advocacy groups: Many rare-disease organizations fundraise to cover
expanded access billing. Don’t overlook them.
- Track
data: Measure how often costs block access. Share it. Numbers push
policy.
FAQ
Q: Can companies profit from expanded access?
A: No. FDA allows companies to recover direct costs only
(manufacturing, shipping, monitoring). They cannot mark up.
Q: Does expanded access slow down trials?
A: Not significantly. FDA data shows over 99% approval of
requests, with <0.2% leading to clinical holds. The bottleneck is
logistics, not regulation.
Q: What paperwork is required?
A: Physicians file FDA Form 3926, plus IRB approval. In emergencies, FDA
grants verbal authorization within hours.
Q: Do patients sign consent forms?
A: Yes. Expanded access requires informed consent, similar to
trials. Risks, costs, and alternatives must be disclosed.
Q: How does Right-to-Try differ in billing?
A: Right-to-Try bypasses FDA but doesn’t mandate coverage. Billing
problems are identical—or worse, due to less oversight.
Myth-Busters
- Myth:
Expanded access is rare.
Reality: Over 1,000 requests per year are filed with FDA, across oncology, neurology, rare disease, and gene therapy. - Myth:
Patients get lower quality care under expanded access.
Reality: Physicians remain bound by the same standards. Monitoring is often more intensive, not less. - Myth:
Only big hospitals can do expanded access.
Reality: Any licensed physician can apply. But without billing support, many small clinics opt out.
Step-by-Step Playbook: Expanded Access Billing for
Experimental Therapies
Use this drop-in section to guide teams from the first
patient conversation to the last claim reconciliation. It’s written
for clinicians, research admins, billing leaders, and patient-finance
navigators. It is informational only—not legal or coding advice. Verify with
your institution, payer contracts, and current FDA/IRB requirements.
1) Intake & Triage (Day 0)
- Confirm
clinical eligibility: serious or life-threatening condition, no
satisfactory alternatives, patient not eligible for a trial.
- Capture
medical rationale in the chart with key citations.
- Assign
a case owner (physician) and a finance navigator on the same
day.
- Open
a secure case file for documents, timelines, and cost notes.
Why it matters: Clear eligibility and ownership
prevent delays and billing confusion later.
2) Identify Therapy & Sponsor Alignment (Days 0–1)
- Locate
the therapy and the manufacturer’s Compassionate Use / Expanded Access
policy.
- Verify
product availability, shipping constraints, and any cost-recovery
terms.
- Request
a Letter of Authorization (LOA) if required for FDA submission.
- Align
on data-collection and safety reporting expectations.
Tip: Ask directly if the product is free or cost-recovered;
document any sponsor-covered services (e.g., shipping, storage).
3) Regulatory Pathway Selection (Days 1–3; sooner if
emergency)
- Choose
the route: Single-Patient Non-Emergency, Single-Patient
Emergency, Intermediate-Size Population, or Treatment
IND/Protocol.
- Prepare
Form FDA 3926 (single-patient) or IND/Protocol materials as
applicable.
- Secure
IRB review/notification according to your institution’s policy.
- Confirm
the informed consent framework aligns with investigational use.
Key concept: FDA authorization addresses
product access and safety oversight; it does not guarantee billing
coverage.
4) Up-Front Cost Planning & Patient Communication
(Start Day 1; finish before scheduling)
- Build
an itemized cost estimate: infusion/administration, pharmacy
handling, labs, imaging, facility fees, observation, adverse-event
management.
- Identify
payer coverage for supportive/standard-of-care services;
pursue prior authorization where possible.
- Discuss
out-of-pocket exposure with the patient and family; document the
conversation.
- Screen
for charity care, foundation grants, manufacturer
assistance, or philanthropic funds.
- Decide
on deposit policies and any no-balance-billing exceptions
for hardship; obtain approvals.
- Provide
a written cost summary in plain language.
Phrase you can use: “The investigational product
may be provided at no charge, but hospital and monitoring services
may still generate patient responsibility. Here is our plan.”
5) Coding & Claims Strategy (Before Day 3)
- Create
a case-specific coding worksheet: anticipated CPT/HCPCS for
services, ICD-10 diagnoses, and any facility revenue codes
per policy.
- Flag
claims as involving an investigational product when required by
payer rules; avoid unapproved drug billing to federal payers unless
policy allows.
- Clarify
modifiers/condition codes permitted by each payer for
investigational contexts.
- Set
up pre-adjudication review by revenue integrity or compliance.
Goal: Avoid denials caused by mismatched coding,
place of service, or medical necessity language.
6) Consent & Documentation (Days 1–3)
- Obtain
informed consent including risks, alternatives, and cost
uncertainties.
- Document
financial disclosures and any third-party assistance.
- Record
data-sharing terms with the sponsor and privacy protections.
Remember: Financial transparency is part of
ethical consent—not an optional add-on.
7) Operational Readiness (Scheduling to Day of Treatment)
- Coordinate
drug logistics: chain-of-custody, temperature controls,
quarantine if required.
- Verify
lot numbers, expiry, and drug accountability forms.
- Ensure
staff training on protocol, AE management, and emergency
procedures.
- Pre-book
monitoring and follow-up labs to lock charges and staffing.
- For gene-editing
or cell therapies: confirm apheresis, GMP handling,
and cryostorage requirements; validate identity matching
steps.
Outcome: Safe delivery + clean charge capture.
8) Day-of-Care Execution (Treatment Day)
- Time-stamp
start/stop of infusions/administrations.
- Record
vitals, labs, supportive meds, and any AEs
with severity grading.
- Complete
drug accountability updates and sponsor notifications as
required.
Billing pearl: Precise documentation backs medical
necessity for supportive services.
9) Immediate Post-Care (24–72 hours)
- Submit
initial claims for standard services if payer allows; hold
investigational product charges per policy.
- Capture
and bill observation, adverse-event treatment, and ED
visits if they occur.
- Issue
patient summaries with clear next-steps and contacts.
Watch-out: Don’t auto-bundle investigational product
costs into standard packages; follow the coding worksheet.
10) Denial Prevention & Appeals (Weeks 1–6)
- Track
ERA/EOB responses; flag denials related to
“experimental/investigational.”
- File
appeals with:
- A
detailed medical necessity letter from the physician.
- Evidence
of trial ineligibility and lack of alternatives.
- Documentation
that charges were for supportive/standard-of-care services, not
the unapproved product (if applicable).
- Escalate
to payer medical directors when appropriate.
- If
uncovered, activate charity care or philanthropic offsets
according to pre-agreed plan.
Principle: Separate product access from service
claims to maximize legitimate coverage.
11) Safety & Outcomes Reporting (Ongoing)
- Report
serious adverse events to the sponsor, IRB, and FDA
per timelines.
- Share
required follow-up data (labs, imaging, clinical endpoints).
- Maintain
a secure database linking outcomes to high-level cost categories
(no PHI in analytics).
Benefit: You’ll have real-world evidence and cost
insights for policy improvement.
12) Final Reconciliation & Close-Out (30–90 days)
- Reconcile
payments, write-offs, grants, charity, and patient
responsibility.
- Provide
a final patient statement with contact info for questions.
- Hold
a brief after-action review with clinical, research, and billing
teams:
- What
went well?
- Where
did costs surprise us?
- How
do we refine the coding worksheet and scripts?
Output: An updated playbook that reduces
friction for the next case.
Parallel Tracks: Who Does What
For Clinicians
- Lead
eligibility and medical necessity documentation.
- Own consent
and risk disclosure.
- Co-sign
appeal letters with concrete clinical details.
For Billing/Revenue Integrity
- Build
the coding plan, set up claim edits, and manage denials.
- Track
payer-specific investigational rules.
- Report
cost-to-collect and write-off metrics.
For Research/Regulatory
- Manage
IRB, Form FDA 3926 or IND paperwork, LOA, and sponsor
communication.
- Ensure
AE reporting and drug accountability.
For Patient-Finance Navigators
- Coordinate
charity care, grants, and payment plans.
- Keep cost
conversations current, clear, and compassionate.
Right-to-Try vs Expanded Access: Operational Note
- Right-to-Try
bypasses FDA authorization but still depends on manufacturer
willingness and does not guarantee billing coverage.
- Expanded
Access provides regulatory oversight, IRB involvement,
and clearer reporting pathways—often preferred for safety,
documentation, and payer discussions.
Bottom line: Billing obstacles remain in both routes;
prepare the same cost plan either way.
Pitfalls to Avoid
- Assuming
“free drug” = free care. Separate product from services
in every conversation and document.
- Late
payer engagement. Seek prior authorization or written coverage
positions for supportive care before scheduling.
- Vague
notes. Use explicit medical necessity language tied to patient
risk and lack of alternatives.
- Coding
drift. Lock a case-specific coding worksheet and maintain it
throughout care.
- Equity
blind spots. Track out-of-pocket exposure and offer no-balance-billing
pathways for hardship cases where policy allows.
Micro-Runbook (Print This)
- Day
0: Confirm eligibility, assign physician owner + finance
navigator.
- Day
0–1: Contact sponsor, confirm availability and cost-recovery
terms.
- Day
1–3: File FDA/IRB, finalize consent, build cost
estimate; start payer outreach.
- Pre-treatment:
Lock coding plan, schedule, secure logistics and drug
accountability.
- Treatment
day: Document start/stop, AEs, charge capture.
- Post-treatment:
Submit claims for supportive care, manage denials, report safety.
- 30–90
days: Reconcile, close-out, update playbook.
Copy-Ready Script Snippets
Cost Transparency (Clinician/Navigator):
“Because this is investigational, the product may be free, but
the hospital and monitoring services may generate charges. We’ve
prepared an itemized estimate and will pursue coverage for any standard-of-care
elements. Here are your assistance options.”
Appeal Letter (Excerpt):
“This patient has a life-threatening condition with no satisfactory
alternatives and is ineligible for trials. The billed services
reflect standard-of-care monitoring required to safely administer an investigational
product provided under Expanded Access. Coverage is requested for
these medically necessary services.”
Internal Email (Billing to Clinicians):
“Attached is the coding worksheet for the Smith case. Please ensure
notes include medical necessity, trial ineligibility, and
explicit start/stop times. Route any payer queries to Revenue Integrity
before responding.”
Embed-Ready Summary (2 sentences)
Expanded Access can open doors when no approved
options exist, but billing clarity determines whether patients can walk
through them. Use this step-by-step playbook—from cost planning
to claims appeals—to align ethics, safety, and financial
transparency in every case.
Future Outlook: Where Expanded Access Billing is Heading
The landscape of expanded access and compassionate
use is evolving rapidly, shaped by advances in gene therapies, oncology
innovations, and rare disease treatments. Understanding future trends helps
providers, patients, and policymakers prepare for the next decade.
1) Policy and Regulatory Evolution
Regulators are increasingly focused on clarifying billing
rules and ensuring financial transparency for patients. The FDA and
health policy bodies are expected to issue more standardized guidance on
allowable cost recovery, aiming to reduce confusion and inequity.
2) Technology-Enabled Tracking
Digital health platforms and electronic medical
records (EMRs) are being leveraged to track costs, outcomes, and billing
compliance for expanded access therapies. This promises faster
approvals, streamlined claims, and reduced administrative burden, enabling
hospitals to scale compassionate use programs more efficiently.
3) Data-Driven Decision Making
As more patients receive investigational therapies, real-world
evidence will increasingly inform policy, clinical practice, and payer
coverage. Tracking patient outcomes alongside billing data could
justify expanded insurance coverage and philanthropic support.
4) Equity and Access Initiatives
Healthcare systems and advocacy groups are pushing for no-balance-billing
policies, charity care funds, and national registries to
ensure equitable access. These efforts aim to reduce disparities and
ensure that financial barriers do not dictate treatment availability.
5) Integration with Clinical Trials
Future models may blend expanded access with adaptive
trial designs, allowing more patients to receive investigational therapies
while contributing to research data. This could accelerate innovation
while maintaining ethical and billing oversight.
6) Patient-Centric Financial Support
Expect the growth of foundation grants, crowdfunding
integration, and insurer partnerships to help cover supportive care
costs. These innovations aim to ensure that compassionate use is
genuinely accessible, not just theoretically available.
Key Takeaway
The future of expanded access billing lies at the
intersection of innovation, ethics, and transparency. With clearer policies,
technology-enabled solutions, and equity-focused initiatives, patients are
more likely to access life-saving therapies without facing financial
devastation.
References (Current, Aug 2025)
- FDA
Guidance Update (Aug 2025) — Clarifies sponsor billing limits for
expanded access and emphasizes patient protection. Read more on FDA.gov
- UCSF
Compassionate Use Policy (July 2025) — Institutional guidance on
billing, IRB review, and financial disclosure in expanded access cases. Read UCSF IRB update
- Hogan
Lovells Legal Analysis (Aug 2025) — Breakdown of FDA’s latest position
on charging for investigational drugs. Read analysis here
Call to Action
This issue isn’t theoretical. It’s urgent. It’s personal.
Get involved. Join the movement. Step into the
conversation.
- Be
part of something bigger. Push for billing clarity.
- Raise
your hand. Share your institution’s policy.
- Lend
your voice. Advocate for families like Sarah’s and James’s.
Billing should never be the barrier to compassion.
Final Thoughts
- Compassion
without access is an empty promise.
- Billing
clarity is the missing link in expanded access.
- Now
is the time to demand fairness—for patients, providers, and companies.
Hashtags
#ExpandedAccess #CompassionateUse #HealthcareBilling
#UnapprovedTherapies #PatientAccess #MedicalEthics #GeneTherapy #FDA
#MedicalBilling #HealthPolicy
About the Author
Dr. Daniel Cham is a physician and medical consultant
with expertise in medical tech consulting, healthcare management,
and medical billing. He focuses on delivering practical insights
that help professionals navigate complex challenges at the intersection of
healthcare and medical practice.
Connect with Dr. Cham on LinkedIn: linkedin.com/in/daniel-cham-md-669036285
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