The Compassionate Freedom of Choice Act: Ill-advised “right to try” goes federal

On paper, the idea sounds irresistible: if you’re dying, you should have the “right to try” any experimental drug
that might help, free from government red tape. Who could be against that? That simple slogan powered a wave of
state-level “right to try” laws and ultimately inspired federal proposals like the Compassionate Freedom of Choice
Act and, later, the federal Right to Try Act. Behind the stirring rhetoric, however, is a messy policy story that
worries many clinicians, ethicists, and science communicators, including writers at Science-Based Medicine, who have
long argued that these laws are less about compassion and more about deregulation and political theater.

To understand why the Compassionate Freedom of Choice Act was called “ill-advised” by science-based critics, we
have to look past the slogans and into the fine print: how the bill was structured, how it compared to the existing
FDA “expanded access” (or compassionate use) pathway, and what actually happened once the federal Right to Try law
was passed in 2018. Spoiler: the new laws didn’t create a true right to access experimental drugs, they didn’t
meaningfully improve access for most patients, and they risked weakening important safeguards that protect some of
the sickest, most vulnerable people in the healthcare system.

How the “right to try” movement began

The “right to try” movement gained momentum around 2014, powered by emotionally compelling stories of terminally
ill patients who were ineligible for clinical trials but desperate to try promising new drugs. Advocacy groups
argued that the FDA’s rules were too slow and too strict, and that patients facing death should be able to accept
more risk than regulators would normally allow. Legislatures in dozens of states quickly passed “right to try”
laws, often with bipartisan support and little oppositionafter all, voting against a bill with “compassion” or
“freedom of choice” in the title is a tough look for any politician.

Federal lawmakers wanted in on the action. Representative Morgan Griffith of Virginia introduced the
Compassionate Freedom of Choice Act (versions appeared in 2014 and 2015) to enshrine a federal
“right to try” by limiting the FDA’s power to block or oversee access to investigational drugs, biologics, and even
medical devices for terminally ill patients.

What the Compassionate Freedom of Choice Act proposed

The Compassionate Freedom of Choice Act (CFCA) essentially took the themes of state “right to try” laws and scaled
them up to a national level. While details varied slightly between versions, the core concepts were strikingly
consistent:

• Who qualified? Terminally ill patients, often defined as those with a disease likely to cause
  death within a limited time frame, who had exhausted approved treatment options.
• Which products? Drugs, biologics, and sometimes devices that were still investigational
  meaning they had not yet been fully approved by the FDA. Some versions required the product to have passed Phase 1
  safety testing; others were even looser.
• Bypassing usual oversight: CFCA would have sharply restricted the FDA’s ability to interfere
  with a physician’s decision to prescribe, and a company’s decision to provide, an investigational product to an
  eligible patient.
• Liability shields: The bill sought to protect manufacturers and clinicians from legal liability
  related to harms from these unapproved products, except in very narrow circumstances.
• Minimal data collection: CFCA proposals limited requirements to collect and report outcomes to
  the FDA, meaning less systematic information on safety and effectiveness would flow back into the regulatory
  system.

To critics at Science-Based Medicine and elsewhere, this wasn’t just a small tweak to existing compassionate use
rules. It was an effort to sidestep the FDA entirely and weaken the evidence-based framework that underpins modern
drug regulation, all while being marketed as an act of mercy.

But don’t patients already have access? The FDA’s expanded access program

The most important fact that “right to try” campaigns rarely highlight is that a pathway to experimental treatments
already existed long before these laws: the FDA’s expanded access program, often called
“compassionate use.” Through this pathway, patients with serious or life-threatening conditions can receive
investigational drugs outside of clinical trials when certain conditions are met.

Under expanded access, a treating physician works with the manufacturer and the FDA to request the drug. The FDA
reviews the request to make sure:

• The potential benefits justify the potential risks for the patient.
• Providing the drug won’t interfere with ongoing clinical trials needed to prove safety and effectiveness.
• There’s at least some evidence (often from early-phase trials) that the drug might help.

Here’s the part that often surprises people: the FDA says it authorizes over 99% of expanded access
requests, and clinical holds due to safety concerns in expanded access are extremely rareabout
0.2% in a review of more than 10,000 cases.

In other words, the main bottleneck isn’t usually the FDA. It’s whether the manufacturer is willing and
able to provide the drug, plus practical issues like cost, logistics, and the patient’s condition. Expanded access
already gives patients and physicians a lot of flexibility, with ethical oversight via institutional review boards
(IRBs) and regulatory review to maintain some guardrails.

Numbers that rarely make headlines

Studies of expanded access in oncology and other fields show thousands of patients per year are treated through this
route, with serious adverse events reflecting both the severity of their illnesses and the experimental nature of
the drugs. But importantly, these events are systematically reported and evaluated, helping regulators get a clearer
picture of risks while not blocking approval of valuable drugs.

By contrast, “right to try” laws add a parallel path that removes much of that oversight and data
collectionwithout clearly improving access.

The federal Right to Try Act: CFCA’s spirit goes national

The Compassionate Freedom of Choice Act never made it into law, but its ideas didn’t disappear. In 2018, Congress
passed, and President Trump signed, the federal
Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act.

The federal Right to Try Act created a new pathway that:

• Applies to patients with life-threatening diseases who have exhausted approved treatments and cannot access a
  clinical trial.
• Allows access to “eligible investigational drugs” that have completed Phase 1 testing and are in active
  development.
• Does not require FDA approval of individual requests and does not require IRB review.
• Limits how the FDA can use data from these uses in its regulatory decisions.
• Provides broad liability protections for manufacturers, prescribers, and others, except in cases of willful
  misconduct.

Supporters portrayed this as a historic victory for patients’ rights. But from an evidence-based standpoint, Right
to Try looks a lot like CFCA-lite: it borrows CFCA’s rhetoric and core structure while softening some of the most
extreme provisions.

Expanded access vs. Right to Try: what’s the difference?

For patients and clinicians, the two pathways can look confusingly similar, but the differences matter:

• Oversight: Expanded access involves FDA review and IRB oversight; Right to Try largely bypasses
  both.
• Data: Expanded access generates systematically reported safety data; Right to Try requires only
  limited annual summaries from manufacturers.
• Scope: Both pathways depend on manufacturers’ willingness to provide the drugneither creates a
  true legal “right” to obtain it.
• Practical use: Expanded access is used thousands of times each year; Right to Try has been used
  rarely, with only a handful of products reported in the law’s first several years and likely only hundreds of
  patients nationwide.

If you’re keeping score, the path with more protections, better data, and far more real-world use is the one that
existed before Right to Try.

Why Science-Based Medicine calls CFCA “ill-advised”

It promises a “right” that doesn’t actually exist

Neither CFCA nor the federal Right to Try Act forces any company to say “yes” to a patient request. Manufacturers
can still decline because they have limited drug supply, worry about liability (even with shields), anticipate
negative publicity, or simply don’t think the drug will help. Insurers aren’t required to cover the drug or related
care, either.

So the term “right to try” is misleading. It’s really the “right to ask,” paired with fewer safeguards and weaker
data requirements. As some critics have put it, these laws risk giving patients false hope rather than a
meaningful new option.

It undermines a system that mostly works

Science-Based Medicine writers have long emphasized that the problem Right to Try claims to fix is largely
imaginary. The FDA already approves the overwhelming majority of expanded access requests and has streamlined
paperwork to make the process faster and more physician-friendly.

By creating a parallel pathway outside FDA oversight, CFCA-style proposals and the federal Right to Try Act do
something more dangerous: they weaken the feedback loops that help us learn from experimental treatments. If serious
adverse events aren’t systematically reported, regulators and researchers may miss important safety signalsor, just
as bad, overreact to anecdotes that lack proper context.

It opens the door to bad actors

Most physicians and drug companies genuinely want to help patients, but not everyone in the health marketplace is a
hero. Critics have warned that looser “right to try” rules could be exploited by fringe clinics and for-profit
ventures selling unproven stem-cell products or other experimental treatments at high prices, wrapped in the
language of freedom and hope.

Without strong oversight and data requirements, it becomes easier to “sell help, not hope”to borrow the phrase
from a Nature commentary on the CFCA erawhile shifting financial and medical risk onto patients and families who
are already desperate.

It uses powerful stories to advance weak policy

Politically, “right to try” is savvy branding. The movement leans heavily on heartbreaking individual stories:
children with untreatable cancers, adults with ALS or advanced heart disease, families who feel the system has given
up on them. These stories are real and deserve compassion. But as Science-Based Medicine points out, good health
policy can’t be built only on anecdotes, especially when they’re selected to support a deregulatory agenda rather
than to reflect typical patient experiences.

When the cameras are off, most patients still rely on clinical trials and the FDA’s expanded access system. Right to
Try, meanwhile, sits mostly on the bookssymbolically powerful, practically underused.

What patients and families actually need

None of this means we should be complacent about access to experimental therapies. Patients with terminal illnesses
do face real barriers, and there are real opportunities for improvement. But those improvements look less like
“right to try” slogans and more like boring, unglamorous policy work:

• Better communication and navigation: Many patients and clinicians simply don’t know how to use
  expanded access, or assume it’s impossible. Making the process clearer and more widely understood can help.
• More inclusive clinical trials: Trials often exclude older patients, those with multiple
  conditions, or those in rural areas. Designing more flexible, inclusive studies gives more people access to
  promising drugs in a controlled setting.
• Support for data collection: Ensuring that off-trial use of investigational products still
  feeds reliable safety and outcome data back into the system is crucial for everyone’s benefit.
• Financial protection: Even experimental drugs can come with experimental-size price tags.
  Policies that help limit catastrophic out-of-pocket costs matter as much as (or more than) new legal pathways.

How clinicians can talk about “right to try” with patients

For clinicians, conversations about the Compassionate Freedom of Choice Act, Right to Try, or expanded access often
happen at emotionally charged moments. A few practical steps can help keep those discussions honest and humane:

• Clarify goals: Is the patient hoping for a cure, hoping to live long enough to reach a
  milestone, or mainly focused on comfort and control? Different goals may point toward different choices.
• Map out options: Review standard treatments, clinical trials (for example, through resources
  like ClinicalTrials.gov), expanded access possibilities, andwhere appropriateRight to Try as a backup path, not
  a magic key.
• Explain uncertainty: Emphasize that investigational drugs may not help and can sometimes make
  things worse, especially when patients are very sick.
• Address logistics up front: Who pays? How will the drug be obtained? Can the patient physically
  travel to the site of care?
• Reaffirm support: Make clear that choosing not to pursue an experimental drug is a valid, often
  wise decisionand that the care team will stay engaged either way.

A science-based approach doesn’t mean being coldhearted. It means giving patients real choices, grounded in the best
evidence we have, rather than symbolic “rights” that quietly shift risks onto the people who can least afford them.

Real-world experiences and lessons related to Right to Try

To see how all of this plays out beyond the pages of congressional bills and policy blogs, it helps to look at what
has happened since the federal Right to Try Act became law. Despite the fanfare, available data show that only a
small number of patients and products have used the federal Right to Try pathway, while thousands of patients each
year continue to access experimental drugs through the FDA’s expanded access program.

Imagine an academic oncologistlet’s call her Dr. Riverawho specializes in hard-to-treat cancers. Before 2018, she
was already filing expanded access requests for selected patients who had exhausted standard therapy but were
promising candidates for an early-phase drug. The process required paperwork and IRB review, but over time her
institution built a playbook: a standard set of forms, a dedicated research nurse, and an IRB chair who knew the
drill. Approvals usually came through in days, not months.

After Right to Try passed, Dr. Rivera began fielding questions from patients and their families: “Can we use the
new Right to Try law?” “Does that mean we don’t need the FDA anymore?” The hospital’s legal and ethics teams
quickly realized that, in practice, many manufacturers preferred to stick with the familiar expanded access pathway.
Companies valued the FDA’s input on dosing and safety, and they wanted clear documentation for their own risk
management. So while the door to Right to Try was technically open, most of Dr. Rivera’s real-world cases still
went through expanded access.

For one patientcall him Jason, a 52-year-old with terminal lung cancerthe family arrived brandishing news articles
and advocacy materials about Right to Try. They had identified a small biotech company developing an experimental
drug and wanted Jason to receive it “no matter what.” Dr. Rivera contacted the company, which expressed sympathy but
declined to provide the drug under either pathway: supply was limited, the clinical trial was still enrolling, and
they worried that off-trial use might complicate interpretation of the results. Right to Try did not force their
hand. The law gave Jason the right to ask, but not the power to demand.

In another case, a woman with a rare sarcomalet’s call her Mariaexplored both options with her care team. The
manufacturer was open to providing its investigational drug, but only through a formal expanded access program that
included structured data collection and IRB oversight. Maria and her doctors agreed that this made sense: if she was
going to take on the risks of an unapproved therapy, she wanted her experience to contribute to the evidence base
for future patients. Right to Try would have allowed a looser, less structured arrangement, but there was no obvious
advantage to skipping safeguards that were already working in her favor.

Ethicists and regulatory scholars who have analyzed early Right to Try experience echo these stories. They note that
the pathway has been used very rarely compared with expanded access, and that many of the most publicized cases
have involved highly experimental products at the fringes of mainstream medicinesuch as certain stem-cell
treatmentsrather than drugs with strong early evidence.

At the same time, clinicians who work closely with patients facing terminal illness report that conversations about
Right to Try can still serve a purpose. They create an opening to talk about what’s realistically possible: to
explain that, yes, there is a legal pathway, but it depends on the manufacturer’s cooperation, and it may not be any
fasteror saferthan expanded access. They can use the moment to broaden the discussion to goals of care: what the
patient values most, how they feel about trade-offs between more time and more side effects, and whether the
potential benefits of an experimental drug really align with those priorities.

The overall lesson from these early experiences is sobering but useful. Laws like the Compassionate Freedom of
Choice Act and the federal Right to Try Act make for powerful talking points and emotional headlines. But when you
zoom in on real clinics, real families, and real decision-making, the system that actually carries the weight is the
less glamorous one: FDA-supervised expanded access, backed by IRB review and careful data collection. For patients
and clinicians trying to make science-based, humane choices at the end of life, that’s where the true “freedom of
choice” still lives.

Conclusion

The Compassionate Freedom of Choice Act and its offspring, the federal Right to Try Act, arose from an
understandable impulse: to help people who are out of options. But as Science-Based Medicine and other
evidence-oriented critics point out, good intentions are not enough. When you look at the data, the existing FDA
expanded access program already provides a workable path to experimental drugs for many patients, with high approval
rates and important safety oversight. Right to Try adds symbolism and slogans, but relatively little practical
benefitand it risks weakening the very systems that keep medicine anchored in evidence rather than anecdote.

In the end, the most compassionate policy isn’t the one that shouts “freedom” the loudest. It’s the one that quietly
does the hard work: supporting rigorous trials, making expanded access easier to navigate, protecting patients from
exploitation, and ensuring that every hard-won experience with an experimental drug teaches us something real.

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pitfalls, and what patients really need.

sapo:
The Compassionate Freedom of Choice Act and the federal Right to Try law promise dying patients a “right to try”
experimental drugsbut do they actually deliver? This in-depth, science-based analysis unpacks how these laws grew
out of emotionally charged stories, what they changed (and didn’t) in the real world, and why the FDA’s expanded
access program still does most of the heavy lifting. Learn how the policies work, where they fall short, and what
patients and families should ask their doctors when they’re considering last-chance treatments.