A new drug produces encouraging results in a clinical trial. The headlines call it a breakthrough. Patients start searching for its name.
And then comes the frustrating part:
The drug is not approved yet.
Does that mean a patient has to wait until approval? Not always.
There are several ways an investigational treatment may potentially be accessed before it reaches the market. But understanding those pathways starts with understanding what “promising” actually means.
Promising Does Not Yet Mean Proven
Before a medicine is approved, researchers are still answering fundamental questions: Does it work? For which patients? At what dose? How serious are the risks? Does the benefit outweigh those risks?
An investigational drug may have produced impressive early results and still ultimately fail in later development.
That is why regulators treat these medicines differently from approved therapies. The FDA explicitly notes that an investigational product has not yet been found safe and effective for its proposed use and may cause serious or unexpected side effects.
For patients, however, uncertainty does not necessarily mean there are no options.
The First Route Is Usually a Clinical Trial
When an appropriate clinical trial is available and the patient is eligible, trial participation is generally the preferred route to an investigational medicine.
A clinical trial provides treatment within a structured research environment, with predefined eligibility criteria, safety monitoring, data collection and a protocol designed to answer specific scientific questions.
This is also why finding the drug is not enough.
A patient may know exactly which investigational therapy they want to explore but still be unable to join its trial because of previous treatments, laboratory results, biomarkers, disease stage, other medical conditions or simply geography.
So what happens when the trial is not an option?
There May Be Another Path
In the United States, some patients with serious or immediately life-threatening conditions may be able to receive an investigational drug outside a clinical trial through FDA Expanded Access, sometimes called compassionate use.
This pathway is intended for situations where there is no comparable or satisfactory alternative therapy and obtaining the investigational product through a clinical trial is not possible. The potential benefit must justify the potential risks, and providing access must not interfere with the drug's clinical development.
Expanded Access can apply to a single patient, an intermediate-size group or a larger patient population, depending on the circumstances.
But there is a crucial point patients often do not realize:
FDA authorization alone is not enough.
The Company Has to Say Yes Too
For individual Expanded Access in the U.S., the patient's physician must be willing to oversee treatment. The company developing the drug must agree to provide it. An Institutional Review Board (IRB) generally needs to review the treatment plan, and FDA must allow the request to proceed. Informed consent is also required.
And the manufacturer is not legally required to provide the medicine.
There may be limited drug supply. The company may be prioritizing clinical trials. It may determine that available safety information does not support use in a particular patient. Or it may simply not operate an Expanded Access program for that medicine.
This makes the manufacturer's decision one of the most important steps in the entire process.
99%: An Important Number That Needs Context
Patients sometimes assume that FDA is the biggest obstacle to Expanded Access.
The data tell a more nuanced story.
According to the FDA, on average, 99% of Expanded Access requests submitted to the agency are allowed to proceed. For single-patient requests specifically, FDA states that it allows more than 99% to proceed.
But this figure needs to be interpreted carefully.
It refers to requests that actually reach FDA. It does not mean that 99% of patients who want an investigational medicine ultimately receive it.
Before a request reaches that stage, a physician must support the treatment and the manufacturer must agree to provide the drug.
What About Europe?
There is no single European pathway that works exactly like FDA Expanded Access.
In the European Union, compassionate-use programs are implemented by individual Member States under national rules. The European Medicines Agency (EMA) can provide recommendations for certain group programs, but it does not operate one centralized European access system.
There is also a distinction between compassionate-use programs for groups of patients and access on a named-patient basis, where a physician may seek an unauthorized medicine for an individual patient under national legislation.
EMA emphasizes that patients should generally be considered for an appropriate clinical trial before compassionate use is pursued.
This means the route, requirements and timeline can differ substantially depending on where the patient lives.
What Should a Patient Ask?
If you hear about a promising drug that has not yet been approved, the most useful question is not simply:
“How can I get this drug?”
Start by establishing where the drug actually is in development and whether there is meaningful evidence for your specific disease and situation.
Then ask:
- Is there an active clinical trial that I may qualify for?
- If I am not eligible, why?
- Does the company offer Expanded Access, compassionate use or another pre-approval access pathway?
- Would my treating physician be willing to pursue such a request?
- What is actually known about the potential benefits and risks?
- What additional medical records or biomarker testing would be needed to evaluate the option?
These questions turn a promising headline into something much more useful: a realistic assessment of whether the treatment is relevant and accessible for one specific patient.
The Right2Hope Perspective
When standard treatment options are running out, learning about a promising investigational therapy can create enormous hope.
The difficult part is determining what comes next.
At Right2Hope, we help patients and families explore that question systematically. We review the patient's medical information, search for potentially relevant clinical trials and investigate other possible pathways to investigational treatment, including Expanded Access and compassionate-use options where appropriate.
We do not promise that an experimental medicine will work or that access will be granted.
We help patients understand what options may exist, what evidence supports them, and what steps may be required to pursue them.
Because “not yet approved” does not always mean “not available.”
Sometimes, it means there is another pathway to explore.