Is Right-to-Try the Best Path for Experimental Meds?

Is Right-to-Try the Best Path for Experimental Meds?

What to do when you or your loved one has exhausted all standard medical options.

A federal Right-to-Try Law (RTT) passed last month, but if you are seeking “last hope” medicines, is RTT the best path to follow? The new law did not erase or replace the FDA’s expanded access (EA) program, also known as “compassionate use.” You have to choose which program will serve your interests best.

Both programs work to give access to investigational drugs, which are not yet approved by the FDA, to those who are critically ill and do not qualify for any clinical trials. In many cases, such medications are oncology meds. But keep in mind that the success rate for cancer drugs in trials is very low.

“The chances that a drug makes it from Phase 1 trial through Phase 3 and to market is 10% or less,” according to ASCO (American Society of Clinical Oncology) Chief Medical Officer Richard Schilsky, MD.

The differences between the FDA’s EA program and RTT are important and can affect your success with the drug, your quality of life and your finances. Take the 35 minutes to listen to this podcast from ASCO discussing the differences.

Here is a cheat sheet to review while listening to the podcast — but please don’t use this in place of listening to the podcast and discussing this in detail with your doctor.


Drug company has to offer the medicine to a patient No No
Patient is responsible for costs of the medicine Yes Limits in
Insurance will cover the cost of the drug No No
Patient can try drugs after Phase 1 trial
(Tests for toxicity only)
Yes No
Patient can try drugs after Phase 2 trial
(Some testing on effectiveness completed)
Yes Yes
The drug company has to warn patient about known risks No No
FDA is involved No Yes
FDA warns patients on known risks, side effects No Yes
FDA tells patients what is known about benefits No Yes
FDA gives guidance to doctor about dosage, effect on other patients No Yes
Centralized location for information drug trial use
(Expanded Access Navigator)
No Yes

Whatever route you take, remember to ask your healthcare provider about the possible benefits (Will it give you a few more months? Will it cure you?) of an experimental med.

Just as important, ask what the likely side effects are (Will the quality of your life improve or get worse?). You are the only person who can decide if the hoped-for benefits outweigh the risks.

Suzanne B. Robotti

Suzanne Robotti founded MedShadow Foundation in 2012. Learn more about Su and her mission.

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