Facts About Right to Try
For terminal patients who have exhausted their conventional treatment options,
obtaining access to potentially life-saving investigational medications is often extremely
difficult. The patient can attempt to enroll in a clinical trial, but many of the sickest individuals
do not qualify. In fact, only 3 percent of cancer patients today are enrolled in clinical trials. For
everyone else, their only hope for obtaining potentially life-saving medications is to ask the FDA
for special permission.
Only about 1,200 people make it through the FDA’s “compassionate use” application
process each year. The process is complicated, time-consuming, and expensive. The first step in
the process requires a doctor to complete a complex and time consuming application. After the
doctor submits the application to the FDA, the manufacturer must also submit lengthy
documentation requirements. The FDA then has a month to review the submission and either
grant or deny the request, but if there are any questions the one-month clock starts over. After
the FDA approves a request, a separate committee not affiliated with the FDA, called an
Institutional Review Board, also must approve the patient’s use of the drug. There is no time
limit on when an Institutional Review Board must review and approve or deny an application.
Sadly, there are many documented cases of patients dying while their application is being
considered.
After a year and half of deliberation, the FDA recently shortened its application form. A
shorter application for the first step is helpful, but it only addresses one part of the approval
process. And ultimately, it’s still an application to the government to ask permission to try to
save your own life. If you have a terminal illness, you don’t have time for a multi-step
government process. If your child is dying from a terminal illness and you know there’s an
investigational medication that is already helping other children survive, a shorter form isn’t
good enough.
We need to remove barriers that limit doctors from providing the care they are trained
to give—and this is exactly what Right To Try does.
Right To Try allows terminally ill Americans to try medicines that have passed Phase 1 of
the FDA approval process but are not yet on pharmacy shelves. Right To Try expands access to
potentially life-saving treatments years before patients would normally be able to access them.
Under Right To Try, a terminal patient would be able to access an investigational
medicine if:
✓ The patient has a terminal disease and has exhausted all conventional treatment
options;
✓ The patient’s doctor has advised the use of an investigational medication;
✓ The medication has successfully completed basic safety testing and is part of the FDA’s
on-going approval process;
✓ The patient has provided “informed consent” acknowledging the potential risk of the
drug; and
✓ The company developing the medication is willing to make it available to the patient.
Right To Try includes important protections. The basic safety testing and informed
consent requirements protect the patient. And doctors and the manufacturer are protected
from liability if the investigational medication doesn’t work. But this is not protection from
medical malpractice.
Right To Try is already law in 41 states: Alabama, Alaska, Arizona, Arkansas, California, Colorado, Connecticut, Florida, Georgia, Idaho, Illinois, Indiana, Iowa, Kentucky, Louisiana, Maine, Maryland, Michigan, Minnesota, Mississippi, Missouri, Montana, Nebraska, Nevada, New Hampshire, North Carolina, North Dakota, Ohio, Oklahoma, Oregon, Pennsylvania, South Carolina, South Dakota, Tennessee, Texas, Utah, Virginia, Washington, West Virginia, Wisconsin, and Wyoming. It has passed with overwhelming bipartisan support in each state and has been introduced in 18 additional states. Right To Try isn’t a red or blue issue; it’s a human dignity issue. That’s why lawmakers from both sides of the aisle are coming together to protect their citizens’ right to try to save their own lives.
The FDA says providing dying people with investigational medications should be an
exception. We think it should be the rule. People fighting for their lives should have access to
medicines that could save them without needing a permission slip from the government.
For more information about Right To Try visit RightToTry.org. Or contact RightToTry@goldwaterinstitute.org or (602) 462-5000.