FDA Streamlining Access to Investigational Drugs

What happens when a person is suffering from a life-threatening illness, and the only hope is an untested, untried and experimental drug? For decades, the answer was that experimental drugs were unavailable in the United States unless you were a participant in a drug evaluation program.

Tragically, thousands of patients who have no options other than non-FDA approved, experimental drugs simply do not have the right or ability to have access to them.

Now, the FDA is looking into changing the process and allowing greater access to investigational drugs under certain limited circumstances. Peter Lurie, Acting Associate Commissioner for Policy and Planning, announced February 4th 2015 the use of a simplified draft form that should accelerate patient access to investigational drugs.

This draft document will be used by physicians who seek FDA permission to use of an experimental drug or biological product. The term of art for such request is “compassionate use” of experimental drugs. While the proposed documentation for seeking the requests has been streamlined, the criteria have not. To meet the standard for this compassionate use, the need for the drug must be one of “last resort.”

• The disease must be serious or immediately life-threatening;
• There must be no treatment available, or no approved treatments have helped for the disease;
• The individual must not be eligible for clinical trials of the experimental/investigational drug;
• The doctor agrees that the individual has no other options and may benefit from the experimental treatment;
• The company that makes the drug must agree to provide it to the individual; and
• The doctor feels that the individual is healthy enough to tolerate the medication.

Some states have made this process easier by passing laws that would allow terminally ill patients to access drugs that have not been approved by the government. This is known as the “Right to Try” bill. Arizona passed this in midterm elections in November 2014 and joined the ranks of Colorado, Missouri, Louisiana, and Michigan. February 3, 2015, House of Delegates in Virginia unanimously passed similar legislation. It is suggested another 25 states will soon follow in suit, passing their own, “Right to Try” legislation. The “Right to Try” laws do not eliminate the FDA’s regulatory authority. The state laws essentially say that patients and their doctors may seek to use drugs that have passed the FDA’s first phase of safety tests. Right-to-try laws allow patients and doctors to negotiate directly with drug companies for access to experimental drugs.

Ultimately, there is a real need for the FDA to police the drug manufacturers who have often had a strong financial incentive to push drugs to market, notwithstanding the severe side effects. Of course, when people are clinging to hope that an experimental drug can help them, there is a strong chance that they could be deceived into taking drugs that are costly and only create additional pain – as opposed to cures – for their terminal disease.

Hopefully, the FDA can find a real balance by providing access to experimental drugs that can save lives, while protecting our most vulnerable citizens from spending their life savings on “cure” that simply is not there.

Alexandra Bhatti, student intern Jacoby & Meyers.