February 9, 2016

Parents testify in favor of legislation to allow investigational drugs to terminal patients

A House Committee has heard emotional debate from supporters of a bill that would allow makers of investigational drugs, biological products or devices to make them available to eligible terminal patients.

Representative Jim Neely (right) listens as Melissa and Steve Heil testify about how his bill could help their daughter.  (photo courtesy; Tim Bommel, Missouri House Communications)

Representative Jim Neely (right) listens as Melissa and Steve Heil testify about how his bill could help their daughter. (photo courtesy; Tim Bommel, Missouri House Communications)

The legislation specifies that it applies to patients who have a terminal illness, have considered all treatment options approved by the Food and Drug Administration (FDA), have received a prescription or recommendation from a physician for an investigational medical product and have given written informed consent for the use of that product or have had such permission given by a parent or legal guardian.

It would allow such patients access to medical products that have completed phase one of a clinical trial but have not been approved for general use by FDA and are still being investigated..

View the legislation, HB 1685

The proposal is sponsored by Representative Jim Neely (R-Cameron), a doctor at Cameron Regional Medical Center whose daughter Kristina has metastatic colon cancer.

“I’ve struggled with this for many years,” Neely tells the committee on Professional Registration and Licensing. “I’ve certainly seen people out there that I’m perplexed that we can’t move forward with a treatment plan. I’ve seen medications that have been highly effective that have been pulled from the market because somebody had an issue with it.”

Neely says the legislation is aimed at getting the makers of drugs or devices to allow patients to have them in hopes that providers will create more access to those products. It does not aim to impact federal law.

Committee Chairman Eric Burlison (R-Springfield) says the state should do what it can to allow patients who have no remaining treatment options approved by the FDA to try experimental options.

“If [the federal government] wants to push back then that’s their business but we should at least allow as much as possible within the discretion, especially that between the doctor and the patient.”

No one testified against the proposal.

Those who testified for it included Steve & Melissa Heil of Springfield, whose two-and-a-half year-old daughter is receiving an investigational drug treatment for a brain tumor.

“We are really excited about this bill,” Melissa Heil testified. “Terminally ill patients do not have time to wait for the FDA to approve things.”

The Heils testified that their daughter’s treatment has caused her tumor to shrink by 33-percent, but has been put on a clinical hold by FDA and will be offered to no more patients.

“We know of nine patients out there with brain tumors right now that are in line waiting for the FDA to open up this program,” Steve Heil testified. Eight of those, he says, are children.

Melissa Heil says if no more patients will be allowed to take the medication that has helped her daughter, its maker will cut off production for lack of demand.

“What we have in our home for her,” she says, “may be all that is left and it is only a couple of months until it expires, so we will most likely be using expired medication just to continue these results.”

Rick Suozzi then testified about his daughter who was a senior in college and died last year of brain cancer, after being rejected for several clinical trials for drugs that her family hoped could have helped her.

Suozzi again called the attention of the committee to the Heil’s daughter.

“Look at that girl there,” he told lawmakers. “You all must pass this bill for people like that. It’s too late for my daughter but it’s not too late for these people.”

Burlison says he plans to add an emergency clause to the bill, which would allow it to become effective immediately upon being signed by the governor, and to pass it out of committee next week.