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‘Right To Try’ Advocates Urge Patient Education

Law in N.C. and 25 other states allow terminally ill patients faster access to new drugs

Christina Sandefur of the Goldwater Institute in Phoenix, Ariz., discusses 'Right to Try' Monday at the John Locke Foundation's Shaftesbury Society. (CJ Photo by Don Carrington)
Christina Sandefur of the Goldwater Institute in Phoenix, Ariz., discusses 'Right to Try' Monday at the John Locke Foundation's Shaftesbury Society. (CJ Photo by Don Carrington)

Advocates of North Carolina’s new “Right to Try” law — and similar ones in other states — are raising awareness of the law, which allows terminally ill patients who have exhausted other procedures the opportunity to try new drugs that gone through part but not all of the Food and Drug Administration’s approval process.

“North Carolina is a Right to Try state,” one of 26 across the nation, said Christina Sandefur, executive vice president of the Goldwater Institute in Phoenix, Ariz., during a March 28 presentation to the John Locke Foundation’s Shaftesbury Society. “The problem is, we need to get the word out.”

The advocates hope that as more people learn about the Right to Try option, more states will adopt it. Lacking that option, families like Diego Morris’ in Phoenix, Ariz., have had to uproot themselves to get life-saving drugs.

When Diego was 11, he was diagnosed with osteosarcoma, or a bone tumor, in his left leg.

“Fortunately, we had two very close friends who are physicians,” said Diego’s mother Paulina. The doctors worked with the family to research the disease and optional treatments. They learned there was a drug available to help prevent recurrence of the cancer, but it was not available in the United States.

After undergoing 10 weeks of chemotherapy in Phoenix and having surgery at St. Jude Children’s Research Hospital in Memphis, Tenn., doctors found that the chemotherapy treatment had killed only 50 percent of the tumor. Diego’s prognosis wasn’t good. “It tends to come back in the lungs,” Paulina Morris said.

Diego concluded his treatment in 2012, two years before Arizona approved its Right to Try law. While the FDA allows a “compassionate use” exemption of unapproved drugs for people with life-threatening illnesses, the family learned that they wouldn’t be able to start using the new drug in time to save Diego’s life, because treatment with the new drug, Mifamurtide, or MTP, needed to coincide with postsurgical chemotherapy.

The family communicated with physicians in Israel, Italy, and England, Mrs. Morris said. They went to Mexico City to talk to physicians there who’d used the new drug. They consulted with their congressional office, the drug company, and the FDA.

“We explored every single option and then decided we needed to go to London,” she said. They chose London because extended family members lived there, and there would be no language barrier.

The family spent nine months in London while Diego received drug treatments and chemotherapy. Mrs. Morris said her husband maintained his job in Phoenix to support the family and pay for the medication. He commuted back and forth to be with the family.

“We had plenty of trips to the emergency room,” Mrs. Morris said. “It was horrible for me not to have my husband there, and horrible for Diego not to have his father there.”

Diego, who is now 15, is doing fine, Mrs. Morris said. The family must make four trips a year to St. Jude in Memphis, in part to have the prosthetic in his leg adjusted.

Since concluding his treatment, Diego has become an advocate for Right to Try laws. He served as honorary chairman of the Right to Try petition initiative in Arizona and he’s told his story to a U.S. Senate committee looking into connecting patients to new treatments.

Paulina Morris also is involved in efforts to get Right to Try laws passed. “We were blessed that we were able to take off and go to another country,” she said. But she knows others are not as fortunate.

“We have to change the system here in the United States,” she said.

Spreading the word about Right to Try will save lives, Sandefur said. Patients suffering from terminal illnesses should ask their doctors if they are eligible to take advantage of the new law.

Last year, the N.C. General Assembly passed and Gov. Pat McCrory signed into law the state’s Right to Try bill. The new law passed the House and Senate unanimously and took effect Oct. 1.

The law allows terminally ill patients under certain conditions access to a new drug, biological product, or device that has completed Phase 1 of the FDA’s clinical trials but has yet to gain final approval.

According to the FDA’s website, Phase 1 studies determine a drug’s most frequent side effects, and how the body processes and discharges the drug. The emphasis in Phase 1 is on safety. Later phases focus on effectiveness and how the drug works with different populations, different diseases, and in combination with other drugs.

Under the new law, drug or device manufacturers are allowed but not required to make products that have completed Phase 1 available to patients. Companies making good faith efforts to comply with the Right to Try law are shielded from liability claims.

To be eligible for such a drug, a terminally ill patient must have tried or considered all other treatment options currently approved by the FDA. The patient’s treating physician must recommend the drug and attest that the patient meets all applicable criteria.

In addition, the patient is required to give “informed consent,” acknowledging that he or she believes all currently approved treatments are unlikely to prolong the patient’s life, and acknowledging the risks involved.

“What we really need to do is change hearts and minds,” Sandefur said. “When you’re fighting for your life, you shouldn’t have to fight the government, too.”