From The Editor | June 14, 2017

Could RTT Ease Access To Critical Drugs?

Ed Miseta

By Ed Miseta, Chief Editor, Clinical Leader
Follow Me On Twitter @EdClinical

Could RTT Ease Access To Critical Drugs?

In May 2016 the Trickett Wendler Right To Try (RTT) Act was introduced by Sen Ron Johnson (R-Wis). The goal of the legislation, currently supported by 46 cosponsors in the U.S. Senate, is to ensure terminally ill patients, their doctors, and pharma companies were allowed to administer investigational new treatments to patients when no alternative treatment exists.

In response to the bill, the NYUSOM (New York University School of Medicine) Working Group on Compassionate Use and Pre-Approval Access released a paper noting there were inherent problems with the RTT approach. The proposals are based on three years of feedback received from patients, patient advocates, life sciences industry, healthcare institutions and providers, ethicists, and venture capital. The key findings were that RTT legislation would not allow patients to get drugs faster, as the FDA approves 99 percent of compassionate use requests in one day or less. Furthermore, the paper notes FDA actually helps, rather than harms patients. RTT, it contends, could place drugs with little safety and efficacy information in the hands of physicians.

Of course, not everyone agrees with the findings of the Working Group. One of those individuals is Michael S. Smith, Vice President of the Gastroparesis Patient Association for Cures and Treatments, Inc. and a patient who has lived with Chronic Intestinal Pseudo-Obstruction for nearly 30 years. Regarding the assertion that RTT laws will do nothing to help patients, Smith could not disagree more.

“Close to 30 million Americans suffer from incurable chronic illness,” he says. “These patients are not worried that President Donald Trump or VP Mike Pence are ready and willing to pursue FDA reform. Nor are they concerned about the core mission of the FDA being abandoned. They are concerned about receiving treatments for their disease which are currently being made available by the FDA.”  

Smith knows the issues faced by these patients firsthand. Almost 30 years ago he was assaulted in high school. As a result of that assault, he sustained permanent nerve and muscle damage to his digestive tract and autonomic nervous system. His condition is today known as Chronic Intestinal Pseudo-Obstruction, and is treated by a family of medications known as the GI prokinetics. Medications such as Propulsid and Zelnorm help to modulate and reset the electrical rhythm of the human digestive tract. According to Smith, the side-effects of these treatments are more limited than total parental nutrition and intestinal transplant, the current end-stage treatments for digestive tract paralysis.

This is where FDA enters the picture. The agency has decided to withdraw or not approve both medications, as well as a third medication known as Domperidone. Domperidone, as well as Propulsid and Zelnorm continue to be used extensively and effectively outside the U.S. to treat gut paralysis. Smith notes a recent expose by the Goldwater Institute effectively outlines the concerns addressed by the non-approval of Domperidone.

FDA did have data to justify its decisions. The aforementioned medications were withdrawn from U.S. circulation as the result of Long QT Syndrome, a cardiogenic side effect.

“FDA initiated this withdrawal despite the fact that nearly 100 other medications with a similar side-effect remain on the U.S. market,” says Smith. “Those medications include Eryc and Benadryl. When I read the information on RTT put forth by the NYUSOM Compassionate Use Working Group, I can only think it will lead many patients in the U.S. to believe it is easy to get a medication that is subject to investigational new drug (IND) processes. Unfortunately, we have found that is not the case.”

Smith cites the IND process for Domperidone, which was recently amended and is now a 21 page package of documentation. There is a list of medications (two-pages long) that patients may not take in concert with a medicine that would allow them to eat and function while properly modulating a failed digestive tract.

“Today, for these patients to gain access to a needed medicine would require a three-step screening process, which would take six months to complete,” notes Smith. “The treatment process could potentially pull patients across the country multiple times per year. Those treatments would be administered by some of the nation’s leading experts in digestive diseases located at the Mayo Clinic and Johns Hopkins, and those physicians would be administering treatments they already know to be proper and appropriate based on the condition of the patients and based upon decades of research supporting the validity of the treatments in question.”  

 According to Smith, compassionate use rules in place at FDA are simply unacceptable. Children and adults with digestive tract paralysis deserve better. All they are currently getting to help with their digestive disorders is a decades-long policy debate.  Even without Right To Try, Smith believes at minimum in the need to establish an FDA Office of Patient Advocacy in which patient navigators employed by the FDA through the use of currently unspent PUDUFA funds would help to guide patients and their caregivers through vagaries of current IND process or provide additional support in conjunction with the NIH’s Magunson Rare and Undiagnosed Disease Program should the patient be denied IND access.

“These individuals deserve the right to hope for the treatments that are currently available,” he adds. “Right now government regulation is denying them that opportunity. RTT would provide them with better access to these needed medicines.”