From The Editor | March 21, 2018

RTT Legislation: Where Do We Go From Here?

Ed Miseta

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

RTT Legislation: Where Do We Go From Here?

In what likely came as a surprise to many who were expecting congress to pass a federal Right-To-Try (RTT) bill, the legislation was defeated by a vote of 259-140. The vote was a disappointment to many prominent Republicans, including President Trump and Vice President Mike Pence, who both supported the measure.

The controversial bill, which would provide terminally ill patients with a path around FDA rules currently in place for compassionate use access, was opposed by most Democrats, including Minority Leader Nancy Pelosi and Rep. Frank Pallone, the ranking democrat on the House Energy and Commerce Committee. In the end, only 32 democrats voted for the bill while just two republicans opposed it.

The next step forward for RTT legislation is unclear. At this time, the three most likely options are Republicans taking up the measure anew, or working with democrats to include some of the provisions in a soon-to-be-unveiled omnibus spending measure.

Many in the industry, including myself, have argued that the bill would do little to bring potentially life-saving medicines to patients in need. With this defeat, now seems to be a good time for politicians to look for a new way forward. The Health Care Blog recommends Republican leaders collaborate with the FDA, the pharma industry, patients, and patient advocates to rewrite the draft legislation. According to the Website, the bill was fraught with errors, was poorly written, and lacked concrete actions that would actually provide patients with potentially life-saving treatments.

The Health Care Blog was not alone in its assessment of RTT. Many groups, including patient advocacy organizations, ASCO (The American Society of Clinical Oncology), and the Working Group on Compassionate Use and Pre-Approval Access (CUPA) at the New York University School of Medicine (NYUSOM) all opposed the legislation.   

CUPA was certainly one of the more vocal opponents of the bill. A statement released by the group after the vote noted it was pleased the House did not advance the bill. While it felt terminally ill patients should have every tool at their disposal to seek access to investigational products, including those not approved by the FDA, the group also felt the RTT bill would not accomplish that goal. 

Debunking The Claims

CUPA felt proponents of RTT had made many claims about the bill that were simply untrue. First, some proponents claimed the FDA’s existing Expanded Access program only benefitted around 1,000 patients per year. While many requests submitted to the FDA are for one patient, some are for more than one patient. CUPA notes some will represent thousands of patients. Neither the FDA nor any other entity tracks how many patients are represented via these large-scale programs.

Second, many RTT supporters claimed that drugs completing Phase 1 trials are generally safe. CUPA notes many in the pharma community agree Phase 1 testing is a very low bar to set for drug safety. These trials enroll a small number of volunteers, normally healthy individuals who are paid for their efforts. Also, since compassionate use requests come from patients with serious, life-threatening illnesses such as cancer, certain types of cancer drugs cannot be ethically tested on healthy volunteers. It is also unlikely that serious adverse events would be detected on such a small number of subjects. Additionally, around 75 percent of drugs that pass Phase 1 testing ultimately fail due to issues related to safety of lack of efficacy.

Finally, RTT proponents will claim that RTT laws at the state level are already helping patients. So, why not have a federal law that would help all patients. CUPA notes this talk has centered on one physician who used a state law to treat patients. Although the doctor claims to have treated almost 100 patients under RTT, the investigational product was also available through the FDA’s expanded access program. In fact, the product has recently been approved by the FDA.     

What Should Be Done?

Last year, Lisa Kearns, a senior research associate at the NYUSOM’s Division of Medical Ethics and a member of CUPA, penned an article for The Hill noting 17 real and workable proposals to truly help patients gain access to unapproved treatments. Among her proposals were strengthening the FDA’s expanded access program, preserving the FDA’s role in the process, and requiring the FDA to expand its current Guidance Document on Expanded Access to include clear and specific information regarding the consequences of an adverse event occurring in the context of expanded use.

The recommendations included using the Prescription Drug User Fee Act (PDUFA) to require the FDA to develop an outreach plan to address fears about expanded access across the industry and to better educate healthcare professionals and researchers on the expanded access process and reporting requirements. There are also smaller companies that will be unable to provide experimental drugs due to resource, financial, or supply constraints. RTT did not address the financial feasibility of the legislation for those companies.

Most importantly, there are now 34 states with RTT laws that have created confusion for healthcare professionals and organizations about whether to follow state or federal rules governing experimental drug access.

There are few in the industry, including supporters and detractors of RTT, who would argue that a terminally ill patient should not have access to a potentially life-saving drug. If we can all agree on that, then we have an opportunity to come together, hear the concerns from both sides, and come up with a solution that is acceptable to all. Now is the time for our representatives to put politics aside and do just that.