‘Right to Try’ is not a sham

On Feb. 13, an op-ed titled “Right to Try is a Sham” by Richard L. Plotkin was published on these pages, setting forth several arguments in opposition to the Right to Try, hereafter referred to as RTT, bills recently introduced in Congress.  RTT is now the law in 33 states.  It is not a sham, and understanding that requires some knowledge about the stark realities faced by terminal patients.

The pending bills in Congress are written to federally legalize implementation of state RTT laws, providing physicians, drug companies and others the clarity they need to start helping more patients.  The legislation does not reduce FDA’s existing authority to regulate the development and approval of new medicines, alter or replace the agency’s existing investigational drug access programs or create a federal RTT right. 

{mosads}State RTT laws allow doctors to prescribe medicines to terminally ill patients being tested in FDA-approved clinical trials that are not yet approved for marketing.  This means that patients who have run out of FDA-approved treatments and are unable to enroll in ongoing clinical trials have a pathway to access those same medications outside a clinical trial without needing to navigate a bureaucratic, time-consuming and dysfunctional FDA expanded access process – a process that rarely works and can take more time than the patient has left.

The drugs eligible for use under RTT laws are called “investigational” drugs by the FDA.  They have been extensively tested in laboratory and animal studies, and at a minimum have completed a Phase I clinical trial in patients with a terminal disease. 

The drugs are being carried forward into later phase testing because the drug company, the FDA, an Institutional Review Board, and the physicians leading the trial collectively agree that the drug is both safe and promising enough to give to larger numbers of patients in Phase 2 and 3 clinical trials. 

The patients allowed to enroll in the trials get a chance at extended life while a much larger number of patients with the same disease are excluded for reasons that have little or nothing to do with safety protections or their prospect for medical benefit.  Most are excluded because they don’t precisely fit the entry criteria, selected in no small part by the drug companies to maximize FDA approval prospects and ensure a competitive advantage in the market based on labeling.  Others who do qualify are excluded because the trials are too small to include everyone who would enroll if they could. It usually takes years to complete the trials, analyze the data, and apply for FDA approval. During that time, the excluded terminal patients die from their diseases without ever finding out if the investigational drugs they sought might have helped.

Mr. Plotkin claims that the FDA’s access programs already provide patients a way to access investigational drugs.  An analysis of FDA’s 2015 statistics on access approvals indicate that fewer than seven thousand patients, and possibly a lot fewer, were allowed access to investigational drugs through the FDA’s single-patient and group access programs across all serious and terminal diseases.  The American Cancer Society estimated that 589,432 patients died in the U.S. from cancer alone in 2015, at a time when the Pharmaceutical Research Manufacturer’s Association reported 836 investigational cancer medicines were in development. Clearly, only a small fraction of terminal patients with a legitimate medical need for an investigational drug succeed in gaining access through FDA’s mechanisms.

Mr. Plotkin thinks RTT laws do not provide a broad enough set of legal protections for participating entities, but that claim is far-fetched.  The provision of an investigational drug under RTT would be legal under both state and federal law, just as provision of the same drug in a clinical trial or through an FDA approved expanded access program is legal. RTT laws also provide specific, limited liability protections for drug companies, doctors, hospitals, etc.  There are no statutory protections for those persons and entities under FDA’s programs.

Mr. Plotkin correctly reports that the federal RTT legislation prohibits the FDA from using outcome information gained from investigational drug prescribing under RTT to delay or adversely impact the development path for the drug.  FDA agrees that outcome information from investigational drug access is generally not usable for evaluating safety and effectiveness, but has long refused to provide clarity on this issue in a written policy.  This has caused many drug companies to view access programs as a regulatory risk, limiting their willingness to make their drugs available.  The federal RTT law provides clarity on this issue for medicines provided under state RTT laws.

At any moment in time, thousands of terminal patients have run out of treatment options, are facing death in the near-term and are asking their doctors about investigational treatment options – learning in nearly every case that the investigational drugs they seek are locked behind a wall of FDA regulation implemented in a manner that severely limits the availability of investigational medicines to those who have no other options.

Congress should pass and the president should sign the federal RTT law to allow state laws to function without threat of federal interference.  

Mr. Walker is co-founder of the Abigail Alliance for Better Access to Developmental Drugs (www.abigail-alliance.org), responsible for regulatory, legislative and litigation efforts, scientific direction, advocacy for patient’s rights, spokesperson on regulatory and scientific issues, and author of policy papers, articles and editorials written to educate the public regarding the Alliance’s positions and initiatives.  Mr. Walker is a volunteer advocate and receives no compensation for his work in patient and regulatory advocacy.

The views expressed by this author are their own and are not the views of The Hill.