Time for national Right to Try legislation

The goal of Right to Try laws is to allow patients with terminal diseases access to promising new treatments that are being safely used in clinical trials, but are not yet available on pharmacy shelves. The laws protect doctors and their institutions from the legal ramifications stemming from prescribing therapies that are not yet Food and Drug Administration (FDA)-approved to patients with no alternatives.

{mosads}As Darcy Olsen, president of the Goldwater Institute — the driving force behind the movement — said at a Senate hearing in February 2016, “Patients have the right to die; why don’t they have the right to live?” She is not alone; indeed, 28 states have passed Right to Try laws to date. If the laws are passed by the 10 additional states currently considering it, enough support for an amendment to the Constitution will have been garnered!

Under the current system, the FDA allows experimental drugs to be prescribed to patients under its Expanded Access programs, which include (1) single patient use — regular and emergency access; (2) intermediate-sized populations; and (3) widespread use. The latter two pathways require drug companies to submit formal protocols to the FDA as part of an Expanded Access IND (Investigational New Drug) or Treatment IND, respectively. For single patients, a Single Patient or Emergency IND must be submitted by either a company or, more typically, the patient’s physician, which includes the protocol governing the drug’s use. These are no small efforts; it is estimated that completing the paperwork requires 100 hours from a physician. The existing process is a great deterrent to the submission of a compassionate use request; so, too, is the 30-day waiting period, which is imposed in order to provide the FDA time to review the requests. In the time required to complete the paperwork and to provide the FDA time to review the request, the patient may likely have succumbed to his or her disease.

In February 2015, the FDA released a guidance document outlining a more streamlined process requiring just 45 minutes of paperwork, but the 30-day delay remained. The agency has not implemented the new process, because expanding the number of compassionate use requests is anathema to the FDA, which has publicly expressed this sentiment. In order to deflect critics, the FDA points out that the overwhelming majority of requests are approved. Indeed, from 2010 to 2014, just 33 of 6,082 requests (0.5 percent) were rejected. For terminally ill patients, their families and physicians, it is not about the rate, but the number. And they want more.

Drug companies have to agree to provide drugs and to submit Expanded Access and Treatment INDs. Typically, companies are very hesitant to do so, especially early in the clinical development phase, because compassionate use exposes them to great risk. Considering that terminally ill patients have multiple grave medical conditions, the potential for unwanted toxicity is high. Moreover, these events are likely not to occur in patients for whom the drug is ultimately intended; that is, patients that match those entered on the clinical trials. Just the same, however, adverse events that occur in compassionate use settings must be reported in the approved labeling with the drug, and severe events can even trigger a suspension of all trials — an IND hold — as was experienced by CytRx, a biotechnology company.

There are also costs to the companies for providing drugs: production, tracking dissemination and collecting information from its use, as well as legal liability. And there is little “upside” for the company, apart from the ethical satisfaction, because the results observed in compassionate use patients are not poolable with results from other studies due to the extreme heterogeneity of the patient population in the compassionate use settings. To an investor, compassionate use represents all risk for little, if any, gain. So, the companies need protection, as well, to participate in this labor of love.

Patients are tired of waiting for each state to pass laws that would likely be ignored by the federal government in the prosecution of doctors, families, hospitals and companies that abide by them. This would then require challenges in court, and ultimately, in the Supreme Court, resulting in a huge state-versus-federal-government battle. Patients don’t want battles; rather, they want access to drugs that might help them, even with the knowledge that these compounds have not been fully tested and might actually hurt them.

Therefore, patients have voiced demand for a national Right to Try Law. Rep. Matt Salmon (R-Ariz.) and Sen. Ron Johnson (R-Wis.) are listening. On Tuesday, May 10, Salmon is conducting a House briefing on H.R. 3012, the Right to Try Act of 2015, which “[b]ars the federal government from prohibiting or restricting the production, manufacture, distribution, prescribing, dispensing, possession, or use of an experimental drug, biological product or device that is: (1) intended to treat a patient who has been diagnosed with a terminal illness; and (2) authorized by, and in accordance with, state law.”

Also on May 10, Johnson is announcing his Senate proposal, which addresses similar issues.

Right to Try isn’t just for older patients dying of cancer — it is for patients, young and old, suffering from all manner of disease, who have no hope. For example, children suffering from cancer or from Duchenne Muscular Dystrophy (DMD) — which is a terminal diagnosis — as well as adults with Alzheimer’s and Parkinson’s diseases, or Amyotrophic Lateral Sclerosis (ALS), better known as Lou Gehrig’s disease. Named in honor of the New York Yankee great, who was called the “Iron Horse” for his rugged durability, ALS robbed Gehrig of his strength and rendered him unable to tie his shoelaces, forcing him to retire.

In 2012, Matt Bellina, a U.S. Navy pilot, was diagnosed with ALS when he was 30 years old. There are no effective treatments for ALS, which is a relentlessly progressive disease that destroys neurons that control muscle movement. Only one drug, Rilutek, is approved; it slows the progression of the disease in some patients. But there are no drugs that reverse the course of ALS, which is universally fatal. In a magnificent video of Matt and his wife, the two discuss their frustration with being unable to try experimental therapies. “The hardest thing for me in having a husband who is terminal would be growing old without him,” said Matt’s wife, mother of two young children. May is ALS Awareness Month, and Tuesday, May 10, is Virutal Advocacy Day.

Right to Try has the potential to help many patients who have no hope. Of course, patients would prefer that new drugs were formally approved outright by the FDA much more quickly, or under programs akin to conditional authorization and exceptional circumstances that are available in Europe.

Right to Try is a cry for help in every sense. It is about time that patients’ pleas are heard and real compassion shown.

Gulfo is the executive director of the Rothman Institute of Innovation and Entrepreneurship at Fairleigh Dickinson University and author of “Innovation Breakdown: How the FDA and Wall Street Cripple Medical Advances” (Post Hill Press). He has more than 25 years of experience in the biopharmaceutical and medical-device industries and is the former CEO of Mela Sciences. Follow him on Twitter @josephgulfo.