This is the third piece in a four-part series about Right to Try at the federal and state levels and the response from the private sector. The first piece defines Right to Try and explains its importance to patients. The second piece discusses the passage of federal Right to Try legislation. The fourth piece discusses Right to Try 2.0, which would expand the law to cover patients with rare diseases who need individualized treatment.
Less than a decade ago, the grassroots Right to Try movement was born out of the premise that patients facing a terminal illness should not have to beg the federal government for permission to save their own lives. The concerns of patients, doctors, policymakers and advocates led to a legislative solution: Right to Try, a law that puts the most personal healthcare decisions back where they belong, with patients and their families. Ultimately, 41 states passed Right to Try laws with overwhelming bipartisan support. At last, in 2018, the federal version of Right to Try was signed into law, protecting patients throughout the U.S.
Today, Right to Try is delivering on its promise: Patients across the country have access to treatments they wouldn’t otherwise have. But Right to Try is also paving the way for a better tomorrow by encouraging innovation in the private sector. It allows treatment manufacturers and doctors to treat, learn and innovate in exciting ways that wouldn’t have been possible under the bureaucratic healthcare system before Right to Try.
Long before Right to Try became federal law, physicians were already using it to save lives in the states. For example, Houston-based oncologist Dr. Ebrahim Delpassand successfully treated nearly 200 terminally ill neuroendocrine cancer patients using LU-177, or Lutetium Dototate, a drug that at the time had not yet received final FDA clearance for sale (though it is now approved). Dr. Delpassand had administered a successful clinical trial for LU-177 therapy for five years. But after the final trial phase was completed in 2015, the FDA decided it would not allow him to treat additional patients until the drug received final agency approval—a process that could take months or longer.
One of Dr. Delpassand’s patients, Marc Hayutin, was in the midst of his treatment (and doing well) when the FDA halted the program. Marc—who, before receiving treatment, was told he only had months to live—was desperate and out of options. But a few months later, Texas lawmakers adopted a Right to Try law, giving patients like Marc a new avenue to access this safe and effective therapy. Invoking his and his patients’ rights under the new law, Dr. Delpassand continued administering LU-177 to people suffering from neuroendocrine cancer. Before being treated under Right to Try, many of these patients were only given three to six months to live. But years later, many are still alive, and while Marc unfortunately passed away, the treatment gave him years instead of the mere months he was originally expected to live.
Without Right to Try, it’s possible that some of Dr. Delpassand’s patients might have been able to continue receiving LU-177. But they would have had to travel to a country where the drug was legally available. One of Dr. Delpassand’s patients, grandfather and outdoorsman Jim Burhorn, said that without Right to Try, he “would have had to go on disability to make trips to Switzerland” to get the lifesaving medicine. Another patient, Mark Angelo, would have been forced to travel to Europe “for this same treatment and follow-up appointments every three months.” But thanks to Right to Try, he was able to stay in the U.S. and spend the time with his wife and kids instead. “This law has been a life saver!” he exclaimed.
The adoption of the federal Right to Try law extended these opportunities to patients nationwide. And it has already proved a blessing. Retired Navy pilot Matt Bellina had been an advocate for Right to Try ever since he was diagnosed with amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig’s Disease) in 2014. ALS is a degenerative nervous system disorder that weakens the muscles. Patients ultimately lose the ability to walk, talk, eat and even breathe. There is no known cure, but there are treatments that can slow (and maybe even reverse) the disease’s progression.
By the time Matt was diagnosed, his illness had progressed too far for one FDA-approved treatment to be effective, but not far enough for another. His disease was too advanced for him to qualify for clinical trials. As a result, Matt’s condition continued to deteriorate. He couldn’t stand, speak or breathe without the aid of a machine. He was out of options—and out of hope. But after Right to Try became federal law, Matt was given access to an investigational drug called NurOwn. After the first round of treatment, he could speak and swallow, could pull himself up a standing position and no longer needed to sleep with a breathing machine.
Of course, nobody can know what the future has in store, but these dramatic improvements certainly mean the world to Matt, who has been able to better enjoy time with his wife and three sons. Most importantly, he was able to make that treatment decision himself, rather than be at the mercy of a federal bureaucrat. “For me, Right to Try is as much about moving the needle towards civil liberties as streamlining medical research,” he said. “Today I am a living example of how both are beneficial, and I want so many more people to share in the experience.”
Right to Try’s primary aim was to give desperate patients access to potentially lifesaving treatments—a chance previously enjoyed only by the fortunate few who were either accepted into a clinical trial or could afford to travel overseas for treatment. But it’s also paving the way for a better system of care for everyone by allowing drug manufacturers to provide investigational treatments on a larger scale and modernize drug development.
After Congress passed Right to Try, drug companies did more than treat individual patients—they began to create programs and processes designed to help entire groups of patients take advantage of the new law. In 2019, Epitopoietic Research Corporation (ERC) became the first pharmaceutical company to announce a formal program to treat patients under Right to Try. ERC is the manufacturer of ERC1671 (also known as Gliovac), a vaccine for patients with glioblastoma, an aggressive form of brain cancer with a five-year survival rate of only about five percent. Now, this innovative treatment, which uses “freshly extracted tumor cells” to “stimulate the patient’s immune system to recognize and reject cancer cells,” is giving scores of patients new hope, as ERC announced that it would treat patients under its Right to Try program rather than treating individuals sporadically.
ERC is not the only pharmaceutical company using Right to Try in innovative ways. NeOnc, a cancer biotechnology company focused on the nontraditional delivery of treatments (such as inhalation), issued a Right to Try policy to help patients who can’t participate in a clinical trial. And Throne Biotechnologies partnered with Anova, a company that helps patients access promising new treatments, to help people with life-threatening autoimmune diseases access stem-cell technologies under Right to Try. Another organization, Beacon of Hope, announced that it would be running a Right to Try program to treat ALS patients.
Right to Try has even helped patients receive better treatments through the procedures the FDA had before Right to Try was adopted. As Anova’s report explains, when “one investigational product is provided under Right-to-Try” without FDA pre-approval, federal bureaucrats have sometimes responded by granting approval to a second investigational product. In other words, giving patients an alternative has sometimes helped kick the old system into a faster gear.
Right to Try has also enabled companies to collect real-world data about everyday patients undergoing treatments, outside the rigidities of a clinical trial setting. The use of real-world evidence is becoming increasingly important in the clinical development process, and it can lead to a better understanding about the use, benefits and risks of treatment. Information about patient survival after receiving treatments under Right to Try is already being collected and studied. One study has shown that treatments obtained through Right to Try were more effective than the options available in traditional trials. And Right to Try treatment data can speed the approval of drugs, making them available to a larger population sooner. This year, the FDA granted fast-track status to a cell-based immunotherapy to treat aggressive brain cancer based in part on the real-world evidence collected from Right to Try patients.
Right to Try is improving and saving lives across the country, giving hope to the hopeless and empowering patients to make critical medical decisions on their own terms. But it’s also changing the way that the private sector administers, studies and develops treatments.
By dismantling legal obstacles that block patients, doctors and drug manufacturers from collaborating without a government middleman, Right to Try is doing so much more than safeguarding the right of individual patients to try cutting-edge medicines—it is unleashing the future of medical care, streamlining the systems by which patients can access treatments and transforming drug development.
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