In 2017, Signals’ blog carnival focused on Right to Try (RTT) legislation – laws that allow patients with life-threatening conditions to ask drugmakers for medicines that have cleared some testing hurdles, but still haven’t been approved by the U.S. Food and Drug Administration (FDA). RTT legislation was first introduced in 2014, and Colorado was the first state to pass this law. When Signals last covered the topic, it had already been enacted by 37 U.S. states.
Since then, RTT legislation has been brought in at a federal level. On May 30, 2018, President Trump, signed RTT into federal law, creating a uniform system for terminally ill patients seeking access to investigational treatments. “They had no hope. For 44 years, they’ve been trying to get Right to Try… I got it approved and it wasn’t easy,” said Trump at a Cincinnati rally in August 2019. “They couldn’t get anything – they’d travel to Asia, if they had money. They’d travel to Europe, they’d travel all over the world hoping for a cure. If they had no money, they’d just go home, they’d die. They had no hope.”
How impactful has RTT been?
Though it’s true that, previously, patients would need to ask the FDA for permission to access experimental treatments, the FDA almost always said yes. Dr. Scott Gottlieb, who served as Trump’s FDA commissioner, told Congress in 2017 that the FDA had approved 99 per cent of patient requests. “Emergency requests for individual patients are usually granted immediately over the phone and non-emergency requests are generally processed within a few days,” he testified in October of 2017 before the U.S. House of Representatives’ Subcommittee on Health, Committee on Energy and Commerce.
In his full testimony, Dr. Gottlieb shared how his personal experience informs his understanding of the importance of new treatment options for terminally ill patients when approved options are limited. “As a cancer survivor who used a commercially-available combination therapy in an off-label manner, I understand, on a very personal level,” said Dr. Gottlieb. “Patients who are fighting serious or life-threatening diseases want the flexibility to try new therapeutic approaches, including access to investigational medical products, particularly when there is no other FDA-approved treatment option.” He went on to explain the FDA’s ongoing work in this area, highlighting the agencies Expanded Access program that helps patients access experimental drugs in a safe manner, and also highlighted work to simplify the review process for individual patient treatment requests.
Rebranding what already existed?
“Right to try. That’s such a great name,” Trump said while signing the bill into law at a press conference in May, 2018. “Some bills, they don’t have a good name. Really. But this is such a great name, from the first day I heard it. Right to try. And a lot of the trying is going to be successful. I really believe that. I really believe it.”
Given that 99 per cent of requests were granted before RTT, many immediately by phone, you could be forgiven for thinking that RTT simply rebrands what was already taking place. But removing the requirement to ask the federal government for permission in the first place has been welcomed by RTT advocates as a step in the right direction for patient autonomy.
Critics, however, argue that RTT laws undermine the existing regulatory framework that is designed to ensure patient safety. By bypassing the rigorous clinical trial process, there is a risk of exposing patients to experimental therapies with unknown or unproven benefits and potentially severe side effects. Equity of access is another concern: access to these therapies via RTT legislation often hinges on factors such as financial resources, geographic location, and the ability to navigate complex health-care systems.
Hear from both sides of the debate
Many of the posts featured in the 2017 Signals blog carnival remain relevant today. You can hear from patient Brian Sladek, who makes a strong case for patient access and RTT in A prescription of hope. Equally concerned with patients’ best interests though drawing a different conclusion, stem cell researcher Dr. Paul Knoepfler outlines why he doesn’t support RTT in Will Right-To-Try Morph into Right-To-Profit for Stem Cell Clinics?.
Though RTT is U.S. legislation, it exists in various forms around the world. In Canada, patients can access investigational treatments via a number of routes, including:
- Expanded Access Programs: These programs – sometimes referred to as compassionate use programs or named patient programs – allow patients with serious or life-threatening conditions to access experimental treatments outside of clinical trials, often under specific conditions and regulatory oversight. Patients can inquire directly with the pharmaceutical company developing the drug/therapy or their health-care provider to determine if such programs are available.
- The federal Special Access Program (SAP): Health Canada’s Special Access Program provides a potential pathway for patients to access drugs that are not yet approved in Canada but may be beneficial for their specific medical condition. The SAP allows health-care practitioners to request access to these drugs on behalf of their patients when certain criteria are met. Health Canada evaluates each request on a case-by-case basis, considering factors such as the patient’s medical need and the available scientific evidence.
- Provincial programs such as Ontario’s Exceptional Access Program (EAP): EAP facilitates patient access to drugs not funded on the Ontario Drug Benefit (ODB) Formulary, or where no listed alternative is available. To receive coverage, the patient must be eligible to receive benefits under the ODB program.
(Ed: Signals’ blog carnival will be back August 22, 2023, with a new compelling topic and a variety of bloggers. Save the date so you can join the carnival fun.)
Where do you stand on RTT almost a decade later? Let us know in the comments.
Cal Strode
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