As rare disease patient advocates, we work with terminally ill patients on a daily basis. A new version of the ‘Right to Try’ bill, which purports to help terminally ill patients access experimental medication, is set to be voted on in the House on Tuesday. While we understand the appeal of Right to Try, we also know it will do more harm than good. It is our duty to patients in need to urge Congress to reject Right to Try.
Thirty-eight states, representing 83% of the population in the USA, have signed Right to Try bills into law. These bills align with the model legislation crafted by libertarian think tank, The Goldwater Institute. Promoted as providing “immediate access to the medical treatments” for terminally ill patients outside of clinical trials, the cruel reality with Right to Try legislation is that it will not grant patients the immediate access to treatments they desperately need – and it never has.
Looking past the myths that Right to Try proponents state ad nauseam, and looking past this legislation’s potential to create an unequal access to medication, the simple fact is: although over 270 million Americans are currently living within the boundaries governed with Right to Try laws – providing them with, as Goldwater claims, “immediate access to medical treatments they need” – there continues to be no concrete evidence of a patient ever receiving a life-saving medication under Right to Try legislations that they otherwise wouldn’t have received under the current Expanded Access Program.
Tomorrow’s vote on a newly crafted Right to Try bill from House Energy and Commerce Committee Chairman Greg Walden (R-Ore.) is more limited than Senator Ron Johnson’s earlier iterations, yet remains little more than a feel-good bill that will do nothing to change the landscape with respect to access to life-saving medications for patients in dire need. In essence, the landscape for access to medications for dying patients does not change tomorrow if a Federal Right to Try law is passed. Very clearly, those patients in dire need of help today will wake up tomorrow needing access to the same life-saving treatments, and feel the same despair when they are not given the access they need through Right to Try.
Why? For starters, nothing in the legislation obliges pharmaceutical companies to provide their experimental products to dying patients. Sure, patients have the right to ask for drugs – but they’ve always had that right under the FDA Expanded Access Program. Without this obligation, drug developers will have the same hesitation to provide their investigational agents under RTT than they do under the current FDA program, thereby leaving patients in the same position they were before the passage of RTT laws as they will be after – desperate and fighting for access.
Another flaw in this legislation is the assumption that drug developers don’t provide their products at an early stage due to their fear of reporting adverse events to the FDA, and the belief that such reports could have a negative impact on the future approval of their drug. In reality, all ethical pharmaceutical companies that we know want FDA oversight of their program as they seek to create safe and efficacious products in the fastest way possible. These companies bear all of the risk for making such products available at an early stage, and have every right to be cautious about how and when they make their products available to the patients in need. Including FDA oversight in such a process is necessary and, indeed, welcomed by industry as they work to meet the needs of both current and future patients in need.
We are not alone in this view, which is echoed by rare disease advocates around the nation, including the National Organization for Rare Diseases (NORD), who agree: “these bills would likely do more harm than good” and that “removing FDA from this process is not likely to facilitate increased access to investigational therapies.” The Association of Clinical Research Organizations (ACRO), meanwhile, released a policy statement opposing Right to Try as “deeply flawed,” while The American Society of Clinical Oncology (ASCO), which supports many terminally ill patients, likewise denounces Right to Try as “ineffective and potentially harmful to patients.”
So, if RTT isn’t the answer for our patients, what is? In short – broader collaboration between drug developers, the FDA, patient advocates, and patients to enhance the FDA’s Expanded Access Program would be the most effective way to bring more immediate access to life-saving drugs for patients in need. Protecting our clinical trials process while using “real world evidence” through an enhanced Expanded Access pathway is possible, and will certainly help remove the barriers that patients face as they seek access to the drugs they need.
Lawmakers should be spending their time helping make that collaboration happen because that is how we are going to save our dying patients. Patient advocates, ethicists, and industry agree: there are ways to structure successful Right to Try laws. We all, lawmakers included, must focus on those and not spin our wheels promoting or passing legislation like Right to Try that looks good, and feels good, but will do nothing for those in need. If we do, we are doing a disservice to a large and very vulnerable group of patients now and in the future, and the hope and help these patients have been waiting for will never arrive.
Andrew McFadyen is the executive director of The Isaac Foundation, a non-profit organization that provides support for patients and families impacted by rare and life-threatening diseases, including gaining access to pre-approved and potentially life-saving treatments. He is a member of the NYU Langone Health Working Group on Compassionate Use and Pre-approval Access (CUPA).
Alexandra Hall, MA, is the Managing Director of Policy, Patient Advocacy and Industry Liaison at The Isaac Foundation.