Last week, House Representatives failed to pass “Right to Try” (RTT) legislation, a bill that purported to help terminally ill patients access experimental medications. Opponents of this legislation, including these authors, have long argued that RTT does nothing to bring potentially life-saving medications to patients who are in dire need and, potentially, puts desperate individuals at risk. With the bill’s failure, Republican leaders should rewrite the draft legislation in a collaborative fashion, taking input from the FDA, the biopharmaceutical industry, patients, patient advocates, and bioethicists who are experts in the field of pre-approval access, working to craft legislation that will actually bring help to patients in need.
The failed RTT bill was fraught with errors, poorly written, and lacked any concrete action to give access to potentially life-saving treatments for terminally ill patients outside of clinical trials. Currently, thirty-eight states, representing 83% of the population in the USA, have already 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, 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. Although over 270 million Americans are currently living within the boundaries governed with Right to Try laws, there continues to be no evidence of a patient ever receiving a life-saving medication under Right to Try legislations that they otherwise wouldn’t have received under the FDA’s current Expanded Access Program. The legislation simply doesn’t work, and it never will, for numerous reasons.
First and foremost, RTT does not obligate pharmaceutical companies to make their investigational drugs available to patients. While RTT states that it gives patients the “right to ask” for this access, this is a right that they have had for over 30 years through the FDA’s Expanded Access Program. Second, Goldwater falsely claims that companies are afraid of the FDA, which is why they don’t provide drug under the FDA program. No ethical biopharmaceutical company would be more apt to provide their experimental medicine outside of clinical trials under RTT because that proposed pathway cuts the FDA out of the oversite of potential adverse events. In truth, 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.
As members of NYU’s Working Group on Compassionate Use and Pre-Approval Access, we have been trying to address the true barriers patients face when trying to gain access to potentially life-saving drugs. We have been steadfast opponents of this legislation since its inception, writing journal articles, op-eds, presenting at conferences and testifying at Congressional Hearings designed to seek feedback on the proposed legislation. We have advanced solutions that would address the barriers that exist for patients and address the concerns that pharmaceutical companies grapple with when they are asked to make their investigational drugs available at an early stage.
Our group, consisting of bioethicists, patient advocates, physicians, academics, pharmaceutical CEOs and former officials at the FDA, believe the FDA’s current expanded access program works but can be improved to further enhance pathways to make potentially life-saving drugs available for dying patients. We strongly believe the FDA’s role in expanded access is to ensure treatment safety, which helps to protect both current and future patients in need. To help pharmaceutical companies feel more comfortable providing their early-stage drugs to patients, the FDA should issue clear and specific information about what – if any – consequences the company will face if there is an unexpected or severe adverse event related to their product. In addition, the FDA should develop and implement a plan to address fears and misperceptions about expanded access for all stakeholders – industry, advocacy organizations, patients, doctors, and the public.
Lawmakers have spent a lot of time and effort promoting RTT legislation publicly while admitting behind the scenes that they know the law will have little or no effect on providing dying patients with the help they deserve. We strongly believe that lawmakers could have the broad and immediate impact they seek for patients by facilitating FDA partnerships with industry, academia, and healthcare and advocacy organizations to better inform all stakeholders on the expanded access process, including relevant reporting requirements. At the same time, lawmakers should empower the FDA to work with industry to see if real world evidence from expanded access can serve to advance drug development and supplement clinical trial data.
It is important to recognize that a key reason for the decision not to make an experimental medicine available under expanded access is that they may not know enough about the compound’s safety or efficacy, and unforeseen issues could threaten their drug development timeline due to FDA or Wall Street concerns and derail their ultimate ability to make the experimental medicine available to future patients. These concerns are a very real barrier for patients getting the access they need. It’s imperative that we make expanded access more appealing to big and small pharma and biotech companies alike. For some companies, particularly small ones, who may be unable to provide experimental drugs – even if they wish to – due to expense, legislators and industry must work together to make this financially feasible.
Finally, we believe clinical trials are the safest, most common, and most valuable way for patients to get access to experimental drugs. Legislators must promote awareness of, access to, and equity in, trials to better support all stakeholders help patients get the access they need in an expeditious fashion.
The Goldwater Institute and other proponents of RTT have promoted this failed legislation as a law that “helps patients get immediate access to the medical treatments they need before it’s too late,” and have characterised Right to Try as legislation that “restores life-saving hope back to those who’ve lost it.” While this utopian vision of access to medications is laudable, it’s misleading, unrealistic and simply does not work. If lawmakers truly want to help patients find hope, they should immediately take action, collaborate with the experts in the field who deal with pre-approval access on a daily basis, and draft and pass legislation that will truly address the root causes of the barriers dying patients face while seeking access to potentially life-saving drugs. Our diverse working group stands ready to help make that happen.
Kenneth I. Moch is CEO of Cognition Therapeutics, a company developing new medicines for Alzheimer’s disease, and has been the co-founder or CEO of five companies developing therapies for life-threatening diseases.
Andrew McFadyen is 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.
Arthur Caplan, Ph.D. is a professor and founding head of the Division of Bioethics at New York University Langone Medical Center.
All three authors are members of the NYU Langone Health Working Group on Compassionate Use and Pre-approval Access (CUPA).
Good read and very well-written. Good to know how we can actually bring potentially life-saving drugs to patients in need and how the stakeholders can support and help patients get the access they need in an expeditious fashion.
The practical application of this heart-felt and well-intended strategy is that it must be implemented by an institution that by tradition and nationally mandated rules, administers medication based certain attributes. These attributes require research for defining a predictably beneficent, risk versus benefit outcome for the any therapeutic strategy as a basis for informed consent. Most physicians have witnessed unusual, adverse reactions to newly FDA approved medications. Commonly accepted standards shared by many physicians would be to honor a degree of skepticism for two years after approval for the introduction of a new medication into their prescribing menu. There are rare exceptions as with new “break-through” medications.
Furthermore, even with evidence of harm, who is to assume any legal liability. Hospitals, doctors, and pharmacists could be affected. Maybe Congress itself would assume any liability, although there would be sensible, as well as self-preservation, resistance to this alternative. If passed, the unintended results may represent a further degradation among physicians that many have come to believe that their “autonomy” to “do no harm” is again discounted as unimportant within our nation’s healthcare.
During my 41 years as a Primary Physician, I also served as the Chairperson for a hospital’s Formulary Committee. The medical staff Committee evolved beginning with one hospital, to then 2 hospitals, then to 7 hospitals, and eventually to a 25 hospital enterprise during the last 1-2 years. Trust, Reciprocity and Cooperation were the operational commitments as we navigated the complex marketing of medications, 340B pricing, national compounding disasters, medication shortages,and the introduction of physician, EHR direct-entry for medication orders. Remember, all of these changes occurred as a result of changes initiated beyond the wall of any hospital institution, at all levels of complexity.
Finally, I wonder how many citizens have a clear understanding of how the achievement of Stable HEALTH actually occurs and what are the life-long interactions of various disruptive processes that degrade a person’s HEALTH. To even identify those associations, there is absolutely no currently recognized means to assess their combined level of degradation for a person’s resilience or endurance. I suspect that these will be more clearly identified as we apply the knowledge that already exists for assessing non-linear control systems within biologic homeostasis. Until then, I vote NO.