The Right-to-Try movement—advocates for faster access to potentially life-saving experimental drugs for terminal patients—has gained strong momentum, with the act recently winning easy passage in congress, and, at the time of this article, it is on its way for a senate vote. The legislation is widely anticipated to become law later this year.
As of this writing, 39 states have independently passed their own right-to-try legislation.
In the not-too-distant future, thanks to dedicated individuals at various levels, numerous qualifying cancer patients—those who have exhausted standard treatment options—will be offered easier access to experimental drugs potentially applicable to their particular disease.
Qualifying drugs have undergone basic safety testing and continue to be under study at the FDA, meaning they are going through various phases of clinical trials. In the case of right-to-try, they will be provided outside the trial setting. This is similar to the current compassionate use program, which also provides experimental drugs not yet approved by the FDA.
In some cases, these drugs may offer remarkable life-saving results. Sadly, for most, they will be deemed ineffective at best, and potentially dangerous. After all, these agents are considered ‘experimental’ because they are still being studied—they are not approved for specific conditions for which patients are trying to obtain them.
Bringing anticancer agents to market is a long drawn out, expensive enterprise, with clinical trials and a peer-review publication process. This is why right-to-try is so important. For example, in 2015, former U.S. president Jimmy Carter was dying from advanced melanoma that had metastasized to his brain. Had it not been for Carter’s access to a well-matched experimental drug, Carter would have certainly died.
Instead, Keytruda, the targeted immunotherapeutic drug, had a remarkable impact on his cancer. Today, at 93-years-old, Carter is thriving and continues to work hard at The Carter Center and as a minister at his church.
Right-To-Try Devotees
The Goldwater Institute, an Arizona based libertarian think-tank, created the template for legislation on which the state laws for right-to-try are modeled. The intent of RTT is to return control of medical decisions to a local level. Supporters include patients and their families, and patient advocate groups.
One ethical argument for RTT is that, in instances where patients have the right to decisions, such as physician-assisted suicide or voluntary euthanasia, those patients should also be afforded the right to try unapproved treatments as a valid alternative.
I support the right-to-try bill because, on balance, I believe the pros outweigh the cons. However, there are also several important unanswered questions—the unknowns—that may tip the scale in a negative direction. Here’s my basic understanding of the nuance of right-to-try (RTT):
Pros
- RTT allows easier, less cumbersome, and conceivably faster patient access to experimental drugs than the existing Compassionate Use (CU) program, which, since 2009, has received approximately 1000 requests per year. (To the CU program’s credit, 99% of the requests have been approved, and in some cases with requested changes to support patient safety.) RTT will circumvent having to wait for certain agreements. Currently, under CU, a patient’s physician, as well as the respective drug company, must agree to provide the experimental agent(s). This can take some time. Those with advanced, terminal disease do not have time on their side.
- Certainly there are more than 1000 patients annually who have exhausted standard treatment and are interested in other potentially life-extending options. A negative to providers, but positive to patients, is that the CU process involves largely ‘non-billable’ (uncompensated) work on behalf of oncologists to investigate experimental options, make recommendations, and file paperwork. Right-to-try aims to create easier access and a less onerous, time-intensive process to match patients with applicable experimental therapeutic options.
- Drugs have undergone basic, phase I testing for safety. (This is also listed as a con.)
Cons
- There is no direct oversight from FDA, and therefore fewer opportunities to track and capture data that would be useful to inform drug development. Some say no or greatly diminished FDA oversight is a good thing, but I believe a better balance of FDA involvement would improve the final bill. Importantly, under the compassionate use program, the FDA tracks the results of treatment to judge safety and efficacy. In fact, right-to-try will greatly reduce, or eliminate, that opportunity for oversight.
- If a patient dies from an experimental drug under right-to-try, the FDA cannot consider the incident (or incidents) in deciding whether to approve or reject the therapy. This may allay the pharmaceutical and biopharma developers’ concerns that expanded access could threaten ongoing trials, but is viewed by many as unethical.
- Reporting of adverse events (negative reactions to therapies other than death) will also not affect ongoing clinical trials. This was also a prerequisite from drug-makers; they do not want to risk negatively impacting their ongoing clinical trials.
- Drugs have undergone basic, phase I testing for safety, with just one single trial being required. One trial does not necessarily provide a guarantee of safety, but can speed up the process of patient access, which is why it is also listed as a pro.
- Ultimately, there is a high potential of the treatment/drug not working, while possibly leading to complications, reducing quality of life, and even leading to a death faster than choosing no treatment.
Unknowns Abound
There are myriad questions related to the administration and long-term results of RTT.
- Would patients undergoing a drug therapy through the right-to-try act still qualify for hospice care, or would they lose coverage?
- Will insurance companies be responsible for patients (clients) and all clinical outcomes from a financial standpoint ‘after’ right-to-try clinical care? What if the experimental drug causes hospitalization or leads to additional treatment? Who will be financially responsible for this care?
In Summary, it will be interesting to see what changes will be made to the legislation before the senate votes on its future. My best guess is the FDA will be given some level of oversight power that ultimately adds more safety mechanisms, while allowing for a speedier approval process as compared to the existing CU program. In other words: increase the safety and oversight process while eliminating the cumbersome application and protracted delays of experimental therapies for those patients for whom time is not on their side.
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