Downside of ‘right to try’ legislation
“Right-to-try” legislation billed as making it easier for terminally ill patients to obtain experimental drugs and devices is currently making its way through Congress.
The new legislation, which proposes to increase the availability of investigational treatments that are currently off limits under FDA regulations, is being met with varying reactions.
While advocates argue that individuals of sound mind should be the final arbiters of what they put in their bodies, opponents including the American Society of Clinical Oncology say that such legislation puts patients at risk of exploitation and does little to facilitate patient access to such therapies.
To what extent do current regulations make it too difficult for patients who have exhausted conventional therapies to gain access to experimental treatments? (Patients who are very sick and/or extensively pretreated are often ineligible for formal clinical trials.)
Arthur Caplan, PhD, NYU Langone Medical Center: Very little. Regulations are not the problem. The FDA has sped up its responses. The challenge remains getting industry to engage in compassionate use. In other words, it’s up to the drug companies to decide if they’re going to give out drugs. The FDA has no control in that.
George Grossberg, MD, Saint Louis University School of Medicine: Current “right-to-try” laws, which vary by state, make it difficult for terminal patients to gain access to experimental therapies since they don’t mandate physicians to prescribe and monitor such treatments. Such laws do not require drug manufacturers to make the drug available promptly, at a reasonable cost, and with a minimum of red tape or delay, nor do they mandate insurance coverage.
Manish A. Shah, MD, Weill Cornell Medicine: I think the major impediment is insurance — most insurance companies will not pay for treatment that hasn’t demonstrated a treatment benefit. If federal guidelines state that this should be considered an option, then insurance companies may follow suit, then allowing more patients to get such options.
Jacqueline French, MD, NYU Comprehensive Epilepsy Center: Typically in the epilepsy space this is not an issue of “regulations.” Rather, the access to the drug is determined by the inclusion/exclusion criteria of a clinical trial testing the drug. Often, patients who are very ill will not be included in a trial because they are too high risk.
What specific aspects of current regulations should be changed (if any) to increase such access?
Robert I. Field, PhD, MPH, Drexel University: It is not clear to me that access should be increased. Patients may be lured into trying unproven treatments, and clinical trial protocols could be compromised. These drugs are called experimental for a reason. They have not yet been proven to work. In fact, most drugs in the final stages of clinical trials never make it to market because the testing fails to show they are effective.
Caplan: Federal “right-to-try” legislation will do nothing to increase access. What is needed are better online databases and navigators to let patients, their families, and their doctors find trials, and the creation of financial incentives to encourage companies to give drugs, particularly small companies with the experimental ones, and the creation of a fund to help pay costs that families face for unapproved drugs or for travel and related expenses.
Grossberg: Increasing access would require minimum delay and red tape for these medicines to become available, with drug companies being mandated/incentivized to offer these treatments promptly and at an affordable price.
What safeguards should be in place to prevent patients from being exploited?
French: I understand that some “investigational products” that are actually sold to patients can show up on Clinicaltrials.gov as a “clinical trial” that they can enter. This is inappropriate and should be prevented at all costs.
Field: If we were to increase access to experimental medicines, I would suggest the FDA review requests to ensure that drugs have a sufficient likelihood of being found safe and effective before patients allow themselves to become guinea pigs.
Grossberg: Terminal patients and their families are definitely “at risk” for being exploited. They are desperate and may even agree to treatments that could increase pain and suffering and even hasten death. It is important that informed consent be obtained. As well, ongoing monitoring by licensed, specialty physicians for safety/tolerability of treatments used via “right-to-try” mandates needs to be enforced with regular reporting to the FDA.
Derek Raghavan MD, PhD, Levine Cancer Institute at Carolinas Healthcare System: The most important aspects in designing any “right-to-try” legislation are to ensure patient welfare and common sense in design. It is imperative that the people who craft this type of legislation actually understand the implications of what they are doing, and have some real experience and/or expertise, either from the clinical or patient perspective.
Caplan: Maintain the role of the FDA to keep an eye on ripoff artists and require collection of data for compassionate use to monitor outcomes that should expose charlatans — of whom there are not many, but there are some.