‘Right-to-try’ legislation has been around at the state level in the U.S. since late 2014. It became a federal law one year ago when, with typical ballyhoo and hype, President Trump signed Senate Bill 204 into law on May 30, 2018.
As he signed the bill, the president said that “… countless American lives will ultimately be saved. We will be saving — I don’t even want to say thousands because I think it’s going to be much more — thousands and thousands, hundreds of thousands, we’re going to be saving tremendous numbers of lives.”
That hasn’t happened. The number of patients who have benefited from state or federal right-to-try laws can currently be measured in single digits, and it is exceedingly unlikely that the pathway Trump extolled will ever be widely used.
Hype quickly met reality in the right-to-try world. State and federal legislation targets the FDA as the key obstacle in giving the dying quicker access to unapproved drugs. That is simply ridiculous. The FDA does not make or control access to unapproved drugs. Those drugs are the property of the drugs’ sponsors: companies and academic institutions and their investors. The FDA can’t make them give anything to anyone.
Several real issues exist in getting very sick patients onto the pathway that right to try was meant to create. These include:
- Do patients have access to good medical care so they can be made aware of new drugs?
- Can they convince a doctor to help them seek access to unapproved drugs outside of a clinical trial?
- And, most crucial, is the company or sponsor developing a new drug willing to make it available?
None of those issues do, or ever have, involved FDA participation. All that the Trump administration and the rest of the right-to-try bandwagon have done in enacting these laws is creating feel-good photo ops while leaving people who are dying in the same lurch they were in before the ink had dried on the bills.
My colleagues and I at New York University’s Division of Medical Ethics quickly recognized that right to try could not succeed in meeting the hopes or hype vested in it. For doctors to be willing and able to seek access to experimental drugs, they need education about how to find new drugs and how to work with their sponsors. And for the pharmaceutical or biotech companies fielding these requests — who are the primary gatekeepers to these products — convincing them to be willing to grant access to unapproved drugs means addressing their concerns about possible negative ramifications of failure lest their investors bail on them. They also need to develop ways to permit access ethically and equitably, especially if they are unable to say yes to all requests for reasons of cost, drug supply, or inadequate infrastructure.
When Janssen, the pharmaceutical companies of Johnson & Johnson, approached me wanting input about how to handle requests for access to investigational drugs by individuals who were ineligible for clinical trials, I jumped at the chance to shove right to try into a corner and be involved in a realistic strategy. Our subsequent work with Janssen created a fair and ethical process for granting access to investigational drugs that works for patients.
As a result of Janssen’s willingness to examine its practices regarding single patient requests — starting with a drug for multiple myeloma then in Phase 3 trials — hundreds of people have gotten access to investigational Janssen drugs and vaccines they otherwise would not have been able to get. So what got done to make that possible while right-to-try laws continue to fail?
We worked with Janssen to form the first of what became several Compassionate Use Advisory Committees (CompACs). These are international advisory groups composed of bioethicists, physicians, and patient advocates who were educated about Janssen’s investigational compounds (under a disclosure agreement with information that was confidential and proprietary), who offer feedback on when and in what contexts specific drugs ought to be made available to whom and provide rapid consultations when emergency cases arise.
The CompAC quickly realized that in order to seek access to Janssen drugs still in trials, doctors and patients first had to find them. That meant advising Janssen on creating user-friendly websites across the vast Janssen internet system with clear, meaningful contact information to guarantee that those making inquiries would know they had been received and that a response would be forthcoming. This is in contrast to right-to-try laws, which do nothing to ensure that anyone can find anything or that inquiries will be met with timely responses.
The members of the first CompAC also realized the kinds of social media campaigns where families of children or dying adults try to get a drug by shaming or threatening a sponsor only discouraged its granting access. And social media campaigns are inherently unfair in that not everyone is “mediagenic,” not everyone knows how to mount a campaign, and not everyone draws attention from politicians or the crowdsourcing support often needed to pay for a drug or to travel to get it.
CompAC members asked Janssen to blind all inquiries to make a level playing field among those requesting access to investigational drugs. We also initially blinded the identity of the committee members to make social media campaigns pointless, something that right to try most certainly does not.
The original CompAC committee and its more recent spinoffs developed rules for distributing scare experimental drugs and vaccines. Some of the principles followed include do no harm, maximize the chance to extend both lifespan and quality of life, and insist that those requesting access try both approved agents and enroll in clinical trials when they can.
By supporting Janssen, the CompACs have helped give access to hundreds of people during the time that right-to-try laws have provided access to almost no one. Other companies, large and small, are now instituting similar programs that have already resulted in access to investigational drugs for hundreds more desperate patients. Wall Street investors seem comfortable with the process. The FDA does, too.
Compassion for people who are dying is ethically commendable. Duping them with ideologically driven laws that provide no help is not. When legislators get near issues of access in our health care system they need to know what is involved lest they leave our most vulnerable citizens with nothing but rhetoric and press releases.
Arthur Caplan is professor of bioethics and the founding head of New York University School of Medicine’s Division of Medical Ethics.