By McFADYEN, HALL & McBRIDE-FOLKERS

The McFadyens in 2010. Gabriel, Ellen, Andrew and Issac, aged 6.

In both the House of Representatives and the Senate, legislators have introduced “Right to Try” bills, which purport to give terminally ill patients access to experimental medications prior to Food and Drug Administration (FDA) approval. Vice President Pence recently met with Right to Try advocates, expressing support for the movement in a tweet. Forbes has published perspectives from both sides on the issue: first, a Right to Try proponent, Nathan Nascimento, defended the legislation, while medical ethicist Arthur Caplan’s response illustrated why Right to Try is – at its core – bad policy. A key voice missing from this dialogue, and the one needed most, is that of the patient.

At The Isaac Foundation, we know firsthand what it means for a family to cope with a loved one’s rare disease diagnosis. Over the past decade, our drive to find a cure for Isaac, the organization’s namesake and the son of the foundation’s founder, evolved into a multi-faceted mission to help those suffering from any rare disease. It is from this vantage point that we strongly urge legislators to oppose these proposed Right to Try bills – not only for their inherent flaws – but from creating further inequality for vulnerable patients across the United States.

Right to Try legislation leaves patients’ access to the drugs and devices they seek to the discretion of pharmaceutical and medical device companies. Neither proposed federal bill requires pharmaceutical companies to make their products available to patients who request them. The legislation creates a landscape of unequal access to these potential treatments.

In truth, “Right to Try” is a misnomer and provides nothing to patients in need except the misguided belief that the legislation makes investigational medical products available. A more apt title would be “Right to Beg,” because this is the only “right” this legislation actually gives patients. And this right to beg – or, less provocatively, right to request – has been given to patients in need since 1987, by the very federal agency Right to Try advocates want to abolish – the FDA.

Under the FDA’s expanded access system, to get access to an experimental treatment, patients and their physicians must first petition the pharmaceutical company developing the drug or device they wish to use. Only if the company agrees to provide their still-in-development product does the request reach the FDA. Right to Try advocates insist the FDA is the main barrier to obtaining unapproved drugs, but the agency never gets involved until a company says yes to a request. Nothing in the new legislation changes the primary role of a company. When the FDA is involved in the existing system, it approves over 99 percent of the requests it receives only weeding out the crooks and flimflam artists trying to exploit the dying and their families.  A recent study of the FDA’s expanded access program found that 11 percent of requests were modified after receiving advice from the FDA reviewers about such issues as dosage or dosing schedule showing that the FDA really helps protect patients rather than hindering access.

To help patients, we must remember that it is the company making the new medical product that ultimately determines who, if anyone, gets access. What is a problem worth fixing is patients not knowing how to approach the right company or how to request access to a product. It isn’t difficult to see why patients with the most privilege – those who can leverage social media campaigns directed at the pharmaceutical industry or those who can have important people make phone calls on their behalf – are more likely to get access than are equally sick but less advantaged patients. The proposed federal law does nothing to fix this huge problem.

Much of the pharmaceutical industry performs dismally in making their compassionate use policies known to both patients and physicians. According to a recent report, only 19 percent of the companies investigated had publicly published their policies on compassionate use. A bill passed by Congress this past December aims to solve this problem by requiring that companies publish information on how make a compassionate use request, along with appropriate contact information. This requirement will hopefully level the playing field for access, but the bill provides no specific penalties for noncompliance, Congress needs to fix that as well.

The Right to Try movement is gaining support among the same politicians who wish to repeal and replace the Affordable Care Act (ACA). One measure discussed by Republicans looking to replace the ACA, is to create state-run high-risk insurance pools. The terminally ill would likely receive coverage for their medical care from these pools, while the healthy are insured elsewhere. This system is likely to increase insurance premiums for the sickest people in society. The notion that in certain states, these patients could also purchase unapproved drugs – from profiting pharmaceutical companies who benefit from being able to sell their investigational products to the dying – will create a dangerous inequality between those who can afford the price tag, and those who cannot.

From our experience in and with the patient advocacy community, we understand the unbearable burden of a potentially terminal diagnosis and can see the appeal of Right to Try legislation for those with nowhere else to turn. The Goldwater Institute, a think tank that wrote the model bill on which many state Right to Try bills are based, does a marvelous job of promoting its policy as the last chance for people to extend their lives. Goldwater claims that “Right To Try laws help patients get immediate access to the medical treatments they need before it’s too late,” suggesting their legislation “restores life-saving hope back to those who’ve lost it.”

This vision of access to medications for millions of Americans who desperately need them is laudable. However, our analysis of the state Right to Try bills that have been passed reveals that no one is getting access to anything that they could not have gotten without these laws.  The cruel reality of Right to Try is that it does not grant patients immediate access to any treatments. Right to Try traffics in false hope, and as advocates for desperate patients, we believe they deserve better.

As patient advocates we know that Right to Try laws can’t and won’t help our loved ones, some of whom are fighting for their lives. What supporters tout as a beacon of hope does nothing to change the reality for patients in need – and risks making Americans’ access to healthcare even more unequal..

We urge legislators to focus on measures that will provide assistance, not empty words, to those in need.

Andrew McFadyen is the executive director of The Isaac Foundation and a member of the NYU School of Medicine’s Working Group on Compassionate Use and Pre-Approval Access. Alexandra Hall is the Managing Director of Policy & Patient Support at The Isaac Foundation. Kelly McBride Folkers is a research associate at the Division of Medical Ethics at the NYU School of Medicine.