'Right to try' continues to be a sham

'Right to try' continues to be a sham
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Four years ago, I got involved with investigational drugs and was credited with leading the successful social media fight to get a 7-year-old boy with cancer — who was given two weeks to live — an investigational drug that the manufacturer refused to give to over 300 terminally ill patients who had previously requested the drug. 

The child’s life was extended for two and a half years.

When the issue of giving investigational drugs to terminally ill children was raised at a conference I attended in 2014, I said, as has been stated by President TrumpDonald John TrumpHannity urges Trump not to fire 'anybody' after Rosenstein report Ben Carson appears to tie allegation against Kavanaugh to socialist plot Five takeaways from Cruz, O'Rourke's fiery first debate MORE in supporting the passage of what is known as the "right to try" law (RTT), “What difference does it make, these patients will soon die anyway?”

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After I raised the question, a pediatric cancer oncologist stated, “You would not say that if you witnessed children tortured after being given experimental drugs leading to their deaths.”

 

I have consistently opposed passage of RTT at the state level and have done so at the federal level. A federal bill, S. 204, was passed by the Senate in August.

Last Friday, the House Energy and Commerce Committee issued a revised version of the federal RTT bill that will soon be voted upon by the House. There is enormous pressure on House members to vote in favor of the revised bill from President Trump and Vice President Pence.

Surprisingly, President Trump in his State of the Union address and in his more recent speech to Republican members of Congress in West Virginia trumpeted the passage of RTT, naively stating as I did in 2014, “What difference does it make, these patients will die anyway.”

If passed, RTT would remove the Food and Drug Administration (FDA) from reviewing applications submitted by the drug sponsors.

Typically, drug manufacturers must first approve an application by the treating physicians to give investigational drugs to terminally ill patients. The proponents of RTT falsely state that the FDA unduly delays its approval for such drugs.

In my experience dealing with terminally ill children who have cancer, the FDA moves quickly when applications are received. I have been told that is the rule, not the exception, for all terminally ill patients.

It is true that the FDA approves over 99 percent of the applications submitted to it. What is not so well known is that in a random sample of applications, it was found that in 11 percent of those applications, the FDA made recommendations that were accepted by the sponsors to improve the safety of the drugs.

The FDA is the safety net that shouldn’t be removed from the equation. The revised bill, however, as does S. 204, removes the FDA from the equation in approving the drug for use by patients that qualify.

The revised bill, when compared to S. 204, also expands the population of patients that could receive an investigational drug. Under S. 204, “eligible patients” are those diagnosed with a life-threatening disease. 

The revised bill is more specific, stating that a patient is eligible to receive the drug if there is reasonable likelihood that death will occur within a matter of months. It continues, however, to add the alternative that the patient would be eligible if the disease would “result in significant irreversible morbidity [poor health] that is likely to lead to severely premature death.” I suggest that there are numerous diseases that would fall into the latter category, greatly expanding the use of investigational drugs that, under S. 204, were intended for terminally ill patients.

In addition, RTT excludes manufacturers, sponsors, their agents and representatives from liability. The sponsors are most often the drug manufacturers. That is not the case for the treating doctors (unless the treating doctors are sponsors), clinical investigators or hospitals, which can be sued for unintentional torts. The revised bill is more restrictive than S. 204 because it also included "prescribers, dispensers or other individual entity (other than sponsors or manufacturers).

That is why there are medical assistants, nurses, pharmacies, the Institutional review boards and others in the chain of distribution — including parents and guardians of minors who are given such drugs — who are not included in those covered under the revised bill.

The House should reject the pressure to approve the revised bill and allow the current system to do its job, under what is known as Expanded Access, with the FDA continuing to perform its function as the gatekeeper for the use of investigational drugs — and with those threatened to be sued continuing to have insurance and retaining the defense that the FDA as the safety net approved the use of the drug.

Richard L. Plotkin is the vice chairman of the Max Cure Foundation, which provides financial support to families battling childhood cancer while also supporting research and raising awareness.