“Right to Try” Act is Not Broad Enough

Sen. Ron Johnson (R-Wisc.) and others have introduced legislation that would allow drug companies to provide investigational drugs to terminally ill patients. Given that the FDA already has an expanded access program that allows seriously ill patients to use unapproved drugs, it is not clear that the proposed legislation really would help additional patients. There is a need, though, in that patients who have exhausted other treatment options should be allowed advanced access to drugs currently in clinical trials. Unfortunately, the legislation as drafted does not accomplish this goal.

A few changes should be considered. The draft law is unduly narrow because it applies only in situations where the patient lives in a State that has passed right-to-try legislation. If federal law is changed to allow expanded access, why not make this available to patients in all states on a uniform basis?

Second, the draft law applies only to patients who are “terminally ill.” This scope is too narrow. Patients with a serious illness (such as cancer) should be allowed expanded access even if their death is not imminent.

Third, the proposed legislation allows, but does not require, drug companies to make the drug available to patients. Drug companies are often reluctant to provide the drug because they fear that such provision might jeopardize the drug approval process. In any case, they lack an economic incentive to make the drug available. Legislation at the Federal level can and should require drug companies involved in clinical trials to provide investigational drugs to patients who do not qualify for the trial.

Fourth, the draft legislation does not require insurance companies (or Medicare or Medicaid) to cover the drug, if it is made available by the drug company. If the drug company chooses to make a drug available to a patient without charge, there is no problem, but the drug company may want to at least recover its costs in doing so. Health insurance should pick up the cost, as with any other drugs, and law should mandate this.

Finally, the draft law provides that adverse patient outcomes arising from expanded access may not be used by the FDA to delay or deny drug approval. This makes no sense. Suppose that patients receive advance access and die. Is the FDA really supposed to ignore that and go ahead and approve the drug if the clinical trial evidence calls for this, even if patient deaths under expanded access raise issues? This provision could in any event be removed if drug companies are required to provide the drug, because its main purpose is to overcome drug company resistance to making the drug available.

I have a personal interest in this topic. I am dealing now with an incurable blood cancer, and I would like to get expanded access to new drugs if I need them. At this point, I don’t meet the criteria for expanded access under FDA policy since (1) there are still clinical trials for which I might qualify, and (2) there are drugs available that I have not tried yet. So while I am ok for now in terms of having treatment options available, I would like to have additional options when those run out. There are a number of investigational drugs that I might try at that point. I might not meet all the criteria for the clinical trials in question, so I might need expanded access. Unless the right-to-try legislation is expanded along the lines I suggest, it wouldn’t be of much use to me or others like me.