1 Many states have already passed Right-to-Try drug laws, which give critically ill patients the right to try medications that have not been approved by the US Food and Drug Administration (FDA). These laws have supporters and critics—and I understand you are one of the latter. Can you explain your opposition to Right-to-Try laws, specifically from an ethics perspective?
Caplan: Right-to-Try laws sound good in the abstract, but they are unethical in that they promise much, but deliver nothing. The laws give people a right to ask companies for drugs or devices that have gone through phase I testing without requiring permission from the FDA. But patients have always had the right to ask sponsors/companies for drugs or devices, and these laws do not obligate or compel companies to give anything. The laws are, unfortunately, quick-fix, feel-good political responses to a very serious, very complex problem. Since the FDA approves 99% of requests that companies present to them, the Right-to-Try laws essentially create false hope among the desperate. Tellingly, there is not a single example of anyone in a Right-to-Try state getting anything as a direct result of these laws. Nor do the laws budget any money to pay for drugs, transportation, hotels, time lost from work, or any other costs commonly associated with trying a novel drug. They are, at best, misleading in that they talk of rights but create absolutely no such thing.
2 Even though the FDA Compassionate Use program is meant to facilitate the same access to unapproved drugs as the Right-to-Try laws, there have been complaints that there are too many obstacles for patients to get the drugs they want to try at the time they need them. Are the Right-to-Try laws intended to correct these problems—and if so, how successful are they at doing this?
Caplan: The FDA is not the obstacle to compassionate use. The proponents of the Right-to-Try laws demonize the FDA, but it is almost never the problem. The FDA moves quickly in response to requests and recently made the process even more efficient by streamlining its website. It is pure ideology that blames the FDA for getting in the way of compassionate use.
The main challenge is the drug sponsors and companies rather than any regulatory agency. Companies and their boards may not want to get involved early on with compassionate use. Some companies are too small to manage requests. Some fear bad publicity if someone on compassionate use dies. Some expect payment for making a drug available. Right-to-Try laws do not address any of these issues.
3 What advice and/or tips would you give to clinicians with terminally ill patients who have exhausted all their therapy options and are interested in trying experimental drugs? What information is crucial for them to have before moving forward?
Caplan: They need to monitor the literature to see what trials are going on and should check ClinicalTrials.gov—although that site has many problems, among them a failure to update the status of trials that may be listed as open, but actually have closed enrollment. Visit the drug company’s website, if there is one, to see what they say about compassionate use. The patient should find out the name of the chief marketing officer, and requests should be made specifically to that person.
Requests ought to include all relevant medical and social data, including a statement that any treatment alternatives have been tried or, if not, why; the reasons that the patient cannot get into a clinical trial or employee assistance program; a comment on likelihood of compliance and the practicality of administering a new drug or biologic; a promise to provide information about what happens to the patient if compassionate use is granted; and all contact info for the doctor, patient, and patient’s key family member.
Financial Disclosure: Dr. Caplan serves as the non-voting Chairperson of the Compassionate Use Advisory Committee (CompAC), an external expert panel of internationally recognized medical experts, bioethicists, and patient representatives formed by NYU School of Medicine, which advises Janssen about requests for compassionate use of its investigational medicines.