Science Challenges for Law and Policy
The “Right to Try” Investigational Drugs: Science and Stories in the Access Debate
In 2014, states began enacting laws giving terminally ill patients a “right to try” investigational drugs. Right-to-try laws are the latest policy development in a decades-long struggle between advocates of liberal access to investigational drugs and defenders of access restrictions. According to access advocates, physician opinion and minimal safety testing are an adequate scientific basis for allowing terminally ill patients to try investigational drugs. But science and policy experts are virtually unanimous in criticizing right-to-try laws. According to the experts, more rigorous scientific and regulatory oversight is necessary to justify wide patient access. In defense of their position, experts cite data on investigational drug risks and low success rates, as well as the public interest in a rigorous drug-evaluation system. Access advocates use a different strategy, however—one that highlights stories of patients and families pleading for investigational drugs. These stories strongly influence legislative and public opinion on access policy. To mount an effective response, experts must tell stories illustrating the harm that liberal access can produce. In this arena, experts must convey their concerns in ways that are meaningful to lay decision makers. Introduction In 2014, several state legislatures confronted a novel policy proposal. They were asked to consider bills recognizing the terminally ill patient’s right to try investigational drugs. The bills sought to allow patients to use investigational drugs without the United States Food and Drug Administration’s (FDA’s) permission. Five states adopted right-to-try laws in 2014, and by early 2015, right-to-try bills had been introduced in many other states. Right-to-try laws are the latest policy development in a decades-long struggle between advocates of liberal access to investigational drugs and defenders of access restrictions. According to liberal-access advocates, physician opinion and minimal safety testing are an adequate scientific basis for allowing terminally ill patients to try investigational drugs. But scientific and other experts dispute this claim. Right-to-try laws would do more harm than good, experts say, by exposing patients to risky and ineffective agents. The laws would also pose an unacceptable threat to the larger group of patients who benefit from receiving drugs that have undergone thorough human testing. Experts predict that if terminally ill patients can easily obtain investigational drugs, fewer patients will be willing to participate in the clinical trials that determine which drugs can actually help patients live longer and better lives. Experts are nearly unanimous in opposing right-to-try laws. In defense of access oversight, scientists, FDA officials, and policy experts cite data on investigational-drug risks and low success rates, as well as the need for a rigorous drug-evaluation system. But in the access debate, data and abstract policy considerations go only so far. Access advocates use a different strategy, one that highlights individual patients’ stories. To support their cause, access advocates offer heartrending accounts of terminally ill patients seeking investigational drugs and deceased patients who were denied such drugs. These stories strongly influence legislative and public opinion on the access question.4 It’s inevitable that patients’ stories will shape public and legislative opinion on access policy. But policy decisions should take into account the full range of patient experiences with investigational drugs. Stories illustrating the harm that can come from liberal access belong in the debate too. An adequate response to the right-to-try campaign will require experts to vividly describe the negative impact that liberal access can have on patients.
In this Article, I examine the right-to-try controversy and the role patient stories play in the debate. Part I reviews the history of investigational drug regulation and current rules governing investigational drug access. In Part II, I describe ethical and policy arguments for and against liberal access rules. To illustrate the potential benefits and harms of liberal access, Part III presents a variety of patients’ access stories. Part III also considers interview and survey data examining patients’ attitudes toward and experiences with investigational drugs. I conclude by urging experts to address access in ways that are meaningful to legislators and the public. Experts should use stories and other information on patients’ experiences to describe the full range of effects that investigational drugs can produce. Such information is essential to developing access policies that truly promote patients’ interests. I. Investigational Drug Regulation A. Drug Evaluation In 1938, Congress passed legislation requiring manufacturers to file applications with the FDA attesting to a drug’s safety prior to marketing the drug.5 To gain approval, manufacturers had to submit a New Drug Application for FDA review, giving the FDA the opportunity to bar the drug from being sold if it concluded that the drug was unsafe.6 In 1962, Congress strengthened the FDA approval standard, demanding evidence that drugs were both safe and effective for medical use.7 Federal law prohibits manufacturers from shipping drugs in interstate commerce without FDA permission.8 Evidence from human trials is necessary to demonstrate drug safety and effectiveness.9 But before drug sponsors10 may conduct a trial, they must obtain from the FDA an Investigational New Drug (IND) exemption allowing them to ship drugs for testing purposes.11 Agency officials review clinical-