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The Risks of Citizen Science

Patient-driven drug development gives patients more control over their treatment options than they would otherwise have. Yet it is not uncontroversial that patients should have more control. When patients play a role in development, they expose themselves (or their children) to the risks associated with using an unapproved drug. Patients are not medical experts. Though their experiences with their own diseases and the collective knowledge of disease communities are valuable forms of medical data, patients still do not generally have medical degrees or PhDs in the relevant disciplines. Patients also are not held to the professional codes, regulations, and heightened standards of liability that prevent medical professionals from taking undue risks or exposing people to unnecessary harm. Historically, these codes and regulations evolved to protect patients from being deceived or exploited by untrained medical practitioners. A potential risk of legitimizing patient-driven development is that it would more generally legitimize laypersons role in medicine to the detriment of patients.

In this section I argue that the risks of patient-driven development cannot justify restrictions on patients who seek a greater role in their treatment. Still, patients should consider the risks of participating in the development process when they make treatment choices.

Four kinds of risks merit consideration. First, patients take risks when they access experimental medicines outside the context of the clinical trial. In these cases, I argue that adult patients are generally in the best position to decide whether the risks of an experimental drug are worth taking. Pediatricians, parents, and patients should collaboratively make treatment choices in the interest of pediatric patients, but in some cases, experimental treatment is a child’s interests. Second, patients may put other patients at risk insofar as patient advocates threaten the con?ventional development pipeline. Yet patient advocates are not obligated to restrain their efforts on the grounds that regulators and manufacturers may impermissibly slow or halt development in response to a patient’s reaction to an experimental drug. Third, patients may put other patients at risk by publicizing dangerous or misleading information. Patients who play a role in drug development are obligated to disclose their level of expertise, and if they do, it is not impermissible to encourage other patients to participate in the development process. The fourth risk, which I address in the next section, is that patients could undermine the clinical trial process by accessing experimental treatments.

Consider first the concern that adult patients could harm themselves by making dangerous choices to use experimental drugs. Even if some drug choices are dangerous, the judgment about whether a drug is acceptably risky is one that each patient is uniquely qualified to make. Though physicians, researchers, manufacturers, and regulators all have expert knowledge about the pharmacological properties of a drug, each patient is an expert about whether the medical risks are worth it, given his or her other values and overall prognosis. Just as patients are entitled to refuse treatment against a medical expert’s advice, so too do patients have rights against interference when they choose to try a risky treatment, even if medical experts recommend against it. When public officials prohibit patients from accessing treatment, they interfere with patients’ rights to make treatment decisions (Flanigan 2012). The general principle of respect for medical autonomy also supports deferring to patients who choose to use experimental drugs, even if that choice proves harmful to the patient.

Unlike competent adults, pediatric patients are not entitled to make their own treatment decisions. Rather, treatment choices for children should be made in the interest of the child. The American Academy of Pediatrics recommends that pediatricians seek assent from patients who are not capable of giving consent (e.g., children under age 14) and permission from their parents (Committee on Bioethics 1995). This means that pediatricians and parents of children with rare diseases should work together to make treatment choices in the interest of the child. These standards are not in tension with patient-driven drug development. Josh Hardy’s medical team supported his petition for access to brincidofovir. The Hempels worked with their pediatrician, Dr. Caroline Hastings, to administer experimental cyclodextrin to the twins through a catheter (Marcus 2015b; Thomas 2014).

Still, experimental drugs are more risky than standard treatments because researchers do not know what they will do. It is not always clear to parents or physicians if an experimental drug is in a patient’s interest. Some parents of children with NPC criticized the Hempels’ decision to provide the experimental drug on the grounds that they were taking unnecessary risks with their children (Marcus 2015a, b). One of the twins, Cassi, suffered complications related to the catheter implantation, even though it is typically a low-risk procedure. Cassi developed a blood clot at the site of the catheter that paralyzed the left side of her body.

Yet concerns about the risks of experimental therapy are not unique to patient- driven development. Pediatric patients in clinical trials face similar risks. Two other NPC patients who were enrolled in an NIH clinical trial of cyclodextrin also suffered complications from their catheter insertion. Even when the treatment does not encounter complications, there are risks associated with the new drug. Recent trials found that cyclodextrin infusion causes deafness for NPC patients, though it may also extend their lives (Marcus 2015a). Despite these significant risks, though, parents, patients, and pediatricians may still judge that the risks of failing to treat a progressive and fatal disease are greater. Policies that prohibit patients from making treatment decisions bear an even higher justificatory burden than other forms of paternalism because people’s interests in their health and treatment choices are especially strong.

Critics of patient-driven development also object that activist patients could undermine drug development and thereby harm other patients. They point out that patients are not the only ones who bear the risks of using experimental therapies or publicizing information about breakthrough treatments. In some circumstances, patient-driven development could slow or halt drug development. Parents in the NPC community worried that patients who used cyclodextrin outside the context of a clinical trial were legally required to report any adverse reactions, which could have caused the FDA to prohibit all further testing or to ban anyone with NPC from using the drug ever again. (Marcus 2015b).

These concerns are not unwarranted. In 2014 the FDA ordered the drug manufacturer CytRx to halt Phase II clinical trials of an experimental cancer drug after a patient died while using the drug outside the context of a clinical trial. Though CytRx later modified the trials and the hold was lifted, the incident illustrates how patients can disrupt the traditional development pathway by gaining compassionate access to an experimental drug (Carroll 2015). If so, patient-driven development may harm other patients by depriving them of the opportunity to use a promising drug that would have been developed were it not for a compassionate user’s adverse reaction.

Bioethicist Art Caplan expressed related concerns about creating a trial to accommodate Josh Hardy’s case. Caplan described the balance between promoting a drug’s approval chances and providing expanded access as “a tradeoff between the public good and self-interest” (Cha 2014). However, Caplan’s characterization assumes a false dichotomy. Public officials and researchers can accommodate patient-driven drug development alongside the traditional pipeline, though they may need to reform the current system to do so. Insofar as expanded access to experimental drugs threatens development, it is because (a) regulatory agencies will be reluctant to approve a drug if there were adverse effects outside the clinical trials, (b) manufacturers are held liable for adverse effects outside of clinical trials, (c) manufacturers will suffer reputational harms if they provide experimental drugs and patients have adverse reactions, or (d) refusing to participate in a clinical trial would compromise existing trials (Darrow et al. 2015).

The first threat to development, which regulators would halt development based on the experience of patients who access a drug outside of a trial, assumes that regulators do not trust their approval requirements. If the current approval system is justified on the grounds that clinical trial data is sufficient for establishing safety and efficacy, then regulatory agencies should not be reluctant to approve a drug based on patients’ experiences outside the context of a clinical trial. If data obtained outside the context of a clinical trial were grounds for halting a trial, then regulators should also rethink whether the existing trial system is sufficient to establish safety and efficacy.

The second threat to development is liability. Despite the recent passage of “right to try” legislation, most US states do not limit tort liability for manufacturers who grant patient’s requests for unapproved drugs. Manufacturers cannot require releases of liability as a condition for providing a patient with a drug under an expanded access program. Even if a manufacturer tried to secure a release of liability, courts might not uphold it if they judge that a patient who released the manufacturer from liability was coerced to do so out of desperation. In these cases, patient-driven drug development does not directly threaten clinical trials, but it could undermine development indirectly, if a patient’s adverse reaction requires that the manufacturer pay a costly settlement.

Manufacturers also expose themselves to potential reputational harms if they allow patients to access experimental drugs during the development phase. Bad publicity from a compassionate user’s adverse reaction could shake investor confidence or undermine the drug’s commercial prospects. Manufacturers are not required to support patients’ calls for greater participation in the drug development process, just as they are not more generally required to provide drugs to all patients who could benefit (Huebner 2013). On the other hand, as Josh Hardy’s example illustrated, manufacturers also encounter extremely bad publicity when they exclude patients from the development process by withholding access to experimental drugs.

Third, patient-driven development may lead some people to take undue risks and harm themselves. The online forums that are frequented by cancer patients like Dave deBronkart cite data and anecdotes that are not subject to rigorous peer review. Chris Hempel collected data from other parents whose children were using experimental cyclodextrin and posted it online. Though members of online communities generally disclose their lack of scientific expertise and the limits of the evidence, other patients may defer to unsubstantiated reports out of desperation. There are no guarantees that citizen-scientists will take appropriate measures to respect people’s privacy or to avoid creating unnecessary risks when they share and analyze medical data. Though patient-driven development should abide by ethical standards such as respect for privacy and standards of truthfulness, there are few mechanisms to enforce these standards on online forums.

Whether it is permissible for citizen-scientists to put others at risk by sharing information depends on the kind of information they share. Surely it is permissible for a single patient to discuss her experiences on an online forum. And when members of patient forums aggregate data and anecdotes alongside peer-reviewed studies, as long as they do not deceive readers about the credibility of each source, they do not wrongfully mislead anyone. Patients who are concerned about the quality of online information can consult their physicians or disregard whatever they find. Sharing truthful information about one’s own experience or providing access to information that is provided elsewhere is permissible. In these cases, as long as members of online communities share information in good faith and disclose their lack of expertise, they do not act impermissibly.

One may object to this argument on the grounds that laypeople are not capable of interpreting complex medical data, especially if they are in a vulnerable position (Darrow et al. 2015). Yet physicians, policy-makers, and researchers ought to presume that laypeople are capable of understanding information about their treatment options. Laypeople are required to interpret and educate themselves about complex medical data in order to give informed consent to approved therapies or to participate in clinical trials. Since there is little reason to think their capacity to interpret and understand information would be adequate in these circumstances but not in others, laypeople should be treated as if they are capable of understanding. Even if laypeople are not capable of understanding complex medical information and statistics, that lack of understanding may be a kind of learned helplessness that is caused by a lack of autonomy over their treatment options. Moreover, as Mark Harrington’s story illustrates, patients with rare and emerging diseases may be relatively more educated than nonspecialist physicians and other kinds of patients because they develop specialized knowledge that others have little incentive to acquire.

On the other hand, sharing medical information online is impermissible when it is deceptive or when sharing violates privacy rights. It is generally wrong to deceive people about potential treatments. It is especially immoral to mislead people who are vulnerable, as desperate and dying patients may be. It is also wrong to share personal information about someone without his or her consent, unless she was not entitled to control that information in the first place. Without delving into the justification for privacy rights here, whatever standards and privacy protections ought to inform physicians’ and parents’ disclosure of information about patients should also serve as guidelines for citizen-scientists who post information online. This may require that they remove identifying information about some cases or that patient communities refrain from publicizing information about individual genomes.

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