The Dangers of Magical Thinking: Situating Right to Try Laws, Patient Rights, and The Language of Advocacy.
On February 6, 2017, the first federal "right-to-try" (1) bill, HR 878, "to authorize the use of unapproved medical products by patients diagnosed with a terminal illness in accordance with State law, and for other purposes" was introduced to the United States House of Representatives. HR 878 will join the right-to-try acts that have been adopted in the majority of the United States since Colorado passed the first such law in 2014 with unanimous bipartisan support (Access, 2014; Koontz, 2014). The bill proposes to offer hope to terminally ill patients, enabling them to use experimental medicines under physician supervision. As the Colorado bill's sponsor, Representative Joann Ginal (D) stated, "It [right-to-try] is an option other than death. It is a bill of hope, when all else has failed" (Koontz, 2014, n.p.). HR 878, however, has two additional provisions: no liability and no use of outcomes. These provisions protect manufacturers and prescribers from responsibility for negative effects or damages caused by the use of these medicines, and, further, prohibit the U.S. Food and Drug Administration (FDA) from citing information resulting from unapproved use to delay approvals. No provision in any existing right-to-try bill requires manufacturers to provide medical products for such use. In fact, most state right-to-try laws, like Colorado's, explicitly allow manufacturers and insurers to pass on the costs of supplying these products to patients. Some laws, also like Colorado's, prevent patients using experimental therapies from receiving hospice or other palliative care (Access, 2014).
A broader "Right To Try" discourse claims that the new laws will increase benefits to patients offered by the FDA's current Expanded Access (Compassionate Use) program (Table 1; Goldwater, 2016). The current Expanded Access program mimics a clinical trial and requires FDA to determine that "the potential patient benefit justifies the potential risk" and "the potential risk is not unreasonable in the context" suggested for the expanded use (2017, n.p.). Thus, FDA mandates an analysis to show that patients are not endangered without adequate justification, which seems reasonable given that nearly 90% of products that enter Phase 1 never make it to the market for reasons including unacceptable side effects and lack of therapeutic benefits (BIO, 2014). The complexities of the Expanded Access process are considered by some medical experts to be an obstacle to access (Darrow, 2015), a view shared by Congressional right-to-try advocates. Yet the implication in a broader Right To Try discourse that any medications "that have passed Phase 1 of the FDA approval process and remain in clinical trials" are potentially lifesaving (Goldwater, 2015, n.p.) runs counter to the realities of pharmaceutical development. Thus, right-to-try laws can harm patients and increase their financial liability while reducing their access to palliative or hospice care (Darrow, 2015; Access, 2014). Below, I discuss the implications of the broader Right To Try discourse, including right-to-try laws, as a potential danger to seriously ill patients.
My discussion takes as a departure point a model of articulation as posthuman inquiry. In "Articulation: A Working Paper on Rhetoric and Taxis" Stormer (2004) takes up taxis, or "the linkage of elements within a texts" (p. 260) "as a critical problem" (p. 262) and thereby identifies the separation between words (rhetoric) and things (materiality) as a cultural performance and not a necessary condition of meaning-making. Thereby, Stormer allows for new ways of understanding how bodies--both material and discursive--can be articulated in a context of posthuman, cyborg, and prosthetic ways of thinking. Drawing on the work of theorists such as Donna Haraway, Gilles Deleuze, and Bruno Latour, Stormer develops a model of "situated knowing" that accounts for the possible relationships between bodies and words (or things and words), establishing meaning in and through networks of posthuman actors. These relationships are not fixed, as John Lynch (2009) observes in "Articulating Scientific Practice: Understanding Dean Hamer's 'Gay Gene' Study as Overlapping Material, Social, and Rhetorical Registers." In fact, Lynch sees Stormer's model of articulation as a way of understanding not only how objects can be viewed "primarily as material or symbolic," (p. 437) but also how people can shift focus from one order to another to suit their discursive aims.
The rhetorical practices Stormer and Lynch examine seem to require a fixed point of reference, or at least the intention that relationships between objects and things should aspire to honesty and transparency. What happens when these relationships or intentions are murky? What happens to bodies, of people or knowledge, when scientific and medical practices are used out of context for political aims? I suggest that Right To Try discourse, unlike a significant earlier critique of FDA practice, ACT UP's FDA Action Handbook (1998), undercuts the possibility for what Stormer calls prosthetic thinking by eliding the material from its consideration of medical rights. I suspect that by focusing on a language of constitutional rights and an ill-defined belief in a right to health, Right To Try discourse participates in propagandistic practices like those that Edward Herman and Noam Chomsky (1988) characterize as manufacturing consent, or creating the appearance of personal agency by leveraging public opinion through propaganda. To support my argument, I draw on Lynch's model for discussing the "gay gene," in which he articulates scientific thought and its reception by accounting for overlapping material, social, and rhetorical registers.
I examine the possibility that advocates for right-to-try laws co-opt a language of advocacy and a right to health without material grounding for their claims. My reading maps a broader Right To Try discourse against the register of regulatory language then considers that reading in the light of ACT UP's activist critique of the FDA. A notable characteristic of right-to-try legislation is a division between its stated aim, to treat patients, and the likelihood that the treatment will work, a fundamental condition for responsible medical practice. While Right To Try discourse encourages reduced regulations and increased corporate protections, ACT UP asked the FDA to embrace bioethical principles and take up its regulatory function more strongly. Thus, ACT UP kept the bodies of the sick and of medical knowledge in the forefront of its mission, while Right To Try discourse masks the effects of its practices, as becomes evident when the relationships between words and things are examined more closely.
Conservative Ideology and a Right to Miracles
Despite its claims to protect the sick and bipartisan legislative support, right-to-try legislation is strongly associated with a conservative ideology of deregulation and the elimination of protections for the vulnerable. A key advocate for right-to-try legislation is the Goldwater Institute, a conservative think-tank that seeks to protect constitutional freedoms by litigating against government agencies to reduce interference in personal decisionmaking. Its support of personal decision-making has been tied to ethically fraught behaviors such as state's rights to discriminate against racial minorities or condemnation of trade unions as impinging the on rights of business owners. A nonprofit group founded under the aegis of the Gold-water Institute, RightToTry.org, published a legislation template consistent with existing right-to-try laws in dozens of states, suggesting a heavy influence of their political ideology (2016). Although political ideology may inform the Goldwater Institute's actions, their stated rationale for expanding access to experimental therapies appears to be emotional rather than political or scientific.
In "Dead on Arrival: 'Compassionate Use' Leaves Little Hope for Dying Patients," the Goldwater Institute begins with a powerful appeal to the emotional stakes of right-to-try laws:
YOU ARE DYING AND HAVE NO HOPE Your disease is 100 percent fatal. It's only a short time before it kills you. There are no treatments that have been approved by the federal Food and Drug Administration. There is a new therapy that could save your life. But it is still being tested in people who have the same disease in rigidly controlled studies called clinical trials that you are too sick to qualify for. It will be a decade or more before the new drug is available to your doctor. You will be long dead by then. What are you willing to do, and how much risk are you prepared to take to try to save your life? Those are questions thousands of Americans face every year after being diagnosed with a deadly disease for which there is no cure, at least none that has been approved by the FDA. For them, their only chance at survival will be to get access to an innovative new drug before it's too late. It may be a faint hope, or even a false one. But it is their only hope. The FDA's compassionate use program is supposed to be that one last chance (2016, n.p).
This harrowing narrative derives persuasive power from the desperation of the terminally ill, casting hope as an entitlement, and "that one last chance" (n.p.) as an obligation under the law, a constitutional right. The language skillfully impugns the FDA for failing to provide compassionate use for all patients because even a "100% fatal" disease should be treated in the cause of an undefined "hope" (n.p.). Yet the Goldwater Institute remains nonspecific regarding the practicalities of obtaining, or funding, the medicines that should provide this last hope. The only clear message is that the FDA should cease its rigid control of clinical trials and offer an option other than death to the dying. Absent from this account is the fact that nearly 90% of all experimental medicines in Phase 1 trials fail to provide a favorable set of benefits relative to risks (BIO, 2014), raising the possibility that these options may be more harmful than helpful.
In fact, the Goldwater Institute's narrative parallels Peter Brooks' (1976) "mode of excess," which, according to Linda Williams (1998), informs most popular narratives. In these emotionally overblown works, a struggle between good and evil results in highly charged actions resolved at the last moment by heroic intervention. Yet there is a key difference. In the Gold-water Institute's narrative, the struggle between life and death, the terminally ill and rigid controls that block access to hope, is interrupted when the FDA fails to take up a heroic role, even though "it is supposed to be" entering at the last moment to provide one last chance to a dying patient. In effect, Right To Try elides the regulatory register of biomedicine--the scientific and expert content of medical discourse and the clinical conditions of sickness--replacing it with a Manichaean dialectic that casts deregulation as the inevitable means to address to terminal illnesses. Thus, RightToTry.org manufactures deregulatory propaganda and substitutes emotional appeals for medical benefits. Unfortunately, a failure to account for the realities of medical research means that unchecked risk and faint hopes are substituted for thoughtful approaches to patient care. The relationship between words and things then collapses into the relationship between words and stories.
A critical feature of both the Goldwater Institute's narrative and Ginal's rationale for the Colorado right-to-try bill is a tacit definition of a right to good health or a cure that sociologist Deborah Lupton (2012) describes as characterizing popular views of medicine in the developed world. Advances in the fight against infectious diseases and to improve quality of life led to a series of expectations: "to feel well, without pain or disability, long after middle age... [for] all children to survive birth and infancy, all women to give birth with no major complications, all surgery and medical treatment to be successful" (Lupton, 2012, p. vii). These expectations, Lupton notes, are continually reinforced by ongoing advances and high success rates in delivering on these promises, creating an entitlement she describes as a belief in an "inalienable" right to healthcare (p. vi). A danger arises when expectations for high-quality healthcare are conflated with a belief in a right to health itself, which is impossible to guarantee, despite language from the World Health Organization (2017) suggesting that all people should have access to the best chance to attain health. This semantic slippage allows for a further displacement of the materiality of illness into a litigious language of abstraction that can do little for the sick or dying.
Consistent with the popular ethos Lupton defines, RightToTry.org's FAQ (frequently asked questions page) identifies the "right to life and medical self-preservation" as "constitutionally protected," despite the caveat that "the United States Supreme Court has never addressed Right to Try specifically" (2015, n.p.). The similarity of this language of rights to the Declaration of Independence's invocation of inalienable rights to "life, liberty and the pursuit of happiness," suggests that RightToTry.org may have conflated two important political documents. Such conflation lends weight to a claim that Right To Try discourse uses political language primarily to evoke emotional responses in support of an unrealistic model of healthcare rather than to guarantee constitutional rights.
A belief in FDA deregulation as an answer to incurable illness currently extends to the highest levels of government. In an address to Congress, President Trump characterized FDA regulations as creating barriers to healthcare because a "slow and burdensome approval process... keeps too many advances... from reaching those in need" (Trump, 2017, n.p.; see also Liptak, 2017). His solution, to "slash the restraints, not just at the FDA, but across our government," quickly shifts from a quest to help the sick to the mystical assertion that "we will be blessed with far more miracles" (Trump, 2017) once the FDA is deregulated. In this construction, each experimental medicine is transformed from a possible solution in need of testing to an "advance" or lifesaving promise, regardless of biomedical reality. Ironically, President Trump's speech appends a necessary connection between medical miracles and deregulation onto a story about a man who saved his daughter's life by following FDA regulations and the basic principles of biomedical research. Thus, the President's narrative, like the Goldwater Institute's, undercuts scientific inquiry, eliding the reality that only a small proportion of experimental medicines, fewer than 12%, advance from initial Phase 1 studies to approval, and even those medicines are not universally effective or curative (BIO, 2016).
The equation of experimental medicines with cures, or even miracles, in conservative political rhetoric is only one failure to manage factual information in the broader Right To Try discourse. For example, RightToTry.org justifies the use of unapproved medicines, at least in part, because they are safe. In addressing whether right-to-try therapies are safe, the FAQ (Goldwater, 2015) begins with an unqualified, "yes":
The only treatments available under Right to Try laws are those that have already passed the FDA's basic safety testing and remain within the FDA's approval process. These are no different than the treatments currently available to the 3% of patients who are lucky enough to be accepted into clinical trials. (n.p., emphasis in original)
Here, the evidence for asserting that experimental treatments are safe is that the FDA continues to investigate these medicines. The very same "slow and burdensome" process that prevents access to medicines is the basis for establishing their safety and usefulness to the dying patient. Thus, the same regulatory activity is simultaneously a barrier to and the necessary support for the use of these experimental products.
The Regulatory Register: A Stepwise Approach to Safety Testing
One problem with the model of "basic safety testing" that informs right-to-try laws is that safety and tolerability measures are meaningful only insofar as they are balanced by therapeutic benefits--the benefit-risk analyses that the Goldwater Institute and President Trump find to be burdensome. Safety, like therapeutic benefits, must be tested in a stepwise fashion, and such testing is technically never finished. The drivers for a stepwise analysis of the benefits and risks of experimental therapies are not merely pragmatic, but based on bioethical principles.
As noted by the International Council for Harmonisation of Technical Requirements for the Registration of Pharmaceuticals for Human Use (ICH) (2016), benefit-to-risk assessments are a core concern in evaluating medicinal products. The principle of ensuring that benefits outweigh risks in clinical research dates at least to the Nuremberg Code, and is reflected in the World Medical Association's Declaration of Helsinki (2013), first issued in 1964 and updated periodically to address current ethical concerns in medical research (see Shuster, 1997). The FDA's construction of bioethical responsibilities also includes the principles of beneficence, autonomy, and social justice outlined in the Belmont Report (National Commission, 1978). One of the three main purposes of the Belmont Report was to outline appropriate measures for assessing benefits and risks to research subjects. An important distinction exists between patient care--interventions intended to help individuals based on a high likelihood of success--and experimentation, which should be based on an appropriate balance of benefits and risks intended to protect the research subject from harm (National Commission, 1978). Unlike the language in "Dead on Arrival," the bioethical codes followed by the FDA emphasize that biomedical research is potentially dangerous and that vulnerable persons should be protected from undue risk.
One measure of research protection mandated worldwide (not simply in the United States) is that medicinal products be investigated in laboratory and animal models prior to clinical studies, a discipline called "Safety" by the ICH (2016). In this context, Safety indicates that sufficient nonclinical evidence exists to support small-scale trials in humans and also that likely harms are balanced against the probable benefits for each different use and stage of preclinical and clinical development (ICH, 2016). Phase I studies are used to identify basic product characteristics, like extent of exposure and metabolism, and possible drug interactions before specific uses are investigated in humans. Due to the nature of these investigations, Phase 1 studies (Table 3) may be needed to clarify findings at any phase of clinical development or even after a product is marketed. Thus, Phase 1 is never technically "passed," as suggested by RightToTry.org (ICH, 2016; U.S. FDA, 2015a, 2015b, 2016). Further, since most right-to-try laws specify only that a product has been studied in "a" Phase 1 study, even the assurance in the FAQ above--that the product has "passed basic safety testing"--is overstated in part because more than one such study is needed to justify further investigation. Further, Phase 1 trials generally do not include patients with life-threatening illnesses, which casts further doubt on the accuracy of RightToTry.org's overall narrative.
Proof of concept, or evidence of therapeutic benefits in people with a specific condition, and appropriate dose selection are assessed in Phase 2 studies. If data suggest that the benefits will outweigh harms, then researchers may apply to a regulatory body, like the FDA, for permission to start Phase 3 studies, large-scale trials in a specific indication, (e.g., Stage 2-3 non-small cell lung cancer, severe asthma). Pivotal Phase 3 studies provide the primary basis for a product's general safety and effectiveness profile--the information that appears on product labelling documentation (U.S. FDA, 2016; Centerwatch, 2017). Once a manufacturer has amassed enough data, an application can be submitted for permission to sell the product, initiating the approval process. Even product approval is not a guarantee of safety, in part because Phase 3 studies include relatively few people compared with the general population. Rare side effects, such as those associated with Viagra, are generally undetectable until a product enters general use. In short, the stepwise drug development process is designed to limit exposure to experimental therapies until there is compelling evidence to suggest that their benefits outweigh their risks, but never guarantees safety (ICH, 2016). Designations for approved use are always qualified and limited, based on available information (U.S. FDA, 2015a, 2015b, 2016; ICH, 2016).
Right to Try discourse fails to account for the regulatory register (both language and process) and the bioethical principles that inform it. The result of this failure is a collapse of research realities into an overly simplistic view of safety testing--false equivalencies between testing and proof. Right to Try replaces meaningful evaluations of safety with the semantically faulty assumption that FDA only investigates "safe" therapies. Thus, right-to-try legislation prevents measures to ensure that patients and research subjects are protected from harm and that treatments administered have a high likelihood of benefitting a patient. When Right To Try discourse criticizes benefit-to-risk analyses as unnecessary (Goldwater, 2016), it ignores bioethical principles and patient protections integral to the clinical development process to advance a type of propaganda--language and stories designed to make emotional appeals not grounded in material reality.
Criticizing failed attempts to interpret regulatory language in this Right To Try discourse may seem unfair. After all, regulatory writing is often unclear to nonexperts. Yet, Right To Try discourse undercuts more than the language and process of pharmaceutical regulations; it also ignores a prior history of activism seeking to reframe compassionate use in order to help the sick. State laws mandating a compassionate right to use medical marijuana began to emerge in the 1990's, starting an ongoing trend (NCSL, 2017). However, these compassionate use laws came long after a globally impactful critique of compassionate use and regulated medical research mounted by ACT UP in the late 1980s. Experts in regulatory writing and medicine (see Benau, 2017; Sanghavi, 2013) accept ACT UP as a catalyst for advancing provisions to protect patients and to allow access to information about research studies. To do this, ACT UP generated a language of activism that successfully managed regulatory and scientific registers and kept the embodied welfare of the sick patient in the forefront of its rhetorical and material practices.
FDA Action Handbook: An Early Critique of Compassionate Use
In the 1980s, acquired immune deficiency syndrome (AIDS) was viewed as implacable, uncontrollable, and mysterious, creating public panic and anxiety. For decades, most dangerous infectious agents could be controlled with antibiotics, or at least vaccines, consistent with the narrative of medical intervention Lupton (2012) describes as motivating beliefs in an entitlement to health. AIDS shattered such biomedical expectations and their accompanying rhetorics. ACT UP responded by mastering regulatory and bioethical language and engaging the FDA, not to wrest away its power, but to exert pressure to protect the lives of the sick.
In "Before Occupy: How AIDS Activists Seized Control of the FDA in 1988," professor and activist Douglas Crimp (2011) recounts the vital importance of the mastery of regulatory and scientific discourse to the success of their FDA takeover, "unquestionably the most significant demonstration of the AIDS activist movement's first two years" (2011, n.p.). Crimp emphasizes that ACT UP's focus on the bodies of the sick was always central to its ideology; however, he credits successful management of two registers, regulatory and popular, with the ultimate effectiveness of the campaign. Mastery of complex information was not left to a few; the "entire body of ACT UP was schooled in advance with knowledge of complicated issues" (n.p.). A committee authored the FDA Action Handbook, which was used to educate the media and ACT UP membership about protest and other tactics. The ACT UP slogan "healthcare is right" invokes a concrete and moral call rather than an abstract, constitutional one, and it was linked to easily legible concepts and commonsense notions consistent with a normative construction of compassionate use. These rhetorical practices balanced and incorporated rhetorical, social, and material concerns.
Central to the FDA Action Handbook was a series of demands (Table 3) referenced against the then-current core document of biomedical ethics in the United States--The Belmont Report. The demands were based on two interrelated circumstances that ACT UP deemed unacceptable for research subjects: the failure of drug companies to follow the bioethical rules outlined in the Belmont Report, and corporate justifications for hiding clinical data (ACT UP, 1988). A critical feature of ACT UP's claims was the impossibility of respecting a right to patient autonomy while simultaneously withholding essential research data in an attempt to protect intellectual property and shore up corporate profitability. ACT UP successfully showed why failure to disclose unfavorable research data to protect proprietary interests ran counter to the primary clinical development goal of ensuring a favorable benefit-to-risk ratio. ACT UP called the FDA out on an apparent complicity with corporate interests, a failure to act in defense of the sick, and an omission in enforcing its own guidelines.
Where Right To Try discourse invokes the constitution, ACT UP combined more accessible moral language and values with bioethical and regulatory discourses, reinforcing rather than undercutting the FDA's regulatory function. In other words, ACT UP mastered the regulatory register in order to support its activist agenda by inducing an appropriate government body to engage in ethical behavior. For example, ACT UP's claims about unethical pharmaceutical research in the FDA Action Handbook are clearly tied to the Belmont Report and a reasonably accurate accounting of the drug development and approval process. By noting that treatment protocols in biomedical research should be required to follow basic principles for medical care, ACT UP helped eliminate many placebo-controlled study designs that harmed patients, and not just in the United States: the Declaration of Helsinki now mandates the use of a standard of care in all clinical research trials (World Medical, 2013). ACT UP also concentrated on benefitting patients and maintaining access to medicines that had helped people in clinical studies. In contrast, Right to Try discourses co-opt advocacy and patient care language by referring to all experimental medicines as "treatments" regardless of their actual effects (Goldwater, 2015).
ACT UP's FDA Action Handbook also thoughtfully links particular clinical features of AIDS--such as immune compromise--to the toxic effects of early experimental therapies, keeping the material reality of the disease in the forefront of its demands. Even though ACT UP refused to reduce persons with AIDS to mere sick bodies, those very bodies, or at least the people in them, remained central to this rhetoric. The FDA Action Handbook, and as Crimp indicates, ACT UP, never lost sight of the ultimate goal of their activism: saving lives.
Another fundamental difference between Right To Try advocates and ACT UP is their level of personal responsibility in learning about science and clinical development. The FDA Action Handbook successfully mobilizes biomedical terminology such as "toxicity," human "subjects," "open access," and "prophylaxis" that parallel usage in regulatory settings, lending weight to the argument that AIDS clinical study designs were unethical because they required avoidable patient harms and thereby abused the trust of trial participants. The demands in Table 3 also connect corporate activities to profit motives rather than an obligation to follow basic medical ethics. In other words, ACT UP's language of advocacy and autonomy undercut corporate interests to protect the sick and dying, producing an activist discourse that was legible in regulatory and biomedical registers as well as to patients. The Right To Try patient, in contrast, is cast as desperate to try any remedy, regardless of its likely effects.
Current regulations, guidance for industry, and the most recent iterations of the Declaration of Helsinki all support the demands initially made by ACT UP, not only for HIV-related research, but all clinical development. Today's clinical trial safeguards for patients--including access to a reasonable standard of care and disclosure of trial designs in registries--can be linked directly to ACT UP's work (Benau, 2017). Indeed, the current iteration of the FDA Expanded Access program also stemmed in part from these demands.
As noted in the FDA Action Handbook, then-current practices for gaining access to experimental therapies required opening an Investigational New Drug Application (IND). At the time, two different, complex IND processes were available for compassionate use. Yet ongoing adjustments and developments at the FDA meant that these processes and their regulation were somewhat fluid (ACT UP, 1988). Instead of focusing on these complications, ACT UP accounted for the most serious obstacle to access for experimental medicines--money. Insurance companies' refusal to pay for experimental medicines is one reason that ACT UP specified that clinical trial participants should receive therapies free of charge after studies ended. This position is in contrast to right-to-try laws that specifically allow companies to charge patients for experimental medicines and associated manufacturing costs. Furthermore, ACT UP demanded access to clinical trial data in order to support patient decisions regarding their own healthcare, while right-to-try laws seeks to prevent important clinical results from affecting access to medications or FDA evaluations.
ACT UP managed to constrain a potentially Manichaean narrative within a specific, limited, and therefore reasonably achievable set of objectives in the FDA Action Handbook, maintaining a focus on the material conditions of illness. Referencing the Belmont Report rather than the Constitution, ACT UP demanded a right to information and increased autonomy in specifically defined circumstances, privileging the interests of the embodied patient as well as body of biomedical clinical knowledge. This is not to say that many AIDS patients were unwilling to take great medical risks, but rather that the regulatory remedy that ACT UP sought remained grounded in intellectually and medically responsible practice.
Much of ACT UP's self-identified anarchist model of activism--a necessity at a time when discrimination against persons living with AIDS was legal--may seem to be at odds with some other discourses of patient advocacy. However, ACT UP's rhetoric shares a vitally important feature with the language of more mainstream patient groups: an emphasis on valuing both biomedical expertise and patient knowledge and needs. Advocacy groups often emphasize the unique value of scientific investigation by experts. For example, the current March of Dimes (2017) website differentiates personal, political, and medical interventions into polio:
President Franklin Roosevelt's personal struggle with polio led him to create the National Foundation for Infantile Paralysis.... Better known as the March of Dimes, the foundation established a polio patient aid program and funded research for vaccines developed by Jonas Salk, MD and Albert Sabin, MD. These vaccines effectively ended epidemic polio.... (n.p.)
In this description, President Roosevelt created a foundation whose activities were intended to supplement and support, rather than replace, regulatory functions and the vaccine research undertaken by Salk and Sabin. The vaccines ended the polio epidemic, enabling the March of Dimes to go on to address additional public health problems, such as infant mortality. This recognition of the distinct roles of biomedical development, organizational advocacy, and patient experience remains much closer to ACT UP's rhetoric than that of Right To Try discourse, which collapses expertise and reinvents biomedical science as a series of pathos-driven and patient-made decisions.
In its advocacy for greater access to compassionate use of experimental medicines, ACT UP created specific, evidence-based demands, educated its members, and mastered various rhetorical registers. Its success in refraining HIV/AIDS research and clinical development more broadly is a testament to the power of self-education and tenacity in the face of powerful corporate interests. In contrast, right to try aligns itself with conservative and libertarian values that emphasize the rights of corporations over patient safety, guaranteeing payment for experimental medicines and limitations of liability rather than cost-free access to safe therapies.
Material Implications of Right to Try
In Manufacturing Consent: The Political Economy of the Mass Media, Edward S. Herman and Noam Chomsky (1988) examine the operation of "systematic propaganda" (p. 1) in democracies and the ways in which those propaganda support economic and political inequalities. Mass media produce messages intended to entertain and educate the general public, "to inculcate individuals with the values, beliefs and codes of behavior that will integrate them into the institutional structures of the larger society" (p. 1). Thus, mass media can mask the operations of systems of power and privilege, a de facto oligarchy manipulating the workings of democracy for its own ends. I suggest that Right To Try discourse participates in a similar propagandistic system. Its Manichaean narratives remain nebulously unspecific about the embodied risk to sick patients and their financial ability gain access to medications. Right-to-try legislation instead privileges corporate interests and profit, guaranteeing protections from criminal and financial liability. As I observed in The International Journal of Clinical Practice (DeTora, 2017), right-to-try legislation is a potentially dangerous, unregulated form of direct-to-consumer advertising that co-opts a language of advocacy and ignores the plight of the sick by encouraging the use of products that are much more likely to fail than to succeed.
The language of Right To Try discourse draws on pathos-driven appeals to create a Manichaean dialectic that mimics popular melodramatic narratives founded on the belief that victim-heroes should be rewarded. Semantic conflation of the term "rights" tacitly combines constitutional freedoms with healthcare rights that are, as Lupton indicated, unfounded beliefs in universal entitlement to health. False equivalencies create misleadingly normative uses of terms like "compassion" or "safety" and build an inaccurate picture of right to try legislation and its putative benefits. Together, these elements contribute to a discourse that ignores the work of earlier activist groups, such as ACT UP, eliding their more accurate accounting for biomedical realities and contributing to language that reveals propagandistic tendencies.
In material terms, enforcing right-to-try legislation is likely to harm people. The disempowerment of the FDA, research ethics, and even practicing physicians by such laws has the potential to endanger public health as well as decades of advances in biomedical ethics (Darrow, 2015; Bateman-House, 2015). By mystifying biomedical research and recasting health and illness as a medical melodrama, these discourses mask a series of moves to reject patient rights as constituted by the Patient Protection and Affordable Care Act, and to eliminate the financial protections in ACT UP's conception of compassionate use. Furthermore, these activities may relocate funding for research activities from corporate profits to the resources of individual patients. Dangerously, this discourse participates in a more general war on science, creating narratives that build precedent for opining objections to empirical evidence, justifying laws that explicitly protect corporations while omitting provisions to directly benefit the sick.
In opening his discussion of articulation and taxis, Stormer (2004) notes that the gap between words and things is a cultural performance and that articulation creates various types of bodies as well as the spaces within which they can interact. Right To Try discourse eliminates certain material considerations--the bodies of sick patients and the realities of biomedical research--in favor of theories of constitutional rights and of limiting corporate liability. Unlike ACT UP, which successfully articulated cultural, political, and biomedical realities in its activist discourse, Right To Try treats serious illness as the setting for a melodramatic struggle about a right to medical self-preservation that ultimately protects corporations instead of the sick. By replacing biomedical knowledge with semantically faulty constructions of rights, right to try discourse rejects both the reality of physical bodies as well as conceptual bodies of medical knowledge, effectively eliminating the possibility for meaningful rhetorical articulation. As a result, Right To Try discourse creates a site of resistance, not to abuses of power, but rather to understanding and critique.
What, then, is the solution to this site of resistance with regard to compassionate use? One suggestion, by Art Gertel (2016) in "Preapproval access and right-to-try initiatives: What are we willing to give up?" is that a combination of personal accountability and an objective evaluation of costs and benefits is the clearest way forward on a policy and practical level. However, this solution leaves room for the rhetorical and semantic problems I noted above to create complications. Elsewhere I (DeTora, 2017) suggested that physicians and regulators review their use of terms like "safety" and create fewer potential sites for public misunderstandings of clinical development. However, these measures alone are not enough. I also believe that the community of rhetoric of health and medicine scholars must continue its work to interrogate discourses that claim to benefit the sick and to identify discursive spaces in need of attention in material, policy, and rhetorical registers. Only by accounting for both material and rhetorical realities can we protect the vulnerable from medical practice based on faulty assumptions about a right to health.
This paper was adapted from part of a talk presented at the Society for the Communication of Complex Information in 2017. Francis Crawley of the Good Clinical Practice Alliance provided invaluable advice. I am grateful to Lisa Meloncon and Blake Scott for their early conversation and advice and ongoing support for the development of this paper. John Lynch gave me invaluable advice during the peer review process. I also thank Art Gertel for providing me with a copy of his paper and helpful discussions.
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LISA DETORA is an assistant professor and the director of STEM Writing at Hofstra University. A long-time professional in biomedical and regulatory writing, Lisa's publications span biomedical, scientific, and humanistic venues. Her current works in progress consider bioethics, embodiment, vaccination, and graphic narrative.
(1) In this article I use "Right To Try" to reference the broad concept of the right to use experimental therapies, consistent with usage by RightToTry.org and other participants in public discourse about this concept. I use "right-to-try" to reference U.S. laws and legal discussion of this concept.
Table 1. FDA description of expanded access (excerpted from U.S. FDA Expanded, 2017, n.p.) Under the Federal Food, Drug, and Cosmetic Act (FD&C Act), a patient may seek individual patient expanded access to investigational products for the diagnosis, monitoring, or treatment of a serious disease or condition if the following conditions are met. * The patient and a licensed physician are both willing to participate; * The patient's physician determines that there is no comparable or satisfactory therapy available to diagnose, monitor, or treat the patient's disease or condition; * That the probable risk to the person from the investigational product is not greater than the probable risk from the disease or condition; * FDA determines that there is sufficient evidence of the safety and effectiveness of the investigational product to support its use in the particular circumstance; * FDA determines that providing the investigational product will not interfere with the initiation, conduct, or completion of clinical investigations to support marketing approval; * The sponsor (generally the company developing the investigational product for commercial use) or the clinical investigator (or the patient's physician in the case of a single patient expanded access request) submits a clinical protocol (a document that describes the treatment plan for the patient) that is consistent with FDA's statute and applicable regulations for INDs or investigational device exemption applications (IDEs), describing the use of the investigational product; and * The patient is unable to obtain the investigational drug under another IND or to participate in a clinical trial. Table 2. Trial characteristics by clinical phase articulated against Right to Try (compiled from U.S. FDA, 2016; Centerwatch, 2017; Goldwater, 2015; ICH, 2016) Phase Number of Purpose Participants 1 (*) Up to 100 To evaluate human pharmacology, possible drug interactions, and preliminary safety signals, usually in healthy volunteers. Right to Try suggests access to experimental medicines here 2 Up to 300 To identify an appropriate dose range for Phase 3 trials and to discover whether the product is likely to work as expected (proof of concept) in a particular, clearly-defined setting. Proof of concept and appropriate dosing are identified here 3 1000+ To establish a safety, tolerability and efficacy/effectiveness profile in the target patient population for a specific, defined use (or uses). (*) Additional Phase 1 studies may be conducted at any time in the clinical development process. Table 3. ACT UP's FDA Action Handbook basic demands (excerpted from ACT UP, 1988, section titled "Drug Trials are Healthcare, Too") 1) Persons with life-threatening illnesses are not reducible to "sick bodies" to be injected, studied, and abandoned. Federal statutes must mandate that all testing organizations enter a sustained, responsible relationship with all test subjects through the person's life. 2) Subjects in a drug trial must not be forced to discontinue concurrent prophylaxis as a condition of participation; death or progression of opportunistic infections is an unacceptable corollary of the "science fore treatment" bias of many current trial. 3) The trial drug, if proven efficacious, must be available, at no cost, to all persons in the study upon its completion; this free availability must be continued throughout the subject's life or until the illness has been eradicated or cured. 4) All useful, helpful and pertinent data accumulated in the study must be available to the trial participant and his/her physician promptly upon completion of the trial. Also, trial participants must have access to all future data on the drug. 5) Placebo trials which have as an endpoint the death of members of the control group, or their progression to opportunistic infection, are unethical and unacceptable. 6) Drug trials subjects have the right to any results, even preliminary, from trials their participation has made possible. Putative drug company "proprietary" interests must never be used to deny subjects open access to this data. Informed consent guidelines must be strengthened to guarantee this. Ultimately, the entire AIDS community must have the right to timely, accurate information on any trials, underway or completed. Neither FDA nor drug companies may be permitted to sit on this information pending publication in "prestigious" scientific journals, at conferences or pending NDA approval.
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|Publication:||Rhetoric of Health & Medicine|
|Date:||Jan 1, 2018|
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