Faith Khalik 1/31/20 Faith Khalik 1/31/20

2020 Presidential Candidates: Policies for Addressing the Opioid Overdose Crisis.

Each day, over 130 people die from opioid-related overdoses. This includes both prescription and illicit opioids. The National Institute on Drug Abuse attributes the opioid overdose crisis to unscrupulous pharmaceutical companies, who misled healthcare providers to believe opioid pain relievers were not addictive. Other researchers, while agreeing that increased drug supply was an important factor, argue that economic and social issues fueled the crisis, viewing the issue through the lens of a structural and social determinants of health framework.

This post is first in a series aimed at identifying and exploring some of the public health issues and policies under consideration by candidates in the 2020 Presidential Election.

The goal of this series is to provide a deeper look at the ways in which candidates may or may not be including a public health framework in their healthcare reform policies, and encourage candidates to thoughtfully and purposefully develop nuanced, evidence-based, impactful policies. We also hope to inform the public as they continue to evaluate each candidate’s efforts to articulate plans that address healthcare and public health challenges in the U.S. Our goal is not endorse a particular candidate or political party; rather, our goal, as always, is to research, analyze, inform, and equip people with the knowledge they need to be engaged and thoughtful members of their communities and this nation.

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Public health experts and practitioners are increasingly pursuing support for and implementation of evidence-based harm reduction policies and programs to reduce harmful outcomes related to substance use disorders (SUD) and opioid use disorders (OUD). Our colleagues at the Health in Justice Action Lab (HIJAL) have identified a series of proven hard reduction programs. Below we offer a description for a few such programs and identify which of the 2020 Democratic Presidential Candidates support each program:

Safe injection facilities
Buprenorphine deregulation
Increased access to SUD treatment in prisons and jails
Decriminalization of opiates for personal use

Safe injection facilities

Safe injection facilities, also known as supervised consumption services (SCS), are legal facilities in which people can bring in and consume illicit drugs under the supervision of trained staff. The facilities provide clients with sterile injection supplies and a safe place to consume drugs, as staff members monitor for overdose and provide first aid as needed. Additionally, staff members are available to provide clients with referrals to drug treatment and other support programs. Although many studies have identified myriad public health benefits of safe injection sites, they remain controversial. Safe injection sites exist around the world, but due to legal issues (and specifically the Controlled Substance Act), not yet in the United States. However, that may be changing soon; a federal judge recently ruled that the Controlled Substance Act does not apply to Safehouse, a Philadelphia group seeking to open a safe injection facility

Candidates who have expressed support for safe injection facilities:

Buprenorphine deregulation

Buprenorphine is an opioid partial agonist used in treating opioid use disorder (OUD). Under the Drug Addiction Treatment Act of 2000, prescribers must complete an approved training, attest to their referral capacity, and submit an application to SAMHSA in order to receive what is known as an “X-waiver.” As noted by Kevin Fiscella et al, “X-waivered prescribers face heightened scrutiny by federal and state law enforcement officials, including periodic audits that are intended to minimize diversion and misuse.” Fiscella articulates four reasons for deregulating buprenorphine:

“First, buprenorphine’s comparative safety undermines a critical rationale for regulation. Buprenorphine, regardless of prescribing intent, is safer than commonly prescribed full-opioid agonists.

Second, deregulation would improve access to buprenorphine during the opioid national emergency. Despite promotion and training initiatives by SAMHSA and legislation that has expanded patient limits and waivers to nurse practitioners and physician assistants, access to buprenorphine has not kept pace with the current epidemic, particularly in rural communities.

Third, buprenorphine regulation is premised on the faulty assumption that buprenorphine diversion is driven by a desire to “get high.” Yet, buprenorphine obtained illicitly is mostly used for self-medication to relieve withdrawal symptoms rather than for euphoria… This suggests that regulations constraining access to buprenorphine may paradoxically contribute to a market for illicit buprenorphine among those who seek treatment.

Lastly, deregulation could help integrate opioid disorder treatment into primary care. Regulations reinforce the stigma surrounding buprenorphine prescribers and patients who receive it while constraining access and discouraging patient engagement and retention in treatment.”

Candidates who have expressed support for buprenorphine deregulation:

Increased access to SUD treatment in prisons and jails

A federal report found that 58% of people in prison and 63% of people in jail suffer from drug use disorders (SUD) based on criteria from the Diagnostic and Statistical Manual of Mental Disorders. However, only 28% of people in prison who met this criteria and 22% of people in jail participated in any type of SUD treatment.

Candidates who have expressed support for increased access to SUD treatment in prisons and jails:

Joe Biden

Decriminalization of opiates for personal use

Some advocates have proposed decriminalization as a measure to improve public health. The Drug Policy Alliance lists several benefits of decriminalization on their website:

“Removing criminal penalties for drug possession and low-level sales would:

Save money by reducing prison and especially jail costs and population size
Free up law enforcement resources to be used in more appropriate ways
Prioritize health and safety over punishment for people who use drugs
Reduce the stigma associated with drug use so that problematic drug users are encouraged to come out of the shadows and seek treatment and other support
Remove barriers to evidence-based harm reduction practices such as drug checking, heroin-assisted treatment, and medical marijuana.”

Candidates who have expressed support for decriminalization of opiates for personal use:

Guest User 9/11/17 Guest User 9/11/17

Federal 'Right to Try' Legislation - perpetuating a misguided skepticism towards the FDA [From CRITical Thinking]

George Consortium member Jeanie Kim, with the Collaboration for Research Integrity and Transparency (CRIT) at Yale Law School, has written a detailed look at the proposed federal "right to try" legislation. Wendy Parmet and Elisabeth Ryan wrote about the Senate bill on PHLW a few months ago; Jeanie's commentary serves as a great companion piece, emphasizing the potential dangers of making an end run around the FDA.

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Federal “Right to Try” Legislation – perpetuating a misguided skepticism towards the FDA

September 8, 2017

By Jeanie Kim

This blogpost provides a commentary on the federal “right-to-try” bills. For a succinct primer on “right-to-try,” see this article in Vox.

The “right to try” (RTT) movement presents a narrative that pits patients against the FDA. Supporters of RTT, powered by the libertarian Goldwater Institute, have pushed for laws that let terminally ill patients bypass regulators to access unapproved treatments.

As of September 2017, 37 states have enacted RTT laws. Earlier this year, the Senate and the House introduced federal RTT bills, and on August 3, 2017, the Senate unanimously passed an amended RTT bill without an opportunity for debate. There is pressure on the House to follow suit, but it is unclear whether the House will consider the originally introduced RTT bill(1) (“RTT 1.0”) or the Senate’s amended version(2)(“RTT 2.0”), or even take up the legislation at all.

Despite the recent legislative backing, RTT is not a new concept.(3) It is a variation on an age-old skepticism towards the FDA that has been around as long as the agency’s inception. At the core of RTT is the previously rejected, yet persistent argument that the FDA’s approval standards for safety and efficacy should not matter for terminally ill patients who have nothing to lose.(4)

The RTT bills are the latest tactic to undermine the FDA by suggesting that regulators are standing in the way of terminally ill patients. RTT 1.0 prohibits the FDA from interfering with “the production, manufacture, distribution, prescribing, or dispensing of an experimental [treatment]” that has passed initial safety testing on healthy volunteers and is intended for terminally ill patients who have exhausted medical options.

By adopting the premise that the FDA is a bureaucratic bottleneck, RTT misplaces the problem. The FDA has an efficient expanded access (EA) program for patients with life threatening conditions. Patients, in consultation with their physicians, can request access to experimental medicines from pharmaceutical companies. Once the company approves, patients can submit their applications to the FDA.

According to a recent report by the Government Accountability Office, of the nearly 5,800 EA requests received by the FDA from 2012 to 2015, the FDA allowed 99% to proceed, and for emergency single-patient requests, the agency typically responds within hours.(5) (In many cases, the FDA provides feedback on dosage and other safety issues.)

These numbers show that the FDA is hardly a barrier.

RTT overlooks these facts and undermines a broader principle. Congress empowered the FDA with the scientific authority to evaluate clinical trial evidence and the regulatory authority to approve drugs for marketing. This dual grant of authority is based on the policy that companies should not be able to profit from medicines before proving that the medicines are safe and effective. Even the EA program operates within this framework – early access to unapproved drugs is carefully balanced against the need for information about a drug’s safety and efficacy.

RTT 1.0 contains provisions that could destabilize this system.

First, RTT 1.0 prohibits the FDA from using any clinical outcomes from RTT uses to negatively impact its review of a drug. This absolute bar removes the flexibility that the FDA needs to evaluate safety information. Under the current EA program, agency reviewers generally give little weight to adverse events that occur from EA uses but still have the scientific discretion to consider whether certain outcomes – such as unexpected organ failures – could be useful for future patients in similar situations.(6)

Second, because the bill prohibits the FDA from interfering with the “distribution” of experimental treatments for terminally ill patients, RTT 1.0 leaves open the possibility of permitting companies to market unapproved drugs. The term “distribution,” which has various meanings,(7) is not defined in the bill and thus, could be a camel’s nose for deregulatory efforts to loosen the FDA’s prohibition on selling unapproved treatments.

Together, these two aspects of RTT 1.0 not only remove basic safeguards for current patients seeking access to experimental treatments but also undermine the research process that ensures that future patients will have access to drugs that are proven to be safe and effective. The removal of regulatory oversight is all the more problematic because RTT 1.0 also shields companies from all liability associated with RTT uses.

The Senate made attempts to address these concerns in its amended bill. First, while RTT 2.0 still prohibits the FDA from using clinical outcomes from RTT uses, the amended bill makes an exception for cases where it would be “critical” to determining the safety profile of a drug.

Second, RTT 2.0 requires manufacturers to adhere to FDA regulations concerning experimental treatments – specifically, regulations that prohibit companies from commercially distributing and promoting experimental drugs(8) and from charging more than "direct costs" for experimental drugs.(9)

The amended bill also does not release companies from liability if there was reckless or willful conduct or gross negligence.

A side-by-side comparison of the two RTT bills shows that RTT 2.0 includes several provisions intended to protect patients and maintain some regulatory oversight. See chart here.

The comparison also reveals just how detrimental RTT 1.0 could be, particularly for patient safety, as the original RTT bill would leave patients vulnerable to opportunistic behaviors by companies and physicians.

However, despite these improvements, RTT 2.0 is still based on the same misguided premise of RTT 1.0 – that the FDA is a barrier – and the same skepticism towards the FDA. Under RTT 2.0, RTT is intended to act as “an alternative pathway alongside [the EA program]” such that both pathways are available for patients with “life-threatening diseases or conditions.” The only difference is that the EA pathway preserves the FDA’s role and the RTT pathway minimizes it. While RTT 1.0 is a swift blow to the FDA, RTT 2.0 sets the stage for a gradual weakening of the agency’s scientific and regulatory authority.

For an example, RTT 2.0 does not expressly prohibit companies from selling unapproved drugs but cites FDA regulations for experimental drugs. This presents a loophole where the FDA can be pressured into loosening its regulations on experimental drugs in the same way as under RTT 1.0. Given the current antiregulatory climate, this is not far-fetched. In fact, a former president of Goldwater Institute has suggested that allowing companies to profit from experimental treatments is aligned with the end goals of the RTT movement.(10)

Finally, adding to the point that RTT is merely a shell for antiregulatory sentiments, neither RTT bill would actually improve patient access to experimental drugs. Many of the patient-centered additions in RTT 2.0—such as the transparency and reporting requirements for companies and the FDA—could easily be incorporated into the EA program without legislative action and without removing the FDA’s oversight.

Unsurprisingly, the RTT bills fail to deliver on its promise to streamline patient access to experimental treatments because they remove a “hurdle” that is not much of a hurdle for patients in the first place, but a critical component of the drug development process.

Other barriers for patients exist. Companies, who are the initial decision makers, are generally reluctant to grant access to unapproved treatments. And even after getting EA approval, patients must be able to afford the costs as payers typically do not cover unapproved treatments.

These issues are multifaceted and much more complex than the “solution” that RTT offers. Patient access to experimental treatments can be improved under the current EA program while maintaining the overall regulatory structure that balances access with safety and efficacy. In fact, there are already efforts to increase the transparency of companies’ EA policies and address other legitimate barriers. True solutions will require collaboration from regulators, manufacturers, and patients, and the FDA is best suited to facilitate improvements that will benefit both present and future patients.

Jeanie Kim is a research fellow at Yale Law School and the Collaboration for Research Integrity and Transparency (CRIT).

1 House RTT bill (H.R.878) (as of September 8, 2017).

2 Senate RTT bill (S.204) (as of September 8, 2017).

3 Joshua Sharfstein, Déjà Vu at the FDA, 95 The Milbank Quarterly (2017), available at https://www.milbank.org/quarterly/articles/deja-vu-fda/.

4 The Supreme Court rejected the argument that terminally patients have the right to access unapproved treatments. U.S v. Rutherford, 442 U.S. 544 (1979); see also Abigail Alliance for Better Access to Developmental Drugs v. von Eschenbach, 495 F.3d 695 (D.C. Cir. 2007), cert denied 552 U.S. 1159 (2008). Contrary to the optimism of the RTT movement, 90% of drugs that enter the first phase of clinical testing do not receive approval because of unexpected toxicity or lack of effectiveness, and even drugs that show promise in Phase 2 clinical trials are often not confirmed by Phase 3 trials. Katarzyna Smietana et al., Trends in clinical success rates, 15 Nature Reviews Drug Discovery 379 (2016), doi: 10.1038/nrd.2016.85; U.S. Food and Drug Admin, 22 Case Studies where Phase 2 and Phase 3 Trials has Divergent Results, January 2017, available at link (accessed September 8, 2017).

5 U.S. Government Accountability Office (GAO), Investigational New Drugs – FDA Has Taken Steps to Improve Expanded Access Program but Should Further Clarify How Adverse Events Data Are Used, GAO 17-564, at 17 (July 11, 2017), available at http://www.gao.gov/assets/690/685729.pdf.

6 U.S. Food and Drug Admin., Expanded Access to Investigational Drugs for Treatment Use – Questions and Answers, at 18 (June 2016).

7 Under current FDA regulations, “distribution” is defined as "to sell, offer to sell, deliver, or offer to deliver a drug to a recipient." 21 C.F.R. § 203.3(h).

8 21 C.F.R. § 312.7.

9 21 C.F.R. § 312.8(d)(1).

10 Darcy Olsen, The Right to Try: How the Federal Government Prevents Americans from Getting the Lifesaving Treatments They Need 205-206 (2015) (then-president of Goldwater Institute stated that “[a]llowing companies to charge for investigational drugs would make compassionate use instantly more attractive [for companies]”).

Guest User 9/8/17 Guest User 9/8/17

A Safer Way to Legalize Marijuana [from Health Affairs Blog]

Eight US states, the District of Columbia, and the country of Uruguay have recently legalized the recreational use of marijuana, with Canada and more US states poised to do the same. The new laws include limits on youth access, operation of motor vehicles when using, and high-volume purchases or possession. However, none of the laws consider which kinds of marijuana products should and should not be legally sold.

George Consortium member Professor Rebecca Haffajee of the University of Michigan School of Public Health co-authored "A Safer Way to Legalize Marijuana" on the Health Affairs Blog today, proposing that legalized marijuana should still exclude smokable products in order to reduce health hazards.

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While we take no position on the overall desirability of marijuana legalization, we propose here that policy makers in favor of it consider only permitting the sale of tetrahydrocannabinol (THC) extracts intended for vaporization or eating, and prohibiting combustible marijuana product sales. While implemented laws allow growing of marijuana for personal use, the policy we propose here would prohibit only the sale of marijuana cigarettes and their makings—flowers, stems, and seeds—to discourage their commercialization and mass production.

Those crafting marijuana laws can draw upon lessons learned about the harms of combusted tobacco and the smoking control policies that followed. Given what we already know about the health hazards of combusted marijuana and the difficulty of controlling the sale of commercially established products, policy makers should capitalize on this opportunity to create a legal marijuana market that mitigates potentially significant harms associated with inhaling combusted marijuana while still facilitating desired benefits of recreational marijuana.

Diverging Trends: Recreational Marijuana Legalization Versus Increasing Restrictions On Tobacco Smoking

The trend toward marijuana liberalization has grown since the mid-1990s to most recently encompass recreational use. By 2018, more than 50 million Americans 21 or older will live in states with access to legal recreational marijuana. Proponents of legalization argue that it reduces the high social costs of criminal law enforcement, an ineffective deterrent to use, the burdens of which fall disproportionately on racial minorities. Creating a legal market for marijuana also facilitates taxes on sales, the revenue from which may be used for public benefit. Legalization, moreover, increases access to a drug less prone to dependence than alcohol or tobacco for therapeutic and pleasure-seeking purposes. Public support for legal marijuana access has risen three-fold in the United States since the 1970s to reach 60 percent in 2016. Likewise, in other countries such as New Zealand, Canada, and France, the majority of the public say they favor legalization of marijuana sales. As momentum to broaden marijuana access builds, policy maker focus ought to prioritize ways to reduce potential harms of marijuana products.

Alongside recreational marijuana use liberalization, policy interventions designed to limit tobacco smoking have proliferated, based on what we have learned about the harms of combusted tobacco—both for the user and those exposed second-hand. This apparent policy divergence from marijuana legalization presents an opportunity for those crafting marijuana policy to learn from the tobacco-smoking experience. After all, both products involve psycho-active substances that can be delivered to the bloodstream in multiple ways. Combustible marijuana likely poses similar risks to those of combustible tobacco, while vaporizing or eating marijuana products offers a “cleaner” delivery mechanism. Why repeat the devastating public health harms of smoking tobacco when policy makers can reasonably mitigate similar consequences of smoking marijuana?

Health Concerns Around Marijuana Combustion

Among the chief concerns with marijuana use, although not yet reflected in liberalization policies, are health harms associated with combustion. Combustion of any substance produces hundreds of chemical compounds, including many known toxins. In a recent comprehensive review of the scientific literature, the National Academies of Sciences, Engineering, and Medicine concluded that “smoked marijuana…is a crude THC delivery system that also delivers harmful substances.” The report and other reviews found strong evidence linking combusted marijuana to increased risk for chronic bronchitis.

Emerging studies link marijuana smoking to increased risk of cardiovascular mortality. Evidence identifying marijuana smoking as a risk factor for lung cancer is not as robust, although potential harms cannot be ruled out, particularly because marijuana smoke is carcinogenic. Research on harms from second-hand marijuana smoke is lacking, but analogizing from tobacco, it is reasonable to assume that chemicals and particulate matter released in marijuana smoke would be harmful to bystanders. Although a typical tobacco user smokes more often than a typical marijuana user, the relative harm of marijuana combustion may not be substantially lower than that for tobacco because some toxins released are higher per unit in a marijuana versus a tobacco cigarette.

Several factors undermine the existing evidence base on health outcomes associated with marijuana use, emphasizing the need to prioritize research in this area alongside policy development. The listing of marijuana as a Schedule I drug by the federal Drug Enforcement Agency has hampered fundingand execution of studies of its therapeutic benefits and harms. Additionally, evidence related to the harms of marijuana use generally comes from studies of lower-potency products (that is, with lower THC content) than are marketed today and can be anticipated in the future. Therefore, it is possible that the harms associated with combustion may differ from those reflected in the current literature.

Potential harms of combusted marijuana extend beyond marijuana alone. Combination marijuana and tobacco use, which can involve blending the substances into a single smoked product or smoking tobacco and marijuana separately but in close temporal proximity, is prevalent. Limiting the availability of combustible marijuana may reduce the amount of dual product use, thereby reducing the amount of and hazards associated with tobacco smoking.

Shifting To Vaporized And Edible Marijuana

Edible and vaporized marijuana products offer the potential to deliver therapeutic and euphoric benefits of marijuana while avoiding cardiopulmonary-related harms of combustion. Although precise estimates of the decreased risks associated with this substitution are not available, by analogy the health risks for smokeless and vaporized tobacco products are estimated to be roughly 90 percent less than those of combusted tobacco.

Valid concerns have been raised about the potential health harms from commercially marketed edibles, especially their attractiveness to, accessibility by, and increasing exposure and overdoses among children. We strongly support prohibitions on the sale of marijuana products—including edibles—to minors, clearly labeling product THC content and requiring child-proof packaging. Additionally, if marijuana is only legally available for sale in forms that do not resemble cigarettes, children may be less likely to cross over between products.

Combusting marijuana is an entrenched behavior that may be challenging to modify. Many marijuana users exhibit a preference for smoking, the primary delivery mode for the drug, and may be less satisfied if combustible products cannot be purchased. However, smoking marijuana is not yet as established in legal markets as is smoking tobacco, and newer users could vape or consume edibles instead of developing preferences for combustion. A subset of established marijuana smokers likely will opt for readily available non-combustibles, particularly when educated about the risks associated with combustion and as social preferences change over time. As well, vaping may serve as a harm-reduction strategy that mimics the individual behavior, social aspects, and psychotropic effects of smoking, and is found to be just as satisfying. Finally, extracted THC for sale as oils, waxes, and edibles is amenable to mass production to a greater degree than raw-form combustible marijuana, potentially facilitating lower prices and a shift toward consumption of these products post-legalization. Combatting and policing a black market for combustible marijuana would still be required, although regulatory enforcement would be limited to retail establishments instead of personal use.

Designing A Safer Marijuana Market From The Outset

Imagine that tobacco had been illegal for most of the 20th century, and its legalization were being considered today—and that we possessed our current knowledge about the health consequences of smoking tobacco. Few would advocate for its legal availability in combustible form, given that alternative available delivery modes—e-cigarettes and smokeless tobacco—can deliver the same psycho-active drug, nicotine, in forms far less likely to cause serious disease.

While we do not have the chance to design a legal nicotine market from scratch, our body politic has the opportunity to design the legal market for marijuana from the ground up. The legalization of recreational marijuana sales has thus far included all forms or only combustible forms (as in the case of Uruguay) of marijuana. Product availability in these new legal markets seems to have been guided by economic considerations and voter preferences, instead of measured consideration prior to legalization of the relative risks and harms of different types of marijuana products.

Policy makers in jurisdictions considering legalization are not bound by custom to make available all forms of marijuana for recreational use. Little prior interstate commerce of legal marijuana products exists, and most states have yet to legalize recreational use. The environment is ripe to experiment with different types of markets, and entrepreneurial policy makers could embark on implementing a safer legal marijuana market that omits combustibles, based on our current and developing knowledge.

Action In The Face Of Some Uncertainty

Marijuana policy makers would be wise to draw upon lessons learned from the experience with tobacco. In the tobacco policy realm, there is a broad consensus that it would be difficult to design a consumer product more harmful than a combustible cigarette—the product that kills one in two long-term users. The suspicion that cigarettes were a dangerous product was well-founded early in the 20th century in animal model studies and pioneering epidemiological surveys. While not risk-free, smokeless tobacco and e-cigarettes will not kill nearly so many of their users. Unfortunately, cigarettes are entrenched within the tobacco marketplace. A protracted battle has raged between public health on the one side, which would benefit by shifting nicotine consumption to safer products or eliminating nicotine use altogether, and incumbent commercial interests and established consumer preferences on the other, which favor selling the most dangerous product, with neither side declaring decisive victory.

While uncertainty still exists regarding the relative harms of different marijuana products and robust research is warranted, waiting for perfect scientific consensus about the scope and nature of harms related to marijuana combustion is unwise. The evidence base around marijuana combustion harms is already strong, and growing. Arriving at total consensus will take decades—as it took to link cigarettes to lung cancer—and waiting to embark on an alternative, very likely safer policy regime has real costs, measured in disease and death. Permitting the sale of THC extracts for consumption in edible or vaporized form will neither compromise therapeutic nor euphoric benefits of recreational marijuana use. In addition, creating variation in recreational marijuana policy regimes—between those already enacted that permit marijuana combustion and those enacted in the future that don’t—would create natural experiments ripe to study the differential effects and quantify harms versus benefits. Policy makers in favor of legalization should seize the opportunity to design a new market that permits recreational sale of marijuana only in edible or vaporized form, to minimize the potential for the kind of disease burden associated with smoked tobacco.

Authors’ Note

We thank Daniel J. Clauw, Mark A. R. Kleiman, and Lynn T. Kozlowski, for their valuable insights.

Guest User 8/29/17 Guest User 8/29/17

"Right to Try" May Do More Harm Than Good

By Wendy E. Parmet and Elisabeth J. Ryan

When the Senate approved the “Right to Try Act of 2017” on August 3, Republican sponsor Senator Ron Johnson hailed it as a law that helps “real people facing their mortality with no hope.” The bill allows patients with “a life-threatening disease or condition” who have “exhausted approved treatment options” to go directly to pharmaceutical companies and request access to drugs or devices not yet approved through the traditional process. The bill does not, however, require those drug companies to grant any such requests. It also does not address how much drug companies can charge those patients for access. And the rhetoric hailing it as a savior for patients “with no hope” ignores the fact that the FDA already has an Expanded Access (Compassionate Use) procedure that allows patients to access investigational medical products outside of clinical trials. In fact, the FDA has approved over 99% of such requests, some in as little as 24 hours in emergency situations. So the “Right to Try Act of 2017” wouldn’t actually create any new access rights; rather it would end the FDA’s oversight role. In addition, the “Right to Try Act” would immunize drug companies and prescribing physicians from liability that may arise from a patient’s use of an unapproved drug or device (or from the denial of access to those drugs and devices) except in cases of “reckless or willful misconduct, gross negligence, or an intentional tort.”

Thirty-seven states already have “right to try” laws, which have been pushed heavily by anti-regulatory, libertarian efforts. These laws have often passed with virtually no opposition because many health professionals and politicians fear “being seen as opposing any one patient’s question to save his or her life.” But states don’t actually have the authority to regulate drug approval and such laws affect little in practice. In fact, as Professor Rachel Sachs stated, "It’s telling that although 37 states have adopted these laws, when asked to provide examples of success stories, one of the primary groups pushing for their adoption can only provide the testimonies of six patients who received access to experimental medicines through a single physician in a single state."

Federal legislation has the potential to seriously undermine not only regulatory protections, but also the “integrity of clinical trials, which remain the safest way for patients to try experimental drugs.” To qualify for the bill’s “right to try,” patients must be “unable to participate in a clinical trial,” but what that means remains unclear. If the language is read broadly to include not only patients who fall outside a trial’s parameters, but also those who cannot access trials due to other reasons (such as distance from a trial site), the bill might reduce patients’ willingness to participate in clinical trials which are vital to protecting population health by gathering evidence as to the safety and efficacy of new drugs. Perhaps the better solution would be to expand the population of eligible participants for clinical trials. As Kelly McBride Folkers says in the prior linked article, “Not only are the sickest individuals often denied spots in a clinical trial, but people of color, women, those who live in rural communities, and those without adequate insurance are vastly underrepresented in clinical trial populations. Their absence greatly diminishes the utility of the data gathered from these trials.” Finally, the FDA needs more oversight of unapproved medical devices, not less. “The agency’s guidance protects patients from exploitation, as well as from well-intended but misguided therapeutic attempts that can cause even more harm or pain than patients are already experiencing from their underlying disease or condition.”

Senator Johnson refused to allow a Senate vote on the FDA budget unless the “Right to Try” legislation was attached to it, fast-tracking its approval with minimal debate; the House may subject it to more scrutiny. The bill, however, remains politically risky to oppose, which could result in harm far more difficult to articulate than “hope for the hopeless.”