On Patient Safety: A Right to Try, Not Exploit

In May 2018, the federal Right to Try Act was signed into US law, allowing eligible patients diagnosed with life-threatening diseases greater access to investigational drugs. The law all-but eliminates the FDA’s role in decision-making regarding the use of unapproved and unproven medical products for life-threatening diseases [6]. By signing this piece of legislation into law, our federal government joined 41 states that have already passed similar laws at the state level.

The federal Right to Try legislation was passed in an effort to give patients who are terminally ill more control over their own treatment options. Supporters of these laws argue that the FDA acts as “the arbiter of life and death” [6] and that patients, especially those with terminal illness, should make their own treatment decisions unencumbered by government bureaucrats.

But I believe these various Right to Try laws at both the state and federal levels are dangerous to our patients. Orthopaedic surgeons should advocate to add safety measures to these laws, which create a perfect storm for unscrupulous providers, private equity, and stem cell businesses to exploit, and potentially injure, our patients.

Over the past few years, several worrisome examples of private equity firms allocating capital and launching investment products to support new ventures in order to exploit Right to Try laws have begun [5, 14]. These ventures follow a similar pattern; because costs are uncapped, private equity firms make treatments astronomically expensive, not because their products are worth it, but because these firms believe that patients are desperate enough to pay for them. For example, one private equity firm raising funds to enter the US market using Right to Try laws plans to charge upfront “membership fees” to patients in addition to monthly fees. Fees for administering treatment, general patient care, and the care of any and all complications can also be substantial, and typically, under these laws, none of these fees or costs are covered by insurance [9, 12].

Another stem cell company providing care under Right to Try plans to charge US patients USD 300,000 for treatment [14].

But the most dangerous aspect of the law is the ease in which private equity firms and stem cell companies can aggressively peddle unproven stem cell treatments across the country with little risk to their own business … but huge risk for our patients. The federal Right to Try law grants broad liability protection to manufacturers and prescribers who provide treatments under the protection of this law [3]. These liability protections include negligent usage in certain circumstances and therapeutic decisions influenced by financial conflict of interest [4].

Stem cell companies, backed by private equity, are advertising services specifically for orthopaedic conditions such as osteoarthritis, soft tissue injury, and chronic joint and muscle pain [13, 14], and plan to take advantage of the vague language in the law as well. In an effort to protect insurers from the cost of caring for patients who are injured using unproven treatments, 19 states allow insurance companies to deny coverage for hospice, six states allow denying home healthcare treatment for patients who use investigational products via Right to Try, and three states allow insurers to deny coverage altogether for up to 6 months after the investigational therapy is performed [4].

Indeed, these Right to Try laws have been carefully crafted to protect every party except the patient. They protect the insurer, the manufacturer, and the prescriber. The patient, sadly, remains completely exposed and vulnerable. Patients desperate with life-threatening illnesses or chronic pain and disability will be exploited emotionally, financially, and physically. Families will lose money, emotional trauma will ensue as patient problems are unaffected by largely ineffective unproven treatments, and false hope will be generated time and again only to be crushed by reality.

But there are options that can blunt this emerging problem.

Push the FDA’s Expanded Access Program

An effective and much safer alternative to Right to Try laws already exists—the FDA’s Expanded Access Program [15]—but this program has been made obsolete by Right to Try laws. Advocates for Right to Try claim that these laws are needed because the FDA currently restricts seriously ill patients from choosing their own treatments [6]. But this is untrue; the FDA’s Expanded Access Program provides patients afflicted with terminal illness with access to investigational drugs outside of clinical trials on a case-by-case basis. Its mean decision time is 2.2 days, and it authorizes approximately 99% of requests [9]. Importantly, FDA involvement ensures safety guardrails remain in place—a feature Right to Try laws tear down. Right to Try laws include no standards for informed consent while the Expanded Access Program requires standards for legal and ethical patient consent including a discussion of the risk of treatment, its expected benefit, and alternative therapies [9].

The Expanded Access Program also includes more stringent collection and reporting of safety data, and the FDA can alter dosing of the investigational agent to improve patient safety [12]. In fact, the FDA has required changes to dose, safety monitoring, and/or informed consent in 11% of requests [8]. Language in the federal Right to Try Act purposefully blinds the FDA to patient-safety concerns uncovered in Right to Try usage in an effort to persuade manufacturers to allow Right to Try usage of their investigational drugs [4].

Of crucial importance to the potential exploitation of patients using these pathways, the FDA scrutinizes, through the Expanded Access Program, the documentation justifying the purchase price of all therapeutics to ensure patients are charged in good faith only for direct manufacturing and shipping costs of the intervention [11]—a feature noticeably absent from state and federal Right to Try laws. And the currently available Expanded Access Program is easy to use. A request can be done by fax, phone, or email, and the physician time required in total is estimated to be 45 minutes [3].

Our American Academy of Orthopaedic Surgeons (AAOS) Political Action Committee should lead here, working in conjunction with oncologists—the other medical specialty most likely to be affected by these laws—to push for continued FDA oversight, such as a modified Expanded Access Program, for all medical therapeutics [3].

Amend Vague Language in the Bill

Multiple stem cell businesses have expressed their intention to take advantage of the law, particularly in the Texas marketplace, which has much broader language than the federal Right to Try Act. For example, in 2017, Texas passed Charlie’s Law, which permits the use of “investigational stem cell treatments to patients with certain severe chronic diseases or terminal illnesses” [7]. The vague term “chronic disease” is worrisome language.

State orthopaedic societies and individual surgeons should collectively lobby for the modification of these state laws, or at least the elimination of this language concerning musculoskeletal conditions. Both individual surgeons and our state orthopaedic societies should actively question provisions allowing insurers to suspend their coverage if these laws are invoked during treatment; this would be an excellent avenue to defend our patients’ safety. If Right to Try laws create a situation so risky that insurers’ risk pools need to be protected, how can such a law be safe for the individual patient? If insurers believe that it’s too risky to cover a patient who uses stem cell treatment with a poorly understood risk profile, then these laws need to be amended to require some evidence of safety and efficacy of proposed treatments.

Go Public

The AAOS should publicly denounce the use of stem cells and other biologics for any indication other than those for which the best-available evidence has found them to be effective. The AAOS position statement on “emerging biologic therapies” does not go far enough [1]. Since providers can employ Right to Try laws to circumvent FDA guidance, the AAOS, through its Biologics Dashboard [2], should document those indications and products where high-quality evidence of safety and efficacy are lacking rather than focusing on these products’ regulatory status. Promoting these unproven products by stem cell companies is a grave financial and mental health risk to our patients. Without our action, they may become an unproven and deleterious (since they divert patient focus and money from evidence-based therapies) treatment option for orthopaedic conditions. Already, the biologics industry has successfully lobbied the state of Arkansas to include their products in insurance plans for their government employees and teachers [10]. As a profession, our best response to this is to advocate for evidence-based treatments and repudiating other pseudoscientific interventions. Additionally, each of us as individual orthopaedic surgeons should resist the siren song of hassle-free easy money from unproven stem cell treatments to augment our practice income.

I cannot think of a more potentially exploitative combination of interests than private equity, stem cell companies, and our Right to Try laws. We should take vigorous steps to combat this triad at both federal and local levels by highlighting the patient safety risks of both Right to Try laws and unproven stem cell treatments.