Patients and families are increasingly turning to social media to facilitate expanded access to unproven drugs. This raises many ethical concerns, says Tim K. Mackey, MAS, PhD, director of the Global Health Policy Institute and assistant professor at UC San Diego’s School of Medicine. Researchers examined 23 case studies of social media used to facilitate “compassionate use” of investigational drugs.1 “There was a high degree of variability in how much engagement and media coverage they got,” says Mackey, the study’s lead author. “Even those that did get a lot of attention, did not necessarily get patients the access they sought.”
Patients’ social media discussions centered on the following topics:
- rapid deterioration of patient’s health, indicating an immediate and urgent need for experimental treatment,
- lack of any alternative treatment options,
- description of the devastating effect of the condition on the patient’s family in order to encourage public support,
- identifying a drug manufacturer or the FDA as the primary barrier to access, and
- overwhelmingly positive characterization of experimental drugs petitioned. “This included characterizing an experimental drug as a ‘wonder drug,’” says Mackey.
The study didn’t report the outcome of the patients involved in the social media campaigns. “But we took a look at it in our original data analysis,” says Mackey. Of the 23 case studies, reports from families or the news indicated that at least half of the patients had died from their diseases. This included patients who received access, but who were either disqualified or had to discontinue.
Mackey says this suggests the overall premise that social media works as an alternative forum to gain access to investigational drugs is “questionable at best.”
Mackey expects to see continued attempts to pass federal right-to-try legislation, “despite the fact that there is little evidence that this form of policy intervention actually helps anyone.”
Mackey says the public rhetoric behind “compassionate use” is fueling policy that is not evidence-based. He says policy should instead be focused on the following:
- Ensuring decisions are communicated to patients in a reasonable timeframe. “This is critical to ensuring a patient’s dignity and managing expectations,” says Mackey.
- Greater transparency regarding companies’ expanded access programs (EAPs).
- Creating a standardized, streamlined process for requesting access through myriad EAPs.
“Based on coverage of ‘successful’ social media campaigns — which may have not been successful at all — patients are likely to continue to try to compel access when they don’t have other options,” says Mackey.
This may not be the best use of their time, given the study’s findings. “What seems clear is that patients use social media due to uncertainty and desperation,” says Mackey. He names the following ethical concerns:
- There are inherent risks associated with use of any unproven drug outside of a structured clinical trial. “There is the potential for greater harm to the patient if treatment is not effective or has a poor safety profile,” adds Mackey.
- Scarce resources are being used for access that might not benefit any of the stakeholders: the patient, the clinician, or the manufacturer.
- EAPs may engender false hope among patients and their families. “In fact, the chances that they actually gain access — or even that the drug may work — may be completely unknown and unlikely,” says Mackey.
M. E. Blair Holbein, PhD, BCAP, chief of the division of regulatory science and assistant professor in the department of clinical sciences at UT Southwestern Medical Center in Dallas, is lead author of a paper which concluded that the FDA’s EAP is a reasonable option for patients for whom all other therapeutic interventions have failed.2
The FDA has three expanded access options that provide ways to access investigational drugs. “Having an IRB [institutional review board] included in the approval process assures an unbiased review,” notes Holbein. This can provide at least some protection for a vulnerable patient.
“The FDA has access to information that is proprietary and not available outside the FDA or the manufacturer,” adds Holbein. Thus, the FDA review can consider whether the proposed use exposes the patient to unreasonable risk.
“Most importantly, this process maintains the best scientific, as well as ethical, review possible under the circumstances,” says Holbein.
Ethical issues include assuring the patient that there is a considered estimation of the most beneficial course, that the patient determines his or her choices, and that there is equitable access to investigational products.
“Patients seeking investigational agents are a vulnerable population that requires a heightened awareness of potential ethical problems,” says Holbein.
It is easy to understand that a person with a serious, even life-threatening, disease wants to have access to any potentially beneficial treatment possible. “The question becomes, how do we establish a balance between the risk of the intervention versus the disease?” says Holbein.
Holbein says the very best way to gain access to a drug in development is to participate in a clinical trial. “Every effort should be made to find a trial,” she says.
Multicenter clinical trials have already been carefully reviewed to ensure the best utilization of the investigational drug, and have had a thorough ethics review. “Usually, this would also be the most timely, least expensive, and most equitable access option,” says Holbein.
- Mackey TK, Schoenfeld VJ. Going “social” to access experimental and potentially life-saving treatment: an assessment of the policy and online patient advocacy environment for expanded access. BMC Medicine 2016; 14:17 DOI: 10.1186/s12916-016-0568-8.
- Holbein ME, Berglund JP, Weatherwax K, et al. Access to investigational drugs: FDA expanded access programs or “right-to-try” legislation? Clin Transl Sci 2015; 8(5):526-532.
- M. E. Blair Holbein, PhD, BCAP, Chief, Division of Regulatory Science/Assistant Professor, Department of Clinical Sciences, UT Southwestern Medical Center, Dallas. Phone: (214) 648-5009. Fax: (214) 648-8683. Email: Blair.Holbein@UTSouthwestern.edu.
- Tim K. Mackey, MAS, PhD, Director, Global Health Policy Institute/Assistant Professor, UC San Diego School of Medicine. Email: firstname.lastname@example.org.