Going “social” to access experimental and potentially life-saving treatment: an assessment of the policy and online patient advocacy environment for expanded access

Abstract

Social media is fundamentally altering how we access health information and make decisions about medical treatment, including for terminally ill patients. This specifically includes the growing phenomenon of patients who use online petitions and social media campaigns in an attempt to gain access to experimental drugs through expanded access pathways. Importantly, controversy surrounding expanded access and “compassionate use” involves several disparate stakeholders, including patients, manufacturers, policymakers, and regulatory agencies—all with competing interests and priorities, leading to confusion, frustration, and ultimately advocacy. In order to explore this issue in detail, this correspondence article first conducts a literature review to describe how the expanded access policy and regulatory environment in the United States has evolved over time and how it currently impacts access to experimental drugs. We then conducted structured web searches to identify patient use of online petitions and social media campaigns aimed at compelling access to experimental drugs. This was carried out in order to characterize the types of communication strategies utilized, the diseases and drugs subject to expanded access petitions, and the prevalent themes associated with this form of “digital” patient advocacy. We find that patients and their families experience mixed results, but still gravitate towards the use of online campaigns out of desperation, lack of reliable information about treatment access options, and in direct response to limitations of the current fragmented structure of expanded access regulation and policy currently in place. In response, we discuss potential policy reforms to improve expanded access processes, including advocating greater transparency for expanded access programs, exploring use of targeted economic incentives for manufacturers, and developing systems to facilitate patient information about existing treatment options. This includes leveraging recent legislative attention to reform expanded access through the CURE Act Provisions contained in the proposed U.S. 21st Century Cures Act. While expanded access may not be the best option for the majority of individuals, terminally ill patients and their families nevertheless deserve better processes, policies, and availability to potentially life-changing information, before they decide to pursue an online campaign in the desperate hope of gaining access to experimental drugs.Electronic supplementary materialThe online version of this article (doi:10.1186/s12916-016-0568-8) contains supplementary material, which is available to authorized users.

Pembrolizumab: first experience with recurrent primary central nervous system (CNS) tumors

“…Some individuals turn to social media and groups of patients may petition for expanded access of new medications. It is not always clear if these requests come from adequately informed sources [39]. On the one hand, it is not always feasible to wait for level I evidence (phase III randomized trials) to gain access to new drugs for an "orphan" population; by waiting, lives may be compromised.…”

(R)evolution: toward a new paradigm of policy and patient advocacy for expanded access to experimental treatments

“…Mackey and Schoenfeld examined the ethical and practical questions posed by the evolving landscape of access to experimental therapies, identifying and analyzing the impact of a spectrum of social media campaigns, the right-to-try legislative movement, and the existing regulatory framework [1]. …”

“…Mackey and Schoenfeld examined the evolution of expanded access policy in the United States, as well as more recent trends toward alternative access such as “right-to-try” legislation and social media campaigns [1]. They discussed both the ethical and practical concerns behind manufacturer denials of expanded access [1].…”

“…They discussed both the ethical and practical concerns behind manufacturer denials of expanded access [1]. …”

“…Some reforms seek to loosen the restrictions involved in the FDA expanded access program (EAP) requirements, while others seek to obligate or pressure manufacturers to provide experimental therapies to patients individually or through an EAP. There is little correlation of factors that point to a “winning” strategy for obtaining such access [1]. Significantly, manufacturers, advocacy organizations, and the FDA each appear to be in search of a new paradigm that ethically and practically addresses patient and manufacturer concerns.…”