Expanded Access Programs Require Compassionate Communications to Keep Your Reputation Intact

No company wants to be seen as uncaring, least of all in the healthcare space. Clients I’ve supported in this sector are often passionate about their mission to bring new and better treatment options to patients. But companies’ intentions may be undermined or misconstrued if they fail to develop a communications plan for an expanded access or “compassionate use” program. These are corporate initiatives that enable—or sometimes oblige—companies make medicines available to certain patients before they’re approved by the Food and Drug Administration (FDA).

The fact is, these programs are conceived and carried out in sensitive contexts that involve complex medical, ethical, and business considerations. Unfortunately, in too many cases, conversations about expanded access take place under the glare of media attention. For patients with terminal diseases, an expanded access program (EAP) may offer the only hope for an effective treatment. Companies, for their part, may have limited supplies of the medicine, and they could be juggling other regulatory and resource constraints. In an emotionally fraught public forum, neither side is likely to see its interests served—which is why a well-crafted communications program can be a life saver.

The pressures and politics surrounding compassionate use may be intense. But with a combination of careful planning, third-party support, and principled decision-making, companies can remain true to their mission to help patients, while still protecting their reputations and commercial viability.

Expanded Access Matters Now More Than Ever

Three key trends are increasing the desire of sick patients to pursue expanded access to unapproved medicines. These intensifying pressures make it imperative for medicines developers to create clear and compassionate communications around their EAP policies:

1. Greater information about therapies for rare conditions

Publicity surrounding promising results in Phase II/III clinical trials is one of the key drivers of patient interest in EAPs. But companies must not count on a broad swath of media channels to communicate essential, or even accurate, information. For example, laws such as the Orphan Drug Act have helped incentivize biopharmaceutical companies of all sizes to invest heavily in potential treatments for rare diseases, where there are tremendous unmet medical needs. Unfortunately, it can be hard for reporters to get accurate information on experimental treatments for rare diseases and attendant patient access programs.

On the other hand, patient advocacy organizations in rare diseases, even if they are informal, are often highly sophisticated. These groups, which frequently communicate in closed communities or offline, can quickly share information—or rumors—about pipeline products and expanded access offerings. This makes it doubly important for companies developing treatments to take charge of communications about potential access programs. Messages about investigational medicines must be clear and strike the right tone the first time they are delivered.

2. New tools for accessing information and mobilizing

Motivated patients have more tools than ever to learn about investigational medicines, and to apply pressure for access to them. Even companies that are conservative in their discussion of a pipeline product may be sharing more than they know. For example, educated patients often monitor investor communications for any hint of a prospective therapy’s timeline and data readouts.

Once alerted, campaigns can start on social media and migrate to traditional platforms to bring unrelenting pressure on a company. Perhaps the most famous example was a campaign under the handle #SaveJosh. It started on social media after eight-year-old Josh Hardy was denied access to a medicine to combat a rare viral infection he’d caught while undergoing cancer treatment. Within weeks, Hardy’s story had become national news. The manufacturer eventually worked with the FDA to make the medicine available, but the company’s reputation had already suffered, and the CEO was subsequently replaced. High-profile cases like this one persuaded many other patients with rare diseases that media activism is their best—or only—option.

3. Public policy becoming friendlier to patients seeking compassionate use

New laws and public policy are making expanded access more visible, and shifting nearly all the burden of these gut-wrenching decisions to pharmaceutical companies.

The recently passed 21st Century Cures Act requires that information on companies’ expanded access policies1 be easily available. The provision in the law is meant to lessen the frustration of patients, rather than to punish companies. But the requirement shows how awareness of the issue has grown in policy circles, and it also highlights that fact that many patients have found companies’ EAPs to be opaque.

The FDA itself has been a change agent in this area. Last year, the Agency unveiled a new, simpler way for patients and physicians to apply for compassionate use.2 The FDA also released new guidance offering flexibility for some companies to recoup their manufacturing costs for medicines made available via an EAP.3 These changes help redirect the conversation from the government to the industry.

One example is the budding Right-to-Try movement, which seeks to mostly eliminate the FDA’s role in overseeing individual expanded access cases. The movement has succeeded in passing legislation in dozens of states. Enactment of a federal Right-to-Try law is also more likely, now that the President and Vice President have announced their support.4

Such laws don’t require pharma companies to provide access. However, the combined impact of Right-To-Try and the earlier FDA reforms would turn patients’ full attention to private sector initiatives.

Expanded Access Communications that are Clear and Caring

Even within a complicated media and regulatory environment, life science companies can still establish fair policies around expanded access and communicate them in a way that demonstrates they are fair, caring, and clear.

A few keys are:

1. Ensure third parties are aligned. Pharmaceutical companies can benefit from outside support when setting the criteria for access to an investigational medicine.

Consider working with a respected academic ethicist to help establish policy criteria and provide an outside perspective. This can help companies wade through thorny ethical issues where there may be detrimental impact, regardless of which path the company pursues. For example, what if creating an EAP would divert so many resources at the company that it slows down regulatory review? In the process, that would inhibit access for a larger number of patients. A second, and perhaps related, question: Should medicine be given to the sickest patients, or to those who most closely match the clinical trial profile?

Patient advocates should also have a clear understanding of the principles upon which an EAP is established. Securing feedback from groups within the first two weeks of a new EAP will ensure that a program’s parameters and its application requirements are clearly understood.

2. Describe your plan based on principles, not logistics. Bringing a new medicine to patients is a complicated, expensive, and poorly understood process. But drawing attention to the challenges you face is a poor strategy when explaining your EAP. It’s not likely to resonate with desperate patients seeking access to medicines. Instead, communicate your policies based on principles, such as the need to treat clinical trial participants who may be receiving a placebo. This may not be what patients want to hear, but they will understand that it’s fair.

3. Be prepared to receive a spike in requests around data announcements. The release of exciting data is likely to trigger an uptick in requests for compassionate use. You must be prepared—one way or the other—to respond fairly and consistently to increased demand. If you have chosen to field calls through a third party, make sure they are adequately trained. If they aren’t prepared to respond to callers with certain physical disabilities, for example, their deficiencies will be your deficiencies.

Laying the groundwork for meaningful conversations about expanded access is one of the toughest challenges in healthcare communications. The external forces I’ve described greatly exacerbate the problem. By understanding patient perspectives, carefully considering your offering, and being prepared for inquiries, you can have compassionate conversations and keep your reputation intact.

References:

1. H.R. 34, 114th Cong. (2016) (enacted) https://www.congress.gov/bill/114th-congress/house-bill/34/text#toc-H88B12877B7F34C11B82985F70860CDA8.

2. Press Announcements – Statement from FDA Commissioner Robert Califf, M.D. on the release of the final individual patient expanded access form. (2016, June 2). https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm504579.htm

3. Ibid.

4. Readout of the Vice President’s Meeting with Right to Try Advocates. (2017, February 08). https://www.whitehouse.gov/the-press-office/2017/02/07/readout-vice-presidents-meeting-right-try-advocates.

  • Paul V. Tyahla

    Paul V. Tyahla is an Issues Management Strategist at inVentiv Health. He helps life science companies plan for and navigate sensitive issues, protect their corporate reputation, and respond to crises. He has helped companies strategize around issues related to expanded access, pricing, litigation, and media investigations.

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