Compassionate Use: Experimental Drug Access from the Ebola Crisis to #Save[Patient]

During his visit to the U.S. Centers for Disease and Control today in Atlanta, President Barack Obama announced a major expansion of military and medical resources to combat the spread of the Ebola virus, including the development of 17 treatment centers, training for thousands of healthcare workers and the creation of a military control center for coordination.

Considering short- and long-term treatment options, $58 million will be allocated to speed the production of antibody-based ZMapp, as well as two Ebola vaccine candidates.

ZMapp, founded by Mapp Biopharmaceutical, Inc., was first in the spotlight when two American aid workers who had been based in Liberia received the experimental drug under the FDA’s “compassionate use” regulation, which provides access to experimental drugs outside clinical trials. Of the seven people who have now been treated with the drug, two have died.

Though the Ebola virus is rare among rare diseases, since the AIDS epidemic in the 1980s, patients with terminal and hard-to-treat illnesses have sought access to drugs in development. But the decision to grant this access is far from simple.

To receive a drug through compassionate use, a patient must have support from a physician, FDA consent and a pharmaceutical company willing to provide the treatment – a step where many patients run into roadblocks.

If a request is turned down by drugmakers, the companies are seen as heartless. But several issues must be considered: a poor outcome under compassionate use could put a hold on the approval process for the broader population; a limited supply of the experimental medication; and a lack of data on its safety and efficacy.

Considering ZMapp, Forbes columnist David Kroll raises this question: “Out of thousands infected, how did the company decide which six or seven patients would receive their medicine?”

Chimerix, a small, biopharmaceutical company, based in Durham, North Carolina, faced intense scrutiny in April when it initially refused an experimental medicine to and eight-year-old pediatric cancer patient who developed a viral infection following a bone marrow transplant, and due to kidney complications from the standard course of therapy, the family sought access to brincidofovir, Chimerix’s anti-viral drug still in development.

The family amassed a substantial social media following that led to national coverage and death threats to company leadership. Though the eight-year-old did not receive the medication through compassionate use, Chimerix worked with the FDA to immediately approve a clinical trial for pediatric patients, in which the boy was the first to enroll.

The public relations crisis led to CEO Kenneth Moch’s resignation and questions raised around the concept of crowdsourcing medical decisions. In the era of social media, should patients with more compelling stories or the ability to gain social media traction receive a life-saving treatment over those with the same or greater needs?

In his first public essay since he left Chimerix, Moch, along with NYU ethicist Arthur Caplan, reflect on these challenges in a Health Affairs editorial: “Without a new system and clarity within the regulatory process, medical decision-making will be left to those individuals and mechanisms — to social media, newscasters and print reporters, and politicians — least appropriate to decide complex medical facts.”

Families no longer need to host a community walk to raise funds and awareness for their loved one. Movements now begin with a compelling photo and #Save[PatientName] Twitter hashtag. As healthcare communicators, our role is not only to bring visibility to these untold patient stories, but also to share the perspective of the companies who must make these difficult access decisions.

Drugmakers need strategic guidance to communicate the complexities of the process and limitations, with a greater sense of transparency and understanding. Regulatory agencies need support addressing concerns and risks, and patient advocacy organizations will continue to need their voices amplified.

As Forbes’ Kroll puts it, we are not yet there in finding a middle ground on compassionate use that does not favor those who mount a social media following. But to reach this greater sense of transparency and collaboration, all parties will need cross-channel communications counsel – from combatting the Ebola virus to navigating the difficult challenge of providing a drug to one patient over another.