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W-20: Building Patient Trust: Our Journey to "Radical" Transparency in Compassionate Use

Poster Presenter

Christine MacCracken

Head, Patient Strategies and Solutions, Office of the Chief Medical Officer
Janssen Pharmaceutical Companies of Johnson & Johnson
United States

Objectives

The objective of this initiative is to collaborate with patient representatives to co-create educational and awareness tools which enhance patient, provider and caregiver understanding of the compassionate use process.

Method

Since 2015, Janssen has collaborated with academia and advocacy groups to support the advancement of patient, provider and caregiver understanding of compassionate use through the development of educational and awareness tools.

Results

Janssen has committed to easing the burden of patients and providers who find themselves in the position of needing to make CU requests and as a result, embarked on several key initiatives: EXPANDED ACCESS AND COMPAC INFOGRAPHICS Working with patients and providers to ensure comprehensive while concise information, Janssen created a non-branded infographic depicting the key steps of making an expanded access request to both the company as well as the FDA. In addition, similar formatted tools have been created to provide a streamlined and user-friendly document for providers, patients and caregivers to digest journal publications related to the Janssen/NYU CompAC collaboration. CONVENING PATIENTS FOR PAA DISCUSSIONS To promote productive dialogue with patients, Janssen has conducted a series of patient/provider driven webinars; these events help ensure patient input is considered, discussed and acted upon. Additionally, both EU and US Advocacy pre-approval summits, first in industry, were held in 2016/18 respectively, convening advocacy, policy and industry to collectively discuss compassionate to advance the needs of patients. In 2018, an EU Summit was conducted to capture patient voices globally. TRANSPARENCY FOR PATIENTS In 2018, an important change was made to the Janssen website to reflect not only its policies and procedures but also potential investigational medicine options. RIGHT TO TRY (RTT) In advance of the federal passing of Right to Try legislation in 2018, Janssen convened a cross functional leadership team to consider the handling of Right to Try requests and almost 5 months prior to federal passage, Janssen posted its updated policy.

Conclusion

While the review and evaluation of compassionate use requests is well established for Janssen and other members of bio-pharma, transparency of information and understanding of the process to request investigational medicines remains less so. In recent years, efforts to bridge these awareness gaps have included 21st Century Cures Act, 2016 and more recently the launch of the Reagan-Udall Navigator launched the following year. One creating requirements for transparency in decision making from companies with compassionate use programs, and the other creating a mechanism by which to learn about compassionate use and companies with potential options. Mutually exclusive but related activities, there is indeed an increase in companies with publicly posted policies which address decision-making, and, there is an increase in available information. Yet most parties are stymied when it comes to making an actual request. These efforts while representing important advancements for patients, must be complemented by the adjacent stakeholders who have the medications which are the basis of the requests—pharmaceutical companies. Janssen’s efforts toward increasing transparency and closing educational gaps are examples in the importance of genuine patient engagement. In compassionate use, these measures are intended to partially alleviate some burden during a particularly burdensome time while navigating complex health systems. While there is more collective work to be done across industry and advocacy to ensure this dialogue continues to seek solutions for seriously ill patients in need, the above initiatives represent real progress in advancing this important discussion.