Putting People First

As 2019 comes to a close, I wanted to take a moment to reflect.

As healthcare professionals, I have always felt that it is our responsibility to prioritize people above all else. Every one of us strives to identify new and better ways to ensure the patients and consumers we serve are the inspiration and guidepost for every decision we make.

At Johnson & Johnson we have held steadfast to our commitment to putting people first. That’s one of the reasons we felt driven to create our novel approach to compassionate use nearly five years ago.

Compassionate use requests are urgent requests for investigational medicines from seriously ill patients around the world when clinical trials aren’t available or when they have no remaining treatment options. The review of requests for these investigational medicines as outlined by the FDA requires ethical considerations for everyone involved, including the scientists in industry who are conducting the research studies, the regulators who review medicines to ensure safety and efficacy, the treating physicians who want the best for their patients, the patient advocates, and most importantly, the people whose lives might depend on this decision.

It’s an incredibly difficult decision for all involved.

And, while promising new science and technological advancements bring hope for patients – there are also ethical challenges to be considered. The need for a fair and ethical process to review compassionate use requests made to industry for these investigational medicines is greater than ever before.

These challenges inspired our approach to compassionate use.

In 2015, along with our colleagues at Janssen Pharmaceutical Companies of Johnson & Johnson, we partnered with the Division of Medical Ethics at the New York University School of Medicine to establish the Compassionate Use Advisory Committee or CompAC. This external, independent group of experts reviews compassionate use requests, along with clear, centralized and ethical procedures. The CompAC includes independent physicians, ethicists, and most importantly, patient representatives. When evaluating compassionate use requests, CompAC applies bioethical principles that include not putting patients at risk of unnecessary harm, having all patient requests blinded to ensure fair and objective review, and recognizing the importance of clinical trials to study new medicines with the goal of having an objective review by health authorities and the ability to provide access to an even greater number of patients in need.

The result is a more consistent, transparent and equitable process which balances the needs of society with the needs of individual patients.  

While CompAC began as a pilot for one investigational medicine for multiple myeloma, more than four years later it has evolved to serve a portfolio of investigational medicines for serious and life-threatening illnesses including oncology, infectious diseases and neurological health.

Recognition by Reagan-Udall Foundation’s 2019 Innovations in Regulatory Science Awards last week was an incredible acknowledgement of the impact of an effort between independent groups, working together using a fair and objective process, all on behalf of patients in need.

As we enter a new decade, I am more excited than ever to see what we will accomplish in 2020 and beyond.