Expanded Access Programs 2016
From left to right: Ken Moch (former CEO, Chimerix); Richard Plotkin (Max Cure), Debra Birnkrant (FDA), Brooke Gladstone (NPR, Moderator), Nancy Goodman (KidsvCancer), Meg Tirrell (CNBC)
On October 28, 2015, Richard Plotkin appeared as a panelist at a two-day colloquium at New York Academy of Science sponsored by NYU School of Medicine, NYU Langone Medical Center, and Johnson & Johnson. The subject of the program is identified as, “Pre-Approval Access: Can Compassion, Business, and Medicine Coexist?” The issue of the use of experimental drugs outside clinical trials for terminally ill patients who have exhausted all approved treatments for their diseases is a fast growing phenomena, especially after the family of 7-year old Josh Hardy, through an extensive international social media campaign, was successful in obtaining for Josh an experimental drug manufactured by a small North Carolina Biotech firm, Chimerix. Not only is Josh alive today, almost two years after receipt of the drug, due to his receipt of this drug, but it is reported that 89 other patients, mostly children, who received the drug as part of the clinical trial established specifically for Josh are likewise alive today because of receipt of that drug. Richard was invited to appear on the panel, entitled, “The Case of Josh Hardy: Social Media and Compassionate Use,” because of his leading the effort to save Josh’s life back in March, 2014. The synopsis of the various panel discussions over the two day period is now available online and can be retrieved here. Richard is quoted in the synopsis as stating during his panel presentation, “If this boy didn’t get the drug, I was going to destroy Chimerix and Ken Moch [its CEO]. There was a 7-year old boy who was going to die.”
It has been said that ever since the Josh Hardy matter, “Few topics in modern health care are as ethically complex, emotionally wrenching and financially fraught as compassionate use, or pre-approval access. To enter the world of compassionate use is to meet the sickest patients, to witness last-gasp efforts to save the terminally ill. It is the desperate process of pleading for access to experimental therapies not yet approved by the Food and Drug Administration (FDA) – compounds that may or may not be safe or effective. For patients, the stakes – life and death – could not be higher; for companies and regulators, the financial, ethical and social costs are very high as well.
The general population also has a stake in these decisions that affect which drugs come to market, and when.” The Max Cure Foundation is privileged to have played a leading role in saving the life of Josh Hardy and indeed, is proud that its co-founder, Richard Plotkin, is recognized as the leader in accomplishing that feat and has become one of the leading patient advocates asked to speak often on the ethical and legal issues that have arisen since Josh Hardy received the experimental drug in March, 2014.
On February 23 and 24, 2016, Richard will be appearing as a panelist in a program entitled, “Expanded Access Programs 2016.” Richard’s panel discussion is on February 23rd at 1:30pm at the Doubletree By Hilton Bethesda/Washington, DC. The subject of that panel discussion is entitled, “Stakeholder Collaboration Colloquium, Fostering a United Front for Streamlined Access Programs.” Richard will in part discuss how he transitioned over the last two years from a role he envisioned as a lawyer and advocate for Josh Hardy only (Richard had a 40 year career as a trial lawyer before retiring and starting The Max Cure Foundation) to one as a pediatric cancer advocate for not only terminally ill children with cancer who had exhausted all approved therapies, such as Josh, but ALSO for the countless other children who might need the then experimental drug in the future.