Richard Plotkin, a Grandparents In Action charter member and founder, is taking action in what he believes to be a bad deal for kids fighting #ChildhoodCancer. If you feel as he does, please let your Representative and Senators know.
Congress is considering “Right to Try” (RTT) bills related to the use of unapproved medical products by patients diagnosed with a terminal illness. In essence, these bills seek to remove the use of experimental medical products outside of clinical trials from the oversight of the Food and Drug Administration (FDA) based on the false claim that the FDA moves too slowly. These bills, if enacted into federal law, would potentially injure the most vulnerable of patients, including children with life threatening diseases, and expose numerous unsuspecting parties to lawsuits given they would not have the “cover” and protection given them by the FDA.
My grandson was diagnosed with cancer in 2007. At that time I was a lawyer for a large regional law firm. In 2008, I retired and formed a childhood cancer foundation. In 2014 I was thrust into the public eye as the patient advocate credited with successfully securing an unapproved drug for a young cancer patient. Since that time I have been involved in obtaining experimental drugs for a number of other children with cancer. In each instance the FDA moved expeditiously to assist in obtaining the drugs.
If RTT becomes federal law, if offered the position as General Counsel for a pharmaceutical company or a biotech firm that welcomed the passage of this federal legislation, I would reject the offer. If I were a General Counsel, my company would not consider requests for experimental drugs under the federal RTT law under any circumstances – thus removing the potential benefits to terminally ill patients they might otherwise receive but for the proposed legislation.
Three years ago I attended a conference in Washington, D.C. for advocates for children with cancer. With regard to giving experimental drugs to children outside of clinical trials, I said: “These children are dying, what is the difference if they receive these drugs or not, there is no downside.” A pediatric oncologist responded, “If you had seen, as I have, children with cancer being given experimental drugs who were ‘tortured’ by the drugs as they died, you would not be so cavalier.” Those words changed my perspective and made me realize that while easy access to experimental medical products may seem a godsend to patients, the reality is much more complex.
This proposed legislation that I oppose is a brainchild of the Goldwater Institute which has been the moving force behind the passage in 33 states of what are known as Right to Try laws. The Goldwater Institute is seeking, based on what I believe is false and misleading information, to have Congress pass a federal RTT law that would remove the safeguards provided by the FDA.
They are doing this for desperate patients, patients who are unable to join clinical trials and for whom approved medicines offer no help. But these patients already have a route by which to get access to experimental treatments outside of clinical trials: the FDA’s expanded access program.
The FDA serves a valuable service in its review of the many applications for using experimental medical products outside of clinical trials. From Oct. 1, 2014 through Sept. 30, 2015, the FDA approved 1,256 such requests from a total of 1,262 received. These were not “rubber stamp” approvals: a recent study of 150 randomly-selected cases found that 11 percent of the cases that received FDA approval had alterations in the treatment plan due to input from the FDA, input that improved the safety and efficacy of the procedures. If that 11 percent figure is applied to the 1,256 requests, 138 cases were positively impacted by feedback from the FDA, feedback that would not be available under RTT legislation.
This is particularly important when children with terminal illnesses seek experimental drugs. These children are in poor health and those treating them typically do, and always should, welcome the review by the FDA with respect to, among other things, the dosage of the drug given to these children. That the drug may have completed Phase 1 (safety) or even Phase 2 (efficacy) clinical trials in adults does not portend equally satisfactory results when dealing with sick children. The review by the FDA gives those (adults and children) seeking compassionate use waivers a degree of “cover” from lawsuits that may later be filed against them.
Incredibly, the RTT bills create a pathway by which experimental drugs can be issued to patients with terminal illnesses without any oversight by the FDA. The bills specifically state that the Federal Government shall not take any action to prohibit or restrict the use of such drugs.
Under the proposed federal law, certain parties are not liable. However, physicians, hospitals, institutional review boards (IRB), parents of minor children, the Goldwater Institute and perhaps others not covered in the bills are not in the protected group. These parties can be sued in state and federal courts. They are not “producers, manufacturers, distributors, prescribers, dispensers, possessors or users.”
Finally, the bills under consideration provide that negative outcomes of the use of experimental medical products under RTT cannot be used by a federal agency (like the FDA) in deciding whether or not to approve the products for public use. This is absurd. What this means is that if the FDA is bypassed and an experimental medical product is given to a patient who suffers severe adverse effects as a result, the doctor and the drug company cannot provide this information to the FDA and, if the FDA learns of that situation, it must remain silent and take no action!
I am not suggesting the federal system in place now through the FDA is perfect. Congress through 21st Century Cures Act has improved the system and indeed, the FDA has implemented changes to expedite the approval process and to make applications to it much less burdensome.
If the RTT becomes federal law, any person being considered as FDA Commissioner ought to reject the offer. The law, if passed, would be contrary to public policy and could form the basis for lawsuits against all those involved in the process.
Richard L. Plotkin, Esq., retired from the private practice of law in 2008 and with his family formed The Max Cure Foundation, Inc., a non-profit charity whose mission is to advance the cause of pediatric cancers through research, family assistance and advocacy. Mr. Plotkin is one of the nation’s leading advocates for children with cancer and their families and has been consulted by Congress and by The Reagan-Udall Foundation in seeking his input on prospective changes to the existing federal laws with respect to expanded access/compassionate use when dealing with experimental drugs.