Al's Comment:

 These are my thoughts on the right to try law. The Senate passed it already and the House is considering it. 

 

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Posted on: 02/14/2018

Dear Mr. President, Speaker Ryan, Leader Pelosi and Dr. Gottlieb:

 We are writing to give you our thoughts on the “Right to Try” bills: S.204, The Trickett

Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2017, as well as H.R.878, the Right to Try Act of 2017.

 While we strongly believe in providing access to experimental drugs for life threatening diseases, we believe there is a better way than Right to Try.

 Right to Try, under its current implementation, removes the FDA from the loop, with no protection for the patients and no way to collect and analyze the outcomes.  Each person’s experiences go to waste with people repeating the same mistakes over and over and not learning from each case.

 Our preference is a new pathway to approval, called “Conditional Approval”. It would empower the FDA to approve drugs for dying patients after a phase 1 trial shows they are relatively safe and have some biologic effect.  Patients receiving such drugs would be followed in a “virtual trial” registry so that we can see how they work, alone and in combination with other therapies. We can look for side effects and home in on the best cocktail.  See https://virtualtrials.com/fda2017.cfm for details.  We have had several productive discussions with the FDA about this, but need your help to get it implemented. 

 If you are committed to Right to Try, please at least consider requiring that all patients receiving such treatments participate in a “Virtual Trial” registry, so that their outcomes and side effects can be tracked.  A Virtual Trial is better than a classic phase 2 or phase 3 trial in that every patient on the drug is observed – not just the 2% of patients who fit the rigid qualifications of a trial.  Since these patients are not cherry picked, the FDA should not be allowed to use the results from a Virtual Trial results to deny approval. However, if the Virtual Trial shows a significant improvement over the standard of care, then under the 21^st Century Cures Act, the FDA can use the resulting real world data to grant approval.

 Sincerely,

 

Al Musella, DPM   [Contact Person]
President / Founder
Musella Foundation For Brain Tumor Research & Information, Inc
1100 Peninsula Blvd
Hewlett, NY 11557
888-295-4740
musella@virtualtrials.com

Keith L. Black, MD
Chairman and Professor, Department of Neurosurgery
Director, Maxine Dunitz Neurosurgical Institute

John A. Boockvar, MD
Director, Brain Tumor and Pituitary/Neuroendocrine Centers
Lenox Hill Hospital /Manhattan Eye, Ear and Throat Hospital

Henry S. Friedman, MD
Deputy Director, The Preston Robert Tisch Brain Tumor Center at Duke, NC

Roberta Hayes, PhD.
Associate Professor, St John's University

Neal Houslanger, DPM
Musella Foundation For Brain Tumor Research & Information, Inc

Linda Liau, MD, Ph.D.
Director, UCLA Brain Tumor Program

Mario Lichtenstein
President  - Voices Against Brain Cancer

Lisa Millar
Founder and President - The Brad Kaminsky Foundation

Dellann Elliott Mydland
Founder and President - The EndBrainCancer Initiative

Hideho Okada MD, PhD
University of California, San Francisco
Department of Neurological Surgery

Michael Schulder, MD
Director, Brain Tumor Center
Zucker School of Medicine at Hofstra Northwell

Simone Treiger Sredni, MD, PhD
Associate Professor - Pediatric Neurosurgery
Northwestern University Feinberg School of Medicine

Marty Tenenbaum, PhD
Founder & Chairman - Cancer Commons 

Lee Tessler, MD, FAANS, FACS
Chief, Division of Neurosurgery, NYU Winthrop Hospital
Executive Director, Long Island Brain Tumor Center
Assistant Clinical Professor of Neurosurgery, NYU Langone Medical Center

Paul Zeltzer M.D.
Co-Founder and Director, Navigating Cancer, Inc.

 

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