Why the 21st Century Cures Act is a Good Thing

A Q&A with Mary Woolley, President and CEO of Research!America

Q: You attended the December 2016 signing by President Obama of the 21st Century Cures Act and are recognized to be a strong supporter. Yet harsh criticism of it has quickly appeared in JAMA, BMJ, a variety of other venues, as well as on these pages. Please tell our readers why this is good legislation and how the public health will be protected from exploitation in this very different regulatory world.

A: The bi-partisan 21st Century Cures Act is grounded in a commitment to assuring that our nation’s research ecosystem has the capacity to accelerate the pace at which safe and effective medical advances reach patients. The Act will expand the efficiency, reach and impact of medical discovery in a manner that sustains crucial safeguards against unsafe or ineffective products. The law finances more research, helps to reduce the administrative cost surrounding basic research, and takes additional steps to overcome challenges the Food and Drug Administration (FDA) faces. Patient groups, health care professionals, academic leaders, industry leaders and the FDA and the National Institutes of Health (NIH) were frequently consulted regarding provisions of this bipartisan bill, and their insights were incorporated. We at Research!America were closely involved throughout development of the bill, and are pleased that it crossed the finish line last December. Continue reading…

Q&A: Air Traffic Control for Cancer

David K. Cundiff, MD
Jeff Shrager, PhD

A Q&A with David K. Cundiff, MD, Retired internist and palliative care physician from LA County + USC Medical Center; Email: dkcundiff@whistleblowerdoctor.org. Jeff Shrager, PhD, Director of Research, Cancer Commons; Adjunct Professor, Symbolic Systems Program, Stanford University; Email: jshrager@gmail.com.

Originally published August 16, 2017

Q: After May 17, 2017, you and Jeff Shrager engaged in a robust discussion about the place, if any, for an “Air Traffic Control system” called “Global Cumulative Treatment Analysis” (GCTA) for Cancer. Will you share excerpts with our readers?

DC: I see some major ethical and financial problems with GCTA:

  1. Who is going to authorize and pay for all treatment regimens (TRs) to be available to all patients at all times? USA health care costs are already out of control. An estimated one-third of all medical interventions do not benefit patients according to many evidence-based medical experts.
  2. How will vulnerable, terminally ill cancer patients and their loved ones be protected from ineffective, toxic, and expensive cancer treatments in the guise of advancing medical science?
  3. How will society be protected from the costs of ineffective cancer treatments with GCTA given that patient self-selection for experimental treatments generally means inconclusive results?

JS: There is no patient self-selection for experimental treatments in GCTA — or at least no more than there is now, and probably significantly less so. Under GCTA the patient, via their care team, is offered a set of options, along with rationales and data needed to make a rational choice about these options. This is just as it is today. The only difference is that under GCTA the set of options (rather than what your doctor happens to like) is guided by the GCTA process, which is running a global PROSPECTIVE adaptive trial over the whole equipoise set. Note that this is NOT “every damned thing” – just the things that we don’t know are better or worse.

Continue reading…

Forget Moonshots: Biomedicine Needs an Air Traffic Control System

A Q&A with Jeff Shrager, PhD, Director of Research, Cancer Commons; Adjunct Professor, Symbolic Systems Program, Stanford University

Originally published May 17, 2017

Q: There never seem to be enough patients matched to cancer clinical trials to quickly test new cancer treatments. Might there be a better way, using new communication technology?

A: Among the few things that everyone can agree upon, one is surely that biomedicine is not an efficient engineering system — that is, a system that efficiently (in time, cost, lives, or whatever else you’d like to measure) reaches its goals to alleviate suffering, etc. When we think of efficient engineering systems, companies such as Apple, Boeing, Google, Tesla, NASA, GE, and Toyota come to mind. Perhaps Big Pharma and parts of the VA and military are efficient engineering systems but surely biomedicine, as a whole, is not.

Granted, biomedicine faces the highly complex problem of matching patients to treatments while at the same time creating and ethically testing novel treatments with budgets that are under constant pressure. But this problem is faced by a huge number of extremely smart people. What is the difficulty? I contend that the difficulty is, in large part, lack of coordination. Continue reading…

Cancer Pain and the Opioid Epidemic

A Q&A with Kevin Sevarino, MD, PhD, President-elect of the American Academy of Addiction Psychiatry and Consulting Psychiatrist at Gaylord Hospital in Wallingford, CT

Q: Opioid abuse, addiction, and overdose are huge American problems right now. Many cancer patents experience chronic pain. What is the best way to use opioids to manage chronic pain?

[Note: The views expressed below represent the opinion of the author, and do not necessarily reflect the views of the American Academy of Addiction Psychiatry nor those of Gaylord Hospital.]

A. We live in amazing times. Targeted immunotherapies, stem cell transplants of transfected cells, identification of unique molecular targets in cancer cells through differential gene expression profiling—all promise to expand survival rates (or cures!) with diminished adverse effects compared to the “blunt hammer” approach of chemotherapy, radiation treatments, and more. Continue reading…

Proposed FDA “Conditional Approval”- More Details

A Q&A with Al Musella, DPM, President, Musella Foundation For Brain Tumor Research & Information, Inc., Hewlett, NY. Marty Tenenbaum, PhD, Founder and Chair, Cancer Commons, Los Altos, CA

Originally published May 10, 2017

Q: Your April 5, 2017 blog post that proposed a new “Conditional” category for FDA drug approval elicited a number of positive and negative responses. Please explain the proposal in more detail to enable better reader understanding.

A: In Response to “Conditional Approval: Right Solution for the Wrong Problem” by
Shannon Brownlee:

We appreciate Ms. Brownlee’s comments on our recent blog post, but think she missed our key points—perhaps we weren’t clear enough: Continue reading…

Conditional Approval: Right Solution for the Wrong Problem

A Q&A with Shannon Brownlee, MS, Senior Vice President of the Lown Institute, a think tank in Boston. She is also co-founder of the Right Care Alliance, a social movement for transforming health care.

Originally published April 26, 2017

Q: Musella and Tenenbaum recently proposed a new way, called conditional approval, for the American FDA to move potentially useful drugs to a patient market. They wrote that safety would be covered and efficacy assessed by a registry. What do you think of that idea?

A: Imagine if there were a way to speed up the discovery and testing of drugs for cancer. Al Musella and Marty Tenenbaum, founders of two cancer patient advocacy organizations, think they have just such a plan. Continue reading…

A Proposed New FDA Drug Approval Pathway: “Conditional”

A Q&A with Al Musella, DPM, President, Musella Foundation For Brain Tumor Research & Information, Inc., Hewlett, NY. Marty Tenenbaum, PhD, Founder and Chair, Cancer Commons, Los Altos, CA

Originally published April 5, 2017

Q: The delay time from discovery/observation, through validation to approval and distribution/use of new cancer treatments remains excessive. With promising experimental treatments, advanced computer technology and biostatistics, creative alternatives to traditional randomized clinical trials, and a government seeking efficiencies, might it now be time for the FDA to issue: “Conditional Approvals”?

A: The first advances in oncology occurred at a time when there were no regulations. Doctors had ideas, and put them to work immediately. They adjusted and combined treatments as needed until they were optimized and became standard treatments. Many types of cancer were cured by this work. Continue reading…

How to Tell a Patient Their Cancer Has Spread

A Q&A with crisis communication expert Lisa Dinhofer, MA, CT

Q. As a counselor and communicator, you are expert and experienced in managing serious situational difficulties up to and including coping with sudden unexpected death. How would you think it best to approach a person with cancer who is being told, “your cancer has spread”?

A: I’ll answer this question by posing another—how did you discuss the diagnosis initially? Did you jointly establish expectations for addressing this illness going forward?

How a diagnosis is delivered plays a critical role in future conversations around how the illness is responding—or not—to treatment. This initial conversation is the foundation for many more that could go in various directions dependent on disease progression, regression, and patient tolerance. Continue reading…

How I Survive Cancer

A Q&A with Erin Maloney: Intrepid explorer, Amateur photographer, Aspiring leader; Toronto, ON

Email: erinLmaloney@gmail.com

Originally published Nov. 1, 2017


Q: You have recently disclosed that you have had a diagnosis of cancer and described your experience in some detail on Medium. What does it mean to you to be a “Cancer Survivor”?

A: Calling myself a survivor sometimes feels like an exaggeration. In 2016, I was diagnosed with Stage 1A2 squamous cell carcinoma of the cervix. It began with a routine pap smear and led to a robotic laparoscopic radical trachelectomy seven months later. Every procedure was challenging, and surgery was particularly arduous. However, I did not have to endure radiation, brachytherapy, or chemotherapy. I got to keep my hair, didn’t have to cope with nausea or worry about the lifelong maintenance required after radiation. So, in many ways, calling myself a survivor feels fraudulent. I have it too easy. Continue reading…