Encouraging and Paying for Clinical Trials, Right to Try, and Expanded Access: Part One

A Q&A with Mark Shapiro, PhD,Vice President of Clinical Development at xCures, Inc., Partner at Pharma Initiatives; mshapiro@xcures.com

Q: Treatment of Americans with advanced cancer is complex and challenging and can be very expensive. Many urge greater participation of such patients in clinical trials. In general, who pays the expenses of clinical trials? And, specifically, how are the costs for Right to Try and expanded-access approaches reimbursed?

A: Incorporating clinical research into the clinical care of cancer patients may provide more options, and better outcomes, but participation is quite low. In 2004, only about 3% of American cancer patients participated in clinical trials.

More recent data suggest that the number may now be about 5%, although it is lower for women, children, minorities, and patients in community settings. The low figure should be of concern for a couple of reasons. First, patients are the scarcest resource in cancer research. Low participation in clinical trials represents a lost opportunity to learn and improve care. If every patient were part of systematic research, we could greatly accelerate the pace of cancer research findings. Second, most cancer treatment guidelines recommend a clinical trial as the standard-of-care at some stage in the course of disease. So, with current levels of participation, as many as 95% of American cancer patients are NOT receiving standard-of-care treatment at some point in their care. This deficit is partially attributed to the presence of comorbidities or poor function. Recent research suggests that liberalizing inclusion and exclusion criteria in clinical trials could increase enrollment by about 45%. In the study of common cancers, enrollment of patients with solid tumors could be increased from about 7% to 11%. Continue reading…

At Diagnosis, What Do Cancer Patients Want?

A Q&A with Laura Benson, RN, MS, ANP, president of Conversations in Care, LLC; LauraBensonRN@Gmail.com

Q: In our digital communication world of 2019, some patients may receive the initial message that YOU HAVE CANCER by cell phone, text, email, or even voice mail. When this happens, what do patients most want, and how can that best be accomplished?

A: When I first read your question, I was immediately brought back to a National Cancer Survivors Day around 1988. During the open microphone portion of the program, a patient bravely rose to address the audience. She proceeded to tell us she didn’t know she had cancer until she received the Survivors Day invitation, whereupon she turned to her attending physician and asked, “Well doctor, do I have cancer?” Since then, communication in today’s digital age has not seen a vast improvement. Indeed, a recent report out of California tells the tale of a patient and family learning he was dying via a video robot interaction.

What has not changed over the decades is the need for information that is easily understood, easily accessible, scientifically accurate, and delivered with compassion and connection. The dawn of the “Dr.Google” age has brought new and unique challenges. Continue reading…

Capturing Patients’ Real-World Experiences to Improve Cancer Research and Care

A Q&A with Grace Castillo-Soyao, founder and CEO of Self Care Catalysts; grace@selfcarecatalysts.com

Q: You are well known as a visionary in the field of Real World Experience-Evidence (RWEE). As the founder and CEO of Self Care Catalysts, headquartered in Toronto, how do you see RWEE evolving to favorably impact the field of oncology?

A: I started Self Care Catalysts with some very basic questions. Why is the patient at the farthest end of the care line, treated as simply the recipient of care? It’s a very industrial mindset, a bit like an assembly line; the patient as something to be acted upon. But patients are often experts at their own conditions, including the many kinds of cancer. Why are they not invited to become participants in their own care, in contributing their own experiences? Why are patient experiences not considered to be scientifically valid?

As noted physician Sir William Osler famously said, “It is much more important to know what sort of a patient has a disease than what sort of a disease a patient has.” Continue reading…

The Crucial 90% Missed by Doctors on Computers

A Q&A with Kevin B. Knopf MD, MPH, chairman of hematology and oncology at Highland Hospital in Oakland, California; kevinbknopf@gmail.com

Q: A successful patient-physician relationship depends upon effective bidirectional attention and mutual understanding. Many patients and physicians believe that common current versions of mandated electronic health records (EHRs) severely impede that interaction, especially eye contact. How can a competent and caring clinical oncologist overcome that problem?

A: For all my faults as a doctor, and I’m sure there are many, there is one thing I think I do correctly, and that is I am never on a computer in front of a patient.

I hear from many colleagues that they can be efficient and personable while going back and forth from the patient to their electronic health record (EHR)—and it is true there are various levels of skill here. However, none do as well, in my opinion, as a computer-free patient environment. I say this from my side as a patient having seen dozens of doctors myself—nothing compares to a doctor who spends all of their time looking you in the eye and interacting face to face. This human contact costs nothing, and yet is so vital. Continue reading…

Challenging Oncology Therapies With Moonshot Price Tags

A Q&A with Pramod John, PhD, CEO of VIVIO Health, a specialty drug management company in San Leandro, CA, that aims to provide better outcomes at lower costs; pramod@viviohealth.com

Originally published December 13, 2017

Q: Some American pharmaceutical companies are well-known for pricing drugs at “whatever the market will bear.” In oncology, some specialty drugs seem to have price tags completely unrelated to the proven effectiveness of the drug. Your company has been taking a lead in confronting this problem. What do you envision as possible solutions?

A: New oncology therapies carry astronomical price tags—most people know this. Receiving far less attention is the question of actual therapeutic value. Drug manufacturers spend billions on advertisements and PR, but unfortunately, real-world patient results are frequently unimpressive. Two recent articles in BMJ make this point, 1) No evidence of benefits for popular oncology therapies and 2) Do cancer drugs improve survival or quality of life? Continue reading…

Best Uses of Physical Medicine and Rehabilitation in Patients with Cancer

A Q&A with Val Jones, MD, Medical Director of Admissions, Saint Luke’s Rehabilitation Institute, Spokane, WA

Originally published February 1, 2017

Q: Your principal practice in Spokane, Washington is physical medicine and rehabilitation (PM&R). What do you find to be the best uses of PM&R in patients with cancer at your facility?

A: Rehabilitation medicine is one of the best-kept secrets in healthcare. Although the specialty is as old as America’s Civil War, few people are familiar with its history and purpose. Born out of compassion for wounded soldiers in desperate need of societal re-entry and meaningful employment, “physical reconstruction” programs were developed to provide everything from adaptive equipment to family training, labor alternatives and psychological support for veterans.

Physical medicine and rehabilitation (PM&R) then expanded to meet the needs of those injured in World Wars I & II, followed closely by children disabled by the polio epidemic. In time, people recognized that a broad swath of diseases and traumatic injuries required focused medical and physical therapy to achieve optimal long term function. Today, cancer patients frequently benefit from comprehensive rehabilitation as they recover from the effects of chemo (neuropathy, weakness, and cognitive impairments), radiation (scarring and range of motion limitations), surgery (flaps, plastics procedures, tumor resection, amputations), and brain injuries (edema, debulking, gamma knife and neurosurgery). Continue reading…

The Promise of Plerixafor in Glioblastoma Treatment

A Q&A with Adan Rios, MD; Professor in the Division of Oncology-Department of Internal Medicine of The University of Texas McGovern Medical School at Houston, Texas Medical Center; adan.rios@uth.tmc.edu

Q: Glioblastoma multiforme (GBM) remains a scourge with a typically rapid fatal course resistant to most therapy. All solid tumors must receive sufficient blood supply to grow. Plerixafor is an FDA-approved drug that may inhibit tumor angiogenesis. How might plerixafor be sensibly used off-label as an adjunctive therapy for GBM?

A: Glioblastoma multiforme (GBM) is a CNS (central nervous system) tumor with post-therapy median time to progression of 7 months and median overall survival of 15 months. I decided to use plerixafor for the prevention of recurrence of GBM in one patient treated with standard chemo-radiotherapy five years ago and since then have studied this patient and this subject in depth. Continue reading…

Who Owns Patient Data in Clinical Research?

A Q&A with Charlotte J. Haug, MD, PhD, MSc, International Correspondent, New England Journal of Medicine; Senior Scientist, SINTEF Techology and Society; Adjunct Affiliate, Stanford Health Policy; Oslo, Norway; charlottejohanne@gmail.com

Originally published October 25, 2017

Q: Many people are coming to believe that active patient participation will be a key to more rapid movement forward in cancer research. Data sharing can help. But who owns the data? And what rights and responsibilities are thus conferred? Your recent NEJM article provides helpful background. Can you help us better understand?

A: Exchange of data between patients and doctors is essential for the practice of medicine – and patient data are essential for medical research and progress.

Traditionally, doctors collected patients’ health information (typically the medical history, laboratory tests, drugs prescribed, outcome of treatment, etc.) and sometimes shared that information, in confidence, with colleagues to seek advice and advance science. The medical record was the physician’s property, and still is in many countries and legislations. But do physicians own the patient data? Continue reading…

Might Cancer Be a Metabolic Disease?

A Q&A with Thomas N. Seyfried, PhD, Professor of Biology, Boston College

Q: As a geneticist, you know that the genomic makeup of cancers recently has captivated much of the scientific community with new knowledge and new treatments. And yet, cancer outcomes remain dismal for many patients. You have written about cancer from a very different perspective. Why do you consider cancer to be a metabolic disease, and how might we look at different therapeutic options under that rubric?

A: Over 1,600 people die each day from cancer in the U.S., according to recent data from the American Cancer Society (Siegel et al., 2018). The failure to manage cancer has been due in large part to the dogmatic belief that cancer is a constellation of genetic diseases. Accumulating evidence, however, indicates that cancer is primarily a mitochondrial metabolic disease involving disturbances in energy production through respiration and fermentation (Seyfried et al., 2014).

The disturbances in tumor cell energy metabolism are linked to abnormalities in the structure and function of mitochondria that disrupt ATP synthesis through oxidative phosphorylation (OXPhos) (Seyfried, 2015; Seyfried & Shelton, 2010). Consequently, all cancer can be considered a single disease with a common pathophysiological mechanism involving dysfunction of mitochondrial OxPhos. As reactive oxygen species (ROS) arise from defects in mitochondrial OxPhos, the gene mutations observed in various cancers and all other recognized cancer hallmarks are considered downstream effects, and not causes, of the initial disturbance of cellular energy metabolism (Seyfried, 2012; Seyfried et al., 2014). Continue reading…