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

A Q&A with Mark Shapiro, PhD,Vice President of Clinical Development at xCures, Inc.Partner at Pharma Initiatives; mshapiro@xcures.com. This is the final installment in a three-part series in which Dr. Shapiro has shared his thoughts on the question below. Read part 1 and part 2.

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: In clinical research, agreements between the research sponsor and the treating institution define what aspects of a study protocol are charged as research or related administrative costs, and what items are considered standard-of-care; that is, eligible for billing to insurance. This is made by a coverage review at the institution. While the sponsors provide the study drug freely to the site and patients, they expect to receive valuable data in exchange. In expanded access, which is treatment rather than research—but stills follows a protocol approved by the U.S. Food and Drug Administration (FDA)— sponsors pay the required administrative costs and the free provision of the investigational drug. The drugs are expensive, and the sponsor incurs additional compliance costs when they make an investigational drug available. So, expanded access is largely a charitable act on behalf of the sponsor. While there are regulations allowing sponsors to recoup their costs under expanded access, these are rarely used. Most sponsors, especially larger companies, deliberately plan for expanded access when planning manufacturing campaigns during oncology drug development. In fact, large sponsors report that they approve about 95% of the expanded-access requests that they receive. Continue reading…


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

A Q&A with Mark Shapiro, PhD,Vice President of Clinical Development at xCures, Inc.Partner at Pharma Initiatives; mshapiro@xcures.com. Last week, Dr. Shapiro shared his initial thoughts on the question below. Today, he discusses issues of cost and equitable access to care.

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: In late-stage cancer care, treatment is very expensive. While there is a great deal of focus on the cost of the drugs, many other costs are involved, including the cost of care, the cost of the facility, and the cost of laboratory and other tests. When you add clinical research on top of care, there are additional tasks, but it is normally the research sponsor that pays for those administrative and research costs, which are incurred by physicians and the institutions conducting the clinical trial.

Insurance companies also pay for at least some of the associated costs of care. In fact, sponsors of cancer trials strive to design studies that follow existing standards of care to minimize the additional costs of non-standard procedures. The Affordable Care Act (ACA) specified that standard-of-care procedures delivered during a clinical trial could be charged to insurance for studies conducted under an Investigational New Drug application. Before the ACA, insurers in many states did not cover procedures performed when the patient was in a clinical trial, so the passage of the ACA can be credited with the increase of access to and enrollment in cancer clinical trials in the past few years. Patients also bear many of the costs of their cancer care, even when they are in clinical trials, because they are responsible for insurance copays and deductibles. Continue reading…


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…


Structural and Clinical Barriers Keep 3 of 4 Cancer Patients from Participating in Trials

Excerpt:

“A new meta-analysis led by Dr. Joseph Unger of Fred Hutchinson Cancer Research Center has revealed that structural and clinical barriers prevent more than 3 out of 4 cancer patients from participating in clinical trials.

“The study is part of an ongoing effort to understand why patient participation is so low in cancer clinical trials. Unger, a health services researcher and biostatistician who focuses on disparities in cancer research, published his findings today in JNCI, the Journal of the National Cancer Institute.”

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If you’re wondering whether this story applies to your own cancer case or a loved one’s, we invite you to use our ASK Cancer Commons service.


Yale Cancer Center Scientists Advise Caution in Immunotherapy Research

Excerpt:

“In a new study by Yale Cancer Center, scientists suggest that as the number of clinical trials in cancer immunotherapy grows exponentially, some caution should be exercised as we continue to better understand the biology of these new therapeutic targets.  The findings are published today in the journal Cell.

“Researchers around the world have been racing to create therapies that unleash the power of our immune systems against cancer. The most successful of these immunotherapies, which target a molecular pathway known as PD-1/PD-L1, have brightened the landscape for many people suffering with lung cancer and other types of tumors.”

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If you’re wondering whether this story applies to your own cancer case or a loved one’s, we invite you to use our ASK Cancer Commons service.


ASCO and Friends of Cancer Research Applaud NCI’s Expansion of Clinical Trial Eligibility Criteria

Excerpt:

“ASCO and Friends of Cancer Research (Friends) applaud the National Cancer Institute’s (NCI) recent revision of its clinical trial protocol template to broaden eligibility criteria for cancer clinical trials. The protocol template was expanded to help increase the opportunity for participation in NCI-funded clinical trials for patients with certain health-care conditions, as well as to provide an opportunity for patients younger than age 18 to participate in adult clinical trials in certain circumstances.”

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If you’re wondering whether this story applies to your own cancer case or a loved one’s, we invite you to use our ASK Cancer Commons service.


Brigatinib Confers Intracranial Responses in ALK-Positive Non-Small Cell Lung Cancer

Excerpt:

“Brigatinib conferred substantial intracranial responses and durable PFS among patients with brain metastases and ALK-positive, non-small cell lung cancer previously treated with crizotinib, according to ongoing study results.

” ‘Crizotinib [Xalkori; Pfizer, EMD Serono], the first licensed ALK inhibitor, is very active but has clear central nervous system liability from poor CNS penetration. All of the next-generation ALK inhibitor drugs have started to show CNS efficacy consistent with their superior activity in the brain compared with crizotinib,’ D. Ross Camidge, MD, PhD, director of thoracic oncology at University of Colorado, told HemOnc Today. ‘The whole clinical trials field has had to evolve around these events in terms of how we should capture and present CNS data. Brigatinib [Alunbrig; Takeda Oncology, Ariad] was one of the drugs that helped with this.’ ”

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Hypofractionation Is Ready for Prime Time in Prostate Cancer, but Will It Be Adopted?

“Separate phase III trials presented at the 2016 Genitourinary Cancers Symposium demonstrated that modest hypofractionated radiotherapy is noninferior to conventional radiotherapy for men with intermediate- and low-risk prostate cancer and should be considered a new standard of care.1,2 However, it is not clear how widely adopted hypofractionation schedules will be.

“NRG Oncology RTOG 0415 was a randomized, phase III, noninferiority study comparing two fractionation schedules in men with low-risk prostate cancer: conventional radiotherapy (73 Gy in 41 fractions over 8.3 weeks) vs hypofractionation (70 Gy in 28 fractions over 5.6 weeks).1

“The study enrolled 1,115 patients with low-risk prostate cancer. No androgen suppression was given. Patients were stratified according to Gleason score 2–4 and Gleason score 5–6.”