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…


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…


ASCO Offers Path to Addressing Affordability of Cancer Drugs in New Position Statement

Excerpt:

“The American Society of Clinical Oncology (ASCO) today issued a position statement aimed at contributing to the national dialogue on rising cancer drug prices. The statement, which asserts that any solutions must also preserve patients’ access to care and foster innovation, analyzes a wide array of options and recommends that a panel of stakeholders be established to determine which proposals will be effective and develop a uniform approach for assessing the value of drugs.

“The ASCO position statement highlights that new cancer drugs routinely cost more than $100,000 per year, and prices on many existing treatments continue to rise, causing serious financial hardship even for many patients with insurance. Patients with cancer are more than twice as likely to declare bankruptcy as those without cancer;  nearly six in 10 report being distressed about their finances during treatment.  Many patients forego or delay treatments as a result, potentially compromising their effectiveness. Drugs are the fastest growing component of cancer care costs, which are expected to increase by more than 25 percent between 2010 and 2020.”

Go to full article.

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.


The US Throws Away $3 Billion Worth of Cancer Drugs A Year

“You already know you pay too much for prescription medication. But a new study by Memorial Sloan Kettering Cancer Center reveals one simple factor that may be contributing to high costs: Waste.

“According to the new study, published Tuesday inBMJ, as much as $3 billion worth of cancer drugs is thrown away every year, unused. The reason why is drug manufacturers package single dose vials that contain more medicine than needed so that leftover medication is simply thrown away. As the authors, which includes Peter Bach, director of MSK’s Center for Health Policy and Outcomes, note:

” ‘These drugs must be either administered or discarded once open, and because patients’ body sizes are unlikely to match the amount of drug included in the vial, there is nearly always some left over. The leftover drug still has to be paid for, even when discarded, making it possible for drug companies to artificially increase the amount of drug they sell per treated patient by increasing the amount in each single dose vial relative to the typically required dose.’ “


Removing More Breast Tissue during Surgery Reduces Costs

“A 10-minute procedure to remove a little more tissue during a partial mastectomy could spare thousands of breast cancer patients a second surgery but and also cut costs by as much as $750 per patient, according to a Yale Cancer Center study.

“The findings are scheduled for presentation Dec. 10 at the 2015 Annual San Antonio Breast Cancer Symposium.

“Nearly 300,000 women in the United States are diagnosed with breast cancer each year; more than half undergo breast-conserving surgery with a partial mastectomy to remove the disease. About a third of patients who undergo this procedure have ‘positive margins,’ or cancer cells found at the edge of the removed tissue, and will require a second surgery to ensure that no cancer remains. A Yale study, published online in May in the New England Journal of Medicine, demonstrated that removing more tissue all the way around the tumor site during the initial surgery — known as cavity shave margins (CSM) — could cut the need for a second surgery in half.”


Pertuzumab Not Cost Effective in Metastatic HER2-Positive Breast Cancer

“Though the addition of pertuzumab to docetaxel and trastuzumab as first-line therapy for HER2-positive breast cancer has been shown to yield a substantial survival benefit, a new analysis shows that there is very little chance that pertuzumab would be cost effective in the United States.

“The CLEOPATRA trial showed that pertuzumab along with docetaxel and trastuzumab (THP) resulted in a median survival in HER2-positive metastatic breast cancer patients of 56.5 months, compared with only 40.8 months for the latter two drugs alone (TH). ‘These exceptional results come at a price,’ wrote researchers led by Ben Y. Durkee, MD, PhD, of Stanford University in California. ‘Our work shows that an insurer could expect to pay $4,649 per week for the THP regimen at Medicare rates. Private contractors and smaller entities would pay more.’

“The researchers used a decision-analytic Markov model to evaluate the regimen’s cost effectiveness, based on the study population from CLEOPATRA and the assumed number of patients for whom the THP regimen would be recommended in the metastatic setting. Results were published online ahead of print in the Journal of Clinical Oncology.”


Super Advocate: Laura Gheorghiu Seeks Better Care for Her Mom—and for Cancer Patients Across Canada


Laura Gheorghiu’s mother didn’t usually go to the doctor for just a cold. After she moved to Québec four years ago, it took a long time to get a family doctor through the public healthcare system and, until then, seeing one meant sitting for hours in a walk-in clinic. But she did seek care for a cold late last fall. For one thing, she finally had a doctor. For another, she had a really bad cold. Continue reading…