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.
The new company xCures is working with Cancer Commons to help cancer patients who cannot participate in clinical trials—either because they are too sick, otherwise don’t qualify, or don’t live near enough to a center conducting a suitable trial. To help these patients gain access to medically logical therapy, we create a bridge between the research sponsor’s companies, physicians, and patients to facilitate expanded access. We are focused on reducing the administrative burdens and barriers that inhibit these stakeholders from providing expanded-access treatments.
Right to Try has a similar set of requirements as expanded access but removes some of the administrative time and cost involved, such as oversight from an institutional review board and the U.S. Food and Drug Administration (FDA) or following a formal treatment protocol that defines the safe use of the treatment. It also removes the need to notify the FDA of serious and unexpected adverse drug reactions altogether. That caused ethicists some concern since it is important to learn whether a patient was helped or harmed and share that information with other patients or doctors. It also makes assessing the effects of the law difficult for policymakers.
While the FDA has been clear that expanded access can help and rarely hurts research sponsors, some companies are reluctant to offer expanded access out of fear that they will learn something that might harm their chances for a drug approval. A review of hundreds of non-disclosure agreements and tens of thousands of patients treated under expanded access had laid this fear to rest. But, a clear advantage for sponsors is that under Right to Try, the law disallows the use of outcomes from Right to Try patients to adversely affect the review of the product by FDA. This type of clarification would certainly help encourage expanded access but risks creating a problem of unreported results even as new legal requirements for registration and reporting of clinical trials were implemented under 42 U.S.C. § 282(j)(5)(B).
So, Right to Try is a new tool in the toolkit for seriously ill patients, but the pathway for expanded access is better defined and may be less expensive to implement for most stakeholders, points I will discuss in part three of this series.
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Copyright: This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.
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.
The new company xCures is working with Cancer Commons to help cancer patients who cannot participate in clinical trials—either because they are too sick, otherwise don’t qualify, or don’t live near enough to a center conducting a suitable trial. To help these patients gain access to medically logical therapy, we create a bridge between the research sponsor’s companies, physicians, and patients to facilitate expanded access. We are focused on reducing the administrative burdens and barriers that inhibit these stakeholders from providing expanded-access treatments.
Right to Try has a similar set of requirements as expanded access but removes some of the administrative time and cost involved, such as oversight from an institutional review board and the U.S. Food and Drug Administration (FDA) or following a formal treatment protocol that defines the safe use of the treatment. It also removes the need to notify the FDA of serious and unexpected adverse drug reactions altogether. That caused ethicists some concern since it is important to learn whether a patient was helped or harmed and share that information with other patients or doctors. It also makes assessing the effects of the law difficult for policymakers.
While the FDA has been clear that expanded access can help and rarely hurts research sponsors, some companies are reluctant to offer expanded access out of fear that they will learn something that might harm their chances for a drug approval. A review of hundreds of non-disclosure agreements and tens of thousands of patients treated under expanded access had laid this fear to rest. But, a clear advantage for sponsors is that under Right to Try, the law disallows the use of outcomes from Right to Try patients to adversely affect the review of the product by FDA. This type of clarification would certainly help encourage expanded access but risks creating a problem of unreported results even as new legal requirements for registration and reporting of clinical trials were implemented under 42 U.S.C. § 282(j)(5)(B).
So, Right to Try is a new tool in the toolkit for seriously ill patients, but the pathway for expanded access is better defined and may be less expensive to implement for most stakeholders, points I will discuss in part three of this series.
***
Copyright: This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.
