Kelly McBride Folkers - Academia.edu (original) (raw)

Op-eds/Reviews by Kelly McBride Folkers

Earlier this month, the Pennsylvania House of Representatives unanimously passed legislation know... more Earlier this month, the Pennsylvania House of Representatives unanimously passed legislation known as the "Right-To-Try Act." This bill, sponsored by Rep. Robert W. Godshall, R-Montgomery, promises to give dying patients access to investigational drugs that are not yet approved by the FDA. The bill, now before the Senate's Public Health & Welfare Committee, cannot and will not help the terminally ill and the Senate should not pass it.

Reasonable minds can argue about how and whether the language in the Cures Act –compromises hamme... more Reasonable minds can argue about how and whether the language in the Cures Act –compromises hammered out over two years - sets the stage for harm to patients. But what is undeniable is that over the years, the United States has swung between looser and stricter regulation of medical products and Cures continues an ongoing pendulum swing away from the strict regulation of new drugs and devices.

Papers by Kelly McBride Folkers

The Journal of clinical ethics, 2018

Evolving practice requires peer review of clinical ethics (CE) consultation for quality assessmen... more Evolving practice requires peer review of clinical ethics (CE) consultation for quality assessment and improvement. Many institutions have identified the chart note as the basis for this process, but to our knowledge, electronic health record (EHR) systems are not necessarily designed to easily include CE consultation notes. This article provides a framework for the inclusion of CE consultation notes into the formal EHR, describing a developed system in the Epic EHR that allows for the elaborated electronic notation of the CE chart note. The implementation of the "meaningful use" criteria for EHR, mandated by the Health Information Technology for Economic and Clinical Health (HITECH) Act of 2009, requires that health professionals meet certain standards for quality, efficiency, and safety, all of which overlap with the goals of standardization, peer review, and quality improvement within CE consultation.

The U.S. and Japan have preapproval access systems for patients who have exhausted approved treat... more The U.S. and Japan have preapproval access systems for patients who have exhausted approved treatment options. We compare the systems of both countries and discuss some ethical concerns surrounding non-trial access to investigational drugs. Notable points of comparison of the two countries' systems include cost of non-trial access to investigational drugs and purpose of the non-trial access system. All stakeholders and the public must understand the ethical issues associated with non-trial preapproval access systems. The current non-trial access programs in both countries depends on pharmaceutical companies' voluntary decisions about whether patients get access to investigational products. Moreover, the potential for inequity of access raises ethical concerns. Non-trial preapproval access is an exceptional way to practice medicine with various ethical and safety concerns, so we suggest that the scope and eligibility for using these pathways should thus be limited.

The ethical practice of medicine requires identifying the interests and rights of patients and cr... more The ethical practice of medicine requires identifying the interests and rights of patients and creating a corresponding set of obligations and duties to fulfill those interests and rights. Transgender and gender nonconforming (TGNC) patients have special needs surrounding their gender identity that present unique ethical challenges for medical professionals, family members, and the patient themself. While there is a strong moral imperative to provide gender-affirming care to TGNC patients to mitigate the risk of depression, suicide, and other poor mental health outcomes, these care options must be considered alongside a developing person’s capacity for understanding and consent, especially in a pediatric setting. As bioethical processes and principles are best understood as part of an analysis of the life stories of patients, we explore ethical issues surrounding gender-affirming care for TGNC patients throughout the life cycle in a series of case descriptions.

Journal of law and policy, 2020

This symposium article examines the use of investigational (un-approved) medical products in the ... more This symposium article examines the use of investigational (un-approved) medical products in the United States, with particular focus on who pays for this use. We examine the various forms of preapproval access (“PAA”) to experimental medical products available in the United States—clinical trials and non-trial preapproval access via the Expanded Access (“EA”) and Right to Try (“RTT”) pathways. For each, this paper analyzes which entity—individual, insurer, sponsor, or other—bears the cost and what limitations or caps, if any, exist on these costs. This paper considers various proposed novel payment mechanisms that may permit more equitable use of investigational medical products. Part I outlines payment-related disparities in access in-grained in the current United States healthcare system. Part II fo-cuses on access in the context of clinical trials, which most payers have begun to cover, but where remaining uncovered expenses can disincentivize participation in clinical trials, e...

BMC Research Notes

Objective To evaluate the availability of information regarding patient access to investigational... more Objective To evaluate the availability of information regarding patient access to investigational treatments through clinical trials and non-trial pre-approval access pathways from a sample of patient advocacy organization (PAO) websites in the United States. Results We systematically analyzed the content of 118 randomly selected PAO websites to assess whether they contained information on clinical trials and non-trial pathways—e.g., the U.S. Food and Drug Administration (FDA) expanded access (EA) program and right to try—over the course of two months from February to March 2019. A majority (81%, n = 96) of PAOs provided a link to ClinicalTrials.gov, and 73% (n = 86) had their own clinical trial finder or list of relevant trials. 23% (n = 27) mentioned EA, with 8% (n = 9) providing specific resources for FDA’s EA program. 8% (n = 10) provided a statement on the passage of the federal right to try law. A majority of PAO websites contained information on clinical trials, but a minorit...

The American Journal of Bioethics

Preapproval nontrial access, historically called “compassionate use,” and certain off-label usage... more Preapproval nontrial access, historically called “compassionate use,” and certain off-label usage of approved medical products are two forms of innovative practice, defined by Earl as a situation “...

Therapeutic Innovation & Regulatory Science

JAMA

Medical crowdfunding involves using social media platforms to appeal for help in paying for medic... more Medical crowdfunding involves using social media platforms to appeal for help in paying for medical care. The largest medical crowdfunding platform, GoFundMe, reported that all campaigns raised 3billionby2016,anincreasefrom3 billion by 2016, an increase from 3billionby2016,anincreasefrom1 billion in 2015. 1 Although medical crowdfunding campaigns can fill insurance gaps, they can also raise money for scientifically unsupported, ineffective, or potentially dangerous treatments. We quantified crowdfunding activity for 5 such treatments.

Pediatrics, Jan 6, 2018

Transgender and gender-nonconforming (TGNC) youth who suffer from gender dysphoria are at a subst... more Transgender and gender-nonconforming (TGNC) youth who suffer from gender dysphoria are at a substantially elevated risk of numerous adverse physical and psychosocial outcomes compared with their cisgender peers. Innovative treatment options used to support and affirm an individual's preferred gender identity can help resolve gender dysphoria and avoid many negative sequelae of nontreatment. Yet, despite advances in these relatively novel treatment options, which appear to be highly effective in addressing gender dysphoria and mitigating associated adverse outcomes, ethical challenges abound in ensuring that young patients receive appropriate, safe, affordable treatment and that access to this treatment is fair and equitable. Ethical considerations in gender-affirming care for TGNC youth span concerns about meeting the obligations to maximize treatment benefit to patients (beneficence), minimizing harm (nonmaleficence), supporting autonomy for pediatric patients during a time of ...

The Journal of clinical ethics, 2018

Evolving practice requires peer review of clinical ethics (CE) consultation for quality assessmen... more Evolving practice requires peer review of clinical ethics (CE) consultation for quality assessment and improvement. Many institutions have identified the chart note as the basis for this process, but to our knowledge, electronic health record (EHR) systems are not necessarily designed to easily include CE consultation notes. This article provides a framework for the inclusion of CE consultation notes into the formal EHR, describing a developed system in the Epic EHR that allows for the elaborated electronic notation of the CE chart note. The implementation of the "meaningful use" criteria for EHR, mandated by the Health Information Technology for Economic and Clinical Health (HITECH) Act of 2009, requires that health professionals meet certain standards for quality, efficiency, and safety, all of which overlap with the goals of standardization, peer review, and quality improvement within CE consultation.

Cancer research, Jun 6, 2017

Biomarkers predicting rapalog responses in sarcomas where PI3K and mTOR are often hyperactivated ... more Biomarkers predicting rapalog responses in sarcomas where PI3K and mTOR are often hyperactivated could improve the suitable recruitment of responsive patients to clinical trials. PI3K/mTOR pathway activation drives energy production by regulating anaerobic glycolysis in cancer cells, suggesting a route toward a monitoring strategy. In this study, we took a multi-modality approach to evaluate the phenotypic effects and metabolic changes which occur with inhibition of the PI3K/mTOR pathway. Its central role in regulating glycolysis in human sarcomas was evaluated by short- and long-term rapamycin treatment in sarcoma cell lines. We observed an overall decrease in lactate production in vitro followed by cell growth inhibition. In vivo we observed a similar quantitative reduction in lactate production as monitored by hyperpolarized MRI, also followed by tumor size changes. This non-invasive imaging method could distinguish reduced cell proliferation from induction of cell death. Our res...

The Hastings Center report, 2017

The parents of Charlie Gard, who was born August 4, 2016, with an exceedingly rare and incurable ... more The parents of Charlie Gard, who was born August 4, 2016, with an exceedingly rare and incurable disease called mitochondrial DNA depletion syndrome, fought a prolonged and heated legal battle to allow him access to experimental treatment that they hoped would prolong his life and to prevent his doctors from withdrawing life-sustaining care. Charlie's clinicians at the Great Ormond Street Hospital in London believed that the brain damage Charlie had suffered as a result of frequent epileptic seizures, along with many other severe disabilities, would render any innovative therapy futile, and they disagreed with his parents' wishes to use an experimental therapy. They felt it in Charlie's best interest that he be allowed to die. A battle ensued among Charlie's parents, his doctors, and a guardian who had been appointed to represent him that drew the attention of politicians and prominent persons from all over the world. The case was much in the news over the past year,...

Earlier this month, the Pennsylvania House of Representatives unanimously passed legislation know... more Earlier this month, the Pennsylvania House of Representatives unanimously passed legislation known as the "Right-To-Try Act." This bill, sponsored by Rep. Robert W. Godshall, R-Montgomery, promises to give dying patients access to investigational drugs that are not yet approved by the FDA. The bill, now before the Senate's Public Health & Welfare Committee, cannot and will not help the terminally ill and the Senate should not pass it.

Reasonable minds can argue about how and whether the language in the Cures Act –compromises hamme... more Reasonable minds can argue about how and whether the language in the Cures Act –compromises hammered out over two years - sets the stage for harm to patients. But what is undeniable is that over the years, the United States has swung between looser and stricter regulation of medical products and Cures continues an ongoing pendulum swing away from the strict regulation of new drugs and devices.

The Journal of clinical ethics, 2018

Evolving practice requires peer review of clinical ethics (CE) consultation for quality assessmen... more Evolving practice requires peer review of clinical ethics (CE) consultation for quality assessment and improvement. Many institutions have identified the chart note as the basis for this process, but to our knowledge, electronic health record (EHR) systems are not necessarily designed to easily include CE consultation notes. This article provides a framework for the inclusion of CE consultation notes into the formal EHR, describing a developed system in the Epic EHR that allows for the elaborated electronic notation of the CE chart note. The implementation of the "meaningful use" criteria for EHR, mandated by the Health Information Technology for Economic and Clinical Health (HITECH) Act of 2009, requires that health professionals meet certain standards for quality, efficiency, and safety, all of which overlap with the goals of standardization, peer review, and quality improvement within CE consultation.

The U.S. and Japan have preapproval access systems for patients who have exhausted approved treat... more The U.S. and Japan have preapproval access systems for patients who have exhausted approved treatment options. We compare the systems of both countries and discuss some ethical concerns surrounding non-trial access to investigational drugs. Notable points of comparison of the two countries' systems include cost of non-trial access to investigational drugs and purpose of the non-trial access system. All stakeholders and the public must understand the ethical issues associated with non-trial preapproval access systems. The current non-trial access programs in both countries depends on pharmaceutical companies' voluntary decisions about whether patients get access to investigational products. Moreover, the potential for inequity of access raises ethical concerns. Non-trial preapproval access is an exceptional way to practice medicine with various ethical and safety concerns, so we suggest that the scope and eligibility for using these pathways should thus be limited.

The ethical practice of medicine requires identifying the interests and rights of patients and cr... more The ethical practice of medicine requires identifying the interests and rights of patients and creating a corresponding set of obligations and duties to fulfill those interests and rights. Transgender and gender nonconforming (TGNC) patients have special needs surrounding their gender identity that present unique ethical challenges for medical professionals, family members, and the patient themself. While there is a strong moral imperative to provide gender-affirming care to TGNC patients to mitigate the risk of depression, suicide, and other poor mental health outcomes, these care options must be considered alongside a developing person’s capacity for understanding and consent, especially in a pediatric setting. As bioethical processes and principles are best understood as part of an analysis of the life stories of patients, we explore ethical issues surrounding gender-affirming care for TGNC patients throughout the life cycle in a series of case descriptions.

Journal of law and policy, 2020

This symposium article examines the use of investigational (un-approved) medical products in the ... more This symposium article examines the use of investigational (un-approved) medical products in the United States, with particular focus on who pays for this use. We examine the various forms of preapproval access (“PAA”) to experimental medical products available in the United States—clinical trials and non-trial preapproval access via the Expanded Access (“EA”) and Right to Try (“RTT”) pathways. For each, this paper analyzes which entity—individual, insurer, sponsor, or other—bears the cost and what limitations or caps, if any, exist on these costs. This paper considers various proposed novel payment mechanisms that may permit more equitable use of investigational medical products. Part I outlines payment-related disparities in access in-grained in the current United States healthcare system. Part II fo-cuses on access in the context of clinical trials, which most payers have begun to cover, but where remaining uncovered expenses can disincentivize participation in clinical trials, e...

BMC Research Notes

Objective To evaluate the availability of information regarding patient access to investigational... more Objective To evaluate the availability of information regarding patient access to investigational treatments through clinical trials and non-trial pre-approval access pathways from a sample of patient advocacy organization (PAO) websites in the United States. Results We systematically analyzed the content of 118 randomly selected PAO websites to assess whether they contained information on clinical trials and non-trial pathways—e.g., the U.S. Food and Drug Administration (FDA) expanded access (EA) program and right to try—over the course of two months from February to March 2019. A majority (81%, n = 96) of PAOs provided a link to ClinicalTrials.gov, and 73% (n = 86) had their own clinical trial finder or list of relevant trials. 23% (n = 27) mentioned EA, with 8% (n = 9) providing specific resources for FDA’s EA program. 8% (n = 10) provided a statement on the passage of the federal right to try law. A majority of PAO websites contained information on clinical trials, but a minorit...

The American Journal of Bioethics

Preapproval nontrial access, historically called “compassionate use,” and certain off-label usage... more Preapproval nontrial access, historically called “compassionate use,” and certain off-label usage of approved medical products are two forms of innovative practice, defined by Earl as a situation “...

Therapeutic Innovation & Regulatory Science

JAMA

Medical crowdfunding involves using social media platforms to appeal for help in paying for medic... more Medical crowdfunding involves using social media platforms to appeal for help in paying for medical care. The largest medical crowdfunding platform, GoFundMe, reported that all campaigns raised 3billionby2016,anincreasefrom3 billion by 2016, an increase from 3billionby2016,anincreasefrom1 billion in 2015. 1 Although medical crowdfunding campaigns can fill insurance gaps, they can also raise money for scientifically unsupported, ineffective, or potentially dangerous treatments. We quantified crowdfunding activity for 5 such treatments.

Pediatrics, Jan 6, 2018

Transgender and gender-nonconforming (TGNC) youth who suffer from gender dysphoria are at a subst... more Transgender and gender-nonconforming (TGNC) youth who suffer from gender dysphoria are at a substantially elevated risk of numerous adverse physical and psychosocial outcomes compared with their cisgender peers. Innovative treatment options used to support and affirm an individual's preferred gender identity can help resolve gender dysphoria and avoid many negative sequelae of nontreatment. Yet, despite advances in these relatively novel treatment options, which appear to be highly effective in addressing gender dysphoria and mitigating associated adverse outcomes, ethical challenges abound in ensuring that young patients receive appropriate, safe, affordable treatment and that access to this treatment is fair and equitable. Ethical considerations in gender-affirming care for TGNC youth span concerns about meeting the obligations to maximize treatment benefit to patients (beneficence), minimizing harm (nonmaleficence), supporting autonomy for pediatric patients during a time of ...

The Journal of clinical ethics, 2018

Evolving practice requires peer review of clinical ethics (CE) consultation for quality assessmen... more Evolving practice requires peer review of clinical ethics (CE) consultation for quality assessment and improvement. Many institutions have identified the chart note as the basis for this process, but to our knowledge, electronic health record (EHR) systems are not necessarily designed to easily include CE consultation notes. This article provides a framework for the inclusion of CE consultation notes into the formal EHR, describing a developed system in the Epic EHR that allows for the elaborated electronic notation of the CE chart note. The implementation of the "meaningful use" criteria for EHR, mandated by the Health Information Technology for Economic and Clinical Health (HITECH) Act of 2009, requires that health professionals meet certain standards for quality, efficiency, and safety, all of which overlap with the goals of standardization, peer review, and quality improvement within CE consultation.

Cancer research, Jun 6, 2017

Biomarkers predicting rapalog responses in sarcomas where PI3K and mTOR are often hyperactivated ... more Biomarkers predicting rapalog responses in sarcomas where PI3K and mTOR are often hyperactivated could improve the suitable recruitment of responsive patients to clinical trials. PI3K/mTOR pathway activation drives energy production by regulating anaerobic glycolysis in cancer cells, suggesting a route toward a monitoring strategy. In this study, we took a multi-modality approach to evaluate the phenotypic effects and metabolic changes which occur with inhibition of the PI3K/mTOR pathway. Its central role in regulating glycolysis in human sarcomas was evaluated by short- and long-term rapamycin treatment in sarcoma cell lines. We observed an overall decrease in lactate production in vitro followed by cell growth inhibition. In vivo we observed a similar quantitative reduction in lactate production as monitored by hyperpolarized MRI, also followed by tumor size changes. This non-invasive imaging method could distinguish reduced cell proliferation from induction of cell death. Our res...

The Hastings Center report, 2017

The parents of Charlie Gard, who was born August 4, 2016, with an exceedingly rare and incurable ... more The parents of Charlie Gard, who was born August 4, 2016, with an exceedingly rare and incurable disease called mitochondrial DNA depletion syndrome, fought a prolonged and heated legal battle to allow him access to experimental treatment that they hoped would prolong his life and to prevent his doctors from withdrawing life-sustaining care. Charlie's clinicians at the Great Ormond Street Hospital in London believed that the brain damage Charlie had suffered as a result of frequent epileptic seizures, along with many other severe disabilities, would render any innovative therapy futile, and they disagreed with his parents' wishes to use an experimental therapy. They felt it in Charlie's best interest that he be allowed to die. A battle ensued among Charlie's parents, his doctors, and a guardian who had been appointed to represent him that drew the attention of politicians and prominent persons from all over the world. The case was much in the news over the past year,...