A crisis in <scp>US</scp> drug pricing: Consequences for patients with neuromuscular diseases, physicians and society, part 1

Burns, Ted M.; Crowell, Jason L.; Smith, A Gordon

doi:10.1002/mus.27015

Cited by 7 publications

(5 citation statements)

References 20 publications

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“…Prior to this approval, 3,4-DAP was available only through compounding pharmacies or a compassionate use program requiring individual Investigational New Drug programs. Since the approval of amifampridine phosphate, concern regarding access and drug pricing has raised many questions about the “financial toxicity” of drugs and their role in the management of rare diseases such as LEMS 39,40 . This is increasingly a concern as historically established, previously compounded entities are approved for disease-specific indications as trademarked products.…”

Section: Lambert-eaton Myasthenic Syndromementioning

confidence: 99%

Lambert-Eaton Myasthenic Syndrome and Botulism

Raja¹

2022

CONTINUUM: Lifelong Learning in Neurology

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PURPOSE OF REVIEW: This article reviews the pathophysiology, epidemiology, clinical features, diagnosis, and treatment of Lambert-Eaton myasthenic syndrome (LEMS) and botulism, presynaptic disorders of neuromuscular transmission in which rapid diagnosis improves long-term outcomes.RECENT FINDINGS: Therapy for LEMS has seen significant advances in recent years due to the approval of amifampridine-based compounds. LEMS is likely still underdiagnosed, particularly when no underlying malignancy is identified. Clinicians must have a strong suspicion for LEMS in any patient presenting with proximal weakness and autonomic dysfunction. Botulism is another rare disorder of presynaptic neuromuscular transmission that is most commonly associated with improper storage or preservation of food products. Over the past 2 decades, wound botulism has been increasingly reported among users of black tar heroin. A high degree of clinical suspicion and electrodiagnostic studies can be beneficial in distinguishing botulism from other acute neurologic disorders, and early involvement of state and federal health authorities may assist in confirming the diagnosis and obtaining treatment. When botulism is suspected, electrodiagnostic studies can provide clinical evidence of disordered neuromuscular transmission in advance of serologic confirmation, and providers should not wait for confirmation of the diagnosis to initiate treatment. SUMMARY: A targeted clinical history and a thorough neurologic examination with support from serologic and electrodiagnostic studies are key to early diagnosis of LEMS and botulism. Early diagnosis of both conditions creates opportunities for therapy and improves outcomes.

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Section: Lambert-eaton Myasthenic Syndromementioning

confidence: 99%

Lambert-Eaton Myasthenic Syndrome and Botulism

Raja¹

2022

CONTINUUM: Lifelong Learning in Neurology

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“…access therapies prescribed off-label (10,11). This results in an equality issue where only the wealthy can afford off-label treatment, while patients with lower incomes cannot.…”

Section: Regulations For Rare Pediatric Diseasesmentioning

confidence: 99%

Off-label medication use in rare pediatric diseases in the United States

Fung

Yue

Wigle

et al. 2021

IRDR

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rare diseases, off-label drug use, pediatrics, infants, children.Many pediatric patients with rare diseases use drugs off-label due to limited data in pediatric patients. Off-label treatment remains an important public health issue for neonates, infants, children, and adolescents, especially for pediatric patients with rare diseases. For patients with rare diseases, the majority of medications have no or limited information in labelling for pediatric use. Children present unique considerations in clinical trials due to ethical and clinical concerns, which have limited and even discouraged testing of drugs in the pediatric population. Numerous legislative measures have been enacted to address barriers in pediatric drug testing. This research reviewed off-label medication use in rare pediatric diseases, evaluated recent medication uses in pediatric clinical practice, discussed key regulations for rare pediatric diseases, and summarized recent drug approvals for rare pediatric diseases. This study demonstrates the ongoing medical need for newly approved medications to treat pediatric rare diseases and revealed the positive impact of regulations from the Orphan Drug Act of 1983 to the Research to Accelerate Cures and Equity (RACE) for Children Act on drug development and off-label medication practice in rare pediatric disease management. This article provides informative historical background and current considerations of off-label use of medications in neonates, infants, children, and adolescents with rare diseases.

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“…De acuerdo con el Hasting Center (39), la presencia de patentes ha evitado la producción de medicamentos más baratos en algunos países y a generado altos precios. Si bien las patentes y los altos precios no son los únicos obstáculos para el acceso a los medicamentos, a menudo son las barreras más significativas (43)(44)(45)(46). Los antirretrovirales patentados son un caso ilustrativo de las tensiones entre el sistema de propiedad intelectual, el mercado, los derechos humanos y la equidad en salud.…”

Section: Conflicto De Derechos Conflicto De Intereses: Algunos Antece...unclassified

Propiedad intelectual, patentes y salud

Uribe

2021

Rev. latinoam. bioet

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Artículo de carácter reflexivo y argumentativo que busca confrontar referentes conceptuales desde la Bioética y el derecho para dar solución a estos interrogantes: ¿son los derechos de propiedad intelectual la antítesis de otros derechos? ¿Existe un conflicto de principios bioéticos en dicha tensión? ¿Cómo abordar desde la Bioética este choque de miradas? ¿Es necesario un cambio de paradigma para transformar el individualismo imperante de la contemporaneidad y evitar los daños de una guerra de rapiñas que se enmascara detrás de un eufemismo de la competencia de mercados y la propiedad intelectual? Entre argumentos, cuestionamientos y disensos se busca un abordaje a estos dilemas con un enfoque bioético. Se deja sobre la mesa la cooperación con una base de empatía y compasión para desviarse del camino del individualismo pernicioso y la voracidad subsecuente del dinero y el poder. Por ello, la Bioética como puente hacia al futuro es la disciplina llamada manifestar dicha cooperación, no solo en los ámbitos disciplinarios, sino entre los diferentes actores sociales, entre los protagonistas de esta obra, para que su final no sea trágico, sino esperanzador y saludable.

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A crisis in US drug pricing: Consequences for patients with neuromuscular diseases, physicians and society, part 1

Cited by 7 publications

References 20 publications

Lambert-Eaton Myasthenic Syndrome and Botulism

Lambert-Eaton Myasthenic Syndrome and Botulism

Off-label medication use in rare pediatric diseases in the United States

Propiedad intelectual, patentes y salud

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