Podcasts about ivacaftor

Martín SabadiniHoy vamos a analizaren detalle un fallo sumamente relevante de la Corte Suprema de Justicia de la Nación queabre un debate técnico, médico y legal de enorme impacto en el derecho a la salud: el caso"G. B., R. contra OSDE sobre amparo de salud" [1].En esta causa se discute algo que vemos a diario en la práctica profesional: ¿puede unaobra social o empresa de medicina prepaga sustituir un medicamento importado de altísimocosto por una alternativa de fabricación nacional con los mismos componentes activos [10]?Hoy te contamos la historia de este amparo, la sentencia de la Corte y, fundamentalmente,tres claves que definieron este expediente: lo que verdaderamente dijo el Cuerpo MédicoForense, el llamativo silencio del médico tratante y las exigencias de bioequivalencia de laANMAT [3, 11, 12].Bloque 2: El Origen de la ControversiaPara ponernos en contexto, la amparista, inicialmente menor de edad representada por sumadre, padece fibrosis quística e inició esta acción en el año 2017 para que OSDE lecubriera su medicación [1]. En el año 2020, solicitó la provisión de una terapia triplemoduladora importada, conocida comercialmente como "Trikafta", compuesta por las drogasElexacaftor, Tezacaftor e Ivacaftor, aprobada por la FDA estadounidense [1].A título cautelar, la justicia ordenó la provisión de este fármaco, importándose bajo el régimende uso compasivo de la ANMAT [1]. Sin embargo, la situación cambió cuando la ANMATautorizó en el país la comercialización de "Trixacar", un medicamento de fabricación nacionalque contiene exactamente esos mismos principios activos [2, 10].Ante esto, OSDE pidió sustituir la marca del medicamento provisto, ofreciendo la opciónnacional [2]. Tanto el juez de primera instancia como la Sala I de la Cámara Civil y ComercialFederal rechazaron este pedido, ordenando mantener la cobertura total de la marcaimportada Trikafta [2, 3]. Para decidir esto, la Cámara consideró determinante un argumento:la supuesta falta de estudios de bioequivalencia de la opción nacional frente a la importada[3]. Pero la Corte Suprema consideró que esta decisión fue arbitraria [11, 15]. Veamos porqué.Bloque 3: ¿Qué Dijo Realmente el Cuerpo Médico Forense?El primer punto crucial que destaca la Corte es que la Cámara realizó una valoraciónsesgada y parcial del dictamen del Cuerpo Médico Forense del 17 de agosto de 2023 [3, 11].Es cierto que el Cuerpo Médico Forense remarcó que la amparista había mejorado de formanotable con la medicación importada [11]. Sin embargo, su conclusión técnica y central nofue que ese era el único medicamento viable [11]. Al contrario, el informe médico forensedeterminó expresamente que, desde un punto de vista estrictamente pericial, no existíaninguna contraindicación médica para sustituir el medicamento importado Trikafta por elnacional Trixacar [3, 11]. El dictamen explicaba que ambos poseían la misma composición dedrogas moduladoras [3, 11].Además, la pericia mencionaba que profesionales del Hospital María Ferrer describíanresultados similares entre ambos medicamentos [3]. La Cámara se aferró a la mención deque Trixacar no poseía estudios de bioequivalencia para rechazar la sustitución, omitiendo laconclusión principal del perito médico oficial de que la sustitución era viable y segura [3, 11].Bloque 4: El Silencio del Médico TratanteY aquí llegamos al segundo gran pilar del fallo de la Corte: la falta de respuesta y el silencioen el expediente [12, 13].Durante el trámite judicial, la Cámara dictó una medida para mejor proveer [12]. Esto es, unamedida para aclarar dudas antes de dictar sentencia [12]. El tribunal le ordenó expresamentea la actora que su médico tratante especificara e informara de manera justificada si la terapiadebía ser inevitablemente de la marca importada Trikafta o si podía intercambiarse con lanacional Trixacar [12].Y lo más importante: dado que constaba en la causa que la paciente ya había recibidotratamiento con el medicamento nacional Trixacar durante el mes de febrero de 2022, se lesolicitó al médico que aclarara si la paciente había sufrido algún tipo de desmejoría oestancamiento de salud durante ese período, debiendo acompañar documentaciónrespaldatoria [12, 13].¿Qué pasó con este requerimiento judicial? La actora y su médico tratante nuncarespondieron [12]. Hubo un silencio absoluto frente a una consulta que resultaba crucial pararesolver la controversia [12, 13]. La Corte señaló que este silencio fue ignorado por laCámara, la cual dictó sentencia sin contar con esa aclaración fundamental, a pesar de estaracreditado que la paciente ya había tomado el medicamento nacional [12, 13].La Bioequivalencia y el CierreFinalmente, la Corte analizó el argumento de la bioequivalencia [14]. La Cámara habíadescalificado el medicamento nacional por la supuesta ausencia de estos estudios, pero laCorte deparó que esa exigencia no tenía respaldo normativo [14, 15]. Los principios activosinvolucrados no se encuentran listados por la ANMAT dentro de los ingredientesfarmacéuticos activos que requieren obligatoriamente de estudios de bioequivalencia para sucomercialización en Argentina [14]. La ANMAT, como órgano regulador y científicocompetente, aprobó el producto nacional para su uso seguro en nuestro país sin estaexigencia [14, 15].Por todo esto, la Corte Suprema de Justicia de la Nación hizo lugar a la queja de lademandada, declaró procedente el recurso extraordinario y dejó sin efecto la sentencia de laCámara por considerarla arbitraria, ordenando que se dicte un nuevo fallo con arreglo a lasconstancias de la causa [15, 16].Este fallo nos deja una enseñanza clave para los profesionales del derecho a la salud: losamparos no pueden basarse en respuestas dogmáticas o preferencias sin sustento fáctico. Elrol y la respuesta activa del médico tratante son indispensables para justificar técnicamentepor qué una marca comercial es insustituible. Si no hay fundamentos médicos concretos nirespuestas a los requerimientos del tribunal, la sustitución por la alternativa nacionalaprobada por la autoridad regulatoria se presenta como una opción jurídicamente válida.CV Abogado consultor en derecho y protección de la salud inclusiva

CHEST April 2024, Volume 165, Issue 4 Sonia Graziano, PsyD, joins CHEST Podcast Moderator Gretchen Winter, MD, to discuss how elexacaftor/tezacaftor/ivacaftor affects mental health, cognitive processing, neuropsychological side effects, GI symptoms, and health-related quality of life in people with cystic fibrosis. DOI: https://doi.org/10.1016/j.chest.2023.10.043   Disclaimer: The purpose of this activity is to expand the reach of CHEST content through awareness, critique, and discussion. All articles have undergone peer review for methodologic rigor and audience relevance. Any views asserted are those of the speakers and are not endorsed by CHEST. Listeners should be aware that speakers' opinions may vary and are advised to read the full corresponding journal article(s) for complete context. This content should not be used as a basis for medical advice or treatment, nor should it substitute the judgment used by clinicians in the practice of evidence-based medicine.

Check out this milestone episode as we return to Cystic Fibrosis from a more pediatric perspective! Don't sweat it as Dr. Ina St. Onge returns to walk us through newborn screening,  early clinic visits, and the nuances of CFTR modulator therapy. 

Durante a centésima quinta reunião da Comissão Nacional de Incorporação de Tecnologias no Sistema Único de Saúde, Conitec, foi feita a apreciação inicial do medicamento tezacaftor/ivacaftor, com nome comercial Symdeko. Infelizmente, a Conitec recomendou para a consulta pública com parecer preliminar desfavorável à incorporação no SUS. Com essa decisão e a abertura da consulta pública no site da Comissão, chegou a hora de toda a comunidade da fibrose cística se unir para tentar reverter essa situação. Neste podcast, compartilhamos informações importantes sobre como participar da consulta pública nº 05 referente ao Symdeko até o dia 04/04/2022. Para mais informações, acesse https://contribuacomfc.unidospelavida.org.br/. Roteiro: Kamila Vintureli e Gabriel Johnson Locução e edição: Kamila Vintureli Trilha: Felipe Caldo

The recent development of triple CFTR modulation means that up to 90% of people with CF will be eligible for ‘highly effective’ CFTR modulator therapy. In this podcast, Rachael Moses, Multimedia Editor of Thorax, and Dr Peter Barry, Consultant Respiratory Physician, Manchester Adult Cystic Fibrosis Centre, discuss the long-term adherence to and effects of ivacaftor on lung function, weight and healthcare utilisation in people with cystic fibrosis and the Gly551Asp mutation. Read the related paper: https://thorax.bmj.com/content/early/2021/03/24/thoraxjnl-2020-215556

This episode of Drug Cards Daily is on the drug Trikafta. As of 2020 it is Brand only. Trikafta consists of three drugs which are elexacaftor, ivacaftor, tezacaftor. It is twice a day dosing with 2 tablets in the morning and 1 tablet (12 hours later) in the evening. The morning tablet is orange in color and consists of elexacaftor 100 mg + tezacaftor 50mg + ivacaftor 75 mg while the evening dose (which is a light blue colored tablet) consists of ivacaftor 150 mg. Currently the drug is approved for use in ages 12 years and older. The main concerns are patients must have the F508del mutation in the CFTR gene have healthy liver function. Patients without the F508del mutation will not benefit from this drug. The other concern is that CYP3A inducers and inhibitors affect this drug greatly so that should always be considered when starting a patient on this treatment. Go to DrugCardsDaily.com for episode show notes which consist of the drug summary, quiz, and link to the drug card for FREE! Please SUBSCRIBE, FOLLOW, and RATE on Spotify, Apple Podcasts, or wherever your favorite place to listen to podcasts are. The main goal is to go over the Top 200 Drugs with the occasional drug of interest. Also, if you'd like to say hello, suggest a drug, or leave some feedback I'd really appreciate hearing from you! Leave a voice message at anchor.fm/drugcardsdaily or find me on twitter @drugcardsdaily --- Send in a voice message: https://anchor.fm/drugcardsdaily/message

As consultas públicas referentes aos medicamentos Kalydeco (ivacaftor) e Orkambi (lumacaftor/ivacaftor) já estão disponíveis e o Unidos pela Vida - Instituto Brasileiro de Atenção à Fibrose Cística, traz neste episódio informações importantes sobre como participar. Roteiro: Kamila Vintureli e Verônica Stasiak Bednarczuk de Oliveira Locução: Kamila Vintureli Data de gravação: 12 de agosto de 2020

Drug discovery is a complicated and expensive process that can take years for a drug to finally come to market. In this week's podcast we walk you through the process of drug discovery looking through the lens of cystic fibrosis. We explain the many steps including screens for different drug compounds, the different phases of testing and the vast regulations that drugs in the US are under to protect people! Finally, we link this back to COVID-19 and some of the drugs that are currently being tested to see if they help treat COVID-19 symptoms. // SOURCES:SourcesCystic Fibrosis: https://www.cff.org/aCystic Fibrosis: https://www.cff.org/Research/About-Our-Research/Research-Milestones/Cystic Fibrosis: https://ghr.nlm.nih.gov/gene/CFTR#:~:text=Normal%20Function,%2C%20tears%2C%20and%20digestive%20enzymes.Ivacaftor discovery: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3770629/pdf/dddt-7-929.pdfOriginal Ivacaftor screen: https://pubmed.ncbi.nlm.nih.gov/16443646/Ivacaftor company: https://investors.vrtx.com/news-releases/news-release-details/fda-approves-kalydecotm-ivacaftor-first-medicine-treatIvacaftor clinical trials: https://www.clinicaltrialsarena.com/projects/ivacaftor-treatment-for-cystic-fibrosis/Follow-up characterization: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2773991/Drug discovery process: https://www.ncbi.nlm.nih.gov/books/NBK22930/Drug discovery process http://phrma-docs.phrma.org/sites/default/files/pdf/rd_brochure_022307.pdfDrug discovery process: https://www.fda.gov/drugs/development-approval-process-drugsDrug failure/approval: https://blogs.sciencemag.org/pipeline/archives/2019/05/09/the-latest-on-drug-failure-and-approval-ratesCOVID-19 Treatments: https://www.covid19treatmentguidelines.nih.gov/antiviral-therapy/chloroquine-or-hydroxychloroquine/COVID-19 Treatments: https://www.fda.gov/news-events/press-announcements/coronavirus-covid-19-update-fda-issues-emergency-use-authorization-potential-covid-19-treatmentRight to Try: https://www.fda.gov/patients/learn-about-expanded-access-and-other-treatment-options/right-tryHydroxychloroquine shortage: https://www.arthritis.org/drug-guide/medication-topics/plaquenil-shortageHydroxychloroquine shortage: https://www.lupus.org/news/fda-recognizes-hydroxychloroquine-and-chloroquine-shortages

In this episode, guest host Dr. David Poetker speaks with Dr. Brad Woodworth and Dr. Do-Yeon. They will discuss the article: In‐vitro evaluation of a ciprofloxacin‐ and ivacaftor‐coated sinus stent against Pseudomonas aeruginosa biofilms. Read the article in International Forum of Allergy and Rhinology Listen and subscribe for free on Apple Podcasts , Google Play […]

Nutritional Issues in Cystic Fibrosis

Benefits of CFTR Modification Beyond FEV1 Improvement

The Effects of CFTR-modifying Therapies

Originally presented at the 2013 North American Cystic Fibrosis Conference, this program covers differentiation of CF classes and current/potential therapies and using the CFTR2 database.

Presented at NACFC Anaheim 2011 this symposium presentation offers information on the current treatment options for patients with cystic fibrosis. Mike Boyle, MD, from Johns Hopkins University School of Medicine offers a 25-minute look at the basics of CF, then describes genotype and CF phenotype relationships. The presentation includes a review the influences of severity of lung disease and outcomes in CF, followed up by issues related to adherence.

Presented at NACFC Anaheim 2011 this symposium presentation offers information on the current treatment options for patients with cystic fibrosis. Mike Boyle, MD, from Johns Hopkins University School of Medicine offers a 25-minute look at the basics of CF, then describes genotype and CF phenotype relationships. The presentation includes a review the influences of severity of lung disease and outcomes in CF, followed up by issues related to adherence.

Presented at NACFC Anaheim 2011 this symposium presentation offers information on the current treatment options for patients with cystic fibrosis. Mike Boyle, MD, from Johns Hopkins University School of Medicine offers a 25-minute look at the basics of CF, then describes genotype and CF phenotype relationships. The presentation includes a review the influences of severity of lung disease and outcomes in CF, followed up by issues related to adherence.

Presented at NACFC Anaheim 2011 this symposium presentation offers information on the current treatment options for patients with cystic fibrosis. Mike Boyle, MD, from Johns Hopkins University School of Medicine offers a 25-minute look at the basics of CF, then describes genotype and CF phenotype relationships. The presentation includes a review the influences of severity of lung disease and outcomes in CF, followed up by issues related to adherence.

Presented at NACFC Anaheim 2011 this symposium presentation offers information on the current treatment options for patients with cystic fibrosis. Mike Boyle, MD, from Johns Hopkins University School of Medicine offers a 25-minute look at the basics of CF, then describes genotype and CF phenotype relationships. The presentation includes a review the influences of severity of lung disease and outcomes in CF, followed up by issues related to adherence.

Presented at NACFC Anaheim 2011 this symposium presentation offers information on the current treatment options for patients with cystic fibrosis. Mike Boyle, MD, from Johns Hopkins University School of Medicine offers a 25-minute look at the basics of CF, then describes genotype and CF phenotype relationships. The presentation includes a review the influences of severity of lung disease and outcomes in CF, followed up by issues related to adherence.