[Instruments of official communication by regulatory authorities on risks of drug use]. / Instrumente behördlicher Kommunikation zu Anwendungsrisiken von Arzneimitteln.

Active communication of authorities, such as the Federal Institute for Drugs and Medical Devices (BfArM) and the Paul Ehrlich Institute (PEI), including maintenance of contacts with health care professionals, as well as press and public relations work, are essential prerequisites for ensuring that information on the risks of using medicinal products reaches both affected patients and healthcare professionals quickly and in a targeted manner. The various instruments of targeted communication describe possible risks and also contain recommendations that help to reduce the risk of using a medicinal product. The supplementary public relations work aims to make the tasks and objectives of the authority known to the public and to experts with the goal of creating and expanding trust in the actions of the authorities. To this end, appropriate communication platforms must be established and accepted so that they are used by both experts and the general public and the authority is perceived and appreciated as a reliable source of risk information. The currently available instruments of targeted risk communication, such as Dear Health Care Professional Communication (DHPC), risk management plans, and educational materials are described in this paper as well as broader communication on official websites or towards the media. Finally, PEI's risk communication is highlighted with particular reference to COVID-19 vaccines.

Clinical trials were missing from regulatory documents of extended-release methylphenidate for ADHD in adults: a case study of public documents.

OBJECTIVES: To assess whether drug regulatory agencies decided on applications for extended-release methylphenidate for use in adult ADHD based on select samples of trials. STUDY DESIGN AND SETTING: Case series of publicly available regulatory documents. We matched an index of extended-release methylphenidate trials for adult ADHD with trials appearing in regulatory documents of extended-release methylphenidate applications. Trials and regulatory documents were identified as part of this systematic review (https://doi.org/10.1002/14651858.CD012857). We sought to identify missing trials in the regulatory documents and to clarify regulatory submission requirements. RESULTS: We indexed 18 trials and matched those with 13 drug applications (11 approved, 2 rejected) published by 7 agencies. There were trials missing in 7 (54%) of 13 applications, median 4 trials (range 1-6). The median proportion of missing trial participants was 45% (range 23% - 72%). Regulators seemingly require that all trials must be included in new drug applications, but wording is ambiguous. CONCLUSION: In this sample of extended-release methylphenidate drug applications for adult ADHD, 7 of 13 regulatory decisions were missing entire trials according to public documents, even though regulatory requirements seem to stipulate that all available trials should be included in drug applications.

Medicamentos biosimilares en Colombia: una revisión desde el consumo informado / Biosimilar Medicines in Colombia: An Approach from the Informed Consumption / Medicamentos biossimilares na Colômbia: uma revisão desde o consumo informado

Resumen Introducción: este artículo presenta un análisis sobre medicamentos biosimilares en Colombia con miras a establecer si existe un consumo informado respecto de estos fármacos, teniendo en cuenta la calidad y cantidad de información que circula en relación con su disponibilidad y acceso económico (precios). Desarrollo: el tipo de estudio es cualitativo con un diseño documental a partir de la revisión en bases de datos como Pubmed, Scopus, Scielo, Vlex y Redalyc, bases de información de registros sanitarios de agencias reguladoras de medicamentos (FDA, EMA e Invima) y el Sistema de Información de Vademecum Med Informática. Uno de los biosimilares autorizados en FDA y cuatro de la EMA se encuentran registrados en el Invima; sin embargo, son más los biosimilares no autorizados ni en FDA ni en EMA que se encuentran registrados en el Invima, los cuales aún no aparecen con esta clasificación de biosimilares ni en la agencia reguladora colombiana ni en ningún otro medio oficial o comercial. De los medicamentos biológicos de referencia de estos biosimilares, 12 tienen control de precios y 7 han sido autorizados al menos una vez vía judicial a los usuarios del sistema. Conclusiones: si bien, la reglamentación ha sido de gran avance en relación con el acceso a biosimilares y, en consecuencia, a sus biológicos pioneros, el sistema aún tiene barreras jurídicas, de información, disponibilidad y acceso que dificultan la protección y efectividad del derecho a la salud de la población en términos de un consumo informado de estos.

Abstract Introduction: This article presents an analysis of biosimilar drugs in Colombia, aiming at critically analyzing whether the consumption of biosimilar drugs in Colombia is fully informed or if it rather is characterized for its lack of information. Informed consumption in the sense of taking into account the quality and quantity of the information circulating in relation to the availability and affordability (prices) of biosimilar drugs. Development: This is a qualitative documentary analysis, based on the review of databases such as Pubmed, Scopus, Scielo, Vlex and Redalyc, and sanitary databases of drugs regulatory agencies (FDA, EMA and the Colombian Invima) and the Vademecum Med Informatica. One of the biosimilars authorized by FDA and four of those by EMA were also registered before by the Invima. However, the number of Invima authorized biosimilars is higher than that authorized by FDA and EMA. It is also important to highlight the fact that any biosimilar is not registered as such neither before the Invima nor before any other official or commercial source. Out of the biological reference products with biosimilars, twelve have regulated price and seven have been authorized at least once via court ruling. Conclusions: Although the regulations are progressive in relation to the access to biosimilars and, consequently, to their biological pioneers, the system still has legal, availability and information barriers that undermine the protection and effectiveness of the right to health.

Resumo Introdução: este artigo apresenta uma análise sobre medicamentos biossimilares na Colômbia com vista a estabelecer se existe um consumo informado respeito destes fármacos, tendo em conta a qualidade e quantidade de informação que circula em relação com a sua disponibilidade e acesso econômico (preços). Desenvolvimento: o tipo de estudo é qualitativo com um desenho documental a partir da revisão em bases de dados como Pubmed, Scopus, Scielo, Vlex e Redalyc, bases de informação de registros sanitários de agências reguladoras de medicamentos (FDA, EMA e Invima) e o Sistema de Informação de Vademecum Med Informática. Um dos biossimilares autorizados em FDA e 4 da EMA, se encontram registrados no Invima, no entanto são mais os biossimilares não autorizados nem na FDA nem na EMA que se encontram registrados no Invima, os quais ainda não aparecem com esta classificação de biossimilares nem na agência reguladora colombiana, nem em nenhum outro meio oficial ou comercial. Dos medicamentos biológicos de referência destes biossimilares, 12 têm controle de preços e 7 têm sido autorizados pelo menos uma vez via judicial aos usuários do sistema. Conclusões: se bem a regulamentação tem sido de grande avanço em relação com o acesso a biossimilares e, consequentemente, a seus biológicos pioneiros, o sistema ainda tem barreiras jurídicas, de informação, disponibilidade e acesso que dificultam a proteção e efetividade do direito à saúde da população em termos de um consumo informado dos mesmos.

[Adaptive drug licensing: A chance for patients?]. / Frühere Zulassung von Arzneimitteln - Eine Chance für Patienten?

In recent years, various options have been discussed to accelerate the approval of new drugs, especially for conditions with high unmet needs. There is a trade-off between the earlier availability of new treatment options and a potentially higher safety risk of drugs which have not been investigated in long-lasting clinical trials and a broader patient population. It must also be taken into account that clinical trial data and results must be sufficient to allow for the reimbursement of the new treatment option. The difficult process of weighting the benefits against the potential risks of an accelerated drug approval should be discussed between patients, regulatory authorities, HTA institutions and pharmaceutical companies. Patients might benefit from a fair, balanced accelerated approval.

Estrategia para la preparación de unidades de salud en buenas prácticas clínicas / Strategy for the preparation of health units for good clinical practices

Introducción: El entorno regulatorio mundial es cada vez más exigente para establecer, implementar y mantener el cumplimiento de las buenas prácticas clínicas (BPC). En Cuba, una respuesta necesaria derivada del desarrollo creciente de la industria farmacéutica y biotecnológica nacional fue la creación del Centro Nacional Coordinador de Ensayos Clínicos (CENCEC). Una de las misiones del CENCEC es preparar a las unidades-sitios clínicos seleccionados que realizan investigaciones clínicas, para su posterior certificación en BPC por la autoridad reguladora nacional, con la finalidad de avalar la calidad que corresponde al proceso de investigación clínica que redunda en una esmerada atención y protección al paciente objeto de estudio. Objetivo: Describir la estrategia del CENCEC para la preparación en BPC de los sitios clínicos seleccionados del Sistema Nacional de Salud (SNS) que participan en ensayos clínicos. Métodos: Se revisan más de 250 documentos normativos emitidos por Europa, Estados Unidos, Japón y los países nórdicos relacionados con aspectos prácticos y éticos para la implementación de las BPC. Se elabora una estrategia para la preparación de los sitios clínicos en BPC concebida en 4 etapas: 1) concepción técnica del proceso y organización documental, 2) selección de los sitios clínicos, 3) diagnóstico y evaluación pre-intervención y 4) preparación para la certificación. Resultados: Se identificaron 80 sitios clínicos que realizan ensayos clínicos en Cuba, de los cuales se seleccionaron 11 para la aplicación de la estrategia. Se elaboró un manual de preparación de los sitios en BPC con los aspectos de mayor impacto en el cumplimiento de las BPC. Se realizaron 40 visitas a los sitios clínicos seleccionados, 12 diagnósticas, 24 de seguimiento, una de inclusión de nuevos sitios, y 3 de declaración de Listo para Certificación...(AU)

Background: The global regulatory environment is increasingly demanding to establish, implement, and maintain the compliance with Good Clinical Practices (GCP). In Cuba, The National Coordinating Center for Clinical Trials (CENCEC) was created as a necessary response derived from the increasing development of the national pharmaceutical and biotechnological industry. One of the missions of the CENCEC is to prepare selected clinical units/sites that conduct clinical research for a further certification in GCP by the national regulatory authority in order to guarantee the quality that corresponds to the process of clinical research, resulting in a careful attention and protection of the patient under study. Objective: To describe the strategy of the CENCEC for the preparation of good clinical practices in the selected clinical sites of the National Health System (SNS) that participate in clinical trials. Methods: More than 250 regulatory documents issued by Europe, the United States, Japan and the Nordic countries, related to ethical and practical aspects for the implementation of good clinical practices, were reviewed. A strategy for the preparation of clinical sites in GCP was conceived in 4 stages: 1) technical design of the process and document organization, 2) selection of the clinical sites, 3) diagnosis and pre-intervention evaluation, and 4) preparation for certification. Results: 80 clinical sites that conduct clinical trials in Cuba were identified, of which 11 were selected for the implementation of the strategy. A manual for the preparation of the sites in GCP with aspects of great impact in compliance with GCP was created. 40 visits were made to the selected clinical sites, 12 of them were diagnostic, 24 were follow-up visits, 1 was made for the inclusion of new sites, and 3 for the statement Ready for Certification...(AU)

Estrategia para la preparación de unidades de salud en buenas prácticas clínicas / Strategy for the preparation of health units for good clinical practices

Introducción: El entorno regulatorio mundial es cada vez más exigente para establecer, implementar y mantener el cumplimiento de las buenas prácticas clínicas (BPC). En Cuba, una respuesta necesaria derivada del desarrollo creciente de la industria farmacéutica y biotecnológica nacional fue la creación del Centro Nacional Coordinador de Ensayos Clínicos (CENCEC). Una de las misiones del CENCEC es preparar a las unidades-sitios clínicos seleccionados que realizan investigaciones clínicas, para su posterior certificación en BPC por la autoridad reguladora nacional, con la finalidad de avalar la calidad que corresponde al proceso de investigación clínica que redunda en una esmerada atención y protección al paciente objeto de estudio. Objetivo: Describir la estrategia del CENCEC para la preparación en BPC de los sitios clínicos seleccionados del Sistema Nacional de Salud (SNS) que participan en ensayos clínicos. Métodos: Se revisan más de 250 documentos normativos emitidos por Europa, Estados Unidos, Japón y los países nórdicos relacionados con aspectos prácticos y éticos para la implementación de las BPC...

Background: The global regulatory environment is increasingly demanding to establish, implement, and maintain the compliance with Good Clinical Practices (GCP). In Cuba, The National Coordinating Center for Clinical Trials (CENCEC) was created as a necessary response derived from the increasing development of the national pharmaceutical and biotechnological industry. One of the missions of the CENCEC is to prepare selected clinical units/sites that conduct clinical research for a further certification in GCP by the national regulatory authority in order to guarantee the quality that corresponds to the process of clinical research, resulting in a careful attention and protection of the patient under study. Objective: To describe the strategy of the CENCEC for the preparation of good clinical practices in the selected clinical sites of the National Health System (SNS) that participate in clinical trials. Methods: More than 250 regulatory documents issued by Europe, the United States, Japan and the Nordic countries, related to ethical and practical aspects for the implementation of good clinical practices, were reviewed...