Perla 2021 fernando ferrer mba oct 27 2017
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O documento descreve o programa PERLA 2021, que tem como objetivo promover a cooperação entre países da América Latina para melhorar o tratamento de doenças raras. O programa visa reunir legisladores, associações médicas e de pacientes para acelerar a criação de leis que garantam o acesso ao diagnóstico e tratamento de doenças raras. O programa também pretende incentivar a pesquisa de novas terapias para essas doenças.
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- 1. PERLA 2021 Programa de Enfermedades Raras de Latino América Programa de Enfermidades Raras de Latino América Rare Disease Program of Latin America Fernando Ferrer, MBA 12 Forum Alianza Latina - Auditorium Windsor Marapendi Rio de Janeiro, Brazil, October 26 - 27, 2017 ®
- 2. PrivilegedandConfidential Fernando Ferrer, MBA © Multinational Partnerships LLC 2 Disclaimer The views and opinions expressed in this session are those of the individual presenters and should not be attributed to their employers nor the organizers of this event. Fernando Ferrer, MBA
- 3. PrivilegedandConfidential Fernando Ferrer, MBA © Multinational Partnerships LLC 3 Objective of this Forum Ampliar la participación de organizaciones de pacientes en la mejora de los tratamientos en América Latina y propiciar un espacio de debate y aprendizaje continuo para asociaciones que compartan ideales y misiones similares en América Latina. Ampliar a participação de organizações de pacientes na melhoria dos tratamentos na América Latina e propiciar um espaço de debate e aprendizado continuo, para associações que compartilhem ideais e missões similares na América Latina. Increase the participation of patient organizations in improving treatment in Latin America; to provide a space for ongoing debate, networking and learning from associations that share ideals and similar missions in Latin America.
- 4. PrivilegedandConfidential Fernando Ferrer, MBA © Multinational Partnerships LLC 4 Patient Advocacy ① introduction
- 5. PrivilegedandConfidential Fernando Ferrer, MBA © Multinational Partnerships LLC 5 Patients in Clinical Trials
- 7. Assemblyman Dr Herb Conaway Trenton, NJ. USA Federal Deputy Augusto Carvalho Brasilia, DF. Brazil
- 9. PERLA 2021 Programa de Enfermedades Raras de Latino América Programa de Enfermidades Raras de Latino América Rare Disease Program of Latin America Prof. Fernando Ferrer, MBA AMAVI Multinational Partnerships LLC 12 Forum Alianza Latina Rio de Janeiro, Brazil, October 27, 2017 ® ② PERLA 2021
- 10. PrivilegedandConfidential Fernando Ferrer, MBA © Multinational Partnerships LLC 10 Vision - Visão Tener en América Latina en 2021 leyes promulgadas para la cobertura económica y el acceso al diagnóstico, medicamentos, dispositivos médicos y servicios de salud para las personas con enfermedades raras; y para fomentar la investigación y desarrollo de soluciones clínicas innovadoras para esas enfermedades. Ter na América Latina em 2021 leis promulgadas para a cobertura econômica e acesso ao diagnóstico, medicamentos, dispositivos médicos e serviços de saúde para pessoas com doenças raras; e para promover a pesquisa e desenvolvimento de soluções clínicas inovadoras para essas doenças. To have in Latin America by 2021 promulgated laws for economic coverage and access to diagnosis, medicines, medical devices and health services for people with rare diseases; and to promote the research and development of innovative clinical solutions for those diseases.
- 11. PrivilegedandConfidential Fernando Ferrer, MBA © Multinational Partnerships LLC 11 Misión Promover el encuentro de legisladores, juristas, asociaciones medicas, de pacientes, y de la industria y servicios de ciencias de la vida de América Latina para crear y acelerar los procesos legislativos y regulatorios que protejan y den tratamiento a las personas con enfermedades raras, y que promuevan la investigación y desarrollo de terapias innovadoras. Impulsar la realización de contactos periódicos entre las partes interesadas para tener promulgadas las leyes hasta el final del 2020 y celebrar en 2021 el año de las enfermedades raras de América Latina en donde cada país y la región trabajen intensamente en la implementación de los nuevos marcos regulatorios. Apoyar las iniciativas legales que permitan la cobertura económica y el acceso al diagnostico temprano, medicamentos y dispositivos médicos, atención medico-hospitalar de forma rápida y eficiente a través de profesionales de salud capacitados, en centros de atención medica especializados, y aplicando técnicas avanzadas. Respaldar las regulaciones que fomenten la investigación y desarrollo de terapias innovadoras para las enfermedades raras.
- 12. PrivilegedandConfidential Fernando Ferrer, MBA © Multinational Partnerships LLC 12 Missão Promover a reunião de legisladores, juristas, associações médicas, de pacientes, e da indústria e serviços de ciências da vida na América Latina para criar e acelerar os processos legislativos e regulamentares que protegem e dão tratamento a pessoas com doenças raras, e promover pesquisa e desenvolvimento de terapias inovadoras. Impulsionar a realização de contatos regulares entre as partes interessadas para ter leis promulgadas até o final de 2020 e comemorar em 2021 o ano das doenças raras na América Latina, onde cada país e região trabalhem intensamente na implementação dos novos marcos regulatórios. Apoiar as iniciativas legais para a cobertura econômica e acesso ao diagnóstico precoce, medicamentos e dispositivos médicos, assistência médico-hospitalar rápida e eficiente através de profissionais de saúde treinados, em centros médicos especializados, e aplicando técnicas avançadas. Respaldar os regulamentos que incentivem a pesquisa e desenvolvimento de terapias inovadoras para as doenças raras.
- 13. PrivilegedandConfidential Fernando Ferrer, MBA © Multinational Partnerships LLC 13 Mission Promote the meeting of legislators, jurists, medical associations, patient associations, and life sciences industry and services in Latin America to create and accelerate legislative and regulatory processes that protect and treat people with rare diseases, and to promote research and development of innovative therapies. Encourage periodic contacts between stakeholders to have legislation enacted until the end of 2020 and to celebrate in 2021 the year of rare diseases in Latin America where each country and region work intensely in the implementation of new regulatory frameworks. Support legal initiatives that allow the economic coverage and access to early diagnosis, medicines and medical devices, medical and hospital care in a fast and efficient fashion through trained health professionals, specialized health care centers and applying advanced techniques. Support regulations that foster research and development of innovative therapies for rare diseases.
- 14. PrivilegedandConfidential Fernando Ferrer, MBA © Multinational Partnerships LLC 14 Valores - Values • Compromiso • Humanidad • Evolución • Sentido de urgencia • Solidaridad • Comprometimento • Humanidade • Evolução • Senso de urgência • Solidariedade • Commitment • Humanity • Evolution • Sense of urgency • Solidarity VALUES: CHESS Commitment – personal and professional dedication in every act so as to accomplish the mission Humanity – considering the human race collectively, leaving no one behind Evolution - Aware of the need to evolve and move towards to a better future Sense of urgency – the diseases are moving forward and we need to act proactively right now Solidarity – unity and mutual support towards a higher purpose VALORES: CHESS Compromiso - dedicación personal y profesional en cada acto para cumplir la misión Humanidad - considerando la raza humana colectivamente, sin dejar a nadie atrás Evolución - Consciente de la necesidad de evolucionar y avanzar hacia un futuro mejor Sentido de urgencia - las enfermedades están avanzando y necesitamos actuar de manera proactiva ahora Solidaridad - unidad y apoyo mutuo hacia un propósito más elevado VALORES: CHESS Compromisso - dedicação pessoal e profissional em cada ato de modo a cumprir a missão Humanidade - considerando a raça humana coletivamente, não deixando ninguém para trás Evolução - Consciente da necessidade de evoluir e avançar para um futuro melhor Sentido de urgência - as doenças estão avançando e precisamos agir proativamente agora Solidariedade - unidade e apoio mútuo para um propósito mais elevado
- 15. PrivilegedandConfidential Fernando Ferrer, MBA © Multinational Partnerships LLC 15 Antecedentes • Las personas con enfermedades raras y sus familias se han esforzado por años para tener una solución terapéutica y económica para su condición. • Algunos países han reconocido el derecho a la salud de los ciudadanos y han promulgado legislaciones para asistir a estas personas. • Ha sido un gran esfuerzo que involucró por años diversos sectores de la sociedad. El progreso ha sido lento y desigual en toda la región y las soluciones aún no han sido suficientes. • Es necesario trabajar en la región en un esfuerzo conjunto donde los éxitos sean compartidos, se aprenda de los errores y se genere un marco legal adecuado a la realidad de la salud de los pueblos de América Latina.
- 16. PrivilegedandConfidential Fernando Ferrer, MBA © Multinational Partnerships LLC 16 Antecedentes • As pessoas com doenças raras e suas famílias têm lutado por anos para ter uma solução terapêutica e econômica para sua condição. • Alguns países têm reconhecido o direito à saúde dos cidadãos e promulgaram legislações para amparar o direito dessas pessoas. • Foi um grande esforço durante anos, envolvendo vários setores da sociedade. O progresso tem sido lento e desigual em toda a região e as soluções ainda não foram suficientes. • E preciso trabalhar em um esforço conjunto onde os sucessos sejam compartilhados, aprender com os erros e gerar um enquadramento legal adequado a realidade da saúde dos povos da América Latina.
- 17. PrivilegedandConfidential Fernando Ferrer, MBA © Multinational Partnerships LLC 17 Background • People suffering from rare diseases and their families have struggled for years to have a therapeutic and economic solution for their condition. • Some countries have recognized the right to health of citizens and have enacted legislation to assist these people. • It has been a great effort that involved for years different sectors of society. Progress has been slow and uneven throughout the region and solutions have not yet been sufficient. • It is necessary to work in the region in a joint effort where successes are shared, learn from mistakes, and to create the legal framework appropriate to the reality of the health of the people of the Latin America.
- 18. PrivilegedandConfidential Fernando Ferrer, MBA © Multinational Partnerships LLC 18 PERLA 2021 • PERLA 2021 es un programa dirigido a unir a los pueblos de América Latina en una lucha contra un enemigo común: las enfermedades raras. • PERLA 2021 es un programa integral de estrategias y acciones para acelerar la creación e implementación de leyes para la amplia protección y asistencia a las personas con enfermedades raras. • PERLA 2021 tiene la función primordial de integrar a las partes interesadas en un diálogo constructivo y acelerar los procesos legales y burocráticos para dar soluciones rápidas y efectivas a los millones de enfermos que sufren de las enfermedades raras en América Latina.
- 19. PrivilegedandConfidential Fernando Ferrer, MBA © Multinational Partnerships LLC 19 PERLA 2021 • PERLA 2021 é um programa voltado para para unir os povos da América Latina em uma luta contra um inimigo comum: as doenças raras. • PERLA 2021 é um programa abrangente de estratégias e ações com o intuito de acelerar a criação e implementação de leis para a ampla proteção e assistência das pessoas com doenças raras. • PERLA 2021 possui a função primordial de integrar as partes interessadas num diálogo construtivo e acelerar os processos legais e burocráticos para o fornecimento de soluções rápidas e eficazes para os milhões de pacientes que sofrem de doenças raras na América Latina
- 20. PrivilegedandConfidential Fernando Ferrer, MBA © Multinational Partnerships LLC 20 PERLA 2021 • PERLA 2021 is a program aimed at uniting the people of Latin America in a fight against a common enemy: the rare diseases. • PERLA 2021 is a comprehensive program of strategies and actions to accelerate the creation and implementation of laws for the broad protection of people with rare diseases. • PERLA 2021 has the primary role of integrating stakeholders into a constructive dialogue and speeding up legal and bureaucratic processes to provide fast and effective solutions for the millions of patients suffering from rare diseases in Latin America.
- 21. PrivilegedandConfidential Fernando Ferrer, MBA © Multinational Partnerships LLC 21 ¿Hay realmente dos lados para estar? Con las personas Con la enfermedad Credit: NHGRI Sólo hay 1 lado Las personas deben estar juntas contra la enfermedad Este es un problema de personas Pacientes Familiares Los defensores del paciente Gente de nuestra sociedad civil Profesionales de la salud Proveedores de servicios de salud Investigadores de la salud Legisladores Responsables de la política sanitaria Funcionarios gubernamentales Juristas Empleados de la industria de las ciencias de la vida Periodistas Credit Microsoft
- 22. Credit: NHGRI La falta de acciones en favor del paciente, y para combatir la enfermedad, es una actitud contra las personas y favoreciendo al desarrollo de la enfermedad rara
- 23. PrivilegedandConfidential Fernando Ferrer, MBA © Multinational Partnerships LLC 23 Há realmente dois lados para estar? Com as pessoas Com a enfermidade Credit: NHGRI Há apenas 1 lado As pessoas devem estar juntas contra a doença Esta é uma questão de pessoas Pacientes Familiares Advogados dos pacientes Pessoas da nossa sociedade civil Profissionais de saúde Prestadores de cuidados de saúde Pesquisadores de saúde Legisladores Formuladores de políticas de saúde Funcionários governamentais Juristas Empregados da indústria das ciências da vida Jornalistas Credit Microsoft
- 24. Credit: NHGRI A ausência de ações em favor do paciente, e do combate a doença, é uma atitude contra as pessoas favorecendo o desenvolvimento de doença rara
- 25. PrivilegedandConfidential Fernando Ferrer, MBA © Multinational Partnerships LLC 25 Are there actually two sides to be? With the People With the Disease Credit: NHGRI There is only 1 side People must be together against the Disease This is an issue of people Patients Family – Relatives Patient’s advocates People of our civil society Healthcare Practitioners Healthcare Providers Healthcare researchers Legislators Healthcare policy makers Governmental officials Jurists Employees of life science industry Journalists Credit Microsoft
- 26. Credit: NHGRI The absence of actions in favor of the patient, and to fight against the disease, is an attitude against people favoring the development of the rare disease
- 27. PrivilegedandConfidential Fernando Ferrer, MBA © Multinational Partnerships LLC 27 Proximos pasos en america latina • Comunicar la iniciativa a las diversas partes interesadas • Generar un encuentro de legisladores y pacientes para conversar lo que hace falta • Facilitar el dialogo de todas las partes interesadas para generar la estrategia y plan de acción • Generar los diversos grupos de trabajo y encuentros para implementar la estrategia • Promulgar que 2021 sea el año de lucha contra las enfermedades raras en América Latina ③ Next steps
- 28. PrivilegedandConfidential Fernando Ferrer, MBA © Multinational Partnerships LLC 28 Próximos passos na América Latina • Comunicar a iniciativa para as várias partes interessadas • Gerar uma reunião de legisladores e pacientes para discutir o que é preciso • Facilitar o diálogo entre todas as partes interessadas para gerar a estratégia e plano de ação • Gerar os vários grupos de trabalho e reuniões para implementar a estratégia • Decretar que 2021 seja o ano de combate as doenças raras na América Latina
- 29. PrivilegedandConfidential Fernando Ferrer, MBA © Multinational Partnerships LLC 29 Next steps in Latin America • Communicate the initiative to the various stakeholders • Generate a meeting of legislators and patients to talk about what is needed • Facilitate dialogue among all stakeholders to generate the strategy and action plan • Generate the various working groups and meetings to implement the strategy • To promulgate 2021 to be the year for combating rare diseases in Latin America
- 30. Fernando Ferrer, BSc. MBA Phone + 1 908 326 6417 fferrer@multinationalpartnerships.com Muchas gracias Muito obrigado Thank you ® © Fernando Ferrer Multinational Partnerships® is a Registered Trade Mark of Multinational Partnerships LLC
Notas do Editor
- Exención de responsabilidad
- Trabalhar em conjunto com uma abordagem aberta, holística e pragmática para trazer soluções aos consumidores
- Vision – big picture of what you want to achieve.
- Mission – general statement of how you will achieve the vision.
- Mission – general statement of how you will achieve the vision.
- Mission – general statement of how you will achieve the vision.
- Core Values – how you will behave during the process. Commitment – personal and professional dedication in every act so as to accomplish the mission Humanity – considering the human race collectively, leaving no one behind Evolution - Aware of the need to evolve and move towards to a better future Sense of urgency – the diseases are moving forward and we need to act proactively right now Solidarity – unity and mutual support towards a higher purpose
- Credit: NHGRI https://www.nih.gov/news-events/nih-research-matters/rare-artery-disease-linked-gene-variant An X-ray of a knee from a patient with ACDC, showing calcium buildup in the artery. NHGRI National Human Genome Research Institute (NHGRI) https://www.genome.gov/ February 14, 2011 Rare Artery Disease Linked to Gene Variant Scientists have discovered a genetic cause for a rare disorder that leads to calcium deposits in arteries in the lower half of the body and in the joints of patients' hands and feet. The culprit is a mutation in the gene encoding CD73, a protein that normally helps to prevent calcium buildup. The researchers hope that understanding the basis for this disease, which they call arterial calcification due to deficiency of CD73 (ACDC), will help them find a cure. Currently, 9 patients are known to have ACDC, and all come from 3 families. The patients experienced pain in their buttocks, legs and joints, but doctors could find no signs of rheumatoid arthritis or other recognized problems that might cause the symptoms. A research team that included scientists from NIH’s National Heart, Lung and Blood Institute (NHLBI) and National Human Genome Research Institute (NHGRI) examined members of 2 affected families to search for a common thread. MRI and X-rays showed that members of the 2 families all had calcium buildup in arteries in the lower half of their bodies, but not around their hearts. In one family, 5 siblings but neither parent was affected by the disorder. The researchers suspected that symptoms might be due to a recessive gene variant. Recessive variants only show their effects when found in both copies of the gene. To test this theory, the team, led by Dr. William Gahl of NHGRI, compared DNA from the parents of the family to their children's. They found a region of DNA that was present only once in the parents, but twice in the children. The team then compared this region to the same region in 200 unaffected people. The results were published on February 3, 2011, in the New England Journal of Medicine. The researchers found a variant in a gene called NT5E that unaffected people didn’t have. NT5E makes a protein called CD73, which produces a small molecule called adenosine that helps regulate many processes in the body. The NHLBI team, led by Dr. Manfred Boehm and Dr. Cynthia St. Hilaire, cultured the patients’ cells to examine the effects of the mutation on an enzyme called TNAP (tissue-nonspecific alkaline phosphatase). TNAP is known to degrade a calcium buildup inhibitor and thus contribute to calcium deposits. The researchers found that the mutant cells produced higher levels of TNAP. Adding adenosine to the cell cultures restored normal levels of TNAP. In addition, while mutant cells made more calcium phosphate crystals in culture, restoring CD73 expression in the cells prevented crystal formation. "Vascular calcification often results from poor diet and lack of exercise," says Gahl, who is NHGRI clinical director and director of the NIH Undiagnosed Diseases Program. "The calcium buildup in arteries of our patients, however, arises because the systems to inhibit it are not working in their cells. We hope that an understanding of this faulty mechanism will guide us in providing helpful treatments for these patients."
- Credit: NHGRI https://www.nih.gov/news-events/nih-research-matters/rare-artery-disease-linked-gene-variant An X-ray of a knee from a patient with ACDC, showing calcium buildup in the artery. NHGRI National Human Genome Research Institute (NHGRI) https://www.genome.gov/ February 14, 2011 Rare Artery Disease Linked to Gene Variant Scientists have discovered a genetic cause for a rare disorder that leads to calcium deposits in arteries in the lower half of the body and in the joints of patients' hands and feet. The culprit is a mutation in the gene encoding CD73, a protein that normally helps to prevent calcium buildup. The researchers hope that understanding the basis for this disease, which they call arterial calcification due to deficiency of CD73 (ACDC), will help them find a cure. Currently, 9 patients are known to have ACDC, and all come from 3 families. The patients experienced pain in their buttocks, legs and joints, but doctors could find no signs of rheumatoid arthritis or other recognized problems that might cause the symptoms. A research team that included scientists from NIH’s National Heart, Lung and Blood Institute (NHLBI) and National Human Genome Research Institute (NHGRI) examined members of 2 affected families to search for a common thread. MRI and X-rays showed that members of the 2 families all had calcium buildup in arteries in the lower half of their bodies, but not around their hearts. In one family, 5 siblings but neither parent was affected by the disorder. The researchers suspected that symptoms might be due to a recessive gene variant. Recessive variants only show their effects when found in both copies of the gene. To test this theory, the team, led by Dr. William Gahl of NHGRI, compared DNA from the parents of the family to their children's. They found a region of DNA that was present only once in the parents, but twice in the children. The team then compared this region to the same region in 200 unaffected people. The results were published on February 3, 2011, in the New England Journal of Medicine. The researchers found a variant in a gene called NT5E that unaffected people didn’t have. NT5E makes a protein called CD73, which produces a small molecule called adenosine that helps regulate many processes in the body. The NHLBI team, led by Dr. Manfred Boehm and Dr. Cynthia St. Hilaire, cultured the patients’ cells to examine the effects of the mutation on an enzyme called TNAP (tissue-nonspecific alkaline phosphatase). TNAP is known to degrade a calcium buildup inhibitor and thus contribute to calcium deposits. The researchers found that the mutant cells produced higher levels of TNAP. Adding adenosine to the cell cultures restored normal levels of TNAP. In addition, while mutant cells made more calcium phosphate crystals in culture, restoring CD73 expression in the cells prevented crystal formation. "Vascular calcification often results from poor diet and lack of exercise," says Gahl, who is NHGRI clinical director and director of the NIH Undiagnosed Diseases Program. "The calcium buildup in arteries of our patients, however, arises because the systems to inhibit it are not working in their cells. We hope that an understanding of this faulty mechanism will guide us in providing helpful treatments for these patients."
- Credit: NHGRI https://www.nih.gov/news-events/nih-research-matters/rare-artery-disease-linked-gene-variant An X-ray of a knee from a patient with ACDC, showing calcium buildup in the artery. NHGRI National Human Genome Research Institute (NHGRI) https://www.genome.gov/ February 14, 2011 Rare Artery Disease Linked to Gene Variant Scientists have discovered a genetic cause for a rare disorder that leads to calcium deposits in arteries in the lower half of the body and in the joints of patients' hands and feet. The culprit is a mutation in the gene encoding CD73, a protein that normally helps to prevent calcium buildup. The researchers hope that understanding the basis for this disease, which they call arterial calcification due to deficiency of CD73 (ACDC), will help them find a cure. Currently, 9 patients are known to have ACDC, and all come from 3 families. The patients experienced pain in their buttocks, legs and joints, but doctors could find no signs of rheumatoid arthritis or other recognized problems that might cause the symptoms. A research team that included scientists from NIH’s National Heart, Lung and Blood Institute (NHLBI) and National Human Genome Research Institute (NHGRI) examined members of 2 affected families to search for a common thread. MRI and X-rays showed that members of the 2 families all had calcium buildup in arteries in the lower half of their bodies, but not around their hearts. In one family, 5 siblings but neither parent was affected by the disorder. The researchers suspected that symptoms might be due to a recessive gene variant. Recessive variants only show their effects when found in both copies of the gene. To test this theory, the team, led by Dr. William Gahl of NHGRI, compared DNA from the parents of the family to their children's. They found a region of DNA that was present only once in the parents, but twice in the children. The team then compared this region to the same region in 200 unaffected people. The results were published on February 3, 2011, in the New England Journal of Medicine. The researchers found a variant in a gene called NT5E that unaffected people didn’t have. NT5E makes a protein called CD73, which produces a small molecule called adenosine that helps regulate many processes in the body. The NHLBI team, led by Dr. Manfred Boehm and Dr. Cynthia St. Hilaire, cultured the patients’ cells to examine the effects of the mutation on an enzyme called TNAP (tissue-nonspecific alkaline phosphatase). TNAP is known to degrade a calcium buildup inhibitor and thus contribute to calcium deposits. The researchers found that the mutant cells produced higher levels of TNAP. Adding adenosine to the cell cultures restored normal levels of TNAP. In addition, while mutant cells made more calcium phosphate crystals in culture, restoring CD73 expression in the cells prevented crystal formation. "Vascular calcification often results from poor diet and lack of exercise," says Gahl, who is NHGRI clinical director and director of the NIH Undiagnosed Diseases Program. "The calcium buildup in arteries of our patients, however, arises because the systems to inhibit it are not working in their cells. We hope that an understanding of this faulty mechanism will guide us in providing helpful treatments for these patients."