On December 18, IMPACT joined the “First Seminar on Innovation and Development of Advanced Therapy Products” organized by the Institute of Public Health of Chile (ISP), which brought together important national and international actors in the health sector, together with leading scientists and researchers in the area, in order to promote innovation and development of advanced therapies in the country, ensuring the quality and safety of the products obtained from research.
The activity was led by the director of the ISP, Catterina Ferreccio, and the National Coordinator of the Pediatric Oncology Group (PINDA), Julia Palma, who is also Coordinator of Hematopoietic Progenitor Transplantation and advisor on Childhood and Adolescent Cancer for the Cabinet of the Chilean Minister of Health.
IMPACT’s director, Maroun Khoury, was present as a guest at the panel discussion “Advanced Therapies in Chile: Potential for regional leadership in R&D, production and export”. Also present in this instance were the executive vice-president of Pro Salud Chile, Jean-Jacques Duhart; the resident physician of the Critical Patient Unit of the Arturo López Pérez Foundation, Joaquín Jerez; and the Innovation and Health Manager of the Chilean Chamber of Pharmaceutical Innovation, Francisca Rodríguez.
“These instances of conversation and debate are fundamental. In science, we have been working for decades on research that has laid the foundations for treatments based on advanced cell therapy that we see today available in some developed countries. These are treatments that give hope to critical patients, where traditional drugs no longer provide solutions. We are at an impressive moment. What comes now is to work together, but with a formal framework, i.e., we as researchers, can suggest being part of an advisory committee and the ISP must respond if it agrees. The next step of this seminar must be concrete actions and not just good will. We should not think that it is something only political, but something where we can all contribute, from our knowledge and expertise. If we understand that what we are doing is patient-centered, independent of political colors, we will be able to move forward,” said Maroun Khoury.
Specific regulation: the necessary step
During the conference, researchers, clinicians and authorities agreed on the opportunities that advanced therapies offer in the treatment of diseases. “The world of cancer, above all, points the perspective of children and adolescents to precision therapy, which means making a diagnosis in a person and according to the different characteristics of the host, such as the tumor or the leukemic involvement of the patient, treating him or her in a very specific way, without causing so many side effects. In pediatric oncology we have a cure rate of more than 80% and the increase in this rate depends on having access to advanced therapies, but we have to separate the universes with respect to age,” said Julia Palma.
However, the lack of specific regulation hinders the development and use of these therapies in the country. The ISP recognized the need to have clear and specific regulatory frameworks, ensuring that they are in line with international standards, while maintaining an ethical and safe approach for patients. To this end, the activity included a review of the regulatory systems of different countries such as Brazil and Japan.
During his intervention at the seminar, Joao Batista, Manager of Blood, Tissue, Cell, Organ and Advanced Therapy Product Management at Brazil’s National Health Surveillance Agency (ANVISA), explained that in Brazil there are already 9 authorized therapies. “Of what we have approved, four are against Cancer, specifically to treat lymphomas, leukemia and myelomas; and 5 are for monogenetic therapies, which seek to correct or replace a defective gene in a patient’s cells to restore their normal function.”
In his presentation, Batista highlighted the need to work at a regional level to overcome the challenges that currently prevent Latin America from promoting the development of these therapies. “The products used in advanced therapies have a very high price, so it is essential to be able to count on the help of the Pan American Health Organization to have access to them,” he said.
Among the conclusions of the seminar presented by the experts, were the need to promote more partnerships between universities, research centers, and private sector companies to promote research and development of new therapies; to enhance tax and investment incentives for advanced therapy companies; and to develop public policies that promote equitable access to advanced therapies, possibly through partnerships with the public sector or initiatives to reduce the costs of these innovative treatments, which should be explored as a country, were highlighted.
2024 was a record year for approval of advanced therapies globally. The U.S. Food and Drug Administration (FDA) approved eight of them.