Social pharmaceutical innovation and alternative forms of research, development and deployment for drugs for rare diseases.

Rare diseases are associated with difficulties in addressing unmet medical needs, lack of access to treatment, high prices, evidentiary mismatch, equity, etc. While challenges facing the development of drugs for rare diseases are experienced differently globally (i.e., higher vs. lower and middle income countries), many are also expressed transnationally, which suggests systemic issues. Pharmaceutical innovation is highly regulated and institutionalized, leading to firmly established innovation pathways. While deviating from these innovation pathways is difficult, we take the position that doing so is of critical importance. The reason is that the current model of pharmaceutical innovation alone will not deliver the quantity of products needed to address the unmet needs faced by rare disease patients, nor at a price point that is sustainable for healthcare systems. In light of the problems in rare diseases, we hold that re-thinking innovation is crucial and more room should be provided for alternative innovation pathways. We already observe a significant number and variety of new types of initiatives in the rare diseases field that propose or use alternative pharmaceutical innovation pathways which have in common that they involve a diverse set of societal stakeholders, explicitly address a higher societal goal, or both. Our position is that principles of social innovation can be drawn on in the framing and articulation of such alternative pathways, which we term here social pharmaceutical innovation (SPIN), and that it should be given more room for development. As an interdisciplinary research team in the social sciences, public health and law, the cases of SPIN we investigate are spread transnationally, and include higher income as well as middle income countries. We do this to develop a better understanding of the social pharmaceutical innovation field's breadth and to advance changes ranging from the bedside to system levels. We seek collaborations with those working in such projects (e.g., patients and patient organisations, researchers in rare diseases, industry, and policy makers). We aim to add comparative and evaluative value to social pharmaceutical innovation, and we seek to ignite further interest in these initiatives, thereby actively contributing to them as a part of our work.

A participação social na vigilância de medicamentos / Social participation in drug surveillance

Objective: to analyze the contribution of patients in drug surveillance, considering some stages of development of these technologies and the interest of public health. Methodology: a bibliographic and documentary analysis was performed on patients' contributions and mechanisms of social participation. Results: we identified consolidated initiatives and results that support the importance of patients in drug surveillance in various segments. However, participation is limited due to the low scientific literacy and ineffectiveness of some social participation mechanisms. Conclusion: patient representative organizations can contribute more effectively to social participation initiatives in drug surveillance, as reported in international experiences. In addition, it is essential that regulatory agencies seek to improve participation mechanisms and the use of methods to collect social contributions, as well as to systematize patients' experiences. (AU).

Objetivo: analizar la contribución de los pacientes a la vigilancia de medicamentos, considerando algunas etapas del desarrollo de estas tecnologías y el interés de la salud pública. Metodología: se realizó un análisis bibliográfico y documental sobre las contribuciones de los pacientes y los mecanismos de participación social. Resultados: identificamos iniciativas y resultados consolidados que respaldan la importancia de los pacientes en la vigilancia de medicamentos en diversos segmentos. Sin embargo, la participación es limitada debido a la baja alfabetización científica y la ineficacia de algunos mecanismos de participación social. Conclusion: Las organizaciones representativas de pacientes pueden contribuir más eficazmente a las iniciativas de participación social en la vigilancia de drogas, como se informa en experiencias internacionales. Además, es esencial que las agencias reguladoras busquen mejorar los mecanismos de participación y el uso de métodos para recaudar contribuciones sociales, así como para sistematizar las experiencias de los pacientes. (AU).

Objetivo: analisar a contribuição dos pacientes na vigilância de medicamentos, considerando algumas etapas de desenvolvimento dessas tecnologias e o interesse da saúde pública. Metodologia: foi realizada análise bibliográfica e documental sobre as contribuições dos pacientes e os mecanismos de participação social. Resultados: identificou-se iniciativas consolidadas e resultados que sustentam a importância do paciente na vigilância de medicamentos em diversos segmentos. Todavia, a participação é limitada devido ao baixo letramento científico e à inefetividade de alguns mecanismos de participação social. Conclusões: organizações representativas de pacientes podem contribuir mais efetivamente em iniciativas de participação social na vigilância de medicamentos, conforme relatado em experiências internacionais. Também, é fundamental que as agências reguladoras busquem aprimorar os mecanismos de participação e a utilização de métodos que possibilitem coletar as contribuições sociais, bem como a sistematização das experiências dos pacientes. (AU).