On Wednesday, August 2, 2023, the MEDI.COM-RARE project organized an online webinar focusing on "Access to Clinical Trials for Patients with Rare Diseases in Romania." The event commenced with Dr. Tanaseanu George Regio, President of the Romanian Association of CROs (ACCSCR), discussing the vital role of Patient Associations. Dr. Dumitru Cristina Andreea provided valuable data on rare diseases, and Dr. Cristina Florescu Moraid addressed the innovative concept of decentralized clinical trials.
Key Points Addressed by the Speakers:
The Role of Patient Associations in Clinical Research: Dr. Tanaseanu George Regio emphasized the pivotal role of Patient Associations in supporting the research industry and patients. These associations play a vital role in offering insights into patient preferences, optimizing study designs, identifying suitable research centers, and providing essential support to patients throughout the clinical trial process. Collaborating with Patient Associations facilitates a continuous dialogue that enhances the understanding of study objectives, evaluation visits, the impact on patients' quality of life, effective communication within patient communities, and valuable feedback.
Rare Disease Impact and Current Studies in Romania: Dr. Dumitru Cristina Andreea provided insightful data on rare diseases at both European and global levels. Rare diseases, constituting 72% of genetic origin among more than 6,000 identified diseases, significantly impact patients' lives and the healthcare system. Romania faces challenges in diagnosing rare diseases and identifying doctors and institutions willing to participate in clinical trials. Currently, Romania has three ongoing clinical studies on rare diseases, focusing on Dermatomyositis, Primary Hyperoxaluria, and Hereditary Angioedema.
Decentralized Clinical Trials and Post-Pandemic Research: Dr. Cristina Florescu Moraid addressed the innovative concept of decentralized clinical trials, which gained prominence in the post-pandemic era. These studies involve conducting study procedures remotely, outside the traditional clinical center setting. Through telemedicine, tele-visits, and home visits, patients receive study-related care at their homes, significantly improving access and adherence. Patient involvement in research and development processes is considered essential for ensuring treatment relevance, enhancing drug development, and ensuring optimal patient protection during clinical trials.
Patient Perspectives and Access to Information:
Mrs. Dorica Dan, President of the Alliance for Rare Diseases Romania, provided valuable insights from a patient's perspective. She emphasized that Romania has 31 accredited experience centers, well-equipped and accredited by the Ministry of Health, which are part of the European reference networks. The patients' association facilitates access to information and communication with the patient group when a clinical trial comes to Romania, and the final decision to enroll in the trial lies with the patient.
Importance of Patient Participation in Clinical Trials:
Patient participation in clinical trials is of paramount importance for medical research and the development of innovative treatments. Patients who enroll in these trials play a crucial role in shaping the future of healthcare by contributing directly to the discovery and evaluation of potential therapies. Their participation allows researchers and healthcare professionals to gain valuable insights into the safety and efficacy of new drugs or interventions.
Moreover, for patients with rare diseases, clinical trials often represent a lifeline, offering hope for improved outcomes and a better quality of life. By participating, patients not only have the opportunity to access novel treatments but also contribute to the advancement of medical knowledge in their respective conditions.In conclusion, the webinar reiterated the significance of research in rare diseases, the need to encourage patient participation in clinical trials, and the importance of fostering open and continuous dialogue among all stakeholders involved. By embracing innovative approaches such as decentralized clinical trials, Romania can enhance access to clinical studies, promote patient-centered care, and drive medical progress forward to benefit those affected by genetic and rare diseases.
