Early access to treatments can be crucial for patients suffering from rare diseases, where therapeutic options are often limited. The aim of this project is to improve this access once a rare disease drug has been granted Marketing Authorisation (MA). Working with our client, we have implemented innovative solutions to effectively manage the regulatory and operational challenges associated with this process.
Background
When the project first launched, our client was faced with 2 key challenges: navigating a complex regulatory environment and responding rapidly to the requirements of the competent authorities. Response times between the various authorities posed a major challenge, requiring precise coordination to meet the deadlines crucial for access to treatment. In addition, a new regulatory requirement was introduced, adding to the complexity of the project.
Faced with these challenges, our customer decided to delegate its data management and statistics activities, while ensuring ongoing communication with the clinical sites. The aim of this decision was to free up internal resources and guarantee optimal data collection and analysis. At the same time, methodological support was essential to standardise and improve data entry and analysis, thereby ensuring the quality and reliability of the information collected.
Description of the project
Several solutions were implemented to meet the needs of the project:
- Optimisation of data collection and control processes: A rigorous data collection and control system was put in place. Email alerts were defined to improve communication between the sponsor, pharmacovigilance teams, data management division, and clinical sites.
- Hotline: A hotline, accessible by telephone and email, has been set up to answer questions from centres within 24 hours. This hotline was also responsible for managing compensation agreements with the centres, developing an e-PRO platform with functions specific to the project, monitoring online data, and validating requests for access to treatments.
- Regular monitoring and management: Monitoring lists, including visit schedules and open queries, have been implemented. Regular communications with the French technical agency for information on hospitalisation (ATIH) have made it possible to implement identification via PASREL. The management of treatment orders and the reconciliation of pharmacovigilance (PV) cases were carried out in accordance with IVIDATA Life Sciences’ or the customer’s procedures (SOP).
Results
The actions carried out led to significant results:
- Efficiency in data management: A batch of queries was generated every quarter, and a pharmacovigilance reconciliation was carried out every month. More than 97% of data was completed, and the average time taken to close queries was reduced to less than a month. Response time to prescribers was reduced to less than 4 hours.
- Compliance with regulatory deadlines: The project met all regulatory deadlines, with the first patient enrolled less than a month after the request for access was accepted, and an interim summary report submitted one month after the database lock.
- Cost control: Despite additional requests from the customer, the project was financially controlled with only 3 amendments. The project involved 11 patients across 10 sites, with patient follow-up over 15 months and a total project duration of 20 months.
- The approach adopted not only met regulatory requirements, but also ensured efficient and rapid data management, while maintaining a high level of communication with all stakeholders.
