Clinical Operations

To enhance patient retention in clinical trials, sponsors need to:

1. Ensure clear and ongoing communication, building trust between patients and site staff.
2. Create a patient centered project that focuses on their needs and concerns.
3. Educate patients on the study’s benefits and their role in developing product knowledge.
4. Facilitate patient’s journey and streamline procedures to minimize the burden on patients and utilize decentralized technologies for remote data collection.
5. Provide ongoing education about the study’s progress to maintain patient engagement.

Clinical trials for rare diseases present unique challenges due to the limited number of patients and limited existing knowledge on the disease. Recruitment difficulties often arise, requiring involvement of patient associations. Patient advocacy groups play a crucial role in raising awareness and facilitating study participation.

Regulatory agencies may offer incentives, such as orphan drug designation, to encourage research in rare diseases.

Flexibility in study design and endpoints are necessary to overcome the heterogeneity in rare diseases. Adaptive trial designs allow for real-time adjustments based on new data.

Patients need to be empowered and patient-reported outcomes gain significance in assessing treatment efficacy.