Real World Data
Collecting real world data (RWD) from patients outside of the clinical trial setting can provide valuable insights for Sponsors, regulators, key industry stakeholders and end users of promising new drugs
Why Collect RWD in EAPs?
- Increase understanding of your drug and its value-effectiveness in existing and new patient populations
- Inform future clinical study efforts by evaluating whether new outcomes are significant and warrant additional clinical trials
- Increase awareness among key stakeholders who could be future advocates for and users of your drug
- RWD can be used to support clinical trial data that is submitted to regulatory bodies for product approval
Considerations for Data Collection in EAPs
The regulations for collecting and monitoring data vary from country to country.
It is important to assess the regulatory guidelines in each country in scope to determine whether or not certain data is allowed to be collected.
Clearly defining what the end use of the data will be will help ensure an appropriate and effective data collection plan is designed.
If submission ready data is desired, data must be:
- Coded using CDISC/CDASH
- Monitored
- SAS formatted extraction
It is important to choose an EDC system that has been developed specifically to make data collection compliant and efficient.
This will ensure the integrity of your data and help align on all data goals, regulations, and data input expectations.
Benefits of Collecting Data with WEP
Types of Data We Can Collect
- Demographic patient information
- Safety
- Dosing information
- Efficacy
- Quality of life (QoL)
- Patient Reported Outcomes (PROs)
- Electronic health records (EHRs)
- Claims and billing activities
- Product and disease registries
- Patient-generated data including in home-use settings
- Questionnaires
- Data gathered from other sources that can inform on health status, such as mobile devices
NEED MORE INFORMATION?
Why Collect RWD in EAPs?
• Publish findings from data collection in industry journals and publications
• Helps inform HCPs, patients and patient groups who could be future advocates for and users of commercial product
• Helps raise awareness of your product among patients who could be future clinical trial participants
• Gain further insight into patient compliance and tolerability
– May inform dosing guidelines
– Assess packaging and formulation effectiveness
• Inform off-label uses
• Determine value-effectiveness for future drug reimbursement
• Gain new insights into new patient populations and indications that you are unlikely to gain from RCT population
• A chance to further understand new outcomes gained from clinical trial efforts – assess whether these are significant data sets and warrant additional clinical studies
• Assess interest and potential uptake from ROW countries
• Regulatory bodies encourage RWD collection and use of RWE
• Safety and efficacy data that echo what is being collected in the clinical trial
• Especially significant in rare diseases where every dataset is significant due to the small patient numbers
What is Real World Data & Evidence?
Increase Awareness Among Stakeholders
• Publish findings from data collection in industry journals and publications
• Helps inform HCPs, patients and patient groups who could be future advocates for and users of commercial product
• Helps raise awareness of your product among patients who could be future clinical trial participants
Increase Understanding of Study Drug
• Gain further insight into patient compliance and tolerability
– May inform dosing guidelines
– Assess packaging and formulation effectiveness
• Inform off-label uses
• Determine value-effectiveness for future drug reimbursement
Inform Future Clinical Study Efforts
• Gain new insights into new patient populations and indications that you are unlikely to gain from RCT population
• A chance to further understand new outcomes gained from clinical trial efforts – assess whether these are significant data sets and warrant additional clinical studies
• Assess interest and potential uptake from ROW countries
Support Clinical Trial Data
• Regulatory bodies encourage RWD collection and use of RWE
• Safety and efficacy data that echo what is being collected in the clinical trial
• Especially significant in rare diseases where every dataset is significant due to the small patient numbers
Why Collect RWD in EAPs?
Collecting data in an EAP provides extremely valuable insights that can allow sponsors to
- Gain further insight into drug safety and efficacy
- Gain greater understanding of the varied patient journeys and treatment pathways
- Generate more data to support the drug approval process
- Identify populations with enhanced benefit/risk
- Develop evidence to support expanding the indication for a drug
Sponsor Considerations
What? is the Goal of Data Collection
- What will the data be used for?
- What types of data will you collect?
- Is a protocol needed?
How? - Human Element
- Who will be providing the data?
- How should you engage with them?
- How will you compensate them, if at all?
How? - Technology Element
- What data collection tool will you use?
- What requirements must this tool fulfil
- Do you have the expertise to run in-house?