Benefit Risk Assessment

Question 1

Risk is related to

Answer 1

harm and collected as safety data

Question 2

Risk is

Answer 2

Applies to all studies
Easier to collect
Relative to condition and patient’s acceptance

Question 3

Benefit is

Answer 3

product/project/therapeutic are specific

Related to endpoints, clinical relevance

Often hard to measure

Question 4

FDA Premarketing B/R Assessment During Development risk is assessed using:

Answer 4

Safety reports submitted by sponsor(s) and investigators
-Expedited safety reports
-Annual reports

Benefit is assessed in relationship to risk
- Based on indication

Question 5

The FDA review team evaluates

Answer 5

whether the studies the sponsor submitted show that the drug is safe and effective for its proposed use.
No drug is absolutely safe; all drugs have side effects.
“Safe” in this sense means that the benefits of the drug appear to outweigh the known risks.

Question 6

Adequate Risk Assessment must address

Answer 6

Quantity of safety data
-ensuring that enough patients are studied
-Long term data needed per ICH at 6 mo & 1 yr

Quality of safety data
-Appropriateness and breadth of the patient populations studied and assessments performed
-How results are analyzed

Question 7

US FDA Framework Details

Answer 7

1.Population Characteristics

Analysis of Condition
-Describe the condition that is treated or prevented by the drug.

Unmet Medical Need
-Describe the other therapies used to treat the condition, including approved and off‐label pharmacological therapies and non‐pharmacological therapies.

2.Product Characteristics

Benefit
-Describe the trials (including strengths and weaknesses) that were conducted to establish efficacy.

Risk
-Characterize the safety concerns identified in the clinical trials.

Risk Management
-Identify risks (if any) that will require mitigation or further characterization

Question 8

Future of Benefit/Risk Reporting

Answer 8

Impact and Use of Real-World Evidence/Data (RWE/D)
Enhanced review methodologies to analyze meta-analyses,
Better use of wireless/mobile technologies to transmit drug safety information from the point of care to FDA,
Advances in pharmacogenomics,
Innovative uses of secure access to safety data from electronic health care records,
Improved animal toxicological methods to predict adverse events