Cost-safety modeling provides an adjunct to cost-effectiveness analyses by additionally considering safety. The document discusses the developer's experience building cost-safety models for three medical devices at the request of a CRO. Stakeholders were interested in forecasting costs and safety given their own inputs. The conclusion is that cost-safety modeling will become increasingly demanded by risk-averse stakeholders and user-friendly models backed by rigorous methods can provide credibility.

1. Cost-safety modeling
STRATEGY
Occasionally useful adjunct to cost-
effectiveness
Frederic C. Daoud, MD, MSc, Epidemiologist 1

2. BACKGROUND
2Frederic C. Daoud, MD, MSc, Epidemiologist

3. Usual models
• To summarize data for regulatory submissions,
price justification, clinical presentation:
– Risk-Effectiveness,
– Risk-Benefit,
– Cost-effectiveness and
– Budget-Impact
3Frederic C. Daoud, MD, MSc, Epidemiologist

4. Cost-safety vs Cost-effectiveness
• For centuries: "Above all do not harm"
("Primum non nocere"): physicians’ doctrine
for centuries
• Past decades: Societal demand for improved
efficacy and drive for innovation partially
inreased focus on efficacy and reduced focus
on safety.
4Frederic C. Daoud, MD, MSc, Epidemiologist

5. Safety vs. Effectiveness
• For centuries: "Above all do not harm"
("Primum non nocere"): physicians’ doctrine
for centuries
• Past decades: Societal demand for improved
efficacy and drive for innovation: Priority focus
frequently shifted from safety to efficacy.
• Risk-benefit modeling not always intuitive or
consensual.
5Frederic C. Daoud, MD, MSc, Epidemiologist

6. Recent return to safety regardless
of efficacy combined with costs
• Legal clinician and institution commitments
predominantly about patient safety and
budget constraints.
• Rarely about efficacy or effectiveness.
• Result: GROWING RISK-AVERSION.
6Frederic C. Daoud, MD, MSc, Epidemiologist

7. MY RECENT EXPERIENCE
2013 - 2016
7Frederic C. Daoud, MD, MSc, Epidemiologist

8. Our CRO asked to develop cost-safety
models without efficacy constraints
• Three cases with very different types of
medical devices
– cost-safety model
– Jointly with budget impact model.
8Frederic C. Daoud, MD, MSc, Epidemiologist

9. Stakeholders asked for versions where
they could plug-in their own inputs
• Want to forecast their cost-to-safety and
budget impact given their own inputs.
• Events held as relevant:
– treatment-adverse effects
– Serious, cost-drivers (death, re-operations, etc.)
– Those modifying decision to using the treatment
given unacceptable risk among recruitment
9Frederic C. Daoud, MD, MSc, Epidemiologist

10. Conclusion
• Cost-safety modeling will become increasingly
demanded by risk-avert stakeholders
• User-friendly models (MS Excel) backed by
state-of-the-art template (Bugs, Treeage) and
publication useful for credibility
• Fundamental need to set up task-forces with
Regulatory affairs, Medical Affairs and Market
Access.
10Frederic C. Daoud, MD, MSc, Epidemiologist

11. Cost-safety models included
• Three cases with very different types of
medical devices
– cost-safety model
– Jointly with budget impact model.
11Frederic C. Daoud, MD, MSc, Epidemiologist