OBJECTIVES: Orphan indications have become an area of focus anticipated to constitute up to 45% of new asset launches by 2023. Combined with recent advancement of cell and gene therapies and challenges within an increasingly tightening payer environment, ensuring a return on investment within small patient populations is becoming challenging. Expanding on from our previously published oncology multiple criteria decision analysis (MCDA) framework, we have developed an orphan MCDA based on a variety of different value attributes that drive decision-making within orphan indications to arrive at a value framework.

METHODS: The orphan MCDA framework in US, France and Germany was developed using 21 weighted criteria validated by previously published research, value framework methodologies from ICER (Institute for Clinical and Economic Review) and ASCO for the evaluation of medicines. We have also validated these through targeted primary market research. The MCDA was based on a scoring system on a scale of 0 to 5 depending on value placed on each criterion by individual markets. The orphan framework included value attributes that measure clinical benefit, economic value, formulation and dosing, disease characteristics also covering additional broader considerations such as public health interests and non-health benefits. A linear regression analysis was performed using monthly cost of treatment as the dependent variable and aggregate MCDA score as the independent variable for orphan products.

RESULTS: For 7 products across a range of orphan indications, a strong correlation between the MCDA scores and the average monthly cost of treatment was observed [R²=0.91 US and FR and 0.87 for Germany]. The correlation that statistically measures how close the data are to the fitted regression line (R²) was over 0.8 suggesting a high predictive relationship.

CONCLUSIONS: The study provides a robust framework for estimating the value and price of assets within orphan indications to inform pricing and market access strategy.