Early Pricing and Market Access Forecasting for Orphan Assets

Leveraging a Value-Driven Approach to Price Forecasting

GPI’s Eva Barkhauskaite, Rachel Jao, Preeti Patel and Tom Brockbank look at early pricing and market access forecasting for orphan assets. The research takes an analytical approach to asset price forecasting, and price value optimisation, designed to empower pricing and market access strategy.

Presented at:


Orphan products could contribute to 45% of new asset launches by 2023. With the recent advancement of cell and gene therapies, in addition to challenges in an increasingly difficult payer environment, ensuring a return on investment within small patient populations is becoming tougher.

Expanding on from our previously reported oncology Multiple Criteria Decision Analysis (MCDA) framework in previous ISPOR events, we have expanded the orphan MCDA. The expansion is based on a variety of different value attributes that drive decision-making within orphan indications, to arrive at a value framework.


The original GPI HORIZON orphan MCDA framework in the US, France and Germany was expanded to Italy, Spain and the UK. The framework was developed using 21 scored and weighted value drivers, validated by previously published research, value framework methodologies from ICER (Institute for Clinical and Economic Review) and ASCO for the evaluation of medicines.

The GPI HORIZON MCDA framework leverages three key areas and highly granulised scoring throughout. The three key focus areas are:

  • Disease characteristics – line of therapy specific scoring; including the burden of disease (mortality and quality of life), level of unmet need (availability of alternatives), frequency, formulation and others
  • Clinical setting for products in question – study design, phase of the study, clinical efficacy (across all endpoints), safety (grade 3/4 TRAEs, discontinuation and others), and quality of life benefit for the assets included in the analyses
  • Broader landscape – innovation status (i.e. first in class), disease modifying effect, economic value and public health interest

We have extensively validated our approach through targeted primary market research, to assign market specific weights and to identify the relative importance to payers. The GPI HORIZON methodology was initially focused on scoring attributes from 0-5, but later, it was further expanded in its granularity and is now based on a scoring system on a scale of 0-15. It is also evaluated by the market specific weights for each of the value criteria for individual markets.

A linear regression analysis is then performed using monthly cost of treatment as the dependent variable, and the aggregate MCDA score as the independent variable for orphan products. In the UK, the framework focused on the correlation against QALYs and leveraged the cost-effectiveness thresholds (for the highly technology appraisal process) to identify a price point at which a product would no longer be cost-effective. Notably, out of 7 assets, only 4 were launched in the UK, which may have resulted in the lowered strength of the relationship between QALY gains and the value score.

We have tested our methodology leveraging an analogue analysis for seven orphan assets for which little or no launch precedence exists to identify the relationship between the MCDA value score and the cost of treatment for those assets.

To see the resulting values of correlation with a brief overview of key drivers and weights for markets, download our webinar, presented at HTAi Virtual Conference.

Example Use Cases

There are several benefits to using a granular and analytical approach when analysing orphan asset value through a payer perspective and adding this approach as part of early access and pricing strategy. The benefits include:

  • Helping to identify payer appropriate price points for the asset to inform go/no-go asset decisions from both in licensing and internal development perspectives
  • Identifying the key value drivers for the asset and tailoring the clinical development programme to demonstrate optimal asset benefit
  • Identifying key payer objections and ensuring the evidence development strategy is tailored to optimise gap filling, which ensures optimal asset outcomes
  • Support with indication prioritisation before pivotal trial design, based on the likely asset outcomes


We used 7 products across a range of orphan indications for which no or little launch precedent exists.

A strong correlation between the MCDA scores and the average yearly cost of treatment was observed (R2 within the regression analysis was consistently over 0.8 suggesting a predictive relationship).


The study provides a robust framework, used standalone, or in combination with targeted payer research, and can be leveraged to estimate the value and price of assets within orphan indications to inform pricing and market access strategy.  It can also help tailor the evidence development strategy, in order to ensure payer questions and objections are addressed in advance. 

Further areas of GPI research into the value driven price and access forecasting, include application of the framework across other indications as well expansion to other launch markets.

Download the full webinar