Objective

To provide a review of key reasons for negative reimbursement decisions in rare diseases and use case studies to review strategies to drive to positive reimbursement.

Background

The HTA and reimbursement submission process in major European markets is increasingly difficult to navigate, these difficulties are often more pronounced in rare diseases.

It is not uncommon for negative recommendations to be made in the first or initial stages of negotiations. Often this results in the manufacturer offering price modulation however this is not always the case.

Method

A review was conducted of all published HTA and reimbursement submission and/or resubmissions outcomes. The research covered HTA submissions and market access for therapies in a range of rare conditions. Following on from the review of published data, case studies were researched and reviewed to identify methods that did not require price modulation.

Results

Across published data common reasons for rare disease treatments failing to gain reimbursement or positive recommendations were:

• Not cost effective at current price (69%)
• Not clinically relevant (38%)
• Comparator not relevant for the market (31%)
• Lack of clinical demand (28%)

Strategies used to change reimbursement position were as follows:

• Price Modulation (89%)
• Clinical Demand (41%)
• New clinical data (23%)

In case studies clinical demand is shown to be the major determining factor (80%) in changing decisions. Modified Delphi methodology has been utilised to demonstrate clinical demand.

Conclusions

Whilst price modulation is seen as the traditional way to change reimbursement decisions, other factors may also play an important role in challenging and changing decisions. Clinical demand also plays a major role in challenging reimbursement decisions. There is suggestive link from data assessed that demonstrating clinical demand robustly may lead to a lower price modulation.