ABSTRACT

Objective

To provide perspectives on the effectiveness of the Modified Delphi (MD) technique in providing evidence to support market access and Health Technology Assessment (HTA) submissions in rare diseases. The value of the technique is discussed together with anonymized case studies demonstrating the method’s merits.

Background

The HTA submission process requires evidence of the therapeutic and patients benefits and unmet need, as standard. The MD technique can form an important tool in gathering this real world evidence.

A MD begins with a foundation of previously developed work to achieve the goal of converging towards a consensus. MD’s normally consists of two or more questionnaire rounds and an opportunity to revise thoughts in light of feedback.

Method

A review was conducted of three unrelated MD research projects (2019) and an assessment made of the method in providing insights that contributed to successful HTA submission outcomes and/or improved understanding of the conditions. The research covered HTA submissions and market access for therapies in a range of rare conditions.

Results

Across three case studies valuable insights were gained including:

• Variations in dosing regimens of existing comparator drugs
• Primary opinions on patient disease burden
• Patient pathways
• Standard of Care
• Long term disease consequences and related healthcare resource implications
• A place in therapy
• Understanding of treatment compliance
• Appreciation of patients’ quality of life
• Prescribing practice and opinion to therapy
• Unmet need and trigger point for prescribing

This led to more accurate modelling of the submission data to better reflect use of existing drugs.

Conclusions

The unique nature of rare diseases means Modified Delphi plays an important role in providing vital insights to support market access and HTA submissions. This is especially the case where documented evidence is lacking. It is recommended that Modified Delphi’s form part of all HTA submissions to ensure effective decision-making.