OBJECTIVES:

Conventional value assessment based on regulatory anchored data often fall short when capturing the patient experience. It is often not clearly defined what additional patient centric value elements (PEx) should be collected that are specifically relevant to a disease. This research aims to compare the importance of PEx vs. conventional value elements from patients’ perspective and reveal which are the most important PEx to be considered in value assessment.

METHODS:

During a 2-day workshop, a combined quantitative (coin allocation) and qualitative (moderated group discussion) method was used in Spinal Muscular Atrophy (SMA), as a case study. Participants were asked to prioritize between clinical trial endpoints (e.g. standardized motor scale outcomes) utilized in the approval of SMA treatments (referred to as traditional value elements) and additional PEx; and secondly, to prioritize among PEx to be considered when selecting a disease-modifying treatment for SMA, by allocating 10 coins. Finally, their experience was qualitatively analyzed according to coin-allocation exercises based on the workshop transcript.

RESULTS:

9 participants allotted 41.25% of their coins for traditional endpoints and 58.75% for PEx. However, patients acknowledged that “today’s revolutionary disease-modifying treatments couldn’t have been developed without old endpoints”. Their most highly ranked PEx were 1) Ability to reach personal milestones (“Allowing myself to think about the future”), 46.25% of overall coins; 2) patient’s financial burden (“Without insurance none of this becomes possible at all”); and 3) Value of Hope /Balance timing of risks and benefits (“Hope and the bouncing of that risk is a big mental gain”), 12.5% of coins, respectively.

CONCLUSIONS:

Our holistic approach highlighted differences in prioritizing traditional value elements vs. PEX, from perspective of SMA patients’. Our findings may contribute to improved disease outcomes by emphasizing the importance of including PEx not only in value assessment but already at the regulatory phase.