The Use of Patient Reported Outcomes in Aiding the Reimbursement Decisions of Advanced Medicinal Therapy Products
Author(s)
Young J, Bolzani A
Cytel Inc, Berlin, BE, Germany
OBJECTIVES:
Advanced therapy medicinal products (ATMPs), including gene therapies, cell therapies, and tissue engineering products, are developed to target incurable diseases by interfering with the genetic or cellular mechanism. Measuring patient-reported outcomes (PROs) for ATMPs may help aid reimbursement decisions in the absence of outcome data. Therefore, this research aimed to understand the importance of PROs in evaluating ATMPs.METHODS:
An HTA review of appraisal reports of medically approved ATMPs in Europe was conducted in the following target countries: France, Germany, and the UK (review performed May 20, 2022).RESULTS:
In total, 15 approved ATMPs with market authorization in Europe were identified. Of these 15 approved drugs, three are awaiting review by the HTA, two were not reimbursed in the UK, five were not reimbursed in France and six were assigned a non-quantifiable benefit in Germany. From the 33 HTA appraisal reports reviewed (HAS n=10; NICE n=12; G-BA=11), PROs were mainly used to inform the patients' health-related quality of life (HrQoL). PROs were used as primary or secondary endpoints in 7 of the 12 ATMPs assessed. For most of the PROs investigated (n=6), the HTA could not conclude a patient's HrQoL because of the non-comparative nature of the study designs, limited data and a small number of patients. In one example, HAS considered the use of the PROs in the assessment of Spherox® as subjective and did not reward reimbursement. The UK did not comment on the use of PROs, and there were no differences in opinion between France and Germany.CONCLUSIONS:
The inclusion and classification of PROs in HTA assessments of ATMPs varied between countries. If PROs will play an important role in measuring the value of these therapies, there is a need to use both generic and disease-specific PROs across all ATMPs – and to compare the PRO evidence to meaningful comparators.Conference/Value in Health Info
2022-11, ISPOR Europe 2022, Vienna, Austria
Value in Health, Volume 25, Issue 12S (December 2022)
Code
HTA155
Topic
Health Policy & Regulatory, Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes, Reimbursement & Access Policy
Disease
SDC: Rare & Orphan Diseases, STA: Drugs, STA: Genetic, Regenerative & Curative Therapies, STA: Personalized & Precision Medicine