PD47 Associations Of Orphan Designation And Other Drug Development-Related Factors On Rollout Times And Health-Technology-Assessment Recommendations Of New-Active-Substances

Autor: Belen Sola-Barrado, Tina Wang, Neil McAuslane
Rok vydání: 2022
Předmět:
Zdroj: International Journal of Technology Assessment in Health Care. 38:S107-S108
ISSN: 1471-6348
0266-4623
DOI: 10.1017/s0266462322003063
Popis: IntroductionThe orphan designation has been used by the European Medicines Agency to incentivize the development of drugs treating rare diseases with high-unmet medical needs by supporting their development process and economic returns. This study evaluated the impact of the regulatory orphan designation and other drug development-related factors on the rollout times and Health-Technology-Assessment (HTA) recommendations of new active substances (NASs).MethodsA total of 656 HTA appraisals from 6 European countries were collected for NASs that received regulatory approval between 2012 and 2020. Multivariable logistic (positive and positive with restrictions vs. negative HTA recommendation as dependent variable) and linear regression (rollout time as dependent variable) models examined associations with regulatory orphan designation, expedited process, product type (biotechnological vs chemical), and jurisdiction (France, England, Germany, Poland, Scotland and Sweden). Rollout time was defined as months elapsed from regulatory submission to HTA recommendation (mean± standard deviation).ResultsMultivariable logistic regression analysis identified disparities in HTA recommendations between countries. Every month increase in rollout time conferred a 3 percent reduction in the odds of a positive recommendation (pMultivariable linear regression analysis indicated that orphan products presented a 4.4-month rise in rollout time when compared to non-orphan products (pConclusionsOrphan designated products showed greater odds of receiving a positive HTA recommendation compared with non-orphan. A more detailed review of orphan products could result in their longer rollout time compared with non-orphan counterparts. Considerable differences were found between HTA recommendations and rollout times between jurisdictions.
Databáze: OpenAIRE
Externí odkaz: