Creating the First Database to Measure the Availability of Innovative Medicines in Orphan Disease in Latin America: A Tool to Enable Multistakeholder Engagement

OBJECTIVES: Improving the availability of innovative medicines in Latin America is a priority for all across stakeholders. Since 2004, EFPIA[1] developed the Patient W.A.I.T.[2] Indicator, enabling stakeholders to measure the availability rate of innovative medicines in 37 European countries. This study has been adapted to understand availability in eight Latin American Countries[3].

METHODS: A group of 196 globally approved innovative medicines in orphan disease was selected[1]. The definitions of ‘availability’ and ‘time to access,’ were adjusted across the countries to address health care system design[2]. Full availability has been defined as a national reimbursement listing or the closest approximation to this in non-universal systems. Local pharmaceutical industry associations gathered the data among their affiliates, complementing it with additional public information.

RESULTS: The average availability rate of orphan medicines was 15% of those globally approved, and 53% of molecules locally approved (2014-2023); an average of 29% of molecules were approved locally. In Europe, the average availability rate was 39%[1]. Colombia reported the highest rate of availability at 26% of globally approved molecules, and 93% of those locally approved (28%), whereas Peru had most opportunity for improvement, with 8% availability of globally approved, and 52% of locally approved (15%). The average delay between local market authorization and reimbursement ranged from 1.5 to 3.4 years[2].

CONCLUSIONS: There are substantial challenges for patients in obtaining access to innovative oncology medicines in Latin America, especially compared to Europe. To identify the root causes of the lack of availability or delays to it, efforts to collect and analyze availability data are an important step, but a broader, multi-stakeholder approach is critical to develop an action plan to address these challenges.