White spots in pharmaceutical pipelines–EMA identifies potential areas of unmet medical needs

Abstract

Unmet medical needs are a priority for organizations such as the WHO and major public-private initiatives, such as Innovative Medicines Initiative, were established to speed up the development of better and safer medicines for patients. To assist such projects, the EMA in its 'Road Map to 2015' considered the mapping of unmet medical needs as a priority. This study has identified medical conditions for which the EMA could not identify developments in the pharmaceutical pipelines, that is, 'white spots'. Our analysis was made using external data sources as well as mining data of the EMA. The main areas for white spots were oncology, infectious diseases and certain psychiatric conditions. According to our data and a review of literature, in a number of these white spots, diagnostic tools may even be missing. The identification of those conditions will benefit stakeholders, including regulators, research funding bodies and patients' organizations.

Queries Raised During Oncology Business Pipeline Meetings at the European Medicines Agency: A 5‐Year Retrospective Analysis

“…Molecular markers have paved the way for targeted therapy, leading to the necessity for the development of CDx to identify which patients would benefit from treatment, for which queries led to the adoption of the In Vitro Diagnostics Medical Device Regulation in 2017 21 . The reclassification of tumors based on molecular markers also raised queries about patient groups with unmet medical needs and the need to explore novel trial designs, including the use of RWE, to compensate for smaller cohort sizes 16,22 . Additionally, molecular markers, such as circulating tumor DNA, can be promising novel end points and potential surrogates for overall survival 23,24 .…”

The New Paradigms in Clinical Research: From Early Access Programs to the Novel Therapeutic Approaches for Unmet Medical Needs

“…Although several innovative therapies were recently approved worldwide, there are still therapeutic areas for which unsatisfied therapeutic needs persist (Figure 1). Indeed, the so-called “white spots” – clinical conditions for which no efficacious treatments are approved – still exist among pharmaceutical pipelines (Papaluca et al, 2015). For example, a high unmet clinical need was observed in patients diagnosed with type 2 diabetes mellitus (T2DM), which represents an increasing health concern worldwide and is a leading risk factor for cardiovascular diseases.…”

Unmet medical needs in lupus nephritis: solutions through evidence-based, personalized medicine

“…Unmet medical needs reflect targeted objectives to improve patient-related outcomes [ 1 ]. Defining unmet medical needs is important for patients, doctors, industry, regulators and for those who allocate healthcare budgets [ 1 ].…”

“…Unmet medical needs reflect targeted objectives to improve patient-related outcomes [ 1 ]. Defining unmet medical needs is important for patients, doctors, industry, regulators and for those who allocate healthcare budgets [ 1 ]. Unmet medical needs accrue from patient-related disease effects (quality of life, organ damage, mortality) and management-related challenges (biomarkers for diagnosis and monitoring).…”

“…Unmet medical needs accrue from patient-related disease effects (quality of life, organ damage, mortality) and management-related challenges (biomarkers for diagnosis and monitoring). Unmet medical needs define potential markets for drug or bioassay development, especially in countries that may allocate healthcare budgets to addressing such needs [ 1 ].…”