Edition of 16 October 2023

A new article published in Frontiers in Pharmacology discusses how EU-level evidence-based decision-making and fair pricing and reimbursement negotiations can be leveraged to ensure innovative medicines are safe, effective, and affordable to healthcare systems while respecting the importance of timely access for patients.

Because of the multiple levels of approvals necessary to market medicines in EU countries, the amount of time it takes for patients to have access to new, innovative therapies can be frustrating. First, the EMA must issue an opinion on commercialisation; the European Commission must then officially decide whether or not to issue a marketing authorisation; and national, regional, and local governments must decide on how the medications will be priced, financed, and prioritised. This process can delay access to important therapies, particularly in the case of rare diseases, which are often life-threatening and/or have very few, if any, treatment options.

Initiatives have been introduced at the European level to expedite drug approvals in order to fulfil unmet medical need; however, such processes introduce a higher level of uncertainty with regards to the safety and efficacy of treatments during the approval stage. This can have consequences on pricing and reimbursement negotiations, making it more difficult for national governments to determine if and to what extent such medicines should be financed.

In order to ease this burden while still permitting timely access to medicines for patients in need, the authors recommend an approach which uses strategies such as standards for clinical trial design, and the use of post-marketing real-world evidence. They also call for a balanced pricing and reimbursement system, through a transparent price regulation, which minimises both uncertainty and regulatory delays.

In a recent article published in the Orphanet Journal of Rare Diseases, researchers developed an integrative rare disease profile network in order to identify candidates for drug repurposing or repositioning. In this study, the aim was to identify potential new treatments for glioblastoma; however, the methodology could be adapted to fulfil treatment needs in other rare conditions.

Glioblastoma is a rare, malignant brain tumour with a poor prognosis and few treatment options. In the case of such rare diseases, drug repurposing/repositioning can be promising avenues for the discovery of new treatments. By finding a new use for active pharmaceutical ingredients which have already been investigated or approved for use in the treatment of another disease, the time and money required to develop a new drug can be greatly reduced.

In order to identify potential repurposing candidates for glioblastoma, researchers in this study developed a glioblastoma-based Biomedical Profile Network (GBPN) using biomedical data extracted from the NCATS GARD Knowledge Graph (NGKG). By performing network analysis on the graph, they were able to identify the ten most promising candidates for repurposing or repositioning.

While still requiring further validation, the findings from this study could lead to promising, less invasive treatment options for people living with glioblastoma. Additionally, they demonstrate the potential of the GBPN and other similar networks to identify potential treatments for rare diseases. By applying the methodology developed in this study to other rare disorders, steps can be made towards meeting the high levels of unmet medical need in the rare disease community.