Investigators: Sanae Akodad (PhD researcher), Prof. Hilde Stevens (Promotor), Prof. Nicolas Deconinck (Co-Promotor) and Prof. Delphine De Smedt (Co-promotor)

Abstract:

Rare diseases are commonly debated nowadays. Many rare diseases are life-threatening, chronic and degenerative, making treating the affected patients a public health priority. The question of access to effective treatment in the plethora of rare diseases is prominent. Besides the ongoing medical and biological complexity of developing effective drugs for some of them, in many cases, when an appropriate pathophysiological target is found for a new drug, the drug development path is typically costly and long. Once a drug reaches the market, the potential return on investment is limited due to the small patient population. The lack of transparency over the true costs of innovation poses a real issue. This project will conduct a detailed investigation of the pricing/reimbursement process in the case of Spinal Muscular Atrophy disease. We will draw general lessons from this case study and formulate recommendations for affordable pricing and reimbursement strategies for other rare disease therapies that will reach the market.

In this project, we will take advantage of our expertise in SMA to investigate five key initiatives currently taking place to optimize the pricing/reimbursement process and to facilitate and accelerate access treatment, making the pricing process more transparent and investigate a major issue in this context, the quality of life (QoL) of patients with SMA, each linked to a separate work package : (1) comprehensive value assessment, (2) early dialogues among relevant stakeholders, (3) assessment of societal participation in producing orphan drugs (ODs), (4) innovative reimbursement approaches to allow timely access to ODs, (5) facing new challenges induced by new treatments.

Through our in-depth investigation of the cost of the disease, QOL, reimbursement policies, initiatives in terms of information exchange, early screening of the disease, transparency in terms of pricing and finally Cognition with what it implies in terms of both QOL and cost effectiveness the aim of this project is therefor to reduce the cost of SMA on our healthcare budget by working towards a more cost-effective treatment plan, and through neonatal diagnosis, offer a better disease prospective, and hence QoL for the patient.

The key lessons learned from this project may be applied as a more general model for other rare diseases.