What is it?
The Bernard Pépin Association, which advocates for the needs of people living with Wilson’s disease in France, ran a ten-year campaign to make a specific formulation of a Wilson’s disease treatment accessible to patients; in 2019, they succeeded.
Previously, the only available formulation of the treatment had to be kept refrigerated and could only be accessed via hospital pharmacies. These factors contributed to poor compliance, because some people found it difficult to visit their hospital regularly and keep the medication at the required temperature.
The campaign succeeded in having a discontinued formulation of the treatment – which did not need to be kept refrigerated – manufactured, authorised and made available in community pharmacies. According to a representative of the association, opening up access to the medication has improved quality of life for many patients.
To achieve this, board members of the association worked closely with healthcare professionals from the Rare Disease Reference Centre for Wilson’s Disease (Le Centre de Référence Maladies Rares (CRMR) de la Maladie de Wilson) and a small pharmaceutical company. Afterwards, they carried on working together to encourage regulators to authorise the formulation and make it available in all pharmacies. The Bernard Pépin Association then worked to inform patients about the new formulation and the benefits it could provide.
What can we learn from this case study?
Collaboration between patient advocates and healthcare professionals on a single policy issue can be powerful in achieving shared advocacy goals. The Bernard Pépin Association and CRMR wrote joint letters to the Ministry of Health and held discussions with policymakers, and eventually the decision was made to authorise the medication. Working closely with the pharmaceutical company was also critical in ensuring that the new formulation was produced.
Training Wilson’s disease advocates can also be highly beneficial in preparing them for discussions with policymakers. Those who were involved in the campaign had to quickly learn about medicine production, clinical research and the marketing authorisation process.
