Pharmacovigilance in a rare disease: example of the VIGIAPATH program in pulmonary arterial hypertension
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Spontaneous reporting is the primary method used in pharmacovigilance (PV) to detect drug safety signal. Specific criteria used in pharmacovigilance to prove accountability of a drug are rarely present in rare disease. The low number of alerts also makes it challenging. The aim of this commentary is to raise awareness among pharmacists on issues and opportunities for pharmacovigilance in rare diseases, taking pulmonary arterial hypertension (PAH) as example, from which a subset of cases are drug-induced. It is demonstrated how a dedicated program named VIGIAPATH created to reinforce pharmacovigilance of drug-induced pulmonary arterial hypertension at a national level, led to increase self-reporting and confirm safety signals. Thanks to a specific program such as VIGIAPATH, pharmacists can play an important role in communication with clinicians, patients and regulatory agencies, facilitating the detection of potential safety signals at an early stage in rare disease.
KeywordsRare diseases Pulmonary arterial hypertension Adverse drug reaction Pharmacovigilance
The authors thank the French pulmonary hypertension pharmacovigilance network, VIGIAPATH, supported by the Agence Nationale de Sécurité du Médicament et des Produits de Santé (ANSM), the patient association HTaPFrance and acknowledge the contribution of all healthcare providers from the French PAH Network with special thanks to Florence Parent, Gérald Simonneau and all members of the French Reference Center for Pulmonary Hypertension, Hôpital Bicêtre, Assistance Publique Hôpitaux de Paris, Le Kremlin Bicêtre, France.
Development of the VIGIAPATH program was funded by the Agence Nationale de Sécurité du Médicament et des Produits de Santé (ANSM). This commentary is not funded.
Conflicts of interest
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