Pharmacovigilance in a rare disease: example of the VIGIAPATH program in pulmonary arterial hypertension
- 20 Downloads
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
- 1.European Medicines Agency. Guideline on good pharmacovigilance practices (GVP) Module IX—Signal management [Internet]. EMA; 2012 Jun. http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/document_listing/document_listing_000345.jsp#section2.
- 4.Galiè N, Humbert M, Vachiery J-L, Gibbs S, Lang I, Torbicki A, et al. 2015 ESC/ERS Guidelines for the diagnosis and treatment of pulmonary hypertension: the Joint Task Force for the Diagnosis and Treatment of Pulmonary Hypertension of the European Society of Cardiology (ESC) and the European Respiratory Society (ERS): Endorsed by: Association for European Paediatric and Congenital Cardiology (AEPC), International Society for Heart and Lung Transplantation (ISHLT). Eur Respir J. 2015;46:903–75.CrossRefPubMedGoogle Scholar
- 20.Belge C, Quarck R, Delcroix M. Letter by Belge et al Regarding Article, “Mitomycin-induced pulmonary veno-occlusive disease: evidence from human disease and animal models.” Circulation. 2016;133:e591.Google Scholar
- 21.Savale L, Chaumais M-C, Dorfmuller P, Humbert M, Montani D. Lung transplantation for mitomycin-induced pulmonary veno-occlusive disease. Presse Med Paris Fr. 1983;2017(46):1223–5.Google Scholar