AN ANALYSIS OF HEALTHCARE UTILIZATION AND COSTS ASSOCIATED WITH PATIENTS WITH ACUTE HEPATIC PORPHYRIAS (AHPS) WITH RECURRENT ATTACKS IN EXPLORE- A PROSPECTIVE, MULTINATIONAL NATURAL HISTORY STUDY OF PATIENTS WITH AHP

Author(s)

Gouya L¹, Bloomer JR², Balwani M³, Bissell DM⁴, Rees DC⁵, Stölzel U⁶, Phillips JD⁷, Kauppinen R⁸, Langendonk JG⁹, Desnick R³, Deybach J¹, Bonkovsky HL¹⁰, Parker C⁷, Naik H³, Badminton M¹¹, Stein P⁵, Minder EI¹², Windyga J¹³, Martasek P¹⁴, Cappellini M¹⁵, Ventura P¹⁶, Sardh E¹⁷, Harper P¹⁷, Sandberg S¹⁸, Aarsand A¹⁸, Alegre F¹⁹, Ivanova A²⁰, Talbi N¹, Chan A²¹, Querbes W²¹, Penz C²¹, Agarwal S²¹, Simon A²¹, Anderson KE²²
¹Centre Français des Porphyries, Colombes, France, ²University of Alabama, Birmingham, Birmingham, AL, USA, ³Mt. Sinai Icahn School of Medicine, New York City, NY, USA, ⁴University of California, San Francisco, San Francisco, CA, USA, ⁵King’s College Hospital, London, UK, ⁶Klinikum Chemnitz, Chemnitz, Germany, ⁷University of Utah, Salt Lake City, UT, USA, ⁸University Hospital of Helsinki, Helsinki, Finland, ⁹Erasmus Medical Center, Rotterdam, The Netherlands, ¹⁰Wake Forest University, Winston-Salem, NC, USA, ¹¹University Hospital of Wales, Cardiff, UK, ¹²Stadtspital Triemli, Zentrallabor, Zurich, Switzerland, ¹³Instytut Hematologii i Transfuzjologii, Warsaw, Poland, ¹⁴Univerzity Karlovy v Praze, Prague, Czech Republic, ¹⁵University of Milan, Milan, Italy, ¹⁶Università degli Studi di Modena e Reggio Emilia, Modena, Italy, ¹⁷Karolinska University Hospital, Solna, Sweden, ¹⁸Norwegian Porphyria Centre, Bergen, Norway, ¹⁹Clinica Universidad de Navarra, Navarra, Spain, ²⁰St. Ivan Rilski University Hospital, Sofia, Bulgaria, ²¹Alnylam Pharmaceuticals, Cambridge, MA, USA, ²²University of Texas Medical Branch, Galveston, Galveston, TX, USA

OBJECTIVES : Acute hepatic porphyrias (AHPs) are rare genetic diseases consisting of acute and chronic manifestations caused by mutations in the heme biosynthesis pathway. EXPLORE is the first observational, prospective, ongoing study characterizing clinical management of patients with ≥3 attacks/year or receiving prophylaxis for AHP. The objective of this analysis is to estimate healthcare utilization and costs in this population from the US payer perspective. METHODS : Patients with recurrent attacks or receiving treatment to prevent attacks were enrolled. Patients’ medical history, questionnaires on signs and symptoms, quality of life, and healthcare utilization, were collected at pre-specified intervals and during attacks. Costs were estimated for hospitalizations, emergency room (ER) and physician visits, and hemin use from publicly available US data sources. RESULTS : CONCLUSIONS : AHP is an acute disease with chronic manifestations that has high healthcare utilization and expenditures primarily driven by hospitalizations and hemin use. This is the first study to estimate expenditures associated with AHP and underscores the significant burden to the US healthcare system.

Conference/Value in Health Info

2018-05, ISPOR 2018, Baltimore, MD, USA

Value in Health, Vol. 21, S1 (May 2018)

Code

PHS39

Topic

Economic Evaluation

Topic Subcategory

Cost/Cost of Illness/Resource Use Studies

Disease

Rare and Orphan Diseases

Explore Related HEOR by Topic

Economic Evaluation

Presentation