THE SIGNIFICANCE OF PATIENT-REPORTED OUTCOMES TO FACILITATE MARKET ACCESS DURING A PRODUCT LIFECYCLE

Author(s)

Diana Rofail, MSc, Senior Project Manager1, Linda Abetz, MA, Director1, Rebecca Heelis, MSc, Research Associate1, Chloë Brown, PhD, Director21Mapi Values, Bollington, United Kingdom; 2 Mapi Values France, Lyon, France

OBJECTIVES: To demonstrate the significance of patient-reported outcomes (PROs) in facilitating market access using evidence from deferasirox for iron-overload. METHODS: Reviews of European reimbursement (NICE/French Transparency Commission) and regulatory authority (EMEA/CHMP) guidance and opinions were performed. A PUBMED search was implemented using PRO keywords and iron chelation therapy (ICT). We considered the added value of deferasirox (oral-ICT) demonstrated by PROs at various timepoints of the product lifecycle.  RESULTS: PROs in a product lifecycle can address market access stakeholders’ concerns by demonstrating: 1) disease/treatment burden on patients, and its impact on adherence; 2) clinically meaningful outcomes from clinical trials and benefit to clinical practice; and 3) patient-perceived benefits over current treatments that may increase adherence, potentially reducing healthcare costs. In our example, 28 studies were identified. Medical importance and unmet needs were demonstrated by studies of the burden to iron-overloaded patients receiving infused-ICT. Administration of infused-ICT was time-consuming, inconvenient, negatively impacted patients’ HRQoL, and up to 50% of UK patients had suboptimal treatment adherence, compromising morbidity and mortality.PROs in phase II/III clinical trials comparing the efficacy and safety of deferasirox with infused-ICT revealed that at each timepoint significantly more patients were ‘satisfied or very satisfied’ with deferasirox or found it to be ‘convenient or very convenient’ compared to previously received infused-ICT. Fewer hours were lost from daily activities, 77% preferred deferasirox, and more patients were willing to continue taking deferasirox than infused-ICT (84% vs. 11%). The French Transparency Commission acknowledged moderate improvement in medical benefit for deferasirox versus infused ICT considering improved HRQoL and adherence to ICT. Use of deferasirox is recommended.CONCLUSIONS: PROs can increase key stakeholders’ awareness addressing concerns such as the medical importance of disease and unmet needs, the therapeutic value of products, and the benefits over existing treatments from the patient perspective.

Conference/Value in Health Info

2008-11, ISPOR Europe 2008, Athens, Greece

Value in Health, Vol. 11, No. 6 (November 2008)

Code

PMC44

Topic

Methodological & Statistical Research

Topic Subcategory

PRO & Related Methods

Disease

Multiple Diseases

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