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Title: Access to orphan medication and quality of life in rare diseases
Authors: Abbas, Amar
Keywords: Orphan drugs -- Malta
Quality of life -- Malta
Rare diseases -- Malta
Issue Date: 2018
Citation: Abbas, A. (2018). Access to orphan medication and quality of life in rare diseases (Doctoral dissertation).
Abstract: Over 7000 rare diseases (RD) affect around 60 million patients living in the European Union (EU) and the United States (US). Research on RDs focuses on treatment and care of RDs with limited focus on health related quality of life and accessibility to Orphan Drugs (ODs). The aims of this study were to 1) analyse and compare regulations and policies related to accessibility of ODs in the EU and US 2) describe OD accessibility and the health needs of the Maltese RD population and 3) create a health related quality of life (HRQOL) assessment tool for RD patients and caregivers. The HRQOL tool explores issues of diagnosis, mental health, use of health and support services and general quality of life. The methodology included (1) assessment of OD accessibility policies in 29 countries based on 6 themes (national OD policies, OD designation, marketing authorisation, marketing exclusivity, incentives and pricing) identified during retrospective analysis of literature (2) identifying issues encountered by Maltese RD patients by (i) interviewing policy makers (ii) conducting literature review (iii) and analysing data from RD registers (3) development, validation (by 7 experts) and administration of HRQOL tool to patients and caregivers (N=225) in the EU (n=137) and the US (n=88). The tool included 30 close ended Likert scale questions divided into four sections collecting information on the demographics, personal care and independence, mental and social health and access to treatment. The results showed that out of the 29 countries assessed, 17 EU countries had OD policies and 6 countries (including the US) had financial incentives for OD development. OD designation, marketing authorisation and market exclusivity were centralised in both the EU and US by the EMA and FDA respectively. Malta had no OD plans in place and RD patients in Malta had poor access to ODs when compared to other EU countries. Statistical analysis of the HRQOL indicated a significant difference (p<0.05) between RD patients in the EU and US. A significant difference was observed when RD patients were asked to report how they felt during everyday activities such as dressing (p=0.001), eating (p<0.001) or participating in everyday activities (p=0.014) with the US RD patients scoring lower in all areas. The financial burden of RDs was significantly higher (p<0.001) in the US as the Likert mean score was 3.00±1.065 while EU RD patients reported 4.44±0.512 on the Likert scale (1 = Financial burden, 5 Not a burden). Poor OD accessibility was reported by both groups with 44 (32%) EU respondents and 27 (31%) US respondents reporting it is ‘almost impossible’ to receive treatments for their RD. EMA has granted 140 ODs with market authorisation compared to 415 approved ODs in the US. There are differences between countries on the degree of accessibility, pricing and reimbursement. Poor HRQOL may be related to issues of accessing medicines, diagnosis, psychosocial support, and coping with stigma and uncertainty. US based RD patients scored poorly when compared with their EU counterparts in relation to mental health, personal care and independence and ability to afford ODs.
Description: PharmD
Appears in Collections:Dissertations - FacM&S - 2018
Dissertations - FacM&SPha - 2018

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