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Volunteers welcome to participate in new research study!

April 2023 update: Many thanks to everyone who has contributed to this study.  We have diverse representation from many communities.  We would welcome hearing from parents with children who have tried to access and / or are receiving an orphan drug to treat their rare disease in NI to ensure their views are incorporated to this important project.

Stakeholders’ perspectives toward obstacles and opportunities associated with accessing Orphan Drugs in Northern Ireland - A Qualitative Study

Invitation Posts to participate in the study

Rare diseases affect approximately 6% of the worldwide population, which is >110,000 people across Northern Ireland at some point in their lives.  Orphan drugs are available to treat approximately 5% of rare diseases but patients and their families often report challenges accessing appropriate treatment options, while pharmaceutical companies anecdotally describe issues bringing clinical trials to NI.

The research team - (Prof Amy Jayne McKnight, Dr. Lynne Lohfeld, Ghada Abozaid and Ashleen Crowe) - in the Centre for Public Health at Queen’s University Belfast are conducting a project to explore stakeholder perspectives of obstacles and opportunities associated with accessing orphan drugs to help treat rare diseases in Northern Ireland.

Ghada Abozaid and Ashleen Crowe are PhD students conducting a qualitative study interviewing people in NI aiming to increase orphan drugs accessibility by exploring barriers and enablers associated with the availability and accessibility of orphan drugs in Northern Ireland. 

We welcome hearing stories from people who live in NI with relevant experience about orphan drugs for rare diseases. Your knowledge and experience can contribute greatly to identifying obstacles to accessing orphan drugs in NI and help identify how we may overcome them. The ultimate goal is to work together to help people diagnosed with a rare disease to have a better quality of life for as long as possible. 

A participant information sheet can be accessed at this link:Rare Disease and Orphan Drugs_Participant Information Sheet

It provides further details on what the study is aiming to do and what participants will be asked to do, should they decide to participate. People living with a rare disease or family caregivers keen to help identify what works & barriers to accessing orphan drugs in Northern Ireland, will receive a £100 voucher after participating in an interview (face-to- face, Microsoft Teams, or phone call) that will last approximately one hour, provided they have direct experience of accessing or trying to access orphan drugs for a rare disease in Northern Ireland and meet the inclusion criteria.

This project is supervised by Prof Amy Jayne McKnight, Dr. Lynne Lohfeld, and Dr. Hussain Al-Omar.  For more information, please contact us, Email:



Photo: AJ McKnight
AJ McKnight
AJ McKnight, Centre for Public Health
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Please do contact our rare disease team by email for further information.