Currently, regulators make marketing authorisation decisions based on the benefit-risk balance of a treatment with little input from patients and the public. The aims of this thesis are to apply systematic methods of benefit-risk assessment proposed for use in regulatory decision-making, and examine how they can incorporate patient and public involvement. These aims were met through the investigation of three case studies: efalizumab, rimonabant, and natalizumab. Efalizumab (indication: plaque psoriasis) was withdrawn from the market due to the occurrence of progressive multifocal leukoencephalopathy. I examined the feasibility of applying descriptive and quantitative frameworks to determine the benefit-risk balance of efalizumab, and evaluated where patient and public involvement can be applied in the decision-making process. Rimonabant (indication: overweight and obesity) was withdrawn from the market due to the occurrence of psychiatric disorders. I conducted a discrete choice experiment to elicit patient and public preferences regarding the benefits and risks of treatment, and developed a probabilistic analysis method to analyse the preference data and determine the benefit-risk balance of rimonabant. Natalizumab (indication: relapsing remitting multiple sclerosis) received authorisation, but the benefit-risk balance was reassessed due to the occurrence of progressive multifocal leukoencephalopathy. Authorisation was maintained with risk minimisation measures. I developed a framework to guide the application, reporting, and evaluation of patient and public involvement in benefit-risk assessment. I then tested the feasibility of the framework with protocols designed to elicit patient and public preferences on the benefits and risks of natalizumab. Based on the findings of these case studies, preferences can be elicited from patients and the public and used to determine the benefit-risk balance of a medicine. This thesis provides a methodological foundation to guide the application, reporting and evaluation of PPI in the benefit-risk assessment of medicines and improve the legitimacy, transparency, and quality of regulatory decision-making.
| Identifer | oai:union.ndltd.org:bl.uk/oai:ethos.bl.uk:656505 |
| Date | January 2013 |
| Creators | Hockley, Kimberley |
| Contributors | Ashby, Deborah; Smith, Peter |
| Publisher | Imperial College London |
| Source Sets | Ethos UK |
| Detected Language | English |
| Type | Electronic Thesis or Dissertation |
| Source | http://hdl.handle.net/10044/1/24744 |
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