While most large pharmaceutical companies have now integrated patient-reported outcomes (PROs) into their drug-development planning, they should be thinking about what is most relevant to patients at the overall strategic level as well as in specific product-development projects, a new report suggests.
By doing so, and designing clinical trials with these objectives in mind, industry will be better equipped to meet the needs not only of patients but of drug regulators and reimbursement authorities says the report by business intelligence provider GBI Research.
As GBI Research points out, regulators increasingly see PROs as “valuable assets” and recognise that patients should be incorporated into the regulatory pathway at an early stage to help define an acceptable balance of risks and benefits in particular disease areas.
Both the US Food and Drug Administration and the European Medicines Agency are “working to establish processes to support this”, while reimbursement authorities are “looking to patients to help them define truly valuable new medicines”, GBI Research comments.
One challenge to pharmaceutical companies seeking to measure PROs, it notes, is the need for “a great deal of upfront planning, particularly in cases where a new appropriate measurement instrument must be developed and validated as part of the drug development programme”.
There are also obstacles to successful partnership between industry and patients that will generate meaningful and useful PROs.
According to the EU Patient Partnership Project (PatientPartner), which promotes the role of patient organisations within the context of clinical trials, patient groups may lack expertise in, and awareness of, clinical research as well as the necessary organisational structure, the report observes.
Patient groups may also operate locally, which complicates relationships with pharmaceutical companies operating on a global scale.
Loss of control
For their part, pharmaceutical companies may be worried about potential negative publicity, delays in clinical trial processes or loss of control when proprietary information is shared. They may put up cultural barriers to patient involvement or not see any added value to it, accustomed as they are to the biomedical tradition of interacting with clinicians and scientists.
There are, however, existing examples of greater patient involvement in setting research agendas, such as the UK-based James Lind Alliance. This non-profit making initiative, established in 2004, brings together patients, carers and clinicians who share an interest in a particular health problem to identify ‘uncertainties’ or unanswered questions about the effects of treatments.
Pharmaceutical company strategies may not address the questions that are most relevant to patients or they may formulate research questions in a way that does not provide meaningful outcomes for patients, the GBI Research report notes.
For example, most drugs for Parkinson’s disease are targeted at movement disorders, while patients may be more concerned about pain, sleep problems, lack of bowel and bladder control or sexual dysfunction.
The foundations for the involvement of patients in drug regulation were laid in the 1980s and 1990s, particularly by AIDS advocacy groups.
In the US, activists from the AIDS Coalition to Unleash Power (ACT UP) protested outside the FDA, arguing that patient perspectives should be integrated into clinical study designs. Ultimately, the report adds, these groups managed to bring patient representatives into the FDA process for reviewing new drug applications.