Widening patient engagement for orphan drugs trials

Background
Orphan drugs are medicines intended for diagnosis, prevention or treatment of life-threatening or progressive disorders that are rare. A rare (or 'orphan') disease affects less than 1 in 2,000 people within the general population. An example is Idiopathic Pulmonary Fibrosis (IPF), a life shortening lung disease that gets worse over time making it hard for the person who has it to breathe.

The pharmaceutical industry has recently highlighted the importance of the development of orphan drugs for both medical and financial reasons. The number of rare diseases increases each year as new sub-types of disease are identified, as does the amount of money spent on creating new medicines to treat them.

The UK is a European leader in the early stages of drug development, but falls behind other counties in the later phases which assess the safety of a drug or how it might compare with other treatments. This matters if the UK wants to become a world-leader in drug development. In response to Brexit and the COVID-19 pandemic, the UK Strategy for Rare Diseases and the Association of the British Pharmaceutical Industry have both called for the inclusion of a wider range of patients in clinical drug trials, but research suggests that the needs and experiences of patients who take part and decline or withdraw from orphan drug trials are often overlooked.

What we know about patient experience of orphan drugs is based on survey responses from patients who have had a positive experience of trial participation and/or treatment, doctors who provide these treatments, and patient and public representatives who have played a role in drug/trial development. Survey responses are largely descriptive, and lack in-depth, rich detail needed to inform improvement or innovation.

There are particular gaps in knowledge about the experience of patients in clinical trials of orphan drugs who do not receive the active treatment (placebo or comparator groups), and those who drop out of a trial.

What will we do?
An experienced qualitative researcher will be seconded to a European-wide pharmaceutical company (Galapagos) to facilitate knowledge exchange between the biomedical industry and academia, to identify opportunities for deep learning for the UK pharmaceutical industry and patients with orphan conditions.

We aim to:
- Learn more about orphan drug development and in-depth patient/families experience of IPF.
- Embed qualitative research (e.g. in-depth interviews and focus groups) in pharmaceutical practice to enable a wider group of patients/families to have the opportunity to be involved in orphan drug research.
- Expand orphan drug engagement to widen their reach and benefit, improve clinical trials and medicine use, to benefit both people with orphan conditions and the pharmaceutical industry.

This will be possible through the following research activities:
- In-depth interviews with IPF patients, their healthcare professionals, and people from organisations that recruit patients to drug trials.
- Interviews with staff and observations of Galapagos' values and practice.
- Training Galapagos staff to conduct interviews and focus groups with patients, and assess their effectiveness.
- Patient and Public Involvement (PPI) representatives will regularly review and input into the research findings and co-design accessible resources for patients, and third party organisations that recruit patients to orphan drug trials.

This information will be used to produce a comprehensive understanding of the Galapagos' transformation planning for widening patient engagement. Training and guidance for using qualitative methods to embed patient engagement for knowledge exchange in host's activities will be developed and assessed. Final outputs will address the ABPI objectives and include patient-facing resources for recruitment to trials of orphan drugs, seminars for scientists and academics, and peer reviewed publications.

Aim:
To improve clinical trials and medicine use, to benefit both people with orphan conditions and the pharmaceutical industry.

Objectives:
1) To generate theoretically informed and empirically grounded knowledge to inform wider patient engagement in orphan drug development.
2) To embed the use of qualitative research methods in pharmaceutical developments and practice, to enable a wider group of patients to participate and inform orphan drug research.
3) To synthesise understanding from objectives 1 and 2 to enable the redefinition, expansion and reconceptualising of orphan drug engagement to widen their reach and benefit.

Research questions:
1) How do patients and other members of the public experience engaging with the process of orphan drug development, and how can engagement be optimised?
2) What is the promise of orphan drugs for patients and where are the gaps in understanding patients' experience and perspectives in drug development?
3) What would a broader definition of patient engagement entail and how can it best be embedded in the practice of orphan drug development to maximise productivity?

Methods: I will:
1) Convene a patient advisory group to provide insights throughout the project.
2) Conduct semi-structured interviews with 30 patients with IPF; ten health professionals who support them; and ten members of organisations that recruit patients to trials.
3) Conduct nonparticipant observation of host organisations' staff practice (n=15) and semi-structured interviews with key staff (n=10). Support host organisation to trial and evaluate the use of qualitative methods to embed patient engagement into industry organisational practices.

Outputs:
1) Draft guidance for embedding patient engagement for knowledge exchange in industry activities.
2) Ethnography of host's transformation planning for widening patient engagement.
3) Co-designed resources to recruit patients to orphan drug trials, seminar series and publications.