Patient engagement in preclinical research is a new phenomenon. Dr David Feldman, National Kidney Foundation, says a forthcoming PFMD How-To Guide will help connect researchers and patients in the earliest stages of medicines development

Meaningful patient input in drug development is still in its infancy. Medicines regulators, including the FDA and EMA, began systematically engaging patients less than a decade ago. Much of these conversations have centred on clinical trials, with patients offering valuable insights on protocol design and desired patient outcomes.

Involving patients in the preclinical phases of research is new – and largely uncharted – territory for academics, industry and patients. ‘The success of patient engagement in clinical trials is still relatively recent but has become more accepted in the halls of government in the US and EU,’ explains Dr David Feldman, National Kidney Foundation. ‘Now the challenge is to convince researchers and patient representatives of the possible benefits of working together on preclinical research.’

To achieve this, Dr Feldman is contributing to the PFMD Working Group developing a How-To Module on Early Discovery and Preclinical Research. The guide will provide the practical tools that academics, biotech and pharma researchers need to engage with patients from the beginning of the discovery process.

It’s important but challenging work. ‘Patient engagement in the preclinical phase is untested compared to the experience researchers have built up in the clinical phases,’ Dr Feldman says. ‘Patient organisations may also not appreciate the importance of getting involved at such an early stage because there may be limited understanding of preclinical research. But the fundamental reason for working together is the same as it is for later stage of development: we want drugs that address what matters to patients.’

Step-by-step guide

The document sets out how researchers can identify suitable patients to work with using a survey. Patient advocacy organisations and their databases of patients can be a key contact in this initial phase. Once the research organisation and patients agree to cooperate, they can work together to define the criteria for patient participation and establish a dialogue on the research goals.

‘At this point, patients learn what the research is all about while the researchers begin to understand patient preferences – for example, do they prefer open-ended questions or specific questions,’ Dr Feldman says.

The next step would involve a webinar where the research sponsor gives an overview of clinical research and patients have an opportunity to learn fundamental principles of early-stage investigations – ranging from the difference between in vitro and in vivo testing to animal models and toxicology.

This is followed by a more in-depth exchange of experiences during a visit to the academic or industry site where the researchers are based. Patients have a chance to see science in action as well as learn more about the specific experiments the researchers are working on. An afternoon session would focus on patient input – including testimonies about their experience and the daily burden of disease.  

‘At this point, the patients and researchers are building a bond which can be the foundation for long-term interaction,’ says Dr Feldman. ‘They can discuss the challenges of treating the disease, patient experiences of medication, aspirations for new therapies – patients learn a lot about drug discovery, and researchers gain information about the target disease that’s not available in the medical literature or in textbooks.’

The process can help to shape how scientists think about their work and adds fresh urgency to the preclinical phase of drug development: ‘These exchanges motivate researchers to ramp up their efforts – it can be a truly eye-opening experience.’

While the document itself is still in development, its publication will mark an important milestone on the way to involving patients right across the drug development lifecycle. It will be one of seven such documents to be published by PFMD, with input from 83 experts from 50 organisations, designed to help patient engagement go mainstream. If successful, it would inspire preclinical researchers and patients to work together, and help them to navigate new territory together.