Patient preferences in early drug development

If a pharma company aims to meet the needs of patients, patient preferences should be included at the very beginning: From phase 1/early development, through to the post-marketing phase of any medical product’s life cycle. A recent paper from Novartis, one of the IMI PREFER project partners, argues for aligning stakeholders in early drug development to ensure development plans best meet patient needs.

According to the authors, companies that embrace patient involvement early, before finalising the design of the critical clinical trials for any drug or treatment, are more likely to have their medical products meet the real needs of patients, and thereby be able to provide the therapeutic outcomes that the patients expect. Engaging patients early on can also ease the burden for patients in the later stages of drug development.

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Partnering with patients and other key stakeholders is said to help align drug development with the needs of patients in a more structured and scientific way. Relevant players include the pharmaceutical industry, regulators, the authorities that assess health technologies and pave the way for reimbursement of costs, organisations and authorities that develop guidelines, doctors, nurses and other clinicians, to the healthcare systems themselves. The COPD study is taking this approach and has already received scientific advice on the study design from the United Kingdom’s Institute for Health and Care Excellence (NICE).  

One of Novartis’ contributions to the Innovative Medicines Initiative (IMI) project PREFER is a preference study with Chronic Obstructive Pulmonary Disease (COPD) patients. Nigel Cook, Head of Decision Support, Global Patient Access at Novartis, is one of the authors. According to him, “patient-based evidence and patient preference information can inform interactions between drug developers and HTA or reimbursement bodies. The first examples of early patient engagement having this benefit are emerging”.  

Evidence that is generated throughout clinical development should demonstrate improved outcomes on endpoints (a result that can be measured, like symptom relief) that matter to patients. According to the authors, we need to align stakeholders around these patient-relevant endpoints, and this is best done through generating robust patient-based evidence including quantitative patient preference studies that are structured, evidence-driven, and conducted in collaboration with patients/patient groups. By so doing, the authors advocate that it is possible to focus on outcomes that are relevant for patients and deliver new treatment options that will best meet their needs.

To strengthen and encourage companies to take decisions in their development process that are driven by the interest of patients, we need scientific rigour, robust and early patient research. According to Nigel Cook and co-authors, this means asking enough patients through patient preference surveys (what is known as quantitative research). And to make sure patients are asked the right questions, these surveys should be based on work that asks more open questions to explore what is important to patients (qualitative insights).

The authors propose the need for a more systematic and transparent process for generating patient preference information, throughout the life cycle of a medical product. Companies that embrace this approach and work in partnership with patients and other key stakeholders, can also improve their processes when there is a need. And, which is the goal for all stakeholders: provide solutions for health care that really help patients.

Read article in Frontiers in Medicine: Cook NS, Cave J & Holtforf A-P, Patient Preference Studies During Early Drug Development: Aligning Stakeholders to Ensure Development Plans Meet Patient Needs, published online 24 April 2019

About the Novartis COPD study

One of Novartis’ contributions to PREFER is a preference study with Chronic Obstructive Pulmonary Disease (COPD) patients. A goal of this project is to understand how burdensome patients find different COPD symptoms. The results will influence the design of future clinical trials for COPD. This project very much embraces the principles of engagement with patients early in the development process. It uses  a structured approach that includes qualitative insights and validation through a quantitative patient preference study, with alignment of all key stakeholders around the study objectives.

This study is the first patient preference study ever to receive scientific advice on the study design from the British National Institute for Health and Care Excellence (NICE) 

Anna Holm & Josepine Fernow






  • Disclaimer: This website and its contents reflects the PREFER project's view and not the view of IMI, the European Union or EFPIA.


The Patient Preferences in Benefit-Risk Assessments during the Drug Life Cycle (PREFER) project has received funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement No 115966. This Joint Undertaking receives support from the European Union's Horizon 2020 research and innovation programme and the European Federation of Pharmaceutical Industries and Associations (EFPIA).