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Patient Input and Patient Preference Studies


Patient preferences are increasingly seen as valuable in healthcare policy decision-making, but such studies are not always easy to design and implement, writes Nick Hicks.

Nick Hicks

Gaining regulatory approval and reimbursement of new treatments in many therapeutic areas such as rare diseases is not easy and reflects in part the lack of demonstration of improvement in meaningful health outcomes for patients.The U.S. Institute for Clinical and Economic Review (ICER) value framework states the problem so clearly “all too often what matters most to patients is poorly captured in the available clinical trial data”.

Patient preferences are increasingly seen as valuable in  healthcare policy decision-making.3 Such studies fill an important gap in a company’s access strategy as they can compensate for the lack of real-world evidence at the time of launch and can show the treatment impact on patients not possible in a standard phase 3.4 But such studies are not always easy to design and implement.3 Such studies are useful for example when there is uncertainty in preference sensitive areas decisions such as when (i) there is no superiority between different treatment options, (ii) ambiguity of evidence supporting one particular treatment, and (iii) variation in the way patients view risk benefit or how they differ from that of HCPs. 5

Patient advocacy and engagement considerations

1. The long-term view and a collective understanding

Working with patients at such an early stage shows the need for the long-term view; moving beyond product accessibility, to identifying and addressing patient unmet needs requires creating and maintaining a true, strong institutional partnership. Creating a trust based partnership based on addressing common shared interests takes time. The pharma company involved already has a strong track record in the area of respiratory medicine so it’s likely that such a strong partnership approach already existed. Without such a philosophy the willingness for cooperation between the stakeholders to work together on such a venture would likely be a lot lower. This shows an organizational culture with strong emotional intelligence that puts the patient at its core with a strategy that reflects the increasingly pivotal  role of patient engagement in R&D, rather than the tokenistic approach of discussing a trial protocol over a cup of coffee with patient representatives every six months.   

Though not having inside knowledge of the initiative, it’s very likely that the different stakeholders involved (company, patient group, NICE, and external experts with their respective skills, insights, and backgrounds) developed a strong collective understanding of the patient needs. Allowing the solution being developed to be fitted into the present day and likely future access environments.

Explaining to the patient group representatives, for example, some of the specific nuances of patient preference  studies, such as the importance of accepting higher or lower risk, are made easier when a solid foundation already exists both in terms of scientific understanding and institutional relationship. The patient is seen as an equal partner and such a full engagement culture comes from the top and embeds itself at all levels of the organization.

2. Adapting compliance structures

Patient engagement in early R&D means that companies have to adapt existing compliance structures so they become more flexible and accommodating of the different circumstances surrounding patient involvement. Traditional compliance structures are sales and marketing focused, which is not the case in situations where treatments do not yet exist. The internal block points between the different internal teams being identified at an early stage allowing appropriate solutions to be made in a timely fashion. Company resource to design such specific guidelines and the synergies between internal teams becoming more important for smaller organizations.

3. Patients and patient groups becoming credible research partners

COPD is associated with a very high burden of disease with high unmet patient needs. Patient advocacy groups were traditionally associated with lobbying and disease awareness to communicate on these unmet needs; their ability to contribute on a more scientific level often questioned. But opportunities to understand scientific disciplines and rigor is increasing, no doubt in  some way to their active participation in initiatives such as EUPATI, IMI, and PARADIGM. This patient preference study supports the important role of patients as credible research partners when their contribution is made in a scientifically robust way. 6

Such a willingness to move away from the traditional roles to participation in accelerated drug development will no doubt increase as patient involvement in these and other training initiatives continue. But limited capacity building resource is the brake for most patient groups becoming involved in R&D. Newly formed groups or those where there has been little historical pharma engagement are most likely to struggle or be reluctant to participate in patient preference studies, for example, at the expense of the more traditional, immediately pressing roles. 

Relevance for all HTA Patient Pathways

In conjunction with Phase III trials, patient preference studies offer a scientifically rigorous approach for patient outcomes research and can guide a pharma pipeline strategy, the involvement of patient group input no doubt strengthening the credibility of later stage HTA submissions. This study was UK-driven, traditionally recognized as the center of patient engagement, but an article published in Pharm Exec in December 2018 questioned if patient advocacy/engagement in pharma was “Brexit-ready”.7  Would the value of such Patient involvement be treated the same within the HAS, TLV or GB-A pathways, for example? Would such long-term patient initiatives even be considered relevant in a non UK pathway setting? Or would the NICE backed patient preference study still be viable as a component of a European data package? 

Some European patient groups are changing their constitutions to allow UK groups ongoing participation and pass on their experiences to European colleagues, but it’s still early days. Perhaps an important factor will be to see how such patient engagement initiatives are covered in the PARADIGM initiative in the coming months. But whatever the Brexit pathway, patient preference studies will become more common.

Nick Hicks is an advocacy and patient engagement consultant for the life science industries.


1.     https://www.nice.org.uk/news/article/nice-provides-first-scientific-advice-on-Patient-preference-study-design

2.     https://ojrd.biomedcentral.com/articles/10.1186/s13023-017-0718-x Measuring what matters to rare disease patients – reflections on the work by the IRDiRC taskforce on patient-centered outcome measures

3.     https://implementationscience.biomedcentral.com/track/pdf/10.1186/1748-5908-8-64   Integrating evidence on patient preferences in healthcare policy decisions: protocol of the patient-VIP

4.     https://core.ac.uk/download/pdf/46172631.pdf  Nicod, Elena (2016) Scientific and social value judgments for orphan drugs in HTA. International Journal of Technology Assessment in Health Care  

5.     https://www.fda.gov/downloads/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/UCM446680.pdf

6.     https://www.cambridge.org/core/journals/international-journal-of-technology-assessment-in-health-care/article/gripp-checklist-strengthening-the-quality-of-patient-and-public-involvement-reporting-in-research/0F12FE8D0A37B8E44AEA31099E63DB44 The GRIPP checklist: Strengthening the quality of patient and public involvement reporting in research

7.     http://www.pharmexec.com/Patient-engagement-brexit-ready



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