Working towards drug approval - How about asking the patient what they want?

Angela Rylands, CPsychol, Patient Centred Outcomes Consultant at pH Associates explores the use of patient preference in drug development ...

In drug development, from the get go, sponsors focus on how they will ultimately get their products approved. Whilst there are many obstacles to overcome along the way, something that they really need to do is make sure they go out and actually ask the patients what it is that they want for their treatment and what it is about their product that is of value to patients. In other words, what are the patient preferences for this product?

Of course, sponsors know that patients have unique perspectives on the value of potential benefits and the impact of potential harms and burdens of their medical treatments, but HOW do sponsors go about incorporating the patients’ thoughts on the value of the product into or outside of their pivotal trials? WHO ultimately is interested in this type of information in the approval process of products?

HOW to collect patient preference data?

Reliable and accurate methods are needed in order to effectively incorporate patients’ values into decision-making processes; this is often referred to as ‘patient preference’ information. This information can either be collected as ‘qualitative’ data, i.e. from interviews with patients to provide rich information about the value of a product, or ‘quantitative’ data, which is the quantification of the desirability or acceptability of outcomes or other attributes of medical products, focusing particularly on the trade-offs that a patient might make.

WHO cares about patient preference in drug approval?

The United States Food and Drug Administration (FDA) currently has a ‘Patient Preference Initiative’ and recently held a workshop (in December 2017) demonstrating their commitment to incorporating patient preference studies into product development. The aim of the workshop was to combine industry and patient experts to develop clearer guidelines for using patient preference study data. 

There are also the adopted 2016 guidelines from the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use which global regulators can refer to; this has a brief mention of patient preference data within the broader context of benefit-risk. The recommendation here is to use descriptive information on patient attitudes and preferences with respect to the therapeutic context, benefits and/or risks.

Ultimately it appears that regulators value information about patient preference that is obtained directly from patients or indirectly from other stakeholders using qualitative, quantitative or descriptive methods.   The status of the guidelines available, however, is less well defined.

So, what is the current status for using patient preference data?

Presently, whilst the FDA has limited expertise in working with patient preference research, they are purportedly addressing this by expanding the responsibilities of their Clinical Outcomes Assessment staff (who review PROs) to include patient preference. 

Indeed, last year, we saw the remarkable inclusion of patient preference information on a drug label, approved by the FDA, for Genentech’s Rituxan Hycela subcutaneous rituximab for patients with lymphoma.  This approval was based on data from Genentech’s global randomized, open-label, crossover (PrefMab) study, where preference for the subcutaneous verses intravenous administration of the product was evaluated according to data from the Patient Preference Questionnaire (PPQ) and from patient satisfaction and convenience data assessed using validated satisfaction questionnaires. The sponsors were able to show that 81% of patients, on their trial, completing the PPQ preferred subcutaneous administration of rituximab. 

This is an example of using patient preference questionnaires in the global trial, which can be seen as a tremendous breakthrough in the patient centred outcomes research space. Not only does it demonstrate an innovative way of incorporating the patient voice into the trial; the findings were then used to obtain a patient centric label for the product.  Whilst this work demonstrates a very positive step in the right direction for developing drugs that are of value to patients, it is important to be mindful that the unique crossover design lent itself to the possibility of collecting this type of data and as such careful thought would be required in the design of clinical trials measuring patient preference.

In the UK, the National Institute for Health and Care Excellence (NICE), whose technology appraisal board considers new drug applications, recently demonstrated their commitment to patient preference data by funding a study to explore how patient preferences should be captured and included in Health Technology Assessment (HTA) submissions.  NICE awarded a £120,000 grant to Myeloma UK to undertake a two year methodological exploratory study. The aim of this study is to evaluate best practice in the use of different methods and technologies for capturing information about patient preferences relating to their condition and the treatments they receive for it. 

In summary, the understanding of how to incorporate patient preference research into the drug development process and to ultimately use the results as part of the approval process is in its infancy and there are no current standards for study design, conduct and analysis, along with limited regulators with experience interpreting and understanding how to incorporate the resulting data.  However, herein lies an opportunity to step up and use existing methodologies of collecting patient preference data to truly bring  patient voice into the development of treatments and look towards innovative approaches to measuring this in or outside of the global trial.

At, pH Associates, a specialist Real World consultancy, their Patient Centred Outcomes are regularly receiving requests for expertise around collecting data on patient preference.  This data can be most easily collected from patients in the real world. pH can help you find ways to collect this type of data.  Please contact Angela Rylands if you would like to know more.