Interesting research, articles and other data about patient involvement in R&D

IMI Survey “Patient involvement in IMI projects”

In August 2013, the Innovative Medicines Initiative ( conducted a survey amongst all IMI projects on “Patient involvement in IMI projects”. The results provide interesting learnings on patient (advocacy group) involvement in R&D projects.

The most common reason for not involving patients in research was that this was not envisaged in the project scope, while only a few respondents indicated there was no clear benefit to involving patients. The majority of projects not currently involving patients also did not plan to do so in the future.

Diverse reasons were given in response to the benefits of involving patients, mostly relating to the perspectives patients can provide on which research objectives are important to patients; how to disseminate results outside the scientific community; how patients perceive particular risks and benefits; and empowering patients to build networks and learn more about their condition. The challenges reported are too numerous to accurately reproduce here, but among other things included the burden (financial, time, energy) on patients; handling logistical and ethical requirements associated with data confidentiality, ethical approval and legal considerations especially in an international contexts; overcoming barriers of language and technical terms; and ensuring representativeness when including a relatively small sample of patients to represent the views of all others.

From the concerns raised among participants in this survey, a number of recommendations can be made to facilitate future patient involvement:

  • Support involvement of patients in the early stages (inception phase, consortium building, scoping) to ensure patient participation is an integrated part of the project with adequate funding
  • Map out for different types of projects where patient input is most appropriate and beneficial ?C for example, in clinical trials or biomarker validation studies with rigid protocols, patients do not have much opportunity to provide input (except as “subjects”) once the study protocol has been defined.
  • Provide training for researchers to understand the potential benefits of involving patients in their work and in which areas this can occur. This may be particularly valuable for projects that currently see patients only as “subjects” either as participants in trials or as providing sample material.
  • Ensure the financial and accounting barriers to patient participation are limited, including understanding the limitations patient organisations face with regard to eligibility of expenses (VAT, ad hoc staff/consultants etc.)
  • Ensure that non-patient project partners are aware of the potential special needs of patients, including limitations in travelling (when organising meetings)
  • In areas where expertise among patients is lacking, provide training and support to patients to adequately equip them to contribute meaningfully to research projects.

Full report here:

Literature and References

“Patient-Centeredness in the Design of Clinical Trials”, Mullins et al (June 2014)

VALUE IN HEALTH 17 (2014) 471-47, doi:10.1016/j.jval.2014.02.012

“Patient-Centeredness in the Design of Clinical Trials”, also calling for more patient centeredness in trial design and also more pragmatism, bayesian statistics, adaptive designs etc. I am forwarding this to you as it might be a nice reference both for the white paper and the charter.

As the Orphanet Newsletter summarized it so nicely: “The authors of an article published in Value for Health regret that classical clinical trials generally do not represent patient interests and rarely inform participants of results during the study. Under such conditions, patients often lose interest and drop out of trials. Experience shows that participants’ active involvement in the design and progression of clinical trials results in greater patient retention and more meaningful results. Informed patients are more willing to engage in time-consuming and effort-requiring studies as they feel valued, empowered and capable of assessing therapeutic options.

Mullins et al. suggest methods of designing trials from a patient’s point of view rather than the investigator’s. Pragmatic, Bayesian statistics and adaptive trials can improve patient safety and increase recruitment and retention. Pragmatic trials are designed to result in outcomes that are most relevant and beneficial to participants. Bayesian statistics draw on collected evidence to update knowledge on the treatment and outcome probabilities. This approach aims to produce results that are easily interpreted and reflect the reality of medical decision making. Adaptive trials also evolve on the basis of data accumulated during the trial and allow patients to be transferred to a more effective treatment if it becomes available once the trial has begun.

Mullins et al. emphasise the primary need for sustained efforts to inform and involve patients and advocates at all stages of clinical studies.”

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