Results
For more detailed results please download the full report here.
Current experience of Pharma with patient involvement
Involving patient organisations in recruitment
Almost half of the respondents reports having involved PO for recruitment. Mostly by asking patient organisations to forward to their members information on the opportunities to take part in a clinical trial or by publishing an article on the product in a patient organisation magazine. This was mostly for phase II and III trials. Respondents were generally more negative about patient organisations suggesting members to their company that have been found eligible to participate by their clinician. Only 3 would consider and 2 report to have done this.
Two respondents report to inform PO on website : www.clinicaltrials.gov
One respondent who had experience in involving PO for recruitment reports: "It has sped up the enrolment of the trials and the patient organizations helped in communicating the value of the trials to the patient community. Our company has been in regular contact with some international, European as well as some national patient organizations. Confidentiality agreements were generally signed before the sharing of information."
Another writes: "We worked with a patient group via an established relationship to signpost the details of a phase III trial in Non Small Cell Lung Cancer on their website. This has worked well for us with over 900 viewings of this information within a 6 week timeframe meaning that patients and carers had the opportunity of understanding about a potential treatment option in a therapy area with relatively few options."
The ones who report to have no experience with involving PO for recruitment say they "haven’t had the opportunity" or leave recruitment up to the principal investigator or physician. Some also note that Po have been very resistant to being involved in recruitment because they are anxious it could fall into advertisement.
Suggestions that were made towards PO involvement for recruitment are:
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Well defined criteria for acceptable advertisement.
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Change of legislation to allow national trial sites and content to be published.
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Having a European regional office.
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Establishment of better contacts and finding out a way PO o not feel compromised.
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List of contacted persons of interested patient organizations.
Involving patient organisations as Information providers in clinical trials
Only 4 from the 20 respondents reports having involved PO for information. But respondents are more positive about the possibility to involve PO for info then for recruitment in the future; 8 state they would consider involving them (vs. 5 for recruitment).
The ones that had involved PO were positive:
"It was a very positive experience. Very valuable input provided." "excellent experience. Good insight was provided allowing to improve on protocols and informed consent forms. We have regular Advisory Boards with ECAB (EATG) that provide feedback on our development program. We provide protocols and informed consent forms to EATG as off Phase IIb for input and suggestions. This is done through a standard operating process."
Most respondents who did not involve PO said they had in house expertise or that it wasn’t required. Other reported not having the opportunity or never been approached by PO.
Suggestions for future:
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Ways of disclosing confidential information
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Company:
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positive suggestion of the investigator,
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Internal strategy discussions and decisions
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Patient organizations:
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literacy in medical science
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Administrative back-office to organise Ad Boards and review processes.
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Involving patient organisations as Advisors in clinical trials
5 from the 19 pharma reported that they involved Po for advising on suitability of material provided, the procedure of explaining the information material to the patients, ethical and safety aspects or other aspects in a clinical trial. This was mostly done in phase II and III trials. Over half of the respondents would not consider involving PO as advisors in a clinical trial.
Three respondents note positive experiences:
"It was a very positive experience. Very valuable input provided via 2 channels: sometimes directly to us or via the investigators. Note: At times for specific cases we have received advice from patient organizations on the suitability of the information material provided to patients but not under a formal process and not consistently for all of our clinical trials."
Another: "We have had contact on a local level with professional patient organisations. A certain level of expertise and professionalism is required to have this kind of input. Input can be very helpful." "HIV patient groups routinely review and feedback on protocol design, procedures and informed consent documents."
1 respondent who has experience had a negative experience: "The time taken was very slow and mostly prohibitive to our processes. Trials have very tight timelines on them."
The ones who hadn’t involved PO:
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not required/common practice/use internal resources
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tightly controlled by regulation/medical ethics committee
Involving patient organisations as Reviewers in clinical trials
Very few respondents had experience with involving Po as reviewers and most respondents did not see a possibility in the future for PO to get involved in reviewing. The ones that had involved PO only had involved them reviewing a clinical trial research protocol, but not of a report or scientific paper.
One respondent has long-term relation with HIV groups: "We have established a good working relationship with HIV groups whereby we routinely share clinical trial documentation and procedures to obtain expert input. Given our longstanding partnerships and patient-centric approach frequently the groups are satisfied with the protocol design with limited revisions needed."
Ones respondent who had no experience: "There is some resistance from some parts of the organization thinking that this could lead to slowing down of the process.”Another reason that was mentioned twice was that the company was not the decision maker, and that the headquarters decides upon these matters.Other reasons for not involving PO were about the same as previously noted (lack of knowledge...)."
One respondent makes the suggestion: "We might consider involving patient organisations to perhaps help us look at innovative training methods?"
Another recommendation: "ICH-GCP (International Conference on Harmonisation - Good Clinical Practice) training. The reviewer should be a person who is a scientific expert on that indication."
Involving patient organisations as Co-researchers in clinical trials
The survey revealed that the responding pharmaceutical companies had very little experience of involving patient organisations as co-researchers in clinical trials. Some do note they have co-funded research initiated by patient organizations. And one respondent notes only having co-researched with a Po who had all the conditions to perform clinical trials: "Physicians, Pharmacy, study nurses, study coordinator in accordance to local law and GCP’s."
Another respondent notes: "We have collaborated with patient organizations as co-researchers once in an observational clinical trial. It was a lengthy & complex process to go through together in order to clearly define the role & responsibilities of each party involved in the collaboration. A successful partnership was critical to minimize the risk of harming our long standing relationship. To date this has been a positive experience."
Reasons for not involving PO as co-researchers are mostly that PO do not have the responsibility to perform research: "PO do not have assigned responsibility based on local law", "PO are not accepted by head quarters as qualified", "unsure how EMEA would react to data generated by patient groups-would it be accepted as a pivotal regulatory package?"
Another reason is the lack of knowledge of PO: "I would not involve patients as co-researchers as they are unlikely to have the medical/scientific etc expertise required to work on what are sometimes very complex studies."
Involving patient organisations as Driving force in clinical trials
None of the respondents notes a PO having been the driving force behind a clinical trial conducted by their company and only 4 from the 15 see this as a possibility for the future. Reasons are mainly the same as mentioned earlier: not common practice and not having been approached by PO.
One respondent writes: "Location of clinical trials are largely governed by suitability of appropriately qualified principal investigators, patient populations and robust trial ethics and standards. These aspects are not usually within the patient group's area of expertise."
Another respondent on barriers: "Barriers would be costs of trial if not budgeted. I think the permanent dialogue structure we are in allows for a more regular input on development, which pre-empts suggestions on how/where and when like in this question."
Suggestions made on what is needed: "A patient organization with a clear focus on clinical research backed-up with appropriate organizational capabilities." "Demonstration of good relationships, data on investigators, studies to date, and infrastructure of suggested partners."
Involving patient organisations in clinical trials with children
Most respondents say there is no difference in practice between normal clinical trials and paediatric trials.
One respondent writes: "Very positive experience. We got practical feed-back regarding the feasibility and suitability of some tests/ measurements planned in the protocol. We received also some feed-back on general communication to the patient community. Lastly in certain cases we have collaborated with one specific patient organisation on trial’s participants travel & logistics."
Most respondents do not comment.
One respondent says to have collaborated with a diabetes patient organisation in a phase III and IV study, where genetic samples have been stored in a bio-bank.
Another says: "As for the setting up and maintenance of all medical databanks this can be a large, daunting long term commitment that organisations may not fully appreciate at the time of initiation. It is also challenging to deal with patient privacy laws."
Opinions of pharma on Patient Involvement
Remarks
Pharma that considered involving PO as info, advisors and reviewers:
"Patient organisation as information channel"
"We see an advisory role and information providing role, not the other topics."
"In general patient groups can provide assistance in bridging the information gap between patients and drug development companies."
"Patient organisations could have a role as advisor in clinical trials however they do not have the clinical research background to act as a reviewer, co-researcher or driving force."
"Evidence would suggest patient organisations add greatest value as advisors, reviewers and providers of relevant information rather than driving commercial trials."
"Collaboration with Patient Orgs should be regular, and formal. At that stage it can easily allow for a mutual trust and understanding that gives information and advises on a company's development plans and trials. Reviewing can be an issue before Phase IV because of regulatory restrictions."
Pharma that were less enthusiastic:
"Level of uncertainty about what patient organisations can provide."
"We do not consider to involve patient organisations in any phase."
Barriers towards patient involvement
Within patient organisations:
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Lack of awareness of clinical research and clinical trials.
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Lack of knowledge and expertise of patient organization.
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Lack of organizational structure and point persons.
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Variety in the level of development and relevant experience of patient organisations.
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Locally operating patient organizations vs. globally operating pharma
Within Pharma:
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Lack of awareness. Companies state that patient involvement; “is not a standard procedure” or are uncertain what the benefits are.
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Biomedical tradition of clinicians/scientists involvement only, not seeing the added value of patient involvement.
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Possible delay of the clinical trial process.
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Potential negative publicity of Industry collaborating with patient organizations.
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Code compliance issues.
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Concern over losing the control when proprietary information is shared.
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Funding of the patient organization to be able to participate in the various procedures.
Regulatory
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Strict guidelines of Central Ethics Committee and Local Authorities.
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There is no standard procedure for involving patient organisations.
Ethical
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Independence of patient groups may be lost.
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Financial links.
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One company experienced “biased enthusiasm” of patient organisations created through successful collaboration.
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Due to conflict of interest between two parties:
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Commercial sensitivities may be compromised.
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Regulatory objectives may be compromised.
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Inequalities of access to therapy among patient organizations (one company has experience with this)
Perceived benefits of patient involvement
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Increased recruitment and targeting broader group of patients.
- More reliable data.
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Valuable practical information and feed-back.
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Better insight into patient needs.
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Improved information material such as patient leaflets and informed consent.
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Better designed trials.
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Better outcome measures.
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Increased awareness among patients.
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Better in-trial participation.
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Raised sales.
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Opportunity to develop early long lasting partnerships.
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For patient organisation: financial compensation for activities.
Recommendations towards patient involvement
For patient organisation:
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Proactive attitude; contact pharma and demonstrate how their involvement can add value
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Need for a professionalised organisation
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Dedicated team or group of people on clinical trials within the organisation
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Join to form European or global organisations
For pharma:
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Researchers need to be educated on patient involvement to get acquainted with it.
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Need to differentiate patient organisation's representatives from individual patients
Interaction pharma and patient organisation:
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Set expectations and allocate role and responsibilities clearly for the two parties
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Use of confidentiality agreement
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Ensuring transparency in the collaboration
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Establishment of long-term contacts
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An independent organisation could act as facilitator
For regulators:
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Formalisation of standard operating procedure to a global recommendation for all disease areas
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Ethical rules on how pharma and patient organisations should interact