Project brief

Maryze Schoneveld van der Linde is a Pompe patient and has worked as a patient advocate internationally since many years. She has been consulting on patient engagement with her own company Patient Centered Solutions since 2007.

Note: Several experienced representatives of patient organizations reviewed this case study. It was purposefully written in a very personal style and covers a lot of issues many patients and their relatives experience when being in contact with pharmaceutical companies. 


First contact at a patient event:

I am a patient with a chronic disease. I am wheelchair bound. Recently I was at a conference organized by a self-support group for patients with my disease. Travel, organization and spending two days at the conference with a full program always is exhausting. But I go there because it is really worthwhile to meet people who have the same problems and to exchange the best practices to make life with my disease a little easier. 

Last time, the president of my patient organization introduced me to a very nice medical affairs manager of a pharmaceutical company. She asked our president to help her reach out to patients who would be willing to talk to scientists and managers of their company about their life with the disease. The lady explained that my disease is a new therapeutic area for the company. After a chat she asked me whether I would be willing to talk about my “patient journey” to a group of her colleagues at the company’s office in Amsterdam. I found that very exciting and spontaneously agreed. 

Thinking about it:

When traveling back home, I became nervous. I had never talked about my disease in front of a group with unfamiliar people, especially not from a pharmaceutical company. I was wondering whether I could live up to their expectations. What could I tell these highly trained people that they would not already know? Add on top of that a two hour trip to Amsterdam and you may imagine how stressed I felt. I had a very bad night and spent the whole next day in great pain and anxiety. However, when the lady called a few days later and asked whether we could start planning, I didn’t tell her and agreed.

Organizational steps: 

What followed then was really unpleasant. I was called by a an event manager who explained to me that I would be reimbursed for my travel costs within the limits of their company policy, which she would send to me. I just had to notify when I would arrive and where I would stay. I was perplexed. I had not thought about organizing the trip myself. I asked whether the event location would be barrier-free and whether there would be wheelchair accessible restrooms. She didn’t know. She also didn’t know names of barrier-free hotels. I told her I needed a caretaker to help me during my stay. She thought that might be an issue, because there was no budget for that. It took three calls until I felt comfortable to take the trip, after they agreed to reimburse my sister for joining. A compensation for the fact that my sister took a day off was not ­­­possible from a legal perspective, which seemed unfair to me. 

The legal part of it: 

A few days later the event manager called again, asking whether I had received the email with legal documents for informed consent and data privacy. I had to fill these in, sign them and send them back to her. I couldn’t tell, because I don’t check my email regularly, as I have difficulties typing on the computer due to my finger stiffness. When I opened the email, it contained a 6 page-document with hard to understand legal language and very strict rules about travel costs and reimbursement. I asked my niece who is a law student, to check and tell whether it’s okay for me to sign. I have no printer at home, so that was another hassle.  

And it’s more complex:

When I was close to letting the company know it would be too much for me, the medical affairs manager called again and asked how things were going. I was so relieved she was calling! She was the one I knew and why I had agreed to do this and I knew she would understand my concerns. I told her I had to plan the whole trip myself and how stressful that was. I told her about my sister taking a whole day off without compensation. I also told her that I still had no clear understanding of what the event would actually look like, who would be there and what exactly would be expected from me. 


  • Understanding what it means for a patient in his or her specific situation to be asked to spend time and energy on interaction with a company. 
  • Making a patient feel empowered and comfortable in sharing experiences with highly trained professionals. Providing a fair and appropriate solution that leads to a win-win situation.
  • Trying to keep the stress level for the patient as low as possible and help to make it a worthwhile experience for him or her. 

Note: For the sake of simplicity in regard of legal compliance, the case study assumes that the company has not yet decided to engage in the respective disease area, i.e. no development or commercial product is involved.


What went well

Asking the patient organization for advice and support with an introduction in person is certainly very appropriate. Also, the company representative was very transparent about the purpose and followed up immediately after the event. Finally, checking in again at a later point in time to make sure things are going well is even more important with patients than with key opinion leaders. Patients usually don’t get paid for their effort and -even if so- depending on the disease it may cost them much more energy than a healthy individual. 

What can be improved?

There was no plan and process in place that fit the purpose of working with patients. Of course, certain things in preparation of an event can be delegated, however in this example, the event manager was clearly not well prepared for the case. If a company has no process or policies in place for interactions with patients, make sure to agree on an appropriate process internally rather than imposing standard policies written for health care professionals­­­ or other experts­­­. Patients require a different approach.  

Considerations and recommendations

Preparing for interaction with an individual patient (whether member of a patient organization or not)

When looking for patients to learn about their “patient journey”, it is highly recommended to approach a patient organization (if existing). It is a matter of respect, transparency and good collaboration practice to at least inform the patient organization about your intent to reach out. You may actually find out they can be very helpful in identifying the right patient (representative) to talk to. 

Want to learn more about preparing for patient engagement and making sure you have the right processes in place? Read our case study "First Time Contact to Patients from Industry's View".

In addition, please carefully read the feedback from a patient representative on the questions: “What is important for you in interactions with industry?” and “What does industry need to consider?”


Mapping the Patient Landscape

Following a general understanding of the patient journey and unmet needs and beforesystematically interacting with patients and KOLs in a specific therapeutic area a company should have an understanding of the stakeholder field. The checklist below can help you obtain such an understanding, by:

 Interviews with known patient advocates and medical specialists about their experience, needs and expectations, available information and data, recommendations regarding other specialists and patients, etc.

Analysis of patient groups and individual patient advocates via web, social media and other sources per country on patient journeys and community communication, objectives, capabilities, structure, medical advisors and connectivity with others, etc.

Analysis of major KOLs per country on number of patients treated, national and international reputation and influence level, publications, registries, connectivity with others including healthcare institutions and patient organizations, etc.

If helpful confirmatory interviews with major identified patient advocates/representatives regarding

independence and credibility among patient and medical community and other important stakeholders

  1. willingness and ability to collaborate with industry and 
  2. level of expertise and efficiency

Source: This checklist has been developed by admedicum® Business for Patients GmbH.


Preparing a collaboration

Defining the interaction

Patients, patient representatives and industry should take responsibility to ensure interactions are meaningful by clearly defined processes and actions, progressed to timelines. In addition, all participants should be prepared for the interaction. 

Prior to each interaction, agree mutually on (where applicable): 

  • The objective of project involving patients and/or areas of common interest to establish agreed structured interaction, providing all parties with necessary protection with regards to independence, privacy, confidentiality and expectations (see section 11. written agreement)
  • The type of input and mandate of the involved person
  • The tools and methods of interaction, e.g. types and frequency of meetings, ground rules, conflict resolution, evaluation
  • Desired patient / patient partner organisation to foster long-term working partnerships, with independence ensured (in scope)
  • The profile of the type of patient/s or patient representatives/s to be involved and their number
  • How activity outputs will be used and ownership of outputs
  • How and when the patient/s involved will be informed of outcomes
  • Contractual terms and conditions including consent and compensation (see section 11, written agreement). 
  • Other elements according to the specific project 


European Patients’ Academy on Therapeutic Innovation (EUPATI) (2016): Guidance for patient involvement for industry-led medicines R&D. (12/06/17)


Preparing a collaboration

The four key principles for collaboration:

1. Clarity of Purpose

Each party should be clear about the reason for and the planned outcome of the collaboration – and the ultimate benefit for patients 

2. Integrity

Each party should act and be seen to act honestly and with integrity at all times 

3. Independence

Each party should maintain their independence 

4. Transparency

Each party should be open and honest about the purpose of the collaboration and be able to account publicly for the associated activities and any exchanges of funding  

Using this guide: a checklist

  • Has there been a frank discussion about the purpose and expected benefits of the collaboration, and any risks, addressing all the issues in this guide?
  • Are the objectives and planned outcomes of the collaboration specified?
  • Are the roles of each partner and reporting mechanisms specified?
  • Has a written agreement or contract been put in place, which sets out how each party will adhere to the four key principles?
  • Is there a named senior individual accountable for managing and maintaining the relationship and monitoring adherence to the four key principles?
  • Is information about the collaboration published on the company and charity websites?
  • Can each party confidently explain the collaboration in public? 

Source: National Voices, The Association of the British Pharmaceutical Industry (ABPI) (2015): Working

together, delivering for patients. A guide to collaboration between charities and pharmaceutical companies in the UK. (12/06/17)


Patient identification/interaction

Patient identification/interaction

There are many ways to identify patients to be involved in an interaction. The main routs are through: 

  • existing patient organisations
  • EUPATI or similar project
  • advertising opportunities for patient participation
  • existing relationships with healthcare providers, hospitals and researchers and other agencies 
  • unsolicited requests previously made by interested parties
  • existing advisory boards / groups (e.g. EFPIA Think Tank, Patients and Consumers Working Party at the EMA)
  • this party agencies 

Source: European Patients’Academy on Therapeutic Innovation (EUPATI) (2016): Guidance for patient involvement for industry-led medicines R&D. (12/06/17)


List of useful conferences

Important international multi-stakeholder conferences with patient advocates included 

Patient Summit Europe

Patients as Partners

ECRD: European Conference on Rare Diseases and Orphan Products

Rare diseases conferences (focus industry) with patient advocates included 

Orphan Drug Summit

The Global Orphand Drug Conference and Expo

You may be want to attend conferences from patient groups to learn from their interactions and subjects about their needs. Many of these conferences are open for industry participants but one should keep in mind that these events are from patients for patients and industry should respect the guest status.  

Example for an indication specific conference by and for patients (industry welcome)

European Rett Syndrome Congress (international)

Also many patient organizations welcome industry at their charity events as a sponsor and as participants, like at charity runs. Check out your area of engagement.


Defining role patients

Although you may not have selected the expert patients or patient groups (EP/PGs) yet, outlining their roles and responsibilities at this stage helps to define your needs. Keep in mind that EP/PG roles may vary at different of the program or may evolve in response to new requirements. Once selected, discuss the roles with your EP/PGs to clarify what they can contribute based on their unique expertise and experience and avoid misunderstandings at the outset, e.g., if they’re expecting to have a partnership role but you’ve designed reactor role (see Types of Patient Roles chart below). 

Patient RoleExamples



Partnership role
  • Patients provide a priori and continuous consultation on outcomes of importance, study design, etc.
  • Patients are paid investigators or consultants
  • Patients have a governance role - "a seat at the table"
Advisor role
  • Patients serve as advisory committee members or provide a priori consultation on outcomes of importance and study design, but have no leadership role or governance authority
Reactor role
  • Patient input is collected distally through surveys, focus groups, or interviews, but patients are no consulted directly or a prior on such things as study design and outcomes of importance
  • Patients are asked to react to what has been put before them rather than being the origin of the concepts of interest
Trial or study participant
  • Patients are recruited or enrolled as study participant, but are not asked for input, consultation or reaction

Source: DIA (2017): Considerations Guide to Implementing Patient-Centric Initiatives in Health Care Product

Development. (02/06/17)


Feedback of a Patient Organization

HollandBIO and admedicum asked Marleen Katee, President of PSC Patients Europe, and Maryze Sconeveld van der Linde, Patient Advocate Pompe Disease, the following questions: 

“What is most important to you when working with the industry?”

  • Relationships are built on trust. A “warm” (personal, trustworthy, sustainable) relationship is the best way forward, especially in long-lasting collaborations.
  • Work with us. Patient experts are patients themselves, so we have different needs and priorities than healthy individuals. For example: we aren’t deadline junkies. Give us at least 3 weeks to get back with an answer. 
  • Be clear on what the (mutual) expectations are. 
  • Do not only talk to us, but above all listen to us. Amazing things can happen…
  • Please don’t ask us to translate: we are not a translation agency, we are a patient organization! 

“What does the industry need to consider when collaborating with patients / patient organizations?”

  • Create lay versions of every paper/document you publish or use, including Travel Policies, Informed Consent and Data Privacy documents.
  • Work with the patient organizations on those lay versions. With all due respect, as researcher it might be a bit of a challenge to figure out if a text is well-understood or not. This is your ultimate opportunity to give back to the community!
  • Train your researchers, event managers, legal counsels etc. on patient-related matters, including how to embed the patient’s voice in your research. Make sure they see the benefits of patient involvement, instead of it being an obligation. Learn them to talk with patients as an equal partner. Above all, learn clinicians & researchers to listen to the patients.
  • Don’t expect the patient (or patient organization) to do all kind of things free of charge. You don’t do that either, do you? A fee is great. However, some patient experts cannot accept a fee, as that will have a huge (negative) influence on their finances (e.g. on welfare or on disability leave) . You can offer to donate to a charity of their choice. Solved!  
  • Accommodate patients needs: if there is a visit, make sure there is wheelchair access, a meal matching our dietary needs. If we need to come a day earlier because we are tired, so be it. Don’t discuss, just do it and make it happen.
  • Expect patients to need more extended deadlines. Being ill is a fulltime job already.

Source: This tool has been developed by admedicum® Business for Patients GmbH.