Project brief

Sheila Khawaja is an acromegaly patient and has worked as a patient advocate internationally since 2003. She is the current Vice-Chair of the World Alliance of Pituitary Organizations (WAPO).

Note: The case study solely focuses on the patient market research.


A company considers to develop a new treatment for Parkinson’s patients suffering from “off” periods, during which they don’t respond optimally to their normal medication. The new treatment has the potential to provide for simpler, faster and more tolerable rescue than current standard of care. From first discussions with physicians and payers, the company receives mixed messages regarding the medical need. First patient interactions however tell them there is a very high medical need. 


The company plans a qualitative and quantitative patients’ insights research with physicians, payers and patients. With this analysis, they aim to get a clearer picture about the medical need and the different existing and potential new treatment options. The outcomes of the analyses will influence their decision to develop and invest in the new treatment, or to stay out of it.


Gaining support of patient associations with direct qualitative and quantitative patients’ insights research. Creating a mutual understanding of and respect for the different interests of a company and a patient association. Creating win-win situations.



The company asks a patients’ insights research agency to conduct qualitative interviews and an online survey among physicians, payers and Parkinson's patients in several European countries. The agency reaches out to several Parkinson's patient associations, asking for support with the identification of patients who suffer from “off” periods of Parkinson's . The company does not want their name to be disclosed, because they are concerned about uncovering their interest in the new treatment. 

The agency experiences several issues:

  • All patient organizations insist on full transparency about the company behind the request and the purpose of the interview. 
  • One association wants to review the association’s comprehensive material about the Parkinson's patient journey, before they decide whether any further interviews and a survey can be performed.
  • Several patient representatives strongly recommend involvement of caretakers and Parkinson's nurses in both the interviews as well as the survey, in order to obtain a full picture of the situation.
  • One association wishes to review the full interview outline and survey questionnaire as a condition. After review, they propose significant changes, which the company considers irrelevant for their internal decision process. 
  • One patient organization recommends patient representatives of their association or physicians who their members trust to perform the interviews, rather than employees of the patients’ insights research agency.  


What went well

Investigating the patient’s view on medical need, and the potential patient benefit of a new treatment over existing ones is highly informative. It should be a standard for investment decisions that require such understanding. Approaching patient associations for help to identify the “right” patients is also recommended. 

What can be improved?

So, why did the agency encounter so much resistance? Lack of transparency, about the company behind the agency, the purpose or the content often leads to a difficult start if it is not well explained. Preliminary research and discussions with patient organizations can demonstrate which information already exists. Patient organizations may be able to give a lot of information ahead of any interviews and surveys . Additional interviews and surveys may even become irrelevant or can be designed in a more targeted and meaningful way. 

Sometimes patient organizations have different priorities and interests. Therefore, not every patient organization may be the right partner in the design and conduct of questionnaires or surveys. This should be openly discussed and respected.

Considerations and recommendations

Preparing for a collaboration

Doing some homework first should be mandatory! The case study "First Time Contact with Patients from the Industry's View" and tool "Mapping the Patient Landscape" also deal with this situation.

Transparency is something to strive for if you want to come across as a credible business partner for patient organizations. Additionally, it may be a compliance requirement, see e.g. the EFPIA Code of Practice on Relationships between the Pharmaceutical Industry and Patient Organisations, artice 5 on Transparency (p. 4). Compliance experts should be involved early to provide clarity in regard of which information can and should be made transparent - or not - to the patient organization and to the public. Where the company cannot be transparent, e.g. when a product of the company is involved, or because the survey would be biased by disclosing the company, this needs to be properly explained. However, it is recommended to where and whenever possible and reasonable, show a name and face of the company to the people before involving an agency. 

Co-creation of a questionnaire or survey with patient representatives is powerful, when both parties have the same goal in mind. In addition to information provided by patient organizations and patients themselves, you can also think about involving caretakers and relatives or nurses who can provide a lot of insights and help you understand patient needs. If requested by the patients, accept a caretaker or physician of choice to join them during interviews.  

After your contact, make sure to follow up with (anonymized) results. Visuals showing product profiles and differences between therapeutic options are very helpful to increase understanding, rather than lots of text. You can also give something else in return. This can be a donation, an open door event and/or a webinar that gives the patient organization valuable knowledge in exchange. It will help your company to being perceived as ethical, serious and reliable. Discuss the best way of a fair return with patient representatives prior to the start of the project. See also checklist "Feedback of a Patient Organization".


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)


Mapping the Patient Landscape

Following a general understanding of the patient journey and unmet needs and before systematically 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:

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



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.