Project brief

Nancy Meyerson-Hess is an internationally renown expert for clinical operations and an Associated Partner of admedicum® Business for Patients. She has cooperated directly with patients related to clinical trial design, recruitment and retention on numerous occasions.


A company starts a clinical trial in patients for treating Pain due to Diabetic Neuropathy over a period of 6 months. Several Key Opinion Leaders (KOLs) give input to the clinical development program. Clinical trial recruitment is very slow and several patients enrolled discontinue participation. This has an impact on replacement of trial materials, extension of contracts with vendors, additional recruitment activities, revision of development timeline, etc. The clinical trial team tries to find solutions. 


Understanding the reasons for slow recruitment and low retention from a patient view; adapting the trial set up accordingly.


Understanding what makes a patient accept or reject the burden of a clinical trial; adapting an ongoing clinical trial to patient’s needs.



  • After review with clinical trial providers and investigators, two major aspects seem to drive the resistance to enroll and/or drop out of the trial: a high number of visits to treatment centers far away from home and overwhelming obligations for documentation several times a day. The travel time and inconvenience to the patients and care givers has a negative influence upon motivation to participate in the clinical trial and/or to remain in the trial over an extended time of six months.
  • The company decides to work with a questionnaire to patients who were interested in participating but decided against it, and to patients who dropped out of the trial. The objective of the questionnaire is to confirm the previous findings and to understand which changes are needed to make those patients enroll and stay in the trial respectively.
  • The company requests the involved clinical research organizations and investigators to ask these patients to fill out the questionnaires or to provide information. Most of the patients respond positively to the request and are open to provide their input.
  • With the outcome of the questionnaires, the company sits down with some of the physicians for review and sets priorities for adapting the clinical trial protocol.
  • The revised version is presented to a small group of coordinating investigators and vendors and a patient from each involved site. 
  • The final revised clinical trial protocol reflects the input from patients, investigators and vendors, in as far as possible. The company also introduces new information targeting patients through newsletters, publications and indirectly via investigator information days. All of these changes need to be submitted and approved by the respective ethics committees or review boards as appropriate.
  • Following implementation, recruitment picks up and retention improves, however the clinical trial still takes longer and costs more than expected.


What went well

Once the issue was identified, the company took reasonable measures to understand the patient perspective. The questionnaire was the result of homework with vendors and investigators. Additionally, the company did a good job reaching out to patients they had to convince, rather than those on the trial that are most simple to reach.

What can be improved?

Patient involvement early in the design of the first clinical protocol would have significantly reduced the challenges met. Early engagement is an opportunity for direct exchange, to identify and bridge gaps in understanding and to come up with practical, innovative approaches to drug development.

The company did not have a formal process in place for patient involvement. Establishing such processes is time-saving and forward thinking.

Considerations and recommendations

Involve patients in your design process!

There is evidence, including insights from FDA and EMA, on the positive impact of patient engagement on recruitment and retention in trials. Both aspects are often negatively influenced by the logistics of the trial and frequent visits to the investigator site, which disrupt a normal routine or work. Patient involvement in the design of clinical trial is therefore highly recommended. Here is some evidence that can help your internal review of the benefits:

  • The DIA Patient Engagement Study reported reduced screen failure rates, faster patient recruitment rates, improved subject retention rates, reduced numbers of protocol amendments, and a greater number of patient relevant endpoints as positive aspects of patient involvement.

  • The Tufts Center for Drug Development Impact Report reported that 57% of all protocols, across all phases, have at least one substantial global amendment. The most frequent changes stem from amendments associated with modifications and revisions to study volunteer demographics and eligibility criteria. Based on the additional time and costs, it seems more cost efficient to reduce amendments by involving patients early on in the trial design and protocol discussions. 


The US based Clinical Trials Transformation Initiative (CTTI) developed some useful tools:

Please keep in mind that stakeholder involvement means really giving patients a chance to understand and express themselves, rather than guessing what a minimally burdensome trial design is. The patient knows best!

Get early management buy-in for the co-creation with patients

Involving patients in clinical trial planning yields a number of opportunities that go beyond successful recruitment and retention alone. Examples are the critical review of primary and secondary clinical trial endpoints, patient reported outcomes and the patient view on benefit-risk assessment. However, all of this requires resources, time and budgets. Therefore getting senior management buy-in early on is a key to success.

Feedback across the board! 

Remember that although there is an obligation to publish your clinical trial, this does not provide individual trial participants with information regarding “their” participation in the clinical trial. Please don’t forget the patient experts you worked with, once the collaboration comes to an end. Prepare final information regarding what suggestions will be implemented, which ones will not, and provide reasons why.. It is good to have agreement on feedback to involved patient experts before trial commencement. And again, patient experts and investigators can probably tell you best what they would like to know from this excersize. 

Further reading

If you want to go deep we recommend the following multi-stakeholder best practice sources:


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

Conferences with and for patients

Patient Summit Europe

Rare diseases conferences with and for patients

Orphan Drug Summit

The Global Orphand Drug Conference and Expo

Indication specific conferences with and for patients

European Rett Syndrome Congress


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)