Archives 2017

Patient-Centered Drug Development: Reflections, Challenges, & Solutions: The Success Story Continues

Following the success of our inaugural event in May 2017, and an overwhelming demand to host a second roundtable workshop, we were pleased to host our second invitation-only event “Patient-Centered Drug Development: Reflections, Challenges, & Solutions” on November 8th, 2017 at the Glasgow Science Center, Glasgow, Scotland. During this exclusive event we brought together strategic decision-makers to share their experience and discuss a roadmap for success.

We were delighted to be joined by guest speakers Professors John Browne and Anna Mayhew, and Dr Jean-François Ricci. Professor Browne presented on the efficient use of PROMs in a real-world setting, Professor Mayhew spoke about her experience in clinical trials in paediatric rare diseases, and Dr Ricci summarised his views on the current challenges for HTA. Finally, our own Dr Stefan Cano talked about facing the challenges of patient-centered outcome measurement, including interpretability, legacy instruments, and treatment benefit. During this lively interactive morning, all participants were given the opportunity to ask questions and put forward their views about how best to advance the measurement of patient-centered outcomes.

Given another great success, we fully intend on having a third event in Baltimore in May 2018. If you are interested in attending, please let us know. If the last two events are anything to go by, seats will go very quickly. So please book early!


Patient-centered outcomes in rare diseases: breakthrough paper co-authored by Thomas Morel (KU Leuven) and Stefan Cano (Modus Outcomes)

Learn in this article how a concept-driven measurement approach addresses the many challenges of evaluating direct treatment benefit in rare diseases.

Modus Outcomes’ team has developed successful concept-driven measurement solutions for sponsors in rare diseases. More specifically, we can help you:

  • Run a tailored workshop on the lessons from the paper
  • Apply these solutions to your clinical programs
  • Design analytical strategies for small populations

Please contact us for more information.

To access freely the article, click here.



Antoine Regnault, Global Lead Statistics at Modus Outcomes has joined ISOQOL’s Board of Directors!

This week is ISOQOL’s 24th Annual Conference in Philadelphia and this year is quite special for Modus Outcomes, as it will be the first year with Antoine as a member of the Board of Directors.

This prestigious position is a recognition from our peers of our leadership in patient-centered outcomes research and contribution to the development of our field. This appointment just follows a successful meeting with FDA in September where Modus presented its concept-driven measurement approach for evaluating and developing COA instruments (see news below).  This approach is at the core of our method to improve the accuracy and interpretability of COA and better understand and communicate the benefit of treatment to a range of stakeholders.

If you are planning on attending ISOQOL in Philadelphia this year, please feel free to ask Antoine about our concept-driven measurement approach.


When FDA met Q-PORTFOLIO: why the rave reviews are relevant to Modus Outcomes

Last week, Modus Outcomes’ own Stefan Cano (, together with Professors Anne Klassen (McMaster University; and Andrea Pusic (Memorial Sloan Kettering Cancer Center;, visited the Center for Devices and Radiological Health (CDRH) at the US Food and Drug Administration. This is the third time the trio has been invited by FDA to present on Q-PORTFOLIO instruments (e.g., BREAST-Q, FACE-Q

During the two-hour open meeting, with more than 50 FDA staff in attendance: Andrea spoke about the background and widespread use of the measures in clinical practice; Anne presented a detailed description of content development; and Stefan described how to maximize clinical interpretability and best practice guidelines for constructing PRO instruments. FDA feedback was very positive, including: high praise for the quality of the research; strong encouragement to qualify the Q-PORTFOLIO instruments; and a request for further training in concept-driven measurement, which is the cornerstone of Modus Outcomes’ methodology for evaluating existing, and developing new PRO instruments. Andrea, Anne and Stefan look forward to further interactions with FDA, with Modus Outcomes’ scientific team supporting teaching and training requirements.

If you are interested in learning more about what the FDA is raving about, please contact Andrea Murison ( to schedule a training session with our experts.


Stefan Cano invited by FDA

On September 13th, together with Professor Anne Klassen (McMaster University) and Dr Andrea Pusic (Memorial Sloan Kettering Cancer Center), Stefan will be visiting the Center for Devices and Radiological Health (CDRH) at the US Food and Drug Administration. This will be the third time Stefan, Anne and Andrea have been invited by FDA to present on Q-PORTFOLIO measures. This time they have been asked to talk about interpretation of the BREAST-Q (, FACE-Q (, BODY-Q scores (responder definitions; cut-scores), use in clinical practice and clinical trials, and best practice guidelines for developing and evaluating PRO instruments.


SPIRIT-PRO Consensus meeting Birmingham, UK, 11-12 May 2017

Antoine Regnault, Modus Outcomes’ Global Lead – Statistics, was a panel member of the SPIRIT-PRO consensus meeting that was held in Birmingham, UK on May 11-12, 2017.

SPIRIT (Standard Protocol Items: Recommendations for Interventional Trials) is an international initiative that aims to improve the quality of clinical trial protocols by defining an evidence-based set of items to address in a protocol.
The SPIRIT-PRO extension will provide guidance on the Patient-Reported Outcome content of clinical trial protocols. It is informed by different data sources (systematic review of published guidance, evaluations of over 200 clinical trial protocols, 2-round Delphi process and a stakeholder survey).
The consensus meeting involved about 30 experts from academia, regulatory agencies, industry, institutional agencies, and scientific journals and will lead to the publication of a checklist of specific PRO protocol items to be included in trial protocols and ethical reviews.
Modus Outcomes is proud to be part of this initiative and contribute to raising the quality standards for the demonstration of treatment benefits in terms of PRO in clinical trials.

More information on SPIRIT-PRO:
And on twitter: @CPROR_UoB #SPIRITPRO


FDA-Duke Margolis COA workshops April 4 and 5, 2017, Washington, DC

Modus Outcomes’ CEO, Patrick Marquis, MD MBA, participated in two invitation-only workshops that convened regulatory, academic and industry experts to discuss the interpretation of meaningful within-patient change and personalized COAs – both increasing important elements in the quest to gather robust and meaningful patient and caregiver input to inform medical product development and regulatory decision-making. The two workshops explored both traditional and new technical approaches to meaningful within-patient change and personalized COAs.

Patrick presented Modus Outcomes’ approach to these questions. Our approach moves toward providing a holistic answer that addresses fundamental conceptual and measurement issues, addressing current uncertainty about the best ways to interpret or personalize COAs. This new way of interpreting change moves from existing “black box” approaches to a more transparent model, making clear the connections between the concept measures, the ruler, and the experience of patients. To learn more please contact Andrea Murison (

A link to the workshops can be found here:


Modus Outcomes: 2016, a year in review 

2016 brought many changes and exciting projects to Modus Outcomes.

In 2016, Modus Outcomes doubled in size! By increasing our team to 16 members, we have expanded our services and reinforced our skill-set in qualitative and quantitative science. We opened three locations for our Global Team: Letchworth Garden City, UK; Lyon – France; and our newest office in Cambridge, MA, USA.

Last year, our clients kept us very busy, and we:

  • conducted more than 30 concept-driven measurement projects
  • worked in more than 10 therapeutic areas (e.g. Oncology, Immunology, CNS, Rare Diseases, Pediatrics).
  • interviewed more than 390 patients
  • collaborated with 17 KOLs
  • conducted more than 75 Rasch models on 17 instruments
  • contributed to the design (including selection of outcomes, and definition of endpoints), specification of the analysis and interpretation of over 10 clinical trials, with 4 involving regulatory interaction (FDA, EMA)

In 2016, we also continued to work hard to promote the science of COA evaluation and development by:

  • carrying out 9 oral presentations,
  • attending 10 conferences,
  • and publishing 25 papers in peer reviewed journals.

Follow us on LinkedIn to find out all the exciting news for 2017!

Best wishes for 2017!

The Modus Outcomes’ Team


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