In collaboration with Mapi Research Trust, Sanofi is organizing a free educational webinar dedicated to the Perception of Anticoagulant Treatment Questionnaire (PACT-Q) on Thursday, September 27, 2018. The PACT-Q is a valid and reliable instrument that allows the assessment of patients' expectations and satisfaction regarding anticoagulant treatment, as well as their opinion about treatment convenience of use. The objective of this webinar is to present the rationale for developing the PACT-Q, its development and validation, and its conditions of use, as well as its description among other PRO questionnaires within the Sanofi PRO questionnaire website. Please register today to this event and join our expert webinar hosts from Sanofi and Mapi to gain insight into how the health research and pharmaceutical community can use the PACT-Q in clinical research studies.
A recent research paper highlights the heterogeneity in peoples opinions about their health status. A small qualitative study, published in Expert Review of Pharmacoeconomics & Outcomes Research, explored the personalised health perspectives of 14 adults with self-reported diagnoses of cardiovascular disease (CVD). People were asked to segment a 0-100 scale (from the EuroQol 5-dimension questionnaire) into sections to show the boundaries between their own definitions of “poor”, “fair”, “good” and “excellent” health. There was a notable variability in where people drew the boundaries; the poor-fair boundary ranged between 10 and 50; fair-good between 40 and 75; and good-excellent between 60 and 91.
Usually, when looking at PRO data, it is inherently assumed that a self-reported health score has the same meaning for all i.e. a score of 40 for three separate respondents would be indicative of the same level of health status. However, the above study suggests that if three respondents provided a rating of 40 on the scale, one may believe that their health was good, another fair, and the third, poor. Similarly, respondents providing a rating of 60 could consider themselves to be in excellent, good, or just fair overall health.
This finding underscores the challenges in interpreting self-reported health scores. The article proposes use of a personalised framework for gathering individual definitions of health and using this information to interpret clinical meaning of PRO data for an individual patient. We believe that this is essential if PROs are to provide informative and useful data to researchers, healthcare providers, regulators, and patients.
On Jun 24-28, 2017, one member of Sanofi’s Department of Clinical Outcomes Group (COG) attended the 2018 DIA Global Annual Meeting and Exposition, in Boston, MA, USA. It was a great opportunity to collaborate and problem-solve on global and local challenges facing the life sciences field.
Emerging themes of the meeting included the opioid epidemic, artificial intelligence/machine learning/big data, patient engagement, and FDA updates on labeling and clinical outcome assessments.
Including patients in the drug development process; getting patient input on study feasibility and what is being asked of them during the study were noted as important during the DIA meeting.
A new Ecosystem of Patient Advocacy Groups (PAGs) was recognized at the DIA annual meeting. Speakers from the PAGS noted it is especially important to ensure privacy/confidentiality of patient information and to allow patients the option to withdraw at any time from a clinical trial (CT).
The FDA is considering Consumer Brief Summaries (CBS’s); which would provide CT summaries for CT patient participants and to help all patients better understand the package inserts.
FDA presented on the Development of Suitable COAs for Rare Diseases (RDs noting ‘there are numerous opportunities to use suitable COAs to assess symptoms, signs, and impacts of RDs that are important and relevant to patients and to support endpoints to measure clinical benefit’.
FDA recommended the 2009 PRO guidance for industry and the Roadmap to PRO measurement in clinical trials as guides to aid in the selection, modification, development, and evaluation of COAs.
FDA noted COAs that are developed and used in clinical practice are not necessarily suitable for regulatory purposes (reliability and validity in target population needs to be established).
Matt Reaney, Head of the Sanofi COG will be presenting at the forthcoming DIA workshop “Advancing the Science of Study Endpoints’ Sep 20-21, 2018 in Bethesda, MD.
On Jun 22, 2018, one member of Sanofi’s Department of Clinical Outcomes Generation (COG) Team attended the 3rd Annual FDA-ASCO Workshop on PROs in Oncology held at the FDA in Silver Spring, MD. It was a great opportunity to discuss and strategize on PROs/core concepts that should be measured in oncology drug development trials and how to best present these data.
Representatives from FDA, ASCO, NCI/NIH, International Payer and Oncology Research Groups (IQWIG, Health Canada, MHRA [UK], SISAQOL).academia and industry participated in an all day workshop to discuss the PRO core concepts (What to measure) in cancer drug development studies; How to best measure these core concepts, Efforts to standardize the analyses of PRO data, and the use of PRO and patient experience data in regulatory reviews.
PRO core concepts identified included disease symptoms; treatment symptoms, impact of side effects and physical function; recommendations for how to best measure these core concepts included symptom libraries and well-defined functional scale; recommendations were made for international standardization on what and how to measure core concepts in cancer drug development trials and how to analyze, interpret and present these data.
Minutes from the workshop can be found here (after 22-Aug 2018)
The inaugural AI Health & Pharma Summit was held recently as part of London Tech Week. The one-day meeting bought together the key stakeholders in pharmaceutical drug development and healthcare to discuss how AI technology can enhance scientific research through better decision-making, optimized innovation, improved efficiency of clinical trials, and new tool creation for physicians, consumers, insurers, and regulators. The event attracted start-up and established AI companies as well as pharmaceutical research and development, medical and commercial participants. Case studies were presented on how AI has been used to expedite timelines, identify data gaps and inform precision drug targeting, streamline clinical trials, and enhance patient safety monitoring. Examples were also give on how AI can improve patient-physician communication, enhance note taking, and predict patient outcomes in routine practice.
The use of AI for healthcare has been actively growing and we are excited about the role of AI data in the development of products and services to enhance patient care and patient-relevant outcomes.
In accordance with the 21st Century Cures Act, the US Food and Drug Administration (FDA) is developing a series of four methodological PFDD guidance documents to address how patient experience data and other relevant information from patients and caregivers can be systematically and robustly collected and submitted for consideration in the regulatory review of medical products. The first guidance (Guidance 1) was released in draft for public consultation on June 12th. This guidance discusses sampling methods that could be used when planning to collect patient input, providing a general overview of the relationship between potential research question(s) and method(s), management and analysis. The remaining guidances will propose specific methods to elicit detailed, unbiased, and comprehensive input from patients, patient groups, and caregivers (Guidance 2; draft in 2019), explore how patient input should be used in development of clinical outcomes assessment strategies (Guidance 3; draft in 2020) and in developing and interpreting patient-focused endpoints for clinical trials (Guidance 4; draft in 2020).
The PRO team at Sanofi are excited about the increasing acknowledgement of patient experience in regulatory decision making as indicated by the development of these guidances. We hope that the FDA will be cognisant of the broad implementation challenges that have come from the stringent requirements in the FDAs PRO guidance from 2009 and will not impose similar restrictions on generating patient experience data.
Detail about the PFDD guidance series is available here: https://www.fda.gov/Drugs/DevelopmentApprovalProcess/ucm610279.htm
The draft of Guidance 1 is available here: https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM610442.pdf
The International Society for Pharmacoeconomics and Outcomes Research (ISPOR) held its 23rd Annual International Meeting in Baltimore in May. The conference, attracting nearly 4000 delegates from over 70 countries, was focused on real-world evidence, digital health, and the new landscape for health decision making. Patient perspectives on health, wellness, medication and healthcare was a central theme. Specifically, there were numerous discussions about the use of patient insights, experiences and preferences for defining outcomes (i.e. patient-defined value); collection of outcome data outside a trial to give confidence to the generalisability of trial data; and definitions of populations to allow for precision-medicine development/indication. It was encouraging to see so many patients and patient groups active in the ISPOR community, and the acknowledged important of such perspectives across all sessions. Matt Reaney from the Sanofi Clinical Outcomes Generation team was invited to present during one of the three plenary sessions focused on patient preference information in regulatory decision making. This session is summarised in a press release from ISPOR: http://press.ispor.org/index.php/ispor-2018-drew-nearly-4000-stakeholders-in-health-economics-and-outcomes-research/
Sanofi Clinical Outcomes Generation team members participated at the Ninth Annual Patient-Reported Outcome Consortium Workshop, April 25-26, 2018, Silver Spring, MD. The meeting brings together industry, FDA, Critical Path Institute members, consultancies and those interested in COA measurement/issues. Highlights of the meeting included FDA update on their role as an active participant in drug development, broadening their traditional regulatory role. Also an update on expanded efforts to enhance drug development with four main areas highlighted: 1. Patient Focused Drug Development (collect and analyze patient experience data, in order to use in designing drug development programs (endpoints in studies/design studies), use to support regulatory decision-making (risk/benefit considerations); 2. Novel innovative trail designs (use of complex and adaptive and other novel trial designs – and how such clinical trials can satisfy the substantial evidence standard); 3. Real World Evidence (use data regarding use or potential benefits and risks of a drug derived from sources other than randomized clinical trials – in support of new indications and post-approval study requirements); and 4. Drug Development Tools: biomarkers and COAs. The COA Qualification Program was discussed with presentations from FDA and of therapeutic working groups that have developed and qualified COAs. Five guidance documents FDA working on were also discussed; these include Guidance 1: Identifying research questions and developing a sampling strategy to collect representative patient input: operationalizing data collection, management and analysis (Dec 18, 2017 Workshop); Guidance 2: Methods to elicit detailed, unbiased and comprehensive input from patients, patient groups and caregivers (Patient experience and patient preferences will inform what and how to measure, how to use COA clinical trial data in regulatory reviews); Guidance 3: developing or identifying COAs (still under development) fit for purpose COAs, Using patient input to develop appropriate COAs for use in clinical trials; Guidance 4: Developing COA-related clinical trial endpoints based on patient input; interpreting these endpoints (interpretable, important); Guidance 5: developing guidance on guidance; topic of guidance to develop and submit proposed draft guidance relating to patient experience data. Details of workshop including meeting transcript and slides to be posted by May 30, 2018 at: https://c-path.org/ninth-annual-patient-reported-outcome-consortium-workshop/
On March 7th 2018, Sanofi’s PRO Team attended the 5th Winter Symposium of the Human Motion Project. The vision of the project is to improve human health by an open collaborative technology platform for the mobile medical monitoring of human motion. The 2018 symposium focused on novel endpoints generated by mobile accelerometry for use in clinical trials. PRO research has highlighted the significance of reduced physical capacity to patients with chronic illness. Accelerometry is a technique for objectively quantifying movement patterns during walking and has more applicability in the real world compared to more traditional gait analysis or laboratory-based testing environment. However, its use in clinical trials has been restricted by questions around its sensitivity, specificity, precision and algorithms for analysis, as well as a lack of standardization of endpoints. The 5th Winter Symposium of the Human Motion Project included presentations and discussions focused on these topics. Specific topics included defining standards in accelerometer implementation and endpoints, testing of new algorithms for step detection and speed estimation, and linking accelerometer data to hard morbidity and mortality outcomes.
This is an important area for the future of clinical research and we are excited about developments allowing for more mainstream use and consistency in specification and interpretation.
On Dec 9-11, 2017, a member of Sanofi’s Department of Clinical Outcomes (CO) Team attended the 59th American Society of Hematology (ASH) Annual Meeting and Exposition in Atlanta, GA, USA. It was a great opportunity to gather information on hematological treatments and methodological advances in the hematology field; particularly hematological malignancies including multiple myeloma.
There were multiple presentations on patient perspectives, symptom experiences, health-related quality of life, financial burden, health economic models, resource utilization, costs, development of PROs, cost-effectiveness of treatment and economic burden of disease. Sanofi’s PRO Strategy for three Isatuximab studies were also discussed and presented at the meeting (ICARIA,IKEMA and IMROZ, NCT02990338, NCT03275285 and NCT03319667). In addition, the meeting provided information about cutting edge technology regarding treatment advances and measurement of the endpoints that matter to patients, providers, payers and employers; for example wearable devices to assess impact on and improvements in mobility and functionality due to disease and treatment.
The insights gained during this meeting will help us to develop clinical outcome, publication and reimbursement strategies at Sanofi.
More news coming soon…