Sanofi is proud to announce the availability of the Injection-Treatment Acceptance Questionnaire (I-TAQ) patient-reported outcome (PRO) questionnaire, developed in collaboration with Regeneron Pharmaceuticals Inc., and evaluating treatment acceptance with a subcutaneous injection therapy. This questionnaire was developed with patients at high cardiovascular (CV) risk who had experience of self-administering their treatment for lowering low-density lipoprotein cholesterol (LDL-C) via subcutaneous injection, and items are generic enough to be used for any adult patients using subcutaneous injections. Its development and validation have met high methodology standards as outlined by the FDA (FDA PRO guidance, Dec. 2009). Following a literature review, concept elicitation interviews were conducted with 29 patients and then cognitive debriefings. The i-TAQ includes 22 items grouped into five domains of Perceived efficacy, Acceptance of side effects, Injection self-efficacy, Injection convenience and Overall acceptance. During its psychometric validation, it was found to be a reliable and valid instrument in high CV risk patients using a subcutaneously administered treatment for LDL-C lowering. Developed in US English, it was also linguistically validated in more than 35 languages. Please visit the i-TAQ page for detailed information !
On Oct 15-16 2018, Sanofi colleagues from the Global Clinical Outcomes and Risk Benefit and Epidemiology teams attended the Patient Focused Drug Development (PFDD) Public Workshop at the FDA in Silver Spring, MD.
The purpose of the meeting was to obtain feedback from stakeholders on two FDA Guidance documents in development under the PFDD initiative entitled Methods to Identify What is Important to Patients (Guidance Document 2) and Selecting, Developing or Modifying a Fit-for-Purpose Clinical Outcome Assessment (COA) (Guidance Document 3).
Representatives from all divisions of FDA (CDER, CDRH, CBER), the Pharmaceutical and Biotech Industry, Public/private partnerships, Academia, Patients and Patient Advocacy Groups were represented to provide input on:
One of the key questions that was discussed and debated over the 2-day meeting related to the target audience for the guidance documents—specifically whether the guidance documents should be targeted at scientists working in the field of PFDD or at a broader audience. While the emphasis on robust measurement would not change the level of technical detail would differ.
The FDA is particularly interested in feedback on the presentation of the text/tables, examples regarding what has worked, what has not worked (lessons learned), how sponsors have modified a COA and how to best estimate clinically important changes at the individual level.
Sanofi will offer a response to assist FDA in finalization of these important guidance documents.
The 2018 DIA Study Endpoints Community conference was held in September in Bethesda, Maryland. The conference brought together health care professionals involved in the selecting, developing, analysing, and interpreting of study endpoints. The focus was on advancing the scientific development and evaluation of study endpoints and educating other health care professionals about these developments. The congress agenda was broad, but all sessions emphasised patient-centricity, which has afforded significant innovations in how patients can be involved in endpoint development and selection in the past few years. Of particular interest was discussion around whether regulatory, payer and patient perspectives on the value of study endpoints can align, and whether core outcome sets for consistent measurement and analysis would facilitate this alignment. Core outcome sets are being developed by numerous organisations, including the Center for Medical Technology Policy (CMTP) and the International Consortium for Health Outcome Measurement (ICHOM) and recently the FDA send a request for information to facilitate their consideration of the value of core outcome sets in certain disease areas. It will be interesting to see how this develops, and the central role of patient-reported outcome (PRO) measures in these. Another topic discussed in detail – and resonating with the Sanofi team - was the importance of measuring positive well-being; something which is rarely done. Rather we focus on the absence of negative well-being, which of course is not the same thing at all.
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/
More news coming soon…