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/
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.
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.
In December 2017, a collaborative workshop was hosted by the Centers of Excellence in Regulatory Science and Innovation (CERSIs) and the Food and Drug Administration (FDA) titled “Advancing Use of Patient Preference Information as Scientific Evidence in Medical Product Evaluation”. The workshop aimed to discuss the current regulatory landscape for the collection, analysis, interpretation and presentation of patient preference information (PPI) and identify future research and capacity needs in order to improve the use of PPI in a regulatory context.
Plentiful research highlights that patients have unique perspectives about the value of the potential benefits and the impact of potential harms and burdens of their medical treatments. The consideration of patients’ needs, values and preferences is one of the core principles of evidence-based medicine, but reliable and accurate methods are needed in order to effectively incorporate these into the benefit-risk appraisal of a new technology. PPI is qualitative input or quantitative data elicited from patients to provide estimates of how much different outcomes, endpoints or other attributes are valued by patients, and the trade-offs that patients state or demonstrate they are willing to make among them.
The workshop focused primarily on eliciting PPI through stated preference methods - in which preferences are elicited by offering choices or posing contingent valuation questions to study participants - and revealed-preference methods in which patient preferences are obtained from the actual observed choices made by patients. Little discussion focused on qualitative data or data derived from quantitative patient-reported outcome instruments.
Matt Reaney from the Sanofi PRO team was invited to provide an industry perspective on eliciting and utilising PPI. Matt welcomed the discussion of PPI and its potential use in individual decision-making. He noted that industry regularly conducts research to elicit PPI in order to develop target product profiles and probability of success documents, inform unmet need and disease burden, make investment decisions, and define what constitutes a meaningful benefit. However, Matt did identify some challenges in generating robust data specifically for regulatory decision-making, namely:
In addition, there are some institutional challenges in generating PPI for regulators. For example, different methods may be utilised to answer different questions and qualitative research, stated or revealed preference studies, cross-over trial design, and PROs have traditionally been managed by separate domains in the industry. In order for the right method to be selected based on the question asked, knowledge of all “preference methods” would be required within a single function. External guidance documents outlining the utility of PPI data in principle, and the research requirements and evidentiary standards to inform the role of PPI in decision-making would assist in the provision of such a function. Matt’s comments were included in a media piece for Biocentury.
We hope that this workshop was the beginning of meaningful collaboration between patients, regulators, academics and industry in identifying how to make use of PPI in regulatory decision making – something which is important to all.
The International Quality of Life Research (ISOQOL) is a well-established non-profit society to advance the scientific study of health-related quality of life and other patient-centered outcomes to identify effective interventions, enhance the quality of health care and promote the health of populations. Sanofi representatives including one member of the Clinical Outcomes Team attended the 24th annual meeting held in Philadelphia, Pennsylvania US, October 18-21, 2017.
The Congress was themed “Embracing Complexity: Using Patient-Reported Outcomes to Generate Real World Evidence" andprovided a platform to discuss the challenges the field is facing with regards to theory, methodology and implementation of results in health care policy and clinical practice. Many presentations focused on oncology and spinal injury studies, although their methods were applicable to other therapeutic areas in general. Of the highlights of this congress were having a higher percentage of studies with focus to the adolescent and pediatric populations commonly less presented within the broad-based congresses.
The presentations were centered on the real world challenges of PRO-based clinical decision making, use of electronic PROs and interpretation of PRO data. Within the contextual consideration of developing and using electronic PROs, challenges of design, tool development, and implementation for both adults and pediatric populations were discussed. Overview of methods (parametric & none-parametric analyses), challenges and considerations in both quantitative and qualitative methods to interpret PRO data were topics, especially for clinically meaningful change and responder definitions.
The International Society for Pharmacoeconomics and Outcomes Research (ISPOR) is a global scientific and educational organization for health economics and outcomes research. The team at Sanofi recently attended the 20th Annual European Congress to present and discuss research findings.
The congress was themed “the evolution of value in health care”; a timely reminder that definitions of value are changing, and an endorsement of the increasing role of patient organisations and representatives in defining value. This change is welcome and shows a progression often lacking in drug development research. However, the question of whether a clear and consistent definition of value can be provided and empirically supported remains open. Indeed it was apparent from presentations and discussions at the congress that some seek to embrace heterogeneity, while others seek simplicity and consistency. It is likely that in the future public and private organisations will come together to provide some clear guidance on defining value, outlining whose perspectives to embrace and when. Although the totality of this guidance remains unknown, it is clear that the patient perspective will be central to this.
In order to support this, the Clinical Outcome Assessment community at ISPOR and elsewhere need to think “bigger” than traditional quantitative outcomes. We need to embrace ways of engaging patients to understand experiences and perspectives beyond traditional endpoints. This will help ensure that we develop technologies to improve people’s lives, not just their health. The Sanofi team look forward to helping shape this movement.
More news coming soon…