Precision medicines can command premiums, in part due to the preferences of providers, opportunities afforded to payers, and the rising influence of the patient–consumer.
This series is dedicated to assessing a gamut of strategies for bringing precision drugs, devices, and diagnostics to market. In the first instalment, we explored emerging biomarker-based approaches, from genomics to wearable sensors.
In this instalment, we will explore the potential of stratifying patient populations through an alternative strategy: patient preference information. These data may be more valuable than they first appear, in part due to emerging regulatory precedent.
New precision paths to approval
A remarkable evolution in the recent past has been the U.S. Food and Drug Administration’s decision to consider patient–consumer preferences for product approval and labelling.
The FDA set a new precedent in January 2015 by approving EnteroMedics’ Maestro® Rechargeable System, an implantable device for weight loss. The device missed its weight loss endpoint in its pivotal trial; the experimental group needed to lose 10% more excess weight than the control, but lost only 8.5%.
However, the device was approved in part because a patient preference survey indicated that a subset of eligible patients were willing to accept the risks of the device in exchange for prolonged weight loss. For this device, the patient preference survey instrument became means to not only obtain approval, but to also generate a precise label with risk-benefit information for a well-defined stratum of patients.
Later in 2015, as part of its Patient Preference Initiative, the CDRH formed its first ever Patient Engagement Advisory Committee and issued a draft guidance on patient preference information (PPI). PPI is defined by CDRH as “qualitative or quantitative assessments of the relative desirability or acceptability of attributes that differ among alternative or diagnostic therapeutic strategies.” [For a discussion of patient preference methods and how they differ from patient-reported outcomes, read this ISPOR paper.]
Through this guidance, the agency encourages voluntary submissions of PPI with PMAs, HDE applications, and de novo requests, as well as the inclusion of PPI in device labelling. The guidance also provides considerations for the design of scientifically rigorous PPI collection tools, as well as examples illustrating how PPI aids in the FDA’s assessment of a device’s risk-benefit profile.
Patient preferences in protocol design and product development
Patient preferences may also provide an alternative vehicle to stratify patient populations and thereby enhance the efficiency of a product's development lifecycle, from prototyping to trial design.
Currently, the Medical Device Innovation Consortium (MDIC), a public-private partnership between the FDA, NIH, CMS, industry, and patient advocacy groups, has adopted this philosophy and is developing standards for defining PPI and employing the data in clinical trials. MDIC has catalogued methods for collecting PPI through the entire product lifecycle and created a framework, called the Patient Centered Benefit-Risk Assessment Framework, that guides the incorporation of those methods into protocol designs and regulatory submissions.
MDIC’s research operates under the principle that incorporating PPI into clinical trial design enhances not only the appeal, but also the benefit of trials to the patient-consumer. PPI-focused trials offer more value to the patient-consumer because they incorporate the patient-consumer’s own opinions into the trial design and therefore give the patient-consumer more control over their own treatment and overall lifestyle.
Based on their research, MDIC has outlined several benefits to collecting and utilising PPI in clinical development:
First, MDIC argues that PPI can be useful in maximising the efficiency of a protocol’s design. Personal risk tolerance and benefit evaluations can identify potential heterogeneity among patient perspectives, as well as subpopulations that would be more willing to accept a risk-benefit trade-off. This information can provide grounds for certain trial enrolment criteria and help to refine primary and secondary endpoints, thereby enhancing a trial's utility for building a case for why certain patients may benefit from a technology over alternatives.
Another key application of PPI, according to MDIC, is to inform investigators of design limitations that affect a target population, providing an opportunity to refine and improve the product before a first-in-human application. Patient feedback can inform, for example, patient-sensitive design controls in early prototyping, pre-clinical design verification testing, and the design of user instructions, thereby enhancing the product's benefit and appeal to the consumer.
MDIC also suggests that more products will reach the market if patients' willingness to use the product is factored into the final assessment of the product's regulatory burden. Information regarding a patient's willingness to trade a certain level of risk for certain benefits, in particular, can help reviewers frame decisions based on a the patients' needs. Ultimately, this information can be used as evidence in support of a product’s approval.
Finally, MDIC's standards aim to clarify how PPI can supplement clinical data during reimbursement discussions. PPI can elucidate how patients process risk-benefit information, providing manufacturers and regulators insight into how to best to relay that information back to patients and to payers. The data can also enhance a developer's decisions about the scope of product launches and reimbursement.
While the use of PPI in clinical trials is still in early stages, the MDIC's extensive patient preference data initiative indicates a range of PPI applications can enhance the development of a precision medicine or device.
Patient-centric trials
The impetus behind collecting patient preference information is to produce medical devices that better meet patients’ needs, as well as to collect data that build a case for regulatory approval and reimbursement. Patient engagement continues to be a strategic priority of CDRH and ultimately reflects the industry’s shift toward patient-centric trials.
This blog is part two of our ‘Precision Medicine for the Patient-Consumer’ series. The next instalment of this series will focus on apps and other technologies that interact directly with patients not only to personalize their experience of healthcare and clinical trials, but also to catalyse new relationships with device manufacturers.
In this section
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Digital Disruption
- AI and clinical trials
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Clinical trial data anonymisation and data sharing
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Clinical Trial Tokenisation
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Closing the evidence gap: The value of digital health technologies in supporting drug reimbursement decisions
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Digital disruption in biopharma
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Disruptive Innovation
- Remote Patient Monitoring
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Personalising Digital Health
- Real World Data
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The triad of trust: Navigating real-world healthcare data integration
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Patient Centricity
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Agile Clinical Monitoring
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Capturing the voice of the patient in clinical trials
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Charting the Managed Access Program Landscape
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Developing Nurse-Centric Medical Communications
- Diversity and inclusion in clinical trials
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Exploring the patient perspective from different angles
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Patient safety and pharmacovigilance
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A guide to safety data migrations
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Taking safety reporting to the next level with automation
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Outsourced Pharmacovigilance Affiliate Solution
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The evolution of the Pharmacovigilance System Master File: Benefits, challenges, and opportunities
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Sponsor and CRO pharmacovigilance and safety alliances
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Understanding the Periodic Benefit-Risk Evaluation Report
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A guide to safety data migrations
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Patient voice survey
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Patient Voice Survey - Decentralised and Hybrid Trials
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Reimagining Patient-Centricity with the Internet of Medical Things (IoMT)
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Using longitudinal qualitative research to capture the patient voice
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Agile Clinical Monitoring
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Regulatory Intelligence
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An innovative approach to rare disease clinical development
- EU Clinical Trials Regulation
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Using innovative tools and lean writing processes to accelerate regulatory document writing
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Current overview of data sharing within clinical trial transparency
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Global Agency Meetings: A collaborative approach to drug development
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Keeping the end in mind: key considerations for creating plain language summaries
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Navigating orphan drug development from early phase to marketing authorisation
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Procedural and regulatory know-how for China biotechs in the EU
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RACE for Children Act
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Early engagement and regulatory considerations for biotech
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Regulatory Intelligence Newsletter
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Requirements & strategy considerations within clinical trial transparency
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Spotlight on regulatory reforms in China
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Demystifying EU CTR, MDR and IVDR
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Transfer of marketing authorisation
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An innovative approach to rare disease clinical development
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Therapeutics insights
- Endocrine and Metabolic Disorders
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- Cell and Gene Therapies
- Central Nervous System
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Glycomics
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- Paediatrics
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Respiratory
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Rare and orphan diseases
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Advanced therapies for rare diseases
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Cross-border enrollment of rare disease patients
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Crossing the finish line: Why effective participation support strategy is critical to trial efficiency and success in rare diseases
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Diversity, equity and inclusion in rare disease clinical trials
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Identify and mitigate risks to rare disease clinical programmes
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Leveraging historical data for use in rare disease trials
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Natural history studies to improve drug development in rare diseases
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Patient Centricity in Orphan Drug Development
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The key to remarkable rare disease registries
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Therapeutic spotlight: Precision medicine considerations in rare diseases
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Advanced therapies for rare diseases
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Transforming Trials
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Accelerating biotech innovation from discovery to commercialisation
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Ensuring the validity of clinical outcomes assessment (COA) data: The value of rater training
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Linguistic validation of Clinical Outcomes Assessments
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Optimising biotech funding
- Adaptive clinical trials
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Best practices to increase engagement with medical and scientific poster content
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Decentralised clinical trials
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Biopharma perspective: the promise of decentralised models and diversity in clinical trials
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Decentralised and Hybrid clinical trials
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Practical considerations in transitioning to hybrid or decentralised clinical trials
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Navigating the regulatory labyrinth of technology in decentralised clinical trials
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Biopharma perspective: the promise of decentralised models and diversity in clinical trials
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eCOA implementation
- Blended solutions insights
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Implications of COVID-19 on statistical design and analyses of clinical studies
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Improving pharma R&D efficiency
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Increasing Complexity and Declining ROI in Drug Development
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Innovation in Clinical Trial Methodologies
- Partnership insights
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Risk Based Quality Management
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Transforming the R&D Model to Sustain Growth
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Accelerating biotech innovation from discovery to commercialisation
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Value Based Healthcare
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Strategies for commercialising oncology treatments for young adults
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US payers and PROs
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Accelerated early clinical manufacturing
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Cardiovascular Medical Devices
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CMS Part D Price Negotiations: Is your drug on the list?
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COVID-19 navigating global market access
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Ensuring scientific rigor in external control arms
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Evidence Synthesis: A solution to sparse evidence, heterogeneous studies, and disconnected networks
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Global Outcomes Benchmarking
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Health technology assessment
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Perspectives from US payers
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ICER’s impact on payer decision making
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Making Sense of the Biosimilars Market
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Medical communications in early phase product development
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Navigating the Challenges and Opportunities of Value Based Healthcare
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Payer Reliance on ICER and Perceptions on Value Based Pricing
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Payers Perspectives on Digital Therapeutics
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Precision Medicine
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RWE Generation Cross Sectional Studies and Medical Chart Review
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Survey results: How to engage healthcare decision-makers
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The affordability hurdle for gene therapies
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The Role of ICER as an HTA Organisation
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Strategies for commercialising oncology treatments for young adults
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