StemCell Therapy

In 2022, personalized medicines (PM) made up more than 34% of new therapeutics approved by the Food and Drug Administration (FDA). PM is defined by the Personalized Medicines Coalition as the use of diagnostic tests to determine which medical treatments will work best for each patient or [the] use [of] medical interventions to alter molecular mechanisms that impact health. This emerging approach to healthcare is growing rapidly and having an important impact on patients, practitioners, and health systems. While many healthcare providers have already discovered the value of PM for their patients, others may remain unsure or unconvinced. The primary rationale for using PM is that the standard-of-care therapy may work well for some patients, but for others, it may have lower efficacy or higher risk for side effects due to patients biological differences. , This is the subset of patients who could benefit the most from PM.

At USP, the Healthcare Quality & Safety (HQS) Center of Excellence has developed a science-based roadmap for personalized medicine that considers how USP can evolve standards to address new modalities of medicine and close important gaps in these treatments that help ensure quality patient care. Waypoints on this roadmap include examining established standards, building collaborations with key stakeholders, identifying volunteers for future DTx standards work at USP, holding roundtable discussions, drafting Stimuli articles, and developing workplan focus areas for the HQS Expert Committees. USP has a long-standing history of healthcare standards including those for compounding preparations that are tailored to meet the unique needs of patients who may not otherwise have access to their medications. Currently, USP is exploring other specialized areas within PM, including pharmacogenomics, digital therapeutics, and 3D-printed medications to name a few.

Pharmacogenomics

Pharmacogenomics (PGx) is the study of how a patients genes can affect drug therapy. From a sample of saliva, cheek cells, or blood, scientists can extract a patients DNA and sequence it to understand how that individuals genes are similar to or different from genes of other patients. The individuals genetic results are then considered by healthcare providers, in combination with other data about the patient and their medical condition, to select the most appropriate drug therapy for them.

Based on decades of research with populations around the globe, healthcare providers can now use PGx to make predictions of an individuals personalized response to medications. For example, PGx can help predict the amount of drug available in the patients body, which can determine both its therapeutic effect(s) and likelihood to cause side effects. This is based on the observation that multiple people who take the same dose of the same medication may metabolize, transport, bind, or otherwise interact with drugs differently, leading to different amounts of drug in their bodies.

PGx presents opportunities for USP to collaborate with key stakeholders who are already developing PGx standards and guidelines and to apply its standards-setting process to establish alignment and consistency in PGx standards. Specifically, some of the standards and guidelines that USP could help develop are the 1) naming of genetic biomarkers and PGx terminology, 2) labeling of medicines to incorporate PGx information, 3) incorporation of PGx into healthcare information technology such as electronic health records and clinical decision support, and 4) diversification of clinical trials so that PGx information is not limited to patients of common ancestries, such as those of European ancestry. USP is well-positioned to engage a wide audience while increasing the reliability of and confidence in the utilization of PGx. This will help support PGx implementation, including payment and reimbursement, both nationally and globally.

Digital Therapeutics

Digital therapeutics (DTx) are defined by the International Organization for Standardization as health software intended to treat or alleviate a disease, disorder, condition, or injury by generating and delivering a medical intervention that has a demonstrable positive therapeutic impact on a patients health. The DTx landscape is evolving rapidly, with more than 40 prescription digital therapeutics already available in the U.S. DTx is projected to have a cumulative annual growth rate of up to 25.4% in the U.S. market by 2030, and this growth is not limited to the U.S., as the global DTx market is expected to expand by 31.6% by 2027. Due to this burgeoning expansion, DTx products are increasingly in need of robust standards to underpin the creation of high-quality products and the delivery of comprehensive care on a large scale.

USP is actively engaged in researching the landscape of DTx and has started an investigation into opportunities for DTx standards to improve the quality of care provided to patients. USP has identified areas for potential standards that include establishing, or supporting the establishment of, global DTx definitions including outcomes used in clinical studies of DTx products. These potential standards may also include key aspects such as security and privacy, promoting awareness and education among healthcare providers and patients, adopting consistent labeling practices, setting standard technology proficiency requirements, addressing issues related to data integrity and code authenticity to deter counterfeit DTx products, facilitating interoperability among the various clinical systems used in healthcare around the world, and integrating DTx into healthcare delivery systems such as existing software and devices.

These elements would create a comprehensive framework for seamlessly integrating DTx within the healthcare industry, as well as for its development and regulation. USP is also maintaining awareness of the current position of DTx, understanding the potential role of standards in DTx product growth, identifying factors that can expedite progress, recognizing and addressing barriers, and determining the competencies needed to navigate the ever-changing, technology-driven market.

What comes next

USP is currently engaging with stakeholder leaders to develop Stimuli Articles related to its research involving potential standards for PGx and DTx. These future articles will feature in the USP-PF and will solicit public comment to promote stakeholder engagement. In the meantime, USP encourages interested parties to reach out for more information as USP approaches its new 2025-2030 cycle.

For further information about USPs work on personalized medicine, visit our webpage, sign up for the HQS newsletter, or contact:

Blaine GroatEmail: blaine.groat@usp.org

Yasmin HaidarbaigiEmail: Yasmin.haidarbaigi@usp.org

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i Personalized Medicine Coalition. Personalized Medicine at FDA: the Scope & Significance of Progress in 2022.report.pdf (personalizedmedicinecoalition.org) Accessed November 10, 2023. Personalized Medicine Coalition.ii Personalized Medicine 101.https://www.personalizedmedicinecoalition.org/personalized-medicine-101/. Accessed November 6, 2023.iii Schork, N. Personalized medicine: Time for one-person trials. Nature 520, 609611 (2015).https://doi.org/10.1038/520609a.iv US Department of Health and Human Services. National Action Plan for Adverse Drug Event Prevention.Washington (DC): 2014; pharmacogenomics working group whose mission is to develop a 56.v International Organization for Standardization. "Health Informatics Personalized Digital Health DigitalTherapeutics Health Software Systems." ISO/TR 11147:Edition 1, 2023, https://www.iso.org/obp/ui/#iso:std:iso:tr:11147:ed-1:v1:en.vi Liesch J, Murphy D, Singh V. Under Pressure: Prescription Digital Therapeutics - How an analysis of the U.S. PDTlandscape indicates mounting pressure for a make-or-break next 3 years. Blue Matter. 2022.vii Digital Therapeutics Market Size, Share, and Analysis Report. Grand View Research, 2023. Accessed viahttps://www.grandviewresearch.com/industry-analysis/digital-therapeutics-market on Sep 05, 2023.viii Digital Therapeutics Market Revenue Forecast: Latest Industry Updates. Markets and Markets, 2023. Accessed via https://www.marketsandmarkets.com/Market-Reports/digital-therapeutics-market-51646724.html on Sep 05, 2023.

Link:
Innovating for Individual Care: The Impact of USP on Personalized Medicine

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