Tag: Healthcare provider

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Use of Electronic Patient Record Systems for Rapid Response to an MHRA Public Assessment Report: Retrospective Observational Study

Background: Digital health data and infrastructure facilitate rapid analysis to provide actionable data, thereby fulfilling the principles of a learning health system. In response to a report from the UK Medicines and Healthcare Products Regulatory Agency (MHRA), a rapid service evaluation was carried out to identify patterns of modified-release (MR) opioid use after elective surgery. Objective: We aimed to describe the prescribing patterns of MR opioids, methods to repurpose existing infrastructure, and the experience of collaboration between clinical and research teams using shared data pipelines. Methods: A retrospective case-control study was conducted at a tertiary care organization across multiple hospital sites in London, United Kingdom. Prescription and administration data for adult patients undergoing elective surgery between March 31, 2019, and June 20, 2025, were extracted from a standardized research data pipeline within 4 weeks of the publication of the MHRA report. Patients were screened for MR opioid prescriptions in the postoperative period and at hospital discharge. Counts and proportions of encounters in which MR opioids were administered or prescribed were evaluated across the study period. Reflections on the application of the infrastructure for this purpose were also documented. Results: Of 126,882 elective surgeries screened, 102,879 (81.1%) met the eligibility criteria. Over the study period, patients received a new MR opioid prescription after 7525 (7.3%) of the 102,879 eligible encounters, with 2438 (2.4%) encounters receiving a new MR opioid prescription at hospital discharge. Postoperative administration of MR opioids and prescribing at discharge have declined since 2020. As a result of this study, a new context-aware alert system was developed to monitor and reduce MR opioid prescribing in this surgical cohort. Reflections on the implementation experience demonstrated how collaboration between clinical and research teams in conjunction with integrated and seamless research pipelines allowed rapid knowledge generation. Key issues raised were the difficulty of validation between parallel data extraction systems and how the two different teams compared nonequitable data points and results. Conclusions: Mature digital and analytical infrastructure within health care institutions can enable swift evaluation of local practices in the context of national medication safety alerts. This can shorten action response times and improve patient care but requires close collaboration between clinicians and research teams. Shared infrastructure between teams across the learning health system improves data quality and provides easy access to the key users. Further work is needed to understand the benefits and challenges of infrastructure built for other use cases and the effectiveness of the intervention.
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Virtual Reality–Based Relaxation Training and Symptom Improvement Among Inpatients With Depressive Disorders: Retrospective Nonrandomized Comparative Study

Background: Virtual reality (VR) is increasingly used for adjunctive relaxation training in psychiatric care. However, evidence remains limited among hospitalized patients with depressive disorders, particularly in routine inpatient settings in China, and little is known about whether improvement varies by session frequency. Objective: This retrospective study examined whether adjunctive VR-based relaxation training was associated with changes in depressive and anxiety symptoms among inpatients with depressive disorders and whether improvement differed by session frequency. Methods: We conducted a retrospective, nonrandomized natural-group comparison using complete anonymized medical records from patients hospitalized in Lishui Second People’s Hospital between January 1 and December 31, 2022. Patients met () diagnostic criteria for depressive episodes or recurrent depressive disorders and were screened using predefined criteria. The analytic sample included 133 inpatients: 63 (47.4%) received adjunctive VR-based relaxation training plus usual care and 70 (52.6%) received usual care only. Usual care included pharmacotherapy and physiotherapy. The VR intervention consisted of 25-minute immersive relaxation sessions delivered approximately 3 times per week. Symptoms were assessed at admission and discharge using the 17-item Hamilton Depression Scale and Hamilton Anxiety Rating Scale. Response was defined as a reduction of 50% or more from baseline, and remission was defined as a total score of 7 or less. Baseline characteristics, outcome scores, response and remission rates, and exploratory session-frequency subgroups were compared. All analyzed variables were checked against complete medical records; no missing values were identified, and no imputation was performed. Results: The VR and control groups did not differ significantly in baseline depressive or anxiety scores. At discharge, adjunctive VR-based relaxation training was associated with lower depressive and anxiety symptom scores than usual care alone. The VR group also showed higher response rates for both depressive and anxiety symptoms and a higher anxiety remission rate, whereas depression remission was similar. Exploratory session-frequency analyses suggested that anxiety improvement may be more consistently associated with VR exposure than depression remission; however, the pattern was not strictly linear and should be interpreted cautiously because treatment frequency was linked to hospitalization duration and routine care factors. Conclusions: This study is innovative in evaluating structured VR-based relaxation training as an adjunct to routine inpatient depression care and in providing preliminary observations on session-frequency patterns in a real-world Chinese psychiatric setting. Unlike many previous VR studies conducted in noninpatient, nonclinical, or short-term experimental contexts, this study reflects everyday clinical practice among hospitalized patients with depressive disorders. The findings contribute practical evidence for integrating immersive relaxation into comprehensive inpatient care, particularly when additional anxiety relief is desired. Because the study was retrospective and nonrandomized, the findings indicate associations rather than causal effects and should be confirmed in prospective randomized controlled trials.
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Access to an mHealth Tool for Symptom Management in Pediatric Oncology Care: Triangulation Study

Background: Digital health offers opportunities to facilitate symptom assessments and communication for children with cancer, particularly after discharge. However, access to these tools must be established to ensure that they effectively support the user. PicPecc (Pictorial Support in Person-Centered Care for Children) is a mobile health tool developed to enable children to remotely assess symptoms and communicate with health care professionals. Understanding access to PicPecc is essential for evaluating its use in pediatric oncology. Objective: The aim was to test a digital intervention with PicPecc in pediatric oncology care through the lens of access to technology. Methods: This study uses a triangulation approach to determine access to digital technology through an intervention, PicPecc outside hospital. Fourteen children (6‐17 y), 5 parents, and 6 nurses from 2 pediatric oncology units in Sweden participated. Children were encouraged to use PicPecc for 2 weeks (achieving a median of 14, IQR 9.75-16 days) following hospital discharge to assess pain, nausea, sleep disturbances, and feelings using an assessment scale, pictures, personal notes, and a chat function. Nurses monitored assessments and responded via the administrative interface. Access was analyzed through interviews and an instrument, and by recording the consumption of PicPecc. Data analysis was based on the 5 dimensions of access (availability, accessibility, accommodation, affordability, and acceptability). Results: The intervention, PicPecc outside hospital, supported availability by enabling children to communicate symptoms in a safe and structured way. Children and parents mentioned feeling safe when they were discharged from the hospital, and nurses perceived it as a valuable complement to follow-up after discharge. PicPecc outside hospital was generally accessible, although initial challenges with log-in procedures related to the PIN code were common. Barriers related to accommodation included interpreting the scale and obtaining an overview of assessments. Affordability was high, as internet access and device availability were not barriers; however, children’s motivation varied depending on symptom burden. Acceptability was strong among children up to 12 years of age, who appreciated the design and gaming function, while the older children found the visual design less age-appropriate. Conclusions: Access to the mobile health tool, PicPecc outside hospital, appears promising for supporting remote symptom assessment in pediatric oncology, particularly among children up to 12 years of age. However, identified barriers, such as motivational factors and integration into the health care system, need to be addressed.
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STAT+: Lawmakers urge HHS to force Eli Lilly to provide 340B drug discounts to hospitals

Dozens of congressional lawmakers are urging the Trump administration to force Eli Lilly to reinstate mandated price breaks to hospitals that participate in a federal drug discount program but have refused to provide the company with claims data.

In a letter to Department of Health and Human Services Secretary Robert F. Kennedy Jr., the bipartisan group of lawmakers argued that Lilly is failing to comply with federal law by eliminating the price breaks. The drugmaker stopped offering discounts last month to reduce what it calls duplicate discounts paid to the hospitals.

At the time, Lilly targeted 50 larger hospital systems among approximately 1,000 hospitals that had not complied with a new policy that was announced this year. The company maintained that roughly 70% of the hospitals that participate in the discount program, or more than 2,300, had previously provided claims data.

Continue to STAT+ to read the full story…

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Elixirgen Builds Rare Disease Pipeline Around Telomere Biology Disorders and DMD

During the 2026 BIO International convention in San Diego, GEN sat down with Aki Ko, CEO of Elixirgen Therapeutics, to discuss the company’s multi-platform technology development efforts. The company, which was founded in 2017, is developing what it believes are breakthrough technologies that target telomere biology disorders (TBD) and aging as well as mRNA-based therapies.

Its therapy for addressing telomere biology disorders, based on its proprietary ZSCAN4 approach, is the furthest to the clinic. The biotech company will also target other aging-related diseases with the technology. Meanwhile, its efforts in the mRNA space are currently focused on Duchenne Muscular Dystrophy (DMD), although there are plans to pursue other targets there as well. 

Ko founded the company with CSO Minoru Ko, MD, PhD, in 2017. Elixirgen’s 15 employees are based in Baltimore in its office space in the Johns Hopkins Medical campus, although there is no affiliation with the university. This location offers some advantages to the company, according to Ko. Specifically, “we have a wet lab and an animal lab” that has “helped us go from in vivo to in vitro very quickly to test concepts or optimize formulations and things like that.” 

Recently, Elixirgen announced an option agreement with Japan’s Nippon Shinyaku focused on DMD. Under the terms of the agreement, Elixirgen will be responsible for the development of an asset dubbed EXG-7001, a locally administered, full-length dystrophin mRNA therapeutic that is currently in preclinical development for the treatment of DMD.

As part of the deal, Nippon Shinyaku will provide funding for the developmental costs of the therapy. Meanwhile, Elixirgen will receive an upfront payment and is eligible to receive additional development and sales-based milestone payments if the option were to be exercised. Also, Nippon Shinyaku may obtain exclusive worldwide rights to commercialize EXG-7001.

“Current approaches for treating DMD focus on delivering or restoring an incomplete dystrophin protein, and there still remains a significant unmet need for a therapy that can successfully deliver a full-length dystrophin protein,” Ko said in comments about the announcement. “By design, EXG-7001 has the potential to deliver the full-length, complete dystrophin protein that is missing in DMD patients, regardless of their genetic mutation.”

EXG-7001 leverages one of Elixirgen’s core technologies. The company has developed a platform for delivering mRNA-based therapies that it claims addresses the major delivery limitations of current methods. “The key features are that it is a lipid nanoparticle-free, localized mRNA therapeutics platform,” Ko explained to GEN. With this approach, “we’re avoiding some of the complications of gene therapies and delivering genes systemically by going local” and avoiding liver accumulation, which remains “a big issue” for mRNA therapeutics. 

The system has two components. The first component, called RNA tether, is designed to ensure that the RNA stays in the tissue that is injected without migrating to the liver. The second component is the mRNA cargo itself, which the company calls Bobcat® mRNA. Though the lead indication for this technology is DMD, there are other diseases involving large genes that the company could target. 

“We’re able to express the full length protein as mRNA as a single strand” and “it stays where you administer it, which is kind of unusual,” Ko said. Combining RNA tether and Bobcat makes it possible to express large genes and localize them to target tissues even without accumulation in the liver. Preclinical data has demonstrated its effectiveness in mice with no safety concerns associated with administration or treatment. “A full length dystrophin being given to kind of key muscles could potentially change quality of life,” particularly for the non-ambulatory population, Ko said. 

Beyond EXG-7001, Elixigen has other candidates in its pipeline that are much closer to the clinic. Its lead candidate is currently in Phase I/II testing at Cincinnati Children’s Hospital Medical Center. This is an ex vivo cell therapy based on the company’s ZSCAN4 technology, which is designed to extend the telomeres of stem cells in “a controlled way” using a telomerase-independent mechanism. EXG-34217 is comprised of autologous CD34+ hematopoietic stem cells that have been treated ex vivo with EXG-001, a non-integrating, non-transmissible, temperature-sensitive Sendai virus vector encoding human ZSCAN4.

The features of that technology were identified by the company’s CSO and his team while he worked at the National Institutes of Health’s National Institute on Aging. In 2024, the U.S. Food and Drug Administration granted Rare Pediatric Disease Designation to the treatment, dubbed EXG-34217, for the treatment of patients with dyskeratosis congenita and related telomere biology disorders. 

“Telomeres obviously have a relationship with aging, and there are in fact genetic diseases associated with short telomeres and telomerase mutations,” CEO Ko told GEN at BIO. People with TBDs are “born with shorter telomeres typically, but also have a mutation in their telomerase so they are not necessarily maintaining them either.” The result is a type of premature aging, so conditions like bone marrow failure and cytopenia happen earlier in the life of the patient. In fact, “bone marrow failure is one of the largest issues” affecting both adults and young children, CEO Ko said. 

One treatment option in these cases is allogeneic hematopoietic stem cell transplantation (HSCT), he continued. However, people with short telomeres have more fragile genomes that are less resistant to chemotherapy and radiotherapy and are at greater risk of cancer even after HSCT treatment. In an ideal scenario, it would be possible to postpone or avoid HSCT for these patients, and the company’s ZSCAN4-based therapy could make it possible to do that. 

The treatment is currently being tested in adult and pediatric patients in Cincinnati. “We started in adults because this is first-in-human,” but the disease is also very severe in children, Ko said. “Our target ultimately is to make sure as many people with TBDs can get this if they need it.” Early clinical results published in 2025 in a paper in NEJM Evidence show durable telomere extension overall with no treatment-related safety concerns observed over a 24-month and 5-month period after infusion. The trial has been going on for some time, and “we have a lot of longer-term data now” and are “looking toward potential accelerated approval.”

But targeting TBDs is just one indication. “Short telomeres manifest in many different ways,” Ko said. Other potential targets for the company’s technology are aging-related diseases, including things like idiopathic pulmonary fibrosis. 

To date, Elixirgen has raised roughly $34 million from existing investors.

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