Unmet Medical Needs Research Articles

Flexible Development Programs for Antibacterial Drugs to Address Unmet Medical Needs.

The US Food and Drug Administration recognizes the unmet medical need for antibacterial drugs to treat serious bacterial diseases caused by resistant pathogens for which effective therapies are limited or lacking. The agency also recognizes that designing and conducting clinical trials to assess the safety and efficacy of drugs to treat resistant infections is challenging, especially for drugs only active against a single or a few bacterial species, and that a more flexible development program might be appropriate. In this article, we discuss several regulatory considerations for flexible development programs for antibacterial drugs intended to meet an unmet medical need. As an example, we use the recent approval of sulbactam for injection and durlobactam for injection (XACDURO) for the treatment of hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia caused by susceptible isolates of Acinetobacter baumannii-calcoaceticus complex.

Rural Alabamians' Unmet Medical Needs: Rethinking the Roles of Online Health Information-Seeking Behaviors.

Unmet medical needs in rural areas are of grave concern in the U.S. With the advent of digital technologies, the Internet has become a critical means for accessing essential health information. However, racial/ethnic minority rural communities experiencing scarcity in healthcare services and access to the Internet are underrepresented in digital health studies. This study examined the association between online health information-seeking behaviors and unmet medical needs in a sample of African/Black American adults living in a rural region of the U.S. Among a sample of 191 adults, we used descriptive analyses to document the level of unmet medical needs and online health information-seeking behaviors of this population and conducted logistic regressions to test the association between online health information-seeking behaviors and unmet medical needs. Most participants were older than 50years old (60.2%), female (68.1%), unemployed (57.6%), and had an annual income of less than $25,000 (60.2%). About 20% of participants experienced unmet medical needs. The mean score of online health information-seeking behaviors was 2.37 (range 0-12). Increasing online health information-seeking behaviors was associated with 5.95 increased odds of experiencing unmet medical needs (OR = 5.95, 95% CI 1.27-27.77). The finding highlights that it is necessary to develop targeted programs aimed at populations with high unmet medical needs, focusing on providing accessible health information and resources. Further research is warranted to investigate the motivations to engage in online health information-seeking behaviors to inform structural and workforce interventions to address unmet medical needs in this under-resourced region.

Assessing Unmet Social and Medical Needs among Latinx in Arizona throughout the COVID-19 Pandemic: Time-Varying Patterns by Social Determinants of Health.

The pandemic exacerbated unmet social and medical needs, disproportionately impacting Latinx communities. This article examines how unmet needs varied from February 2021 to April 2023 among 428 Latinx individuals. Multiple-to-multiple path analysis models of cross-sectional surveys showed significant increases in unmet social (β = 0.17, p = .001) and medical (β = 0.12, p = .001) needs over time, with variations by age, language, and health insurance. Initially, older adults reported more unmet medical needs, but later, younger adults reported higher needs (β=-0.16, p = .001). Among Spanish speakers, both social (β = 0.24, p = .001) and medical (β = 0.20, p = .003) needs increased significantly. Uninsured individuals had significant increases in unmet social (β = 0.20, p = .001) and medical (β = 0.16, p = .013) needs, while those with public insurance experienced decreased social needs (β=-0.12, p = .017) compared to privately insured. These findings highlight growing disparities within the Latinx community, underscoring the need to prioritize vulnerable populations in public health responses and monitor program impacts for equitable access to essential services.

Natural Language Processing–Assessed Unmet Medical and Social Needs in Cancer Crowdfunding Stories

Natural Language Processing–Assessed Unmet Medical and Social Needs in Cancer Crowdfunding Stories

Association Between Objective Social Isolation and Unmet Medical Needs: A Nationwide Cross-sectional Study in Korea.

The aim of this study was to analyze the relationship between objective social isolation (SI) and unmet medical needs (UMN) in adults aged 19 and older. A cross-sectional analysis was conducted of 208 619 adults aged 19 and older, excluding missing data, using the 2019 Korea Community Health Survey. To analyze the association between objective SI and UMN, the chi-square test and logistic regression analysis were performed. The prevalence of UMN was 1.14 times higher (odds ratio [OR], 1.14; 95% confidence interval [CI], 1.06 to 1.23) among those with SI than among those without SI, and the OR for groups with 5 SI types was 2.77 (95% CI, 1.86 to 4.12) compared to those with no SI types. In addition, a stratified analysis by age group showed that the association between SI and UMN existed even in groups under 64 years old. However, among those aged 65 and older, SI was associated with an OR of 1.53 (95% CI, 1.37 to 1.71) for UMN compared to non-SI. As the number of SI types increased, the prevalence of UMN also increased, indicating a strong association between SI and UMN in older adults. This study found that individuals with SI experienced UMN due to fear and anxiety about interpersonal relationships. Therefore, based on the results of this cross-sectional study, it is necessary to investigate the causal relationship between SI and UMN through future longitudinal data.

Lesbian, Gay, Bisexual, and Other Sexual Minority Adults in the United States and Their Unmet Medical Needs and Telehealth Use Due to the COVID-19 Pandemic in 2020

Sexual minority adults—including lesbian, gay, bisexual, queer/questioning, and other sexual minorities (LGBQ+)—face demonstrated barriers to employment, health insurance, and healthcare access. We sought to assess the association between sexual orientation, unmet medical needs, and telehealth use due to the COVID-19 pandemic using the National Health Interview Survey (2020). Using multivariable analyses, we assessed the likelihood of having unmet medical needs and telehealth use in LGBQ+ adults and heterosexual adults, adjusting for socioeconomic, demographic, and clinical factors. We found that LGBQ+ individuals had a significantly higher likelihood of having any unmet medical needs or unmet non–COVID-related medical needs due to the pandemic compared with heterosexual individuals. The LGBQ+ individuals were more likely to use telehealth than their heterosexual counterparts—for both general and pandemic-related reasons. Our findings indicate that disastrous events such as the COVID-19 pandemic likely exacerbate existing healthcare access disparities faced by LGBQ+ individuals. This warrants further research to explore the interventional potential of telehealth for reducing healthcare access disparities, especially for those living in geographic regions lacking culturally competent providers.

Association between Perceived Activity Restriction Due to People's Perception of Aging and Unmet Medical Needs among Middle-Aged and Elderly People: A Population-Based Study.

Background and Objectives: The older members of a population might experience unmet medical needs, despite desiring medical care due to activity limitations driven by their perception of aging. This study conducted a cross-sectional analysis of the association between perceived activity restriction (PAR) due to people's own perception of aging and unmet medical needs (UMN) in late middle-aged and older Koreans based on the Korean National Health and Nutrition Examination Survey (KNHANES). Materials and Methods: The 2016-2020 KNHANES was used to analyze a total of 2008 participants among groups aged 45 years or older by applying individual weights imposed from the raw data. The independent variable of PAR was assessed using self-reported questionnaires based on the global activity limitation indicator. Also, the dependent variable of UMN, referring to the state in which a patient's medical care or service was insufficient, inadequate, or lacking, was assessed using a single question. After excluding missing values, the data on 2008 individuals were analyzed using a chi-square test, weighted logistic regression, and a stratified analysis of gender, age, and the presence of chronic illnesses. Results: The group that experienced PAR had an OR 2.13 higher (odds ratio [OR]: 2.13; 95% confidence interval [CI]: 1.27-3.56) to present UMN than the group that did not experience PAR. Furthermore, the results of the stratified analysis indicated that, in the group of female participants with chronic illness and in the group of elderly people, experiencing PAR was associated with a higher experience of UMN. Conclusions: There was a close association between PAR and UMN. In particular, when PAR occurred in the group of female participants with chronic illness and in the group of elderly people, the incidence rate of UMN was also found to be high. This finding highlights the need for policies and institutional measures to reduce UMN within vulnerable groups with an increased risk of medical inaccessibility due to activity restriction.

The PedAL/EuPAL Project: A Global Initiative to Address the Unmet Medical Needs of Pediatric Patients with Relapsed or Refractory Acute Myeloid Leukemia.

The prognosis of children with acute myeloid leukemia (AML) has improved incrementally over the last few decades. However, at relapse, overall survival (OS) is approximately 40-50% and is even lower for patients with chemo-refractory disease. Effective and less toxic therapies are urgently needed for these children. The Pediatric Acute Leukemia (PedAL) program is a strategic global initiative that aims to overcome the obstacles in treating children with relapsed/refractory acute leukemia and is supported by the Leukemia and Lymphoma Society in collaboration with the Children's Oncology Group, the Innovative Therapies for Children with Cancer consortium, and the European Pediatric Acute Leukemia (EuPAL) foundation, amongst others. In Europe, the study is set up as a complex clinical trial with a stratification approach to allocate patients to sub-trials of targeted inhibitors at relapse and employing harmonized response and safety definitions across sub-trials. The PedAL/EuPAL international collaboration aims to determine new standards of care for AML in a first and second relapse, using biology-based selection markers for treatment stratification, and deliver essential data to move drugs to front-line pediatric AML studies. An overview of potential treatment targets in pediatric AML, focused on drugs that are planned to be included in the PedAL/EuPAL project, is provided in this manuscript.

Prevalence and Correlates of Unmet Medical and Social Needs in Virginia's Medicaid Managed Long Term Services and Supports Program.

Medicaid Long Term Services and Support (LTSS) programs serve individuals with complex medical and social needs. Increasingly, state Medicaid programs are contracting with managed care organizations to administer LTSS programs. Understand the prevalence of and risk factors for unmet medical and social needs among a sample of patients within a Medicaid managed LTSS program. We surveyed a cross-sectional random sample of 798 community-residing individuals over 21 in Virginia who were served by the state Medicaid managed LTSS program. Outcomes of interest include 3 distinct medical needs: medical appointments, medical transportation, and prescriptions; 4 distinct social needs: housing security, food security, utility bills, and nonmedical transportation, and composite measures of unmet social and medical needs. We found that 12.5% of our sample had any unmet medical need, while far more (62.2%) of our sample had any unmet social needs, with food insecurity being the most common. We found that members of color had almost 2 times the odds of having both unmet social and medical needs [social: adjusted odds ratio (aOR): 2.21; 95% confidence Interval (CI): (1.59, 3.09); medical aOR: 2.25 ; 95% CI: (1.34, 3.8)]. Medicaid members may not be fully realizing the potential of LTSS programs and would benefit from both Medicaid agency and managed care organizations' strategies aimed at addressing social drivers of health. To achieve health equity for LTSS members of color, Medicaid agencies may consider policies specifically targeting racial disparities.

PCR40 Unmet Medical Needs and Treatment Preferences for Systemic Treatments in Patients with Moderate to Severe Psoriasis: A Cross-Sectional Survey Study in China

PCR40 Unmet Medical Needs and Treatment Preferences for Systemic Treatments in Patients with Moderate to Severe Psoriasis: A Cross-Sectional Survey Study in China

Unmet Medical Needs Amongst Older Patients Aged ≥60 Years with Newly Diagnosed Acute Myeloid Leukemia Receiving Intensive Chemotherapy: Experience from a Predominately United States Community Setting

Background: Venetoclax (Ven) in combination with hypomethylating agents is approved for treatment in patients (pts) with newly diagnosed (ND) acute myeloid leukemia (AML) who are ≥75 years old or unfit for intensive chemotherapy (IC), based on the longer median overall survival (OS) with Ven+azacitidine (Aza) compared with Aza in combination with placebo in the Phase III VIALE-A study (NCT02993523). Several studies are now evaluating Ven-based regimens in pts eligible for IC, for example Ven+Aza compared with IC (NCT04801797). Additionally, Ven in combination with cladribine (CLAD) and low-dose cytarabine (LDAC) is currently under investigation in a Phase II single-arm study (NCT03586609) amongst older pts (aged ≥60 years) with ND AML. An interim analysis of this trial evaluated 60 pts where complete remission (CR) and CR with incomplete count recovery (CRi) was observed in 93% of pts, and the median OS, overall and by transplant status, was not reached; 1-year OS was 72.9% and 2-year OS was 63.5% (Kadia et al. J Clin Oncol 2022). The aim of this study is to better understand treatment outcomes amongst older pts (aged ≥60 years) with ND AML receiving IC in a routine clinical care setting in the United States (US). Methods: This was a retrospective cohort study using the Flatiron Health electronic health record-derived, US nationwide, de-identified database. Pts aged ≥60 years with ND AML who initiated IC within 60 days of AML diagnosis (Dx) between January 1, 2014, and November 30, 2022, with ≥2 visits recorded within 3 months of AML Dx, and ≥3 months of follow-up before study end date (February 28, 2023) were included. Clinical trial participants and pts with acute promyelocytic leukemia or core binding factor were excluded. Descriptive analysis on patient characteristics was conducted. The proportion of pts who achieved real-world response (defined as <5% bone marrow [BM] blasts), real-world CR/CRi (defined as <5% BM blast with either platelet count <100 x 10 9/L or absolute neutrophil count <1000 x 10 9/L) and received a stem cell transplant (SCT) post-remission were reported. Thirty-day mortality, 60-day mortality, 1-year real-world OS (rwOS), and 2-year rwOS were evaluated using Kaplan-Meier (KM) analyses. The starting points for KM analyses were IC treatment initiation (all pts), SCT (pts who underwent SCT post-remission), and remission (pts who did not undergo SCT post-remission). Pts were censored at the last activity date or SCT date (whichever occurred earliest) for the overall patient cohort, and the last activity date for pts with or without SCT post-remission. Results: A total of 1105 pts met the study criteria. The median (IQR) age was 67 (64-71) years, 57% were male, 27% had secondary AML, 48% had BM blast >50% at Dx, and 66% were treated in the community setting. Excluding 340 pts with unknown cytogenetic and molecular data, 19%, 32%, and 48% of pts had European LeukemiaNet 2017 favorable, intermediate, and adverse risk, respectively. At a median (IQR) follow-up of 13 (6-28) months from AML Dx, 74% (n=821) of pts achieved real-world response, including 66% (n=724) who achieved real-world CR/CRi. For all pts, the 30-day mortality was 3%, 60-day mortality was 6%, 1-year rwOS was 58%, 2-year OS was 34%, and the median rwOS was 14.3 months ( Figure A). A total of 37% (n=306/821) of pts underwent SCT post-remission; 98% (n=300/306) underwent allogeneic-SCT and 2% (n=6/306) underwent autologous-SCT. The median rwOS was 40.4 months for pts undergoing SCT post-remission, and 13.8 months for pts without SCT post-remission ( Figure B). Conclusions: In this large real-world data study, we observed older pts (aged ≥60 years) with ND AML receiving IC treatment had a median OS of <14 months and <35% of pts remained alive 2 years after IC initiation. Findings from this study suggest the need for other novel treatment options, including Ven based regimens, to improve OS in older pts (aged ≥60 years) with ND AML who are eligible for IC. Future studies are warranted to conduct randomized controlled trials to comparatively evaluate IC vs other novel treatment options in older pts (aged ≥60 years) with ND AML, or conduct the appropriate statistical adjustment to minimize confounding in indirect treatment comparisons from different studies.

PCR214 Severe Sialorrhea: Patient Journey and Related Costs Reveal Unmet Medical Needs

Severe sialorrhea (drooling) can be observed in up to 60% of children with chronic neurological disorders. As little information is available on the management and costs of patients with severe sialorrhea, this study aimed at better understanding the care pathway and related costs associated with the management of this condition in France.

Essential Thrombocythemia in Adolescents and Young Adults: Clinical Aspects, Treatment Options and Unmet Medical Needs.

Current treatment of essential thrombocythemia (ET) should primarily prevent thrombo-hemorrhagic events, without increasing the rate of fibrotic progression or leukemic evolution, and secondarily control microvascular symptoms. Unlike other classic BCR::ABL1-negative myeloproliferative neoplasms, ET is frequently diagnosed in adolescents and young adults (AYA), defined as individuals aged 15 to 39years, in up to 20% of patients. However, since the current risk stratification of this disease is based on models, including that of ELN, IPSET-Thrombosis and its revised version, mainly applied to an older patients' population, international guidelines are needed that specifically consider how to evaluate the prognosis of AYAs with ET. Furthermore, although ET is the most frequent MPN among AYA subjects, there is a lack of specific recommendations on how to treat it in this subgroup of patients, as management decisions are typically extrapolated from those for the elderly. Accordingly, since AYAs with ET represent a unique disease subset defined by attenuated genetic risk, more indolent phenotype, and longer survival than their older counterparts, treatment selection requires special attention to specific issues such as the risk of fibrotic/leukemic transformation, carcinogenicity, and fertility. This review article will provide a comprehensive overview of the diagnosis, prognostic stratification, and possible therapeutic approaches for AYA patients with ET, including antiplatelets/anticoagulants and cytoreductive agents, with a focus on pregnancy management in real-life clinical practice.

Strategy for Pre-Clinical Development of Active Targeting MicroRNA Oligonucleotide Therapeutics for Unmet Medical Needs.

We present here an innovative modular and outsourced model of drug research and development for microRNA oligonucleotide therapeutics (miRNA ONTs). This model is being implemented by a biotechnology company, namely AptamiR Therapeutics, in collaboration with Centers of Excellence in Academic Institutions. Our aim is to develop safe, effective and convenient active targeting miRNA ONT agents for the metabolic pandemic of obesity and metabolic-associated fatty liver disease (MAFLD), as well as deadly ovarian cancer.

Nirmatrelvir/ritonavir in COVID-19 patients with haematological malignancies: a report from the EPICOVIDEHA registry

Nirmatrelvir/ritonavir treatment decreases the hospitalisation rate in immunocompetent patients with COVID-19, but data on efficacy in patients with haematological malignancy are scarce. Here, we describe the outcome of nirmatrelvir/ritonavir treatment in a large cohort of the latter patients. This is a retrospective cohort study from the multicentre EPICOVIDEHA registry (NCT04733729) on patients with haematological malignancy, who were diagnosed with COVID-19 between January and September 2022. Patients receiving nirmatrelvir/ritonavir were compared to those who did not. A logistic regression was run to determine factors associated with nirmatrelvir/ritonavir administration in our sample. Mortality between treatment groups was assessed with Kaplan-Meier survival plots after matching all the patients with a propensity score. Additionally, a Cox regression was modelled to detect factors associated with mortality in patients receiving nirmatrelvir/ritonavir. A total of 1859 patients were analysed, 117 (6%) were treated with nirmatrelvir/ritonavir, 1742 (94%) were treated otherwise. Of 117 patients receiving nirmatrelvir/ritonavir, 80% had received ≥1 anti-SARS-CoV-2 vaccine dose before COVID-19 onset, 13% of which received a 2nd vaccine booster. 5% were admitted to ICU. Nirmatrelvir/ritonavir treatment was associated with the presence of extrapulmonary symptoms at COVID-19 onset, for example anosmia, fever, rhinitis, or sinusitis (aOR 2.509, 95%CI 1.448-4.347) and 2nd vaccine booster (aOR 3.624, 95%CI 1.619-8.109). Chronic pulmonary disease (aOR 0.261, 95%CI 0.093-0.732) and obesity (aOR 0.105, 95%CI 0.014-0.776) were not associated with nirmatrelvir/ritonavir use. After propensity score matching, day-30 mortality rate in patients treated with nirmatrelvir/ritonavir was 2%, significantly lower than in patients with SARS-CoV-2 directed treatment other than nirmatrelvir/ritonavir (11%, p=0.036). No factor was observed explaining the mortality difference in patients after nirmatrelvir/ritonavir administration. Haematological malignancy patients were more likely to receive nirmatrelvir/ritonavir when reporting extrapulmonary symptoms or 2nd vaccine booster at COVID-19 onset, as opposed to chronic pulmonary disease and obesity. The mortality rate in patients treated with nirmatrelvir/ritonavir was lower than in patients with targeted drugs other than nirmatrelvir/ritonavir. EPICOVIDEHA has received funds from Optics COMMIT (COVID-19 Unmet Medical Needs and Associated Research Extension) COVID-19 RFP program by GILEAD Science, United States (Project 2020-8223).

Unmet Medical Needs In Short Bowel Syndrome With Intestinal Failure Identified From A Qualitative Survey Of Patients And Healthcare Providers

Unmet Medical Needs In Short Bowel Syndrome With Intestinal Failure Identified From A Qualitative Survey Of Patients And Healthcare Providers

Community Health Collaborative Facilitates Health System and Community Change to Address Unmet Medical and Social Needs in New Jersey.

Community Health Collaborative Facilitates Health System and Community Change to Address Unmet Medical and Social Needs in New Jersey.

Identifying the Group Vulnerable to Unmet Medical Needs Due to Food Security: According to Children in the Household.

Adults may experience unmet medical needs for various reasons. The purpose of this study was to examine the effect of food security on unmet medical needs according to the presence of children in the household of adults, as well as to identify the medically vulnerable group considering individual and household characteristics. This study was conducted using data from the National Health and Nutrition Examination Survey for 2013-2015 and 2019-2020. The subjects of the study were 23,069 adults 19 years of age or older, and were divided into two groups according to whether or not children were included in the household. In order to observe the association between food security and unmet medical needs, multiple logistic regression analysis was performed. In addition, a subgroup analysis was performed in consideration of individual and household characteristics. When food security was unstable for households with children, or without children, there was a high correlation with unmet medical needs. Considering individual and household characteristics, in groups with lower age and household income level, or higher number of members in household and subjective health status, food security was strongly correlated with unmet medical needs in households with children. Contrarily, households without children showed a high correlation in the opposite characteristics of households with children, excluding household income level. Food security was highly correlation with unmet medical needs regardless of whether or not children were included in the household. However, according to the individual and household characteristics of households with and without children, the relationship between food security and unmet medical needs was found to be different. Therefore, it is necessary to prepare a health policy that can increase access to medical services in consideration of food security and individual and household characteristics depending on whether or not children are included in the household.

Modeling Approaches to Reimagine Stability (MARS) for Enabling Earlier Access to Critical Drugs for Patients with Unmet Medical Needs

FDA's experience to date has shown that completion of stability data requirements is one of the most observed challenges for applicants of New Drug Applications (NDAs) with an expedited review designation. Since NDAs submitted under these expedited pathways often have limited available real-time stability data from the primary batches, Modeling Approaches to Reimagine Stability (MARS) have been proposed to support establishment of tentative retest periods of the drug substance and/or expiration dating period (shelf-life) of the drug product. MARS incorporate statistical principles and available tools as a part of the predictive models. In this study, a data mining exercise has been conducted with regulatory submissions of Investigational New Drug (IND) Applications, NDAs, and Abbreviated New Drug Applications (ANDAs) containing MARS data. The case studies presented herein demonstrate how MARS data has been applied to regulatory scenarios involving prediction of retest and/or shelf-life, bridging major development changes, and confirming that no degradation has been observed or predicted. Using the assumption of a linear time trend for those cases that do not display sufficient degradation to conduct MARS for projection of an expiration date, an analysis of covariance (ANCOVA) model is developed and described herein to test the hypothesis of zero slope by a p-value method. Our results show that the application of MARS adequately supported establishment of a tentative commercially viable retest date/shelf-life, thus enabling earlier access to critical drugs for patients with unmet medical needs.

Identifying the Unmet Medical Needs of HIV-Positive Subjects in Korea: Results of a Nationwide Online Survey.

An online survey was conducted in Korea to identify the unmet medical needs of people living with human immunodeficiency virus (HIV) (PLWH). Participants (n = 105) were mostly male (93.3%), aged >40 years (75.2%), and treated for ≥6 years post-diagnosis (61.9%). Most PLWH (71.4%) were very satisfied/satisfied with their HIV management. Areas of concern were quality of life (QoL) and mental health. Characteristics of a long-term therapeutic agent were 'low risk of resistance', 'high long-term viral suppression efficacy', and 'high degree of safety'. Pre-consultation QoL and mental health screening would be beneficial for the long-term success of HIV management.