Prescription Products Research Articles

503 Examination of Labeling for Geriatric Sub-Populations in Recently Approved Type 2 Diabetes Drugs

OBJECTIVES/GOALS: To assess labels of drugs approved for Type 2 Diabetes (T2D) for the inclusion of geriatric sub-population data (ages 65-74, 75-84, ≥85) since January 1, 2013, in accordance with international guidance and US regulations in recognition of an aging populations and global demographics. METHODS/STUDY POPULATION: Utilizing FDA Guidance for Industry: Labeling for Human Prescription and Biological Products - Implementing the Physician’s Labeling Rule (PLR) Content and Format Requirements and the International Council for Harmonization of Technical Requirements (ICH) E7 guidance “Studies in Support of Special Populations: Geriatrics” as reference for assessing labels. The Center for Drug Evaluation and Research (CDER) new drugs/biologic approvals database was filtered for drugs approved between Jan. 1, 2013 and Dec. 31, 2022 with approved T2D indications. Examined original drug labels and supplements from Drugs@FDA for geriatric use efficacy and safety wording in Section 8.5 (Use in Specific Populations, Geriatric Use), for labels. Subpopulation data in labeling for ages 65-74, 75-84, and ≥85 was analyzed. RESULTS/ANTICIPATED RESULTS: Seven T2D drugs (Trulicity, Tresiba, Adlyxin, Ozempic, Steglatro, Kerendia, Mounjaro) approved within the specified time period were analyzed. In the current examination, all labels contain information regarding efficacy differences between ages 65+ and 75+, however, none contain information on efficacy for ≥85 populations. Four of the seven drugs have been updated with increased data from further efficacy trials for older adults conducted after initial approval. The remaining three drugs have only been reworded, or not changed at all, with no further efficacy trials conducted. DISCUSSION/SIGNIFICANCE: This research shows the gap in representation of older adults in clinical trial data and T2D drugs’ labeling. Despite having a higher usage of T2D drugs compared to the general population, older adults and especially the oldest-old (≥85) are underrepresented. Additional demographic requirements ensuring diversity in clinical trials is needed.

Overview of popular cosmeceuticals in dermatology.

The eternal pursuit to prevent ageing and maintain a youthful appearance has resulted in a rapidly expanding cosmeceutical industry. Cosmeceutical products, particularly of natural origin, are in high demand due to claims of efficacy for signs of ageing and other skin conditions. Consumers often include cosmeceutical products in their skin care regime as they are readily available, and a more affordable option compared to prescription products. However, many cosmeceutical ingredients lack clinical evidence regarding their efficacy and safety as these products are not regulated by the U.S. Food and Drug Administration. This review provides a brief overview of several popular cosmeceutical ingredients with regards to their potential indications, targets and mechanisms of action.

504 - Investigator- and patient-rated local tolerability in phase 3 trials of topical roflumilast in patients with psoriasis, seborrheic dermatitis, and atopic dermatitis

Abstract Introduction Formulating a topical medication that does not irritate the skin is an important factor contributing to patient treatment adherence and satisfaction. Many topical prescription products use penetration enhancers (including propylene glycol, polyethylene glycol, and ethanol) to overcome barrier properties of the skin. However, these excipients may irritate the skin causing tolerability reactions such as burning and stinging, which can reduce patient treatment adherence. Topical roflumilast is a highly potent (Kd∼0.7 nM) phosphodiesterase 4 inhibitor formulated as a water-based cream or foam that does not contain penetration enhancers or fragrances. Objectives We present prospectively assessed investigator- and patient-rated local tolerability from Phase 3 trials of topical roflumilast for patients with psoriasis (DERMIS-1, DERMIS-2, ARRECTOR) seborrheic dermatitis (SD; STRATUM), and atopic dermatitis (AD; INTEGUMENT-1, INTEGUMENT-2). Methods Patients were randomized to apply topical roflumilast (DERMIS: 0.3% cream; ARRECTOR and STRATUM: 0.3% foam; INTEGUMENT: 0.15% cream) or vehicle once daily for 8 weeks (DERMIS, ARRECTOR, and STRATUM) or 4 weeks (INTEGUMENT). Investigators assessed local tolerability on an 8-point scale (0 [no evidence of irritation] to 7 [strong reaction spreading beyond application site]) in the clinic before investigational product (IP) application. Patients reported local tolerability on a 4-point scale (0 [none: no sensation] to 3 [severe: hot, tingling/stinging sensation that has caused definite discomfort]) in the clinic 10-15 minutes after IP application. Tolerability was also assessed by reviewing documented adverse events. Results As assessed by investigators, ≥96.5% of patients in the roflumilast-treated groups had no evidence of irritation at the application site across all trials at all timepoints. Patient-rated local tolerability was favorable and improved with treatment: across all trials, 1% of roflumilast-treated patients reported a score of 3 (severe; defined as a “hot tingling/stinging sensation that has caused definite discomfort”) after the first application (day 1) and <1% at each subsequent assessment. Rates of adverse events, including those at the application site, were low for all trials. Conclusions Roflumilast cream and foam formulations demonstrated favorable local tolerability based on investigator- and patient-rated assessments in patients with psoriasis, SD, and AD, including application to sensitive areas such as the face and intertriginous areas. ClinicalTrials.gov Identifiers: DERMIS-1: NCT04211363; DERMIS-2: NCT04211389; ARRECTOR: NCT05028582; STRATUM: NCT04973228; INTEGUMENT-1: NCT047​73587; INTEGUMENT-2: NCT04773600.

Investigator- and Patient-Rated Local Tolerability in Phase 3 Trials of Topical Roflumilast in Patients With Psoriasis, Seborrheic Dermatitis, and Atopic Dermatitis

Investigator- and patient-rated local tolerability in phase 3 trials of topical roflumilast in patients with psoriasis, seborrheic dermatitis, and atopic dermatitis
 
 Christopher G. Bunick1 on behalf of the authors
 
 INTRODUCTION: Formulating a topical medication that does not irritate the skin is an important factor contributing to patient treatment adherence and satisfaction. Many topical prescription products use penetration enhancers (including propylene glycol, polyethylene glycol, and ethanol) to overcome barrier properties of the skin. However, these excipients may irritate the skin causing tolerability reactions such as burning and stinging, which can reduce patient treatment adherence. Topical roflumilast is a highly potent (Kd~0.7 nM) phosphodiesterase 4 inhibitor formulated as a water-based cream or foam that does not contain penetration enhancers or fragrances. We present prospectively assessed investigator- and patient-rated local tolerability from Phase 3 trials of topical roflumilast for patients with psoriasis (DERMIS-1, DERMIS-2, ARRECTOR) seborrheic dermatitis (SD; STRATUM), and atopic dermatitis (AD; INTEGUMENT-1, INTEGUMENT-2).
 METHODS: Patients were randomized to apply topical roflumilast (DERMIS: 0.3% cream; ARRECTOR and STRATUM: 0.3% foam; INTEGUMENT: 0.15% cream) or vehicle once daily for 8 weeks (DERMIS, ARRECTOR, and STRATUM) or 4 weeks (INTEGUMENT). Investigators assessed local tolerability on an 8-point scale (0 [no evidence of irritation] to 7 [strong reaction spreading beyond application site]) in the clinic before investigational product (IP) application. Patients reported local tolerability on a 4-point scale (0 [none: no sensation] to 3 [severe: hot, tingling/stinging sensation that has caused definite discomfort]) in the clinic 10-15 minutes after IP application. Tolerability was also assessed by reviewing documented adverse events.
 RESULTS: As assessed by investigators, ≥96.5% of patients in the roflumilast-treated groups had no evidence of irritation at the application site across all trials at all timepoints. Patient-rated local tolerability was favorable and improved with treatment: across all trials, 1% of roflumilast-treated patients reported a score of 3 (severe; defined as a “hot tingling/stinging sensation that has caused definite discomfort”) after the first application (day 1) and <1% at each subsequent assessment. Rates of adverse events, including those at the application site, were low for all trials and <0.5% of roflumilast-treated patients discontinued due to adverse events at the application site across all trials.
 CONCLUSION: Roflumilast cream and foam formulations demonstrated favorable local tolerability based on investigator- and patient-rated assessments in patients with psoriasis, SD, and AD, including application to sensitive areas such as the face and intertriginous areas.
 Sponsored by Arcutis Biotherapeutics, Inc.
 ClinicalTrials.gov Identifiers: DERMIS-1: NCT04211363; DERMIS-2: NCT04211389; ARRECTOR: NCT05028582; STRATUM: NCT04973228; INTEGUMENT-1: NCT04773587; INTEGUMENT-2: NCT04773600

Prevalence and Characteristics of Medicinal Cannabis Use among Chronic Pain Patients; A Post- Legalization Study in a Tertiary Care Setting in Thailand

Objective: Cannabinoid products have been applied for numerous medical conditions, including chronic pain. Thailand was the first country in South East Asia to legalize medical cannabinoids. This study aims to explore prevalence, characters, attitude, side effects of medical cannabinoid use, and pain-related outcome among the chronic cancer and non-cancer pain population at Siriraj Hospital. Materials and Methods: 200 chronic cancer pain and 670 chronic noncancer pain patients were collected by questionnaires and interviews. Data included demographic data, clinical diagnosis, pain treatment, knowledge, attitude, pattern of use, side effects and quality of life of cannabinoid extracts. Results: Prevalence of active cannabis user was 15% in chronic cancer pain and 3.1% in noncancer pain. Oil extract sublingual was the most common form. Pain control was the most common initial reason for usage. No serious side effects were reported. Common side effects were dry oral mucosa, drowsiness, and headache. The most common source was obtained from friends. 36% of the patients believed they had enough understanding of medical cannabis, while 68.5% agreed that it is appropriate to use in Thailand. In cancer patients, the Edmonton Symptom Assessment System (ESAS) subscale for lack of appetite, anxiety, and subscale for a brief pain inventory (BPI) for enjoyment of life were higher among active users. In patients with noncancer pain, only the mood subscale BPI was lower among active users. Conclusion: Medical cannabis usage is common compared with general population in Thai patients with chronic pain and may be associated with increased pain interference and cancer-related symptoms. Nonmedical license prescription and nonmedical license cannabis products were common in Thailand.

Insights into acne and the skin barrier: Optimizing treatment regimens with ceramide-containing skincare.

Acne is a common, complex, multifactorial inflammatory skin disease associated with epidermal barrier dysfunction. Beginning in childhood, acne affects many adolescents and adults. Acne is associated with lower self-esteem, anxiety, and depression and may cause scars and pigmentary sequelae. The review explores the relationships between acne and the skin barrier function and discusses nuances in the prevention, treatment, and maintenance of acne and its impact on the skin barrier. The advisors' previous publications addressed prescription and nonprescription pediatric acne treatment and skincare using cleansers, moisturizers, and a practical algorithm for treatment and maintenance, including skincare recommendations for pediatric acne patients and an algorithm for skin of color patients with acne. Before the meeting, literature was culled on the relationship between the skin barrier and acne and current best practices in acne, addressing prescription and nonprescription acne products and skincare as monotherapy, adjunctive, and maintenance treatment. After discussing 13 draft statements, the advisors applied the selected literature and drew from their clinical knowledge and experience, and agreed on five statements. The follicular epithelial barrier is directly involved with changes that occur during both comedogenesis and in stages of inflammation, especially with follicular rupture compromising the barrier's integrity. In acne-affected skin, sebaceous glands are larger, sebum excretion and filaggrin expression higher, and stratum corneum lipids are reduced. Educating patients and clinicians about inflammation's central role in acne and measures to reduce inflammation is essential. Skin irritation and xerosis from acne and treatments lead to poor treatment adherence. A skincare regimen should be included in the acne prevention, treatment, and maintenance care regimen and should be ongoing. Maintenance treatment with topical agents and skincare using gentle ceramide-containing cleansers and moisturizers is a recommended strategy after successfully controlling the disease. Epidermal barrier dysfunction contributes to acne exacerbation. Using the appropriate treatment and skincare helps to minimize irritation and inflammation, enhance treatment adherence, and improve patient outcomes.

A Detailed Analysis of the Past 20 Years of US FDA-Approved Prescription to Over-the-Counter Switches

ObjectivesThis evaluation assesses the quantity, uniqueness, and innovative nature of the past 20 years of Rx-to-OTC (RTO) switches, where a current prescription (Rx) product is reclassified for over-the-counter (OTC) status. Broadening access to more OTC drugs with well-established safety and efficacy could help to reduce healthcare expenditure and address public health challenges.MethodsThe FDA-maintained RTO switch list website was accessed to generate the primary dataset. Each product listed was assessed for the current OTC availability in the United States of its active ingredient, pharmacological class, and indication to determine its innovative quality. Descriptive statistics were employed in this study.ResultsFrom January 2002 through August 2022 there were 45 RTO switches. Among these, 51.1% involved a new to OTC active ingredient, 22.2% involved a new pharmacological class, 6.6% involved a new indication, and 82.2% were considered follow-on products that introduced a new to OTC active ingredient or new dosage form of an already marketed active ingredient to treat an existing OTC indication. A small minority (6.6%) were considered an exceptional innovation that would offer US consumers a genuinely novel OTC product, providing a new to OTC active pharmaceutical ingredient, pharmacological class, and indication. Overall, there was 1 exceptional innovation every 6.7 years.ConclusionsOver 40 RTO switches have come to the OTC market in the past 20 years; however, exceptional innovations that expand access to new to OTC active ingredients for new indications are rare. Policies and strategies that result in more innovative switches that can benefit consumers and public health should be evaluated.

N-acetylcysteine Pharmacology and Applications in Rare Diseases-Repurposing an Old Antioxidant.

N-acetylcysteine (NAC), a precursor of cysteine and, thereby, glutathione (GSH), acts as an antioxidant through a variety of mechanisms, including oxidant scavenging, GSH replenishment, antioxidant signaling, etc. Owing to the variety of proposed targets, NAC has a long history of use as a prescription product and in wide-ranging applications that are off-label as an over-the-counter (OTC) product. Despite its discovery in the early 1960s and its development for various indications, systematic clinical pharmacology explorations of NAC pharmacokinetics (PK), pharmacodynamic targets, drug interactions, and dose-ranging are sorely limited. Although there are anecdotal instances of NAC benefits in a variety of diseases, a comprehensive review of the use of NAC in rare diseases does not exist. In this review, we attempt to summarize the existing literature focused on NAC explorations in rare diseases targeting mitochondrial dysfunction along with the history of NAC usage, approved indications, mechanisms of action, safety, and PK characterization. Further, we introduce the research currently underway on other structural derivatives of NAC and acknowledge the continuum of efforts through pre-clinical and clinical research to facilitate further therapeutic development of NAC or its derivatives for rare diseases.

The Role and Benefits of Dermocosmetics in Acne Management in Japan.

In Japan, as in other countries around the world, acne vulgaris is a common disease and a frequent reason for patients to consult dermatologists. For optimal management of acne, it is important to understand how available products to support skin health can be used both with and without prescription products. Dermocosmetics can be defined as skincare agents with dermatologically active ingredients that directly support or care for the symptoms of various skin conditions (distinct from vehicle effects). There are products with active ingredients-including familiar ones such as niacinamide, retinol derivatives, and salicylic acid-that target important aspects of acne pathophysiology. Others, including ceramides, glyercin, thermal spring water, and panthenols, may have positive effects on skin barrier function that are useful in managing acne. This publication will discuss the roles of dermocosmetics in acne either as monotherapy to manage the milder forms of acne and help prevent relapses, or as adjuncts to prescription therapy to increase efficacy or adherence and assist in prevention of local adverse effects. Dermocosmetics may also have active ingredients that positively impact the skin microbiome.

Network pharmacology and experimental studies for deciphering the molecular targets and mechanisms of Chaihu Shugan powder in the treatment of functional dyspepsia.

Chaihu Shugan powder (CSP) is a prevalent prescription product used in the treatment functional dyspepsia (FD) in China. However, the underlying pharmacological mechanisms involved in the treatment of FD remain unclear. To explore the key components of CSP and their molecular targets and mechanisms in the treatment of FD. Active compounds for CSP were identified from the TCMSP and SymMap databases, and the relevant targets were predicted. FD-related targets were obtained from the CTD database. In addition, using the protein-protein interactions (PPI) analysis, the common targets were obtained. Furthermore, the compound-target networks were created with Cytoscape. Finally, molecular docking was performed to identify the core targets and validate them experimentally. In total, 78 active compounds and 671 related targets of CSP were obtained. PPI network analysis identified 15 key FD-related compound targets. Molecular docking revealed that sitosterol and hyndarin exhibited good binding activities with AKT1 and IL6, respectively. Animal experiments have shown that CSP effectively increased the protein levels of AKT1 and reduced the serum levels of IL-6 in FD rats. This study provides a theoretical evidence for the analysis of the molecular targets and mechanisms of the action of CSP in FD.

Self-care and self-medication as central components of healthcare in Germany – on the way to evidence-based pharmacy

In Germany, self-care is, above all interpreted as the prevention and treatment of minor injuries and illnesses by the patients themselves – that is, without a physician's prescription or medical advice. Maintaining one's health in the sense of a preventive approach through non-medicinal measures also plays an important role. Self-medication in this context is the treatment with approved over-the-counter-(OTC)-medications. In addition, other OTC-products such as dietary supplements as well as complementary and alternative medicines including homeopathic medications are frequently requested options by pharmacy customers. OTC-medications are central components of the German healthcare system, with expert advice from pharmacists in community pharmacies (CP) enabling safe and effective treatment. Additionally, screening for appropriate self-medication by pharmacists ensures that serious illnesses receive timely medical attention.In addition to prescribed medication, self-medication is an important part of the CP business in Germany. In contrast to prescription products, the price of OTC-products is not regulated. As a consequence, the price of OTC-products (including also pharmacy-only drugs) is influenced by competition among CPs and mail-order pharmacies, respectively. The sales of OTC-products for self-medication outside pharmacies, e.g. in drugstores and supermarkets, is restricted to a limited number of specific products.Evidence-based counseling in CPs, while generally advocated still remains a challenge. The evidence for the usage of OTC-products from clinical studies is not yet optimally integrated into everyday pharmacy practice. Information tools such as EVInews offering regular newsletters and a database have been developed to reduce the evidence-to-practice gap and to improve the overall counseling quality. Furthermore, the switching of drugs from prescription-only to pharmacy-only status also challenge CPs to provide adequate and updated guidance.

Trends in amphetamine prescriptions given at discharge in emergency departments: A national analysis (2012–2019)

ObjectivesIn parallel with the opioid epidemic, there has been a resurgence in abuse, medical complications, and deaths related to amphetamines. The opioid epidemic began with increasing rates of prescription products that evolved overtime to include heroin and more recently, fentanyl analogues. Current trends in amphetamine prescriptions are less well described. We sought to determine if there has been a change in amphetamine prescriptions given at discharge in U.S. emergency departments (EDs) in recent years. MethodsWe conducted a retrospective review of data provided by the National Hospital Ambulatory Medical Care Survey (NHAMCS) from 2012 to 2019. We computed total number of visits that were given amphetamine prescriptions (amphetamine salts, methylphenidate derivatives, and dexmethylphenidate) at discharge for each year. We computed the total number and rate of visits (of all ED visits) that had both amphetamines and opioids prescribed at discharge over the years. We computed data normality using Shapiro Wilke's test and used descriptive statistics such as mean to describe the data distribution as applicable. We used spearman's rho (SR) or pearson's correlation (PC) as applicable to describe trends in data. All p-values were one-tailed and were reported at a 0.05 significance level. All analyses were conducted in IBM SPSS version 28. Results/findingsFrom 2012 to 2019, there were an estimated 817,895 ED visits where an amphetamine prescription was given at discharge, with an overall strong increase in rate over time (SR = 0.71, p = 0.02). At the beginning of the study period (2012) there were 83,503 (0.06%) visits and in 2019 there were 186,539 (0.12%) visits (123% absolute increase). On average, there were 102,237 (SD: 52,725) visits with discharge amphetamine prescriptions per year. There was a strong, linear increase in number of visits that involved a discharge amphetamine salt prescription (PC = 0.92, p = 0.001). In 2012, there were a total of 23,676 visits and in 2019, a total of 124,773 visits (427% increase). There was no trend in visits where both an amphetamine and opioid were prescribed (PC: 0.61, p = 0.06). ConclusionThere have been increases in discharge prescriptions for amphetamines in the ED over time. This was largely driven by prescriptions for amphetamine salts. Future research initiatives should continue to monitor this trend and in prescriptions and associated abuse in the setting of rising amphetamine abuse.

Eyes Instead of Ears: Eye Injuries Following Ocular Exposure to Otic Medications.

To determine the epidemiology of ocular exposures and toxicoses in dogs and cats from otic products, 79 dog and cat cases with an ocular exposure to a topical otic medication were retrieved from the American Society for the Prevention of Cruelty to Animals Animal Poison Control Center database. Prescription products were involved in 75/79 (95%) of cases, and over-the-counter products in 4 (5%). Clinical signs included conjunctivitis, blepharospasm, epiphora, ocular discharge, and corneal ulceration. Medication error, specifically involving mistaken identification (i.e., an otic product confused with an ophthalmic product), occurred in 68/79 (86%) of cases. In 4 of these 68 cases, an otic instead of an ophthalmic medication was mistakenly dispensed to the pet owner. Unintentional delivery (i.e., accidental ocular exposure in the course of an otic application) occurred in 9/79 (11%) of cases, and 2 (3%) cases involved intentional delivery of otic products to the eyes. Because mistaken identification was the most common cause of ocular toxicoses from otic products, separate storage and/or distinctive packaging for ophthalmic versus otic products could reduce medication errors. Animal poison control center epidemiological data can be used as a source of information regarding veterinary medication errors.

RWD113 Differences in Adverse Events Reporting Between Natural Products Versus Prescription Products in the United States and Canada

Natural dietary supplement sales saw a 17% increase in 2020 from the year prior. However, it is well established that these products present risks of adverse events. Considering the lack of requirements by regulatory bodies in the United States and Canada for reporting adverse events, the quality, volume, and sources of reports may vary compared to prescription medications. The aim of this study is to examine adverse event reporting patterns in both the Unites States and Canada, comparing top-selling natural products with top-selling prescription medications.

Microsponge Drug Delivery System: Emerging Technique in Novel Drug Delivery System and Recent Advances

A number of advancements have been made in the drug delivery system in order to achieve the goals of improved efficacy and cost-effectiveness in therapy. Controlling the rate of release of active drugs to a predetermined site in the human body has been one of the pharma industry's most challenging tasks. Microsponges are porous cross-linked, non-collapsible microspheres with a size range of 5-300µm that can entrap a variety of drugs and then be incorporated into a formulated product like gel, cream, powder, or liquid. Controlled release of drugs onto the epidermis with the confidence that the drug remains primarily localized and does not enter the systemic circulation in substantial amounts is a field of research that the microsponge delivery system is progressively exploring. In order to remove systemic exposure and minimize local cutaneous reaction to active drugs, microsponge technology has been introduced in topical drug products to facilitate the controlled release of the active drugs into the cell. Also, numerous studies have shown that microsponge systems are non-irritating, non-mutagenic, non-allergenic, and non-toxic. MDDS technology is being used currently in cosmetics, over-the-counter (OTC) skin care, sunscreens, and prescription products and has recently been used in oral drugs as well as biopharmaceuticals (protein, peptides, and DNA based therapeutics) drug delivery. The purpose of this article is to provide information about microsponges such as the method of preparation, mechanism of drug release from microsponges, characterization, applications of microsponges, and information about microsponges updated research.

Patient satisfaction and impact of salivary flow rate on effectiveness of xerostomia products

To determine the types of products used to treat dry mouth and their perceived effectiveness, the relationship between salivary function and xerostomia symptoms, and whether salivary function predicts response to management strategies. Cross-sectional study of 87 patients with dry mouth and documented unstimulated whole salivary (UWS) and stimulated whole salivary (SWS) flow rates. Participants completed a questionnaire assessing dry mouth complaints and symptoms and effectiveness of specific dry mouth products. Mean (SD) age was 61.7 (12.9) years including 78 (90%) females. 47 (54%) participants had Sjögren's disease. Oral dryness symptoms (0-10 scale) rated highest with a mean (SD) of 7.2 (2.17); other symptoms scored from 3.4 to 5.1. Lower levels of UWS and SWS were associated with worse dry mouth and difficulty speaking, while lower levels of SWS flow alone were associated with greater difficulty swallowing and a decline in taste. More than half of the participants (55%, n=48) reported using ≥4 dry mouth products. Participants with normal SWS flow had significantly better responses to lozenges and prescription products. Patients with dry mouth and normal stimulated flow rates (i.e., residual salivary capacity) respond better to stimulatory products (parasympathomimetic and lozenges). Salivary flow rate assessments may help with recommendations of dry mouth products.

MT30 Learnings from DTC Launch of a Digital Therapeutic for ADHD in Response to the COVID-19 FDA Policy for Digital Health Devices for the Treatment of Psychiatric Disorders

ObjectiveTo describe real-world learnings from the launch of direct-to-consumer digital therapeutic in response to the FDA policy for digital health devices during COVID-19 prior to product’s FDA-cleared status.MethodsFrom April to August 2020, Endeavor® 3-month access was provided to children who were 8-12 years old; with a parent-reported ADHD-diagnosis; in the US with access to an iPad®/iPhone® device. Parents agreed to contact their child’s health care provider before using Endeavor. Recommended product use was 5 days/week, ∼25 minutes (5 missions) a day, for at least 4 weeks. Surveys were sent online at treatment start (Day 1) and after 1 month (Day 30). Surveys included: Child cognitive functioning: Patient Reported Outcomes Measurement Information System (PROMIS) parent-proxy 8 selected items. Surveys for ADHD-related impairment: Impairment Rating Scale (IRS) parent-report.ResultsDuring this direct-to-consumer launch, Endeavor was activated 446 times, 419 children started the treatment and 357 children played at least one full Endeavor mission. Parents saw significant improvement in 6 of the 8 selected PROMIS cognitive functioning items. There was also improvement seen in the ADHD-related overall impairment severity.ConclusionsThis demonstration confirmed that Endeavor access under FDA emergency guidance via direct-to consumers was feasible, with approximately 20% of patients requesting reactivation. Compliance rates varied from those reported in the structured setting of previous clinical trials. Caregivers observed improvement in their child’s cognitive functioning and impairment severity. Future studies should provide real-world insights on compliance and efficacy as a prescription product. ObjectiveTo describe real-world learnings from the launch of direct-to-consumer digital therapeutic in response to the FDA policy for digital health devices during COVID-19 prior to product’s FDA-cleared status. To describe real-world learnings from the launch of direct-to-consumer digital therapeutic in response to the FDA policy for digital health devices during COVID-19 prior to product’s FDA-cleared status. MethodsFrom April to August 2020, Endeavor® 3-month access was provided to children who were 8-12 years old; with a parent-reported ADHD-diagnosis; in the US with access to an iPad®/iPhone® device. Parents agreed to contact their child’s health care provider before using Endeavor. Recommended product use was 5 days/week, ∼25 minutes (5 missions) a day, for at least 4 weeks. Surveys were sent online at treatment start (Day 1) and after 1 month (Day 30). Surveys included: Child cognitive functioning: Patient Reported Outcomes Measurement Information System (PROMIS) parent-proxy 8 selected items. Surveys for ADHD-related impairment: Impairment Rating Scale (IRS) parent-report. From April to August 2020, Endeavor® 3-month access was provided to children who were 8-12 years old; with a parent-reported ADHD-diagnosis; in the US with access to an iPad®/iPhone® device. Parents agreed to contact their child’s health care provider before using Endeavor. Recommended product use was 5 days/week, ∼25 minutes (5 missions) a day, for at least 4 weeks. Surveys were sent online at treatment start (Day 1) and after 1 month (Day 30). Surveys included: Child cognitive functioning: Patient Reported Outcomes Measurement Information System (PROMIS) parent-proxy 8 selected items. Surveys for ADHD-related impairment: Impairment Rating Scale (IRS) parent-report. ResultsDuring this direct-to-consumer launch, Endeavor was activated 446 times, 419 children started the treatment and 357 children played at least one full Endeavor mission. Parents saw significant improvement in 6 of the 8 selected PROMIS cognitive functioning items. There was also improvement seen in the ADHD-related overall impairment severity. During this direct-to-consumer launch, Endeavor was activated 446 times, 419 children started the treatment and 357 children played at least one full Endeavor mission. Parents saw significant improvement in 6 of the 8 selected PROMIS cognitive functioning items. There was also improvement seen in the ADHD-related overall impairment severity. ConclusionsThis demonstration confirmed that Endeavor access under FDA emergency guidance via direct-to consumers was feasible, with approximately 20% of patients requesting reactivation. Compliance rates varied from those reported in the structured setting of previous clinical trials. Caregivers observed improvement in their child’s cognitive functioning and impairment severity. Future studies should provide real-world insights on compliance and efficacy as a prescription product. This demonstration confirmed that Endeavor access under FDA emergency guidance via direct-to consumers was feasible, with approximately 20% of patients requesting reactivation. Compliance rates varied from those reported in the structured setting of previous clinical trials. Caregivers observed improvement in their child’s cognitive functioning and impairment severity. Future studies should provide real-world insights on compliance and efficacy as a prescription product.

Osteoarthritis: modern possibilities of therapy

The article discusses the treatment of osteoarthritis (OA). In recent decades, ideas about the pathogenesis of OA have undergone significant changes. If at the dawn of the study OA was presented as a degenerative process associated with age, now that some immunological and genetic aspects of the disease have become known, the idea of the pathogenesis of OA has changed. Currently, there are clinical guidelines developed by professional communities that define approaches to the diagnosis and treatment of patients with OA. In Russia, the Association of Rheumatologists and Orthopedic Traumatologists in 2021 developed clinical guidelines for the management of patients with gonarthrosis and coxarthrosis, but currently no clear consensus has been developed in any of the treatment recommendations regarding the use of nutraceuticals and dietary supplements. While according to published studies of plant and botanical nutraceuticals developed from natural products, promising data on efficacy compared to placebo drugs and their potential for the treatment of patients with OA have been demonstrated. According to a study conducted by N.E. Lane et al., patients with OA regularly use supplements, as well as over-the-counter products in combination with prescription drugs, and the likelihood of using prescription products increases with increasing duration and severity of OA. And currently there is not enough knowledge and information about the possibilities of additional use of nutraceuticals in the treatment of patients. The article discusses the composition of the biologically active additive, which has anti-inflammatory, anti-catabolic and antiproliferative effects and can be considered as an addition to NSAIDs, chondroitin sulfate, glycosamine (sulfate or hydrochloride) and their combinations in OA. Type II collagen peptide, curcumin (curcuminoids 95%), black pepper extract (piperine) and Boswellia pilchata extract in clinical studies have demonstrated efficacy and safety in the treatment of patients with OA. A number of studies have also found a number of pleootropic effects. Effects such as anti-inflammatory, neuroprotective, immunomodulatory, cardioprotective and antitumor effects will be important in patients with comorbidity.

Development of synthetic biotics as treatment for human diseases.

Advances in synthetic biology have allowed the generation of strains of bacteria that are genetically altered to have specific therapeutic benefits. These synthetic biotics, also widely referred to as engineered living therapeutics, have tremendous potential as a new therapeutic modality, and several have advanced into the clinic and human testing. This review outlines some of the unique attributes of synthetic biotics as well as some of the challenges in their development as prescription products. Regulatory considerations are discussed, and a case study of a program that has advanced into Phase 2 testing is provided: SYNB1618 for the treatment of PKU.

First HRT product in the UK to be made available without a prescription

For the first time, women in the UK will be able to access hormone replacement therapy (HRT) in community pharmacies without a prescription, the Medicines and Healthcare products Regulatory Agency (MHRA) has confirmed. Following a safety review and public consultation, in which over 88% of 1,229 respondents were in favour of the move, Gina 10 […]