ABSTRACT
In the arena of clinical research, gender equity accelerates research excellence: we need multiple perspectives and all the brain power we can muster to maximize research productivity and quality. [...]women physician investigators enhance enrollment of women as participants in clinical trials, which is crucial to our ability to generalize from the data and to maintain the health of women. Women are underrepresented among academic grand rounds speakers,14 speakers at medical conferences15, and award recipients from medical specialty societies.16 Time pressure is especially intense on young women faculty. Besides spending more time on domestic chores, they spend more time at work on teaching, service, and mentoring. Female primary care physicians spend more time with patients.22 Elderly hospitalized patients treated by female internists experience lower mortality and readmission rates.23 Patients undergoing coronary artery bypass grafting had shorter hospital length of stay when treated by an all-female physician team as compared with an all-male team.24 Female patients treated by male physicians following acute myocardial infarction have higher mortality than those treated by female physicians.25 Sex discordance between patient and surgeon is associated with increased likelihood of adverse postoperative outcomes-and that observation that is driven by worse outcomes for female patients treated by male physicians.26 Clinical trials play a fundamental role in bringing new medications and interventions to our patients, yet women have often been excluded from participation. Among 60 randomized controlled trials (RCTs) of lipidlowering therapies reported between 1990 and 2018, there was a modest increase in enrollment of women over time, but women remain underrepresented compared with the relative burden of disease.30 In another study of 317 RCTs of heart failure with reduced ejection fraction published in highimpact journals over the past 20 years, only 25% of participants overall were female, and females were under-enrolled in 72% of these trials.
ABSTRACT
Home visits have the power to ease the clinical trial process for patients, but complex study design, tight timelines, busy clinical operations teams, and overburdened sites can sometimes make home health feel like yet another moving piece to manage. Individual variables that play a large role in establishing timelines can include factors like: * Amount of protocol-specific training required. * Level of engagement during a visit. * On-site processing requirements. * Drug or sample stability. * Recruitment goals. [...]all training should be to the full satisfaction of the principal investigator overseeing the study. Since sites are still responsible for the conduct of home visits from a regulatory perspective, there is often a concern about how they can remain in control of the progress without overwhelming the already busy study team and staff.
ABSTRACT
The limited participation of African Americans in clinical trials has been a topic of discussion among medical and scientific researchers for some time. With the testing of coronavirus vaccines, this discussion has continued, particularly given the disproportionate impact of the virus on members of the African American community. With the public health goal of achieving widespread or "herd" immunity, the concept of "vaccine hesitancy" has also been addressed with regard to the population in general, and in relation to the African American community, among others. Vaccine hesitancy has been reported among groups from healthcare workers to rural residents to the poor. As is the case with all segments of society, African Americans are not monolithic. However, there are aspects of the issue of vaccine hesitancy which are unique and specific to the African American community in the U.S. In particular, the nature of the information about the coronavirus itself and about the vaccine, and importantly, the increasing availability of the information about the Tuskegee experiment, Henrietta Lacks, and other cases, along with the prevalence of misinformation and disinformation on aspects of science, such as that involving vaccines, are relevant to understanding the nature of vaccine hesitancy among African Americans.
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3 An important component for the provision of patient-centric healthcare is the ability to collect blood samples remotely in a manner that delivers samples of a quality comparable to that of current standard phlebotomy collection, enabling routine clinical testing, monitoring disease progression and testing drug concentrations.2-4-г, This blood sampling could be performed at the patient's home, at a nearby pharmacy, or at a local clinic, rather than a centralized facility. Examples of commercially available devices include the Neoteryx Mitra,13 Tasso-M20, SST and Tasso+,14 SeventhSense TAP and TAP II,15 Trajan hemaPEN,16 Labcorp Pixel,17 Capitainer qDBS,1" HemaXis DB10 and DX,19 and Drawbridge OneDraw.20 These devices are capable of collecting from as little as 20 microliters (approximately half a drop) to several hundred microliters of blood and have been widely used to determine clinical parameters,21 drug concentrations,5'22 therapeutic drug monitoring,23 and, more recently, COVID antibody levels.24,25 Some of these devices enable the collection of a fixed volume of blood, collected as dried blood, which can then be shipped and handled at room temperatures-avoiding the need for freezers and dry ice for storing and shipping samples-enabling its adoption even in remote areas with limited infrastructure. Patient-centric blood sampling techniques have been gaining popularity for use in pharmaceutical drug development;however, to date they have not been broadly accessible to the general public.26 This can be partially attributed to the "cliniccentric" healthcare model, where reimbursement is dependent on in-person visits and sample collection. [...]the status quo remains and anyone who needs a blood test is required to go to the doctor's office or clinic. [...]studies have demonstrated that the overall cost to society will be lower, by improving health outcomes and allowing broader access and patient convenience.27 The availability and adoption of patient-centric approaches can provide access and treatment options to clinical trial participants not geographically co-located with the investigative sites and improving access in rural or lesser developed communities, globally, potentially improving the health of the general population.
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More recent policies aim to offset years of underenrollment of minority groups by expanding enrollment criteria, including more research sites in minority communities, and engaging investigators with diverse backgrounds. FDA has also sought to promote research diversity through its Drug Trials Snapshots program, established in 2015 to increase the visibility of clinical trial enrollment by age, sex, ethnicity and race. According to a recent report on the program's impact, though, there still may be a ways to go for clinical trials to reflect the diversity of the US population.
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FNIH will manage an ACTIV steering committee to develop an inventory of potential candidates, launch master protocols with a single control arm, and set criteria for ranking potential candidates for first-wave and subsequent evaluation. [...]a third group will tap NIH's extensive clinical trial network infrastructure to build capacity for expediting trials and to study different populations and disease stages. ?o advance vaccine development, another ACTIV group will form a collaborative framework to map epitopes and develop assays, establish protocols for sampling and immunological analyses, collect clinical data on immunological responses and endpoints, and engage with regulators on surrogate endpoints for clinical evaluation. Jill Wechsler MULTIPLE WEBSITES IDENTIFY AND TRACK RESEARCH ON COVID Widespread research activity is available from these and other organizations: * The University of Oxford Center for Evidence-Based Medicines lists more than 1000 clinical trials at http:// covid19.trialstracker.net/index.html * Bill & Melinda Gates Foundation with Cytel identifies more than 600 trials in the US and other regions at https://covid19-trialscom * TranspariMed offers a guide to multiple trials at https://www.transparimed.org/ * Bi°Century tracks vaccines and therapeutics in its COVID-19 Resource Center, https://www.biocentury.com/ clinical-vaccines-and-therapies * World Health Organization: https://www. who.int/emergencies/diseases/novelcoronavirus-2019/global-researchon-novel-coronavirus-2019-ncov/ * Regulatory Affairs Professionals Society: https://www.raps.org/newsand-articles/news-articles/2020/3/ covid-19-therapeutics-tracker
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Last week, eight prominent biotech industry executives publicly emphasized the importance of rigorous clinical research and complete study data to support any authorization or approval of a new covid vaccine or treatment. Hahn raises concerns These statements aim to offset fears that fda might soften its approval standards due to pressure from the White House to make available a covid vaccine in October. Continuing predictions from the White House about a vaccine being available in two months, and instructions from the Centers for Disease Control and Prevention (CDC) that state public health departments should be prepared to distribute a vaccine by the end of October, heightened concerns that political pressure will lead to some kind of authorization of a new vaccine before the Nov. 3 election.
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The much-anticipated meeting of FDA's Vaccines and Related Biological Products Advisory Committee (VRBPAC) last month addressed a number of critical issues related to testing and approval of vaccines to prevent COVID-19 infection, including policies and data requirements for determining that a pandemic vaccine can be considered safe and effective, particularly when based on more limited, early clinical trial data. While studies sponsored by AstraZeneca and Johnson & Johnson's Janssen unit resumed soon after the meeting, the study pauses were described by researchers as a sign that clinical trial safety systems were working as intended, as the analysts determined the adverse events were unrelated to the test vaccine candidates. The aim is to gain further information on vaccine efficacy and side effects, including rare adverse events and fuller comparisons among patient groups with differences in age, sex, comorbidities, and ethnic characteristics.
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Growing public concerns about politics playing a role in vetting potential vaccines and therapies to combat the COVID-19 pandemic has prompted sponsors of leading clinical trials to make public their study protocols and statistical analysis plans. [...]AstraZeneca's timeframe for enrolling and assessing study participants was delayed by the need to address the report of a serious adverse event in its Phase III study. The disclosure of these usually confidential details on research endpoints, assessment timeframes, and study analysis plans aim to promote information sharing among vaccine developers, and also build public confidence.
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[...]CURES' BILL PROMOTES PANDEMIC PREPAREDNESS Congressional leaders are developing the next version of the 21st Century Cures Act, including provisions to advance research related to the COVID-19 crisis as part of initiatives for bringing innovative therapies to market faster (see https://bit.ly/2SKfA4S). Cures 2.0 continues and updates some of the main themes of the first Cures Act: support development of treatments for rare diseases, patient-focused drug development, diversity in clinical trials, expanded use of digital health systems, increased health literacy, and utilization of real-world data. A public education campaign, moreover, would aim to counter concerns about the safety of vaccines to promote widespread vaccination. Because these treatments are costly and unprofitable for biopharma companies to test and market, the legislation proposes additional financial support for both pre-market studies and post-market production and subsidized higher reimbursement rates for antibiotics that address critical needs.
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Studies in mild-to-moderate cases as well as severe disease leave us still searching for a magic pill
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BackgroundConsideration is needed when using Janus kinase (JAK) inhibitors to treat RA in pts aged ≥65 years or those with cardiovascular (CV) risk factors. The JAK1 preferential inhibitor FIL was generally well tolerated in clinical trials[1];safety has not been determined in a real-world setting.ObjectivesTo report baseline characteristics and up to 6-month safety data from the first 480 pts treated with FIL in the FILOSOPHY study (NCT04871919), and in two mutually exclusive subgroups based on age and CV risk.MethodsFILOSOPHY is an ongoing, phase 4, non-interventional, European study of pts with RA who have been prescribed FIL for the first time and in accordance with the product label in daily practice. Baseline characteristics and the incidence of select adverse events (AEs) are assessed in pts aged ≥65 years and/or with ≥1 CV risk factor (Table 1), and in those aged <65 years with no CV risk factors.ResultsAs of the end of June 2022, 480 pts had been treated: 441 received FIL 200 mg and 39 received FIL 100 mg. Of the 480 pts, 148 (30.8%) were aged ≥65 years;332 (69.2%) were aged <65 years. In total, 86 (17.9%) were former smokers, 81 (16.9%) were current smokers and 203 (42.3%) were non-smokers (data were missing for 110 pts [22.9%]). In addition to smoking, the most frequent CV risk factors included a history of hypertension (32.3%), a history of dyslipidemia (10.2%) and a family history of myocardial infarction (8.5%;Table 1).23 pts (4.8%) discontinued treatment due to AEs. Of the 354 pts aged ≥65 years or with ≥1 CV risk factor, infections affected 64 pts (18.1%), 34 (9.6%) had COVID-19, 2 (0.6%) had herpes zoster, and cardiac disorders (angina pectoris, atrial fibrillation, palpitations and tachycardia) affected 5 pts (1.4%);no cases of malignancies were observed. In the subgroup aged <65 years and with no CV risk factors (n=126), infections occurred in 18 pts (14.3%) (9 [7.1%] had COVID-19;3 [2.4%] had herpes zoster) and malignancies (myeloproliferative neoplasm) affected 1 pt (0.8%);no pts had cardiac disorders. There were no cases of deep vein thrombosis or pulmonary embolism in either subgroup.ConclusionIn this interim analysis of FILOSOPHY, no unexpected safety signals emerged at up to 6 months. Although infections and cardiac disorders affected a numerically greater proportion of pts aged ≥65 years or with ≥1 CV risk vs those aged <65 years with no CV risk, longer follow-up on a broader cohort is necessary to further characterize the safety of FIL in different groups of pts with RA.Reference[1]Winthrop K, et al. Ann Rheum Dis 2022;81:184–92Table 1.Baseline characteristics and CV risk factorsBaseline demographics/CV risk factorsAll FIL-treated pts (N=480)≥65 years or with ≥1 CV risk factor (n=354)<65 years and no CV risk factor (n=126)*Female sex, n (%)351 (73.1)252 (71.2)99 (78.6)Age, years, mean (SD)57.6 (11.5)60.4 (10.8)49.6 (9.6)Rheumatoid factor positive, n (%)†228 (47.5)167 (47.2)61 (48.4)Anti-citrullinated protein antibody positive, n (%)‡243 (50.6)176 (49.7)67 (53. 2)Body mass index, kg/m2, mean (SD)27.6 (5.7) n=43728.0 (5.4) n=33126.3 (6.4) n=106RA disease duration, years, mean (SD)10.4 (9.4) n=47810.5 (9.5) n=35310.0 (8.8) n=125Tender joint count 28, mean (SD)8.6 (6.9) n=4578.7 (7.1) n=3408.3 (6.3) n=117Swollen joint count 28, mean (SD)5.6 (5.2) n=4525.7 (5.4) n=3365.4 (4.4) n=116Former smoker, n (%)§86 (17.9)86 (24.3)0Current smoker, n (%)§81 (16.9)81 (22.9)0Non-smoker, n (%)§203 (42.3)130 (36.7)73 (57.9)Family history of myocardial infarction, n (%)41 (8.5)41 (11.6)0Medical history of: n (%) CV disease33 (6.9)33 (9.3)0 Diabetes35 (7.3)35 (9.9)0 Dyslipidemia49 (10.2)49 (13.8)0 Hypertension155 (32.3)155 (43.8)0 Ischemic CNS vascular disorders11 (2.3)11 (3.1)0 Peripheral vascular disease17 (3.5)17 (4.8)0*Includes 53 pts with missing smoking status data who were aged <65 years with no other CV risk factors.†Missing/unknown in 154 pts;‡Missing in 153 pts;§Smoking status data missing in 110 pts (22.9%).AcknowledgementsWe thank the physicia s and patients who participated in this study. The study was funded by Galapagos NV, Mechelen, Belgium. Publication coordination was provided by Fabien Debailleul, PhD, of Galapagos NV. Medical writing support was provided by Debbie Sherwood, BSc, CMPP (Aspire Scientific, Bollington, UK), and funded by Galapagos NV.Disclosure of InterestsPatrick Verschueren Speakers bureau: AbbVie, Eli Lilly, Galapagos, Roularta, Consultant of: Celltrion, Eli Lilly, Galapagos, Gilead, Nordic Pharma, Sidekick Health, Grant/research support from: Galapagos, Pfizer, Jérôme Avouac Speakers bureau: AbbVie, AstraZeneca, BMS, Eli Lilly, Galapagos, MSD, Novartis, Pfizer, Sandoz, Sanofi, Consultant of: AbbVie, Fresenius Kabi, Galapagos, Sanofi, Grant/research support from: BMS, Fresenius Kabi, Novartis, Pfizer, Karen Bevers Grant/research support from: Galapagos, Susana Romero-Yuste Speakers bureau: AbbVie, Biogen, BMS, Lilly, Pfizer, Consultant of: Sanofi, Lilly, Grant/research support from: Lilly, MSD, Roberto Caporali Speakers bureau: AbbVie, Amgen, BMS, Celltrion, Eli Lilly, Galapagos, Janssen, MSD, Novartis, Pfizer, Sandoz, UCB, Consultant of: AbbVie, Amgen, BMS, Celltrion, Eli Lilly, Fresenius Kabi, Galapagos, Janssen, MSD, Novartis, Pfizer, Roche, Sandoz, UCB, Thomas Debray Consultant of: Biogen, Galapagos, Gilead, Francesco De Leonardis Employee of: Galapagos, James Galloway Speakers bureau: AbbVie, Biogen, Eli Lilly, Galapagos, Gilead, Janssen, Novartis, Pfizer, Roche, UCB, Consultant of: AbbVie, Eli Lilly, Galapagos, Gilead, Janssen, Novartis, Pfizer, Grant/research support from: AstraZeneca, Celgene, Gilead, Janssen, Medicago, Novavax, Pfizer, Monia Zignani Shareholder of: Galapagos, Employee of: Galapagos, Gerd Rüdiger Burmester Speakers bureau: AbbVie, Amgen, BMS, Chugai, Galapagos, Lilly, Pfizer, Sanofi, Consultant of: AbbVie, Amgen, BMS, Galapagos, Lilly, Pfizer, Sanofi.
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BackgroundUpadacitinib (UPA), a Janus kinase inhibitor, was effective and well tolerated in patients (pts) with non-radiographic axial spondyloarthritis (nr-axSpA) through 14 weeks (wks) of treatment.[1]ObjectivesThis analysis assessed the efficacy and safety of UPA vs placebo (PBO) through 1 year.MethodsThe SELECT-AXIS 2 nr-axSpA study included a 52-wk randomized, double-blind, PBO-controlled period. Enrolled adults had a clinical diagnosis of active nr-axSpA fulfilling the 2009 ASAS classification criteria, objective signs of inflammation based on MRI sacroiliitis and/or elevated C-reactive protein, and an inadequate response to NSAIDs. One-third of pts had an inadequate response to biologic DMARDs. Pts were randomized 1:1 to UPA 15 mg once daily or PBO. Concomitant medications, including NSAIDs, had to be kept stable through wk 52. The study protocol outlined that pts who did not achieve ASAS20 at any two consecutive study visits between wks 24 to 52 should receive rescue therapy with NSAIDs, corticosteroids, conventional synthetic/biologic DMARDs, or analgesics. Cochran-Mantel-Haenszel (CMH) test with non-responder imputation incorporating multiple imputation (NRI-MI) was used to handle missing data and intercurrent events for binary efficacy endpoints. Mixed-effect model repeated measures (MMRM) was used to assess continuous efficacy endpoints. NRI was used for binary endpoints after rescue and as observed analysis excluding data after rescue for continuous endpoints. Treatment-emergent adverse events (TEAEs) are reported through wk 52.ResultsOf the 314 pts randomized, 259 (82%;UPA, n=130;PBO, n=129) completed wk 52 on study drug. More pts achieved an ASAS40 response with UPA vs PBO from wks 14 to 52 with a 20% treatment difference at wk 52 (63% vs 43%;nominal P <.001;Figure 1). The proportion of pts achieving ASDAS inactive disease with UPA remained higher than PBO at wk 52 (33% vs 11%;nominal P <.0001;Figure 1). Consistent improvements and maintenance of efficacy were also seen across other disease activity measures. Between wks 24 and 52, fewer pts on UPA (9%) than PBO (17%) received rescue therapy. A similar proportion of pts in each treatment group had a TEAE (Table 1). Infections were the most common TEAE;the rates of serious infections and herpes zoster were higher with UPA vs PBO, although no new serious infections were reported from wks 14 to 52. COVID-19 events were balanced between treatment groups. No opportunistic infections, malignancy excluding non-melanoma skin cancer, adjudicated major adverse cardiovascular events, inflammatory bowel disease, or deaths were reported. Two pts (1.3%) on PBO had adjudicated venous thromboembolic events.ConclusionUPA showed consistent improvement and maintenance of efficacy vs PBO through 1 year across multiple disease activity measures. No new safety risks were identified with longer-term UPA exposure. These results continue to support the benefit of UPA in pts with active nr-axSpA.Reference[1]Deodhar A, et al. Lancet. 2022;400(10349):369–379.Table 1.Safety through week 52Event, n (%)PBO (n = 157)UPA 15 mg QD (n = 156)Any AE103 (66%)107 (69%)Serious AE6 (3.8%)6 (3.8%)AE leading to D/C4 (2.5%)6 (3.8%)COVID-19-related AE22 (14%)24 (15%)Deaths00Infection60 (38%)68 (44%) Serious infection1 (0.6%)2 (1.3%) Herpes zoster1 (0.6%)5 (3.2%)Malignancy other than NMSC00NMSC1 (0.6%)0Hepatic disorder7 (4.5%)6 (3.8%)Neutropenia1 (0.6%)8 (5.1%)MACE (adjudicated)00VTE (adjudicated)2 (1.3%)a0Uveitisb3 (1.9%)2 (1.3%)Inflammatory bowel disease00aBoth patients had non-serious events of deep vein thrombosis in the lower limb with risk factors including obesity and prior deep vein thrombosis in one patient and concomitant COVID-19 infection in the other patient.bThree events of uveitis occurred in each treatment group (among n = 3 patients in the PBO group and n = 2 patients in the UPA group);two events in the PBO group and one in the UPA group occurred in patients with a history of uveitis.AcknowledgementsAbbVie funded this study and participated in the study design, res arch, analysis, data collection, interpretation of data, review, and approval of the . All authors had access to relevant data and participated in the drafting, review, and approval of this publication. No honoraria or payments were made for authorship. Medical writing support was provided by Julia Zolotarjova, MSc, MWC, of AbbVie.Disclosure of InterestsFilip van den Bosch Speakers bureau: AbbVie, Amgen, Galapagos, Janssen, Lilly, Merck, MoonLake, Novartis, Pfizer, and UCB., Consultant of: AbbVie, Amgen, Galapagos, Janssen, Lilly, Merck, MoonLake, Novartis, Pfizer, and UCB., Atul Deodhar Consultant of: AbbVie, Amgen, Aurinia, BMS, Celgene, GSK, Janssen, Lilly, MoonLake, Novartis, Pfizer, and UCB, Grant/research support from: AbbVie, Bristol Myers Squibb, Celgene, GSK, Lilly, Novartis, Pfizer, and UCB, Denis Poddubnyy Speakers bureau: AbbVie, Biocad, BMS, Galapagos, Gilead, GlaxoSmithKline, Janssen, Lilly, MSD, Medscape, MoonLake, Novartis, Peervoice, Pfizer, Roche, Samsung Bioepis, and UCB, Consultant of: AbbVie, Biocad, BMS, Galapagos, Gilead, GlaxoSmithKline, Janssen, Lilly, MSD, Medscape, MoonLake, Novartis, Peervoice, Pfizer, Roche, Samsung Bioepis, and UCB, Grant/research support from: AbbVie, Lilly, MSD, Novartis, and Pfizer., Walter P Maksymowych Consultant of: AbbVie, BMS, Celgene, Galapagos, Gilead, Janssen, Lilly, Novartis, Pfizer, and UCB, Grant/research support from: AbbVie, Novartis, Pfizer, and UCB, Employee of: Chief Medical Officer of CARE Arthritis Limited, Désirée van der Heijde Consultant of: AbbVie, Bayer, BMS, Cyxone, Eisai, Galapagos, Gilead, GSK, Janssen, Lilly, Novartis, Pfizer, and UCB, Employee of: Director of Imaging Rheumatology BV, Tae-Hwan Kim Speakers bureau: AbbVie, Celltrion, Kirin, Lilly, and Novartis., Mitsumasa Kishimoto Consultant of: AbbVie, Amgen, Asahi-Kasei Pharma, Astellas, Ayumi Pharma, BMS, Chugai, Daiichi Sankyo, Eisai, Gilead, Janssen, Lilly, Novartis, Ono Pharma, Pfizer, Tanabe-Mitsubishi, and UCB., Xenofon Baraliakos Speakers bureau: AbbVie, BMS, Celgene, Chugai, Merck, Novartis, Pfizer, and UCB, Consultant of: AbbVie, BMS, Chugai, MSD, Novartis, Pfizer, and UCB, Grant/research support from: AbbVie and Novartis, Yuanyuan Duan Shareholder of: AbbVie, Employee of: AbbVie, Kristin D'Silva Shareholder of: AbbVie, Employee of: AbbVie, Peter Wung Shareholder of: AbbVie, Employee of: AbbVie, In-Ho Song Shareholder of: AbbVie, Employee of: AbbVie.
ABSTRACT
[...]many sponsors have implemented alternative ways to conduct trials or components of them, incorporating remote and virtual activities, which aim to bring studies directly to patients via a range of technologies. Every clinical investigation begins with the development of a protocol that describes h.ow a clinical trial will be conducted-encompassing the objective(s), design, methodology, statistical considerations, and organization of the study-and ensures the safety of the trial subjects and integrity of the data collected. [...]of the pandemic, we will see more hybrid trials that combine site and remote patient visits. In forcing the research community to be more open to using different tools to ensure vital research continues, the pandemic is driving the beginning of a new era of patient and site engagement-one in which a more proactive and efficient approach ensures the lines of communication remain open and data is collected appropriately and with greater speed.
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According to the Association of the British Pharmaceutical Industry (ABPI) there has been a significant decline in the number of industry clinical trials nitiated in the UK. "The COVID-19 pandemic has accelerated the decline in ate-stage industry clinical research in the UK, compared to its global peers," said Richard Torbett, chief executive of the ABPI in a press release (3). Findings from an ABPI report into the state of clinical trials in the UK has shown that consistently slow and variable set up times for clinical trials encountered in the NHS are causing some pharmaceutical companies to look elsewhere for clinical research (4). [...]a close relationship is needed between government and the life sciences sector now more than ever to ensure the country does not fall too far behind its global competitors.
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[...]we've seen more companies diversifying their portfolios and investing in therapies for rare diseases so that advanced therapies, and in particular, gene therapies-which were deemed experimental and risky when I was involved with the European Medicines Agency's Committee for Advanced Therapies some 10-12 years ago-take centre stage," Schneider continues. The year's expectations were unsurprisingly centred around COVID-19 for Martin Lush, global vice president. "Many big pharma companies will continue down the path of externalising services for small molecules and/or known molecules maintenance, to keep their focus on novel molecules," she explains. [...]of this complexity, there can be much negotiation and, hence, delay in access to medicines, he explains.
ABSTRACT
[...]of the significant cost of development, companies seek to recoup finances through data exclusivity and patent protection of intellectual property, such as the drug product's formulation. Bio/pharma companies reformulate existing therapies for a whole host of reasons, such as treating underserved or neglected disease areas, improving patient adherence (particularly for target patient groups, such as paediatrics), reducing the potential of drug abuse, and providing alternative options in crisis situations-as has been apparent during the COVID-19 pandemic. Pentamidine is an anti-infective agent that can be used to treat an earlier stage of the disease;however, it is unable to penetrate the blood-brain barrier sufficiently to treat the secondary stage of HAT. [...]it was hypothesized that a combined pentamidine-Pluronic formulation may be a suitable approach to provide patients with a single therapeutic option for treatment of all stages of HAT. [...]it was concluded that the pharmacokinetic data attained supports the use of safety and tolerability data from the conventional risperidone formulation for further testing of VAL401 (4).
ABSTRACT
While the majority of the world is in isolation to try to slow the spread of the virus responsible for COVID19, companies and regulatory bodies in the bio/pharma industry are facing numerous challenges and committing significant efforts to support the development of novel therapeutics and monitor supply chains. Some notable drugs and vaccines for COVID-19 in development are: * Messenger RNA vaccines, from CureVac and BioNTech, are in preclinical stages. * Remdesivir, from Gilead Sciences, originally developed to treat Ebola, is in Phase III clinical trials. * SNG001, from Synairgen Research, is an inhaled formulation of interferon-beta-1a that is entering Phase II clinical trials. * Chloroquine and hydroxychloroquine are currently being investigated in clinical trials and are being donated by various companies for evaluation. The emptying of the shelves can be attributed to the, as of yet unproven, link with the drug having efficacy in the treatment of COVID-19. [...]in this current climate, it is imperative that pertinent scientific information that can be of reassurance and informative be disseminated with due diligence and care.
ABSTRACT
The bio/pharma sector is an industry that has probably been most affected by the pandemic, not only is there an expectation for bio/pharma companies to step up and develop appropriate treatments quickly, resulting in regulatory flexibility and unprecedented collaborations, but there have also been concerns around supply chain security. Agencies have prioritized protocol consultations, shortened the timelines for clinical trial application review and approvals, and there is some focus on implementing fast-track and priority review processes for the evaluation of marketing authorization applications. Marton (Arriello): Authorities that have more experience in evaluation of data adapted quickly, while other authorities, such as those in the Commonwealth of Independent States area, stopped all activities for a period of time or adapted along the way. Gross (ProductLife): There is already some cooperation between the US and Europe, especially in the regulatory development process and in the evaluation process, so it's possible the current situation could reinforce those efforts and lead to some further joint assessments for clinical trial protocols and marketing authorization reviews.