The Union Ministry of Ayush (MoA) is working closely with the World Health Organization (WHO) to develop an International Herbal Pharmacopoeia (IHP) to set global quality and efficacy standards for

Texas AG Paxton says Pfizer might have lied about efficacy of COVID vaccine, could be 1% effective | 6 Dec 2023 | GOP Texas Attorney General Ken Paxton says that he believes that pharmaceutical company Pfizer may have lied about the efficacy of the COVID-19 vaccine and the effectiveness could be as low as one […]

[spotlight] In June, we heard what could be this year's biggest HIV breakthrough: a twice-yearly injection can prevent HIV infection. Findings from a second large study of the jab has now confirmed that it works. Elri Voigt goes over the new findings and unpacks the licenses that are expected to facilitate the availability of generic versions of the jab in over a hundred countries, including South Africa.

Aim/Purpose: Improving public schools is a focus of federal legislation in the United States with much of the burden placed on principals. However, preparing principals for this task has proven elusive despite many changes in programming by institutions of higher learning. Emerging technologies that rely on augmented and virtual realities are posited to be powerful pedagogical tools for closing this gap. Background: This study investigated the effects of immersive simulation technologies on principals’ self-efficacy after treatment and the perceived significance of the design of the immersive simulation experience as an effective tool for adult learners. Methodology: The investigator employed a multiple-methods study that relied on a purposive sample of graduate students enrolled in educational leadership programs at two small universities in the southeastern United States. Participants completed a two-hour module of immersive simulation designed to facilitate transfer of knowledge to skills thereby increasing their self-efficacy. Contribution: This paper contributes to a small body of literature that examines the use of immersive simulation to prepare aspiring principals. Findings: The findings indicate moderate effect sizes in changes in self-efficacy, positive attitudes toward immersive simulation as a pedagogical tool, and significance in the design of immersive simulation modules. This suggests that immersive simulation, when properly designed, aids principals in taking action to improve schools. Recommendations for Practitioners: Educational leadership programs might consider the use of immersive simulations to enhance principals’ ability to meet the complex demands of leading in the 21st century. Impact on Society: Principals may be more adept at improving schools if preparation programs provided consistent opportunities to engage in immersive simulations. Future Research: Future research should be conducted with larger sample sizes and longitudinally to determine the effectiveness of this treatment.

Aim/Purpose: The aim of this study was to examine the sense of challenge and threat, negative feelings, self-efficacy, and motivation among students in a virtual and a blended course on multicultural campuses and to see how to afford every student an equal opportunity to succeed in academic studies. Background: Most academic campuses in Israel are multicultural, with a diverse student body. The campuses strive to provide students from all sectors, regardless of nationality, religion, etc., the possibility of enjoying academic studies and completing them successfully. Methodology: This is a mixed-method study with a sample of 484 students belonging to three sectors: general Jewish, ultra-orthodox Jewish, and Arab. Contribution: This study’s findings might help faculty on multicultural campuses to advance all students and enable them equal opportunity to succeed in academic studies. Findings: Significant sectorial differences were found for the sense of challenge and threat, negative feelings, and motivation. We found that the sense of challenge and level of motivation among Arab students was higher than among the ultra-orthodox Jewish students, which, in turn, was higher than among the general Jewish student population. On the other hand, we found that the perception of threat and negative feelings among Arab students were higher than for the other two sectors for both the virtual and the blended course. Recommendations for Practitioners: Significant feedback might lessen the sense of threat and the negative feelings and be a meaningful factor for the students to persevere in the course. Intellectual, emotional, and differential feedback is recommended. Not relating to students’ difficulties might lead to a sense of alienation, a lack of belonging, or inability to cope with the tasks at hand and dropout from the course, or even from studies altogether. A good interaction between lecturer and student can change any sense of incompetence or helplessness to one of self-efficacy and the ability to interact with one’s surroundings. Recommendations for Researchers: Lecturers can reduce the sense of threat and negative feelings and increase a student’s motivation by making their presence felt on the course website, using the forums to manage discussions with students, and enabling and encouraging discussion among the students. Impact on Society: The integration of virtual learning environments into the learning process might lead to the fulfilment of an educational vision in which autonomous learners realize their personal potential. Hence they must be given tasks requiring the application of high learning skills without compromise, but rather with differential treatment of students in order to reduce negative feelings and the sense of threat, and to reduce the transactional distance. Future Research: Further studies should examine the causes of negative feelings among students participating in virtual and blended courses on multicultural campuses and how these feelings can be handled.

Aim/Purpose. Higher education institutions face difficulties and challenges when it comes to distance learning. The purpose of this paper is to examine self-efficacy indicators and student satisfaction during online English classes. Background. E-learning has been very relevant since the Covid-19 era and is still relevant today. It is possible for students to study regardless of their location or time. By measuring students’ self-efficacy, instructors can gain valuable insights into their students’ ability to create social interaction, cope with technology, and acquire knowledge and tools to manage the learning process. Methodology. This study uses mixed methods along with two measurements. Before and after the course, quantitative and qualitative data were collected. Higher education students in Israel participated. A total of 964 students enrolled in English as a foreign language courses at the pre-basic, basic, and advanced levels. Contribution. Analyzing self-efficacy from several angles provides insight into students. What influences students’ confidence and belief in their ability to succeed in online courses. Moreover, how students perceive their own learning and how they cope with challenges. Findings. Compared to the measurement before the course, self-efficacy decreased on average. Most significant decreases occurred in ‘creating social interactions’ and ‘acquirement of knowledge and tools’ to manage the learning process. A slight decrease was observed in the ability to cope with technology. Additionally, self-efficacy and satisfaction with the course were positively correlated. Recommendations for Practitioners. An overview is provided of the most effective tools and techniques for teaching languages in digital format in this paper. This will allow instructors to design and deliver courses in a more effective way. Thus, they will be able to make better informed decisions, resulting in better outcomes for students. Recommendations for Researchers. Distance Learning courses should resemble the common digital environments in everyday life, rather than imitating face-to-face courses mainly in the field of social interaction. Impact on Society. Digital tools should be encouraged that facilitate effective learning processes instead of sticking to traditional methods that characterize face-to-face courses. Using common interfaces in daily use among the general population will enable the implementation of these recommendations. Future Research. Future studies could be helpful if they compared the English courses developed in the CEFR model with those taught face-to-face as well as those taught online. In addition, motivation and self-monitoring should be examined in both synchronous and asynchronous courses as well.

Self-efficacy is essential to learning but what happens when learning is done as a result of a collective process? What is the role of individual self-efficacy in collective problem solving? This research examines the manifestation of self-efficacy in prediction markets that are configured as collective problem-solving platforms and whether self-efficacy of traders affects the collective outcome. Prediction markets are collective-intelligence platforms that use a financial markets mechanism to combine knowledge and opinions of a group of people. Traders express their opinions or knowledge by buying and selling “stocks” related to questions or events. The collective outcome is derived from the final price of the stocks. Self-efficacy, one’s belief in his or her ability to act in a manner that leads to success, is known to affect personal performance in many domains. To date, its manifestation in computer-mediated collaborative environments and its effect on the collective outcome has not been studied. In a controlled experiment, 632 participants in 47 markets traded a solution to a complex problem, a naïve framing of the knapsack problem. Contrary to earlier research, we find that technical and functional self-efficacy perceptions are indistinguishable, probably due to a focus on outcomes rather than on resources. Further, results demonstrate that prediction markets are an effective collective problem-solving platform that correctly aggregates individual knowledge and is resilient to traders’ self-efficacy.

The purpose of this research is the exploration of the opinions and level of self-efficacy in the usage of Information Communication Technologies (ICTs) of teachers in Greek pre-schools in the learning process and administration of nurseries. By using the term “usage and utilisation of ICTs in the learning process” we mean the utilisation of the capabilities that new technologies offer in an educationally appropriate way so that the learning process yields positive results. By using the term “self-efficacy” we describe the strength of one’s belief in one’s own ability to use the capabilities he or she possess. In this way, the beliefs of the person in his or her ability to use a personal computer constitute the self-efficacy in computer usage. The research sample consists of 128 pre-school teachers that work in the prefecture of Kavala. Kavala’s prefecture is a representative example of an Education Authority since it consists of urban, suburban, and rural areas. The approach that is deemed to be the most appropriate for the exploration of such research questions is content analysis methodology and correlation analysis. The main findings of the study have shown statistically significant differences regarding the opinions and stances of the pre-school teachers for the introduction of the ICTs in the administration and the usage and utilisation of ICTs in the administration and preparation of teaching. Lastly, there were statistically significant differences between the opinions and stances of the pre-school teachers for the usage and utilisation of ICTs in the learning process. Instead, there were no statistically significant differences regarding the level of self-efficacy of the pre-school teachers in the usage and utilisation of the ICTs in the learning process. The research results could be used in the educational field as well as by Greek Ministry of Education, Research and Religious Affairs in order to take any corrective action, after the effort of Greek Ministry of Education, Research and Religious Affairs, to integrate ICT in the learning process with training courses since 2006.

Aim/Purpose: This study aims to explore levels of Technological Access (ownership, access to, and usage of computer devices as well as access to Internet services) and levels of Technological Efficacy (technology related skills) as they pertain to underserved (UNS) and underrepresented (UNR) students. Background: There exists a positive correlation between technology related access, technology related competence, and academic outcomes. An increasing emphasis on expanding online education at the author’s institution, consistent with nationwide trends, means that it is unlikely that just an increase in online offerings alone will result in an improvement in the educational attainment of students, especially if such students lack access to technology and the technology related skills needed to take advantage of online learning. Most studies on levels of Technological Access and Technological Efficacy have dealt with either K-12 or minority populations with limited research on UNS and UNR populations who form the majority of students at the author’s institution. Methodology: This study used a cross-sectional survey research design to investigate the research questions. A web survey was sent to all students at the university except first semester new and first semester transfer students from various disciplines (n = 535). Descriptive and inferential statistics were used to analyze the survey data. Contribution: This research provides insight on a population (UNS and UNR) that is expanding in higher education. However, there is limited information related to levels of Technological Access and Technological Efficacy for this group. This paper is timely and relevant as adequate access to technology and technological competence is critical for success in the expanding field of online learning, and the research findings can be used to guide and inform subsequent actions vital to bridging any educational equity gap that might exist. Findings: A critical subset of the sample who were first generation, low income, and non-White (FGLINW) had significantly lower levels of Technological Access. In addition, nearly half of the survey sample used smartphones to access online courses. Technological Efficacy scores were significantly lower for students who dropped out of or never enrolled in an online course. Transfer students had significantly higher Technological Efficacy scores while independent students (determined by tax status for federal financial aid purposes) reflected higher Technological Efficacy, but at a marginally lower level of significance. Recommendations for Practitioners: Higher education administrators and educators should take into consideration the gaps in technology related access and skills to devise institutional interventions as well as formulate pedagogical approaches that account for such gaps in educational equity. This will help ensure pathways to sustained student success given the rapidly growing landscape of online education. Recommendation for Researchers: Similar studies need to be conducted in other institutions serving UNS and UNR students in order to bolster findings and increase awareness. Impact on Society: The digital divide with respect to Technological Access and Technological Efficacy that impacts UNS and UNR student populations must be addressed to better prepare such groups for both academic and subsequent professional success. Addressing such gaps will not only help disadvantaged students maximize their educational opportunities but will also prepare them to navigate the challenges of an increasingly technology driven society. Future Research: Given that it is more challenging to write papers and complete projects using a smartphone, is there a homework gap for UNS and UNR students that may impact their academic success? What is the impact of differing levels of Technological Efficacy on specific academic outcomes of UNS and UNR students?

The post Antipsychotic Drugs: A Concise Review of History, Classification, Indications, Mechanism, Efficacy, Side Effects, Dosing, and Clinical Application was curated by information for practice.

Chronic Stress, Volume 8, Issue , January-December 2024. Police officers are frequently faced with chronic and acute stressors, such as excessive workload, organizational stressors and emotionally charged reports. This study aims to examine the relationship between a form of chronic strain (ie, burnout symptoms) and a resource (ie, coping self-efficacy) in a sample of Dutch […]

The post A Loss Cycle of Burnout Symptoms and Reduced Coping Self-Efficacy: A Latent Change Score Modelling Approach was curated by information for practice.

The post Efficacy of a culturally adapted, cognitive behavioural therapy-based intervention for postnatal depression in British south Asian women (ROSHNI-2): a multicentre, randomised controlled trial was curated by information for practice.

The post Efficacy and Safety of Sustained-release Dexamphetamine in Patients With Moderate to Severe Cocaine Use Disorder was curated by information for practice.

As COVID-19 vaccinations continue and accelerate across the U.S., a new rapid expert consultation from the National Academies of Sciences, Engineering, and Medicine offers advice for decision-makers communicating to the public about vaccine efficacy, effectiveness, and equity — drawing on findings from social and behavioral science.

Fast Guard Service sites improper training as the reason behind so many unfortunate incidents surrounding armed security guards.

More.Longevity & Wellbeing is reshaping the wellness industry with its innovative line of wellness blends and capsules, crafted from premium natural ingredients.

Sanofi/GSK launch Phase III trial of COVID-19 vaccine, will test efficacy against variants

Informational text — resources whose purpose is to inform — is essential to daily life and fundamental to literacy. Unfortunately, young children typically have limited exposure to informational text. Two 9-week randomized controlled trials with 263 first-grade children from low-income communities examined whether free educational videos and digital games from the PBS KIDS show “Molly of Denali” supported children’s ability to use informational text to answer real-world questions. Study 1 found significant positive intervention impacts on child outcomes; Study 2 replicated these findings.

Springbok coach Rassie Erasmus wants a tour whitewash and, with the Scotland win, he's a third of the way there. He didn't say it would be pretty, as Sunday's success at Murrayfield showed.

Visual Abstract

Believing you can change your life—and help others overcome setbacks—is an essential part of an effective school culture. Eminent psychologist Albert Bandura explains.

Low-efficacy mu opioid receptor (MOR) agonists may serve as novel candidate analgesics with improved safety relative to high-efficacy opioids. This study used a recently validated assay of pain-depressed behavior in mice to evaluate a novel series of MOR-selective C9-substituted phenylmorphan opioids with graded MOR efficacies. Intraperitoneal injection of dilute lactic acid (IP acid) served as a noxious stimulus to depress locomotor activity by mice in an activity chamber composed of two compartments connected by an obstructed door. Behavioral measures included (1) crosses between compartments (vertical activity over the obstruction) and (2) movement counts quantified as photobeam breaks summed across compartments (horizontal activity). Each drug was tested alone and as a pretreatment to IP acid. A charcoal-meal test and whole-body-plethysmography assessment of breathing in 5% CO₂ were also used to assess gastrointestinal (GI) inhibition and respiratory depression, respectively. IP acid produced a concentration-dependent depression in crosses and movement that was optimally alleviated by intermediate- to low-efficacy phenylmorphans with sufficient efficacy to produce analgesia with minimal locomotor disruption. Follow-up studies with two low-efficacy phenylmorphans (JL-2-39 and DC-1-76.1) indicated that both drugs produced naltrexone-reversible antinociception with a rapid onset and a duration of ~1 h. Potency of both drugs increased when behavior was depressed by a lower IP-acid concentration, and neither drug alleviated behavioral depression by a non-pain stimulus (IP lithium chloride). Both drugs produced weaker GI inhibition and respiratory depression than fentanyl and attenuated fentanyl-induced GI inhibition and respiratory depression. Results support further consideration of selective, low-efficacy MOR agonists as candidate analgesics.

SIGNIFICANCE STATEMENT

This study used a novel set of mu opioid receptor (MOR)-selective opioids with graded MOR efficacies to examine the lower boundary of MOR efficacy sufficient to relieve pain-related behavioral depression in mice. Two novel low-efficacy opioids (JL-2-39, DC-1-76.1) produced effective antinociception with improved safety relative to higher- or lower-efficacy opioids, and results support further consideration of these and other low-efficacy opioids as candidate analgesics.

The phase 3 VISION trial demonstrated that [¹⁷⁷Lu]Lu-PSMA-617 prolonged progression-free survival and overall survival (OS) in prostate-specific membrane antigen [PSMA]–positive metastatic castration-resistant prostate cancer (mCRPC) patients who progressed on taxane-based chemotherapy and androgen receptor–signaling inhibitors (ARSIs). The U.S. expanded-access program (EAP; NCT04825652) was opened to provide access to [¹⁷⁷Lu]Lu-PSMA-617 for eligible patients until regulatory approval was obtained. This study aimed to evaluate the efficacy and safety profile of [¹⁷⁷Lu]Lu-PSMA-617 within the EAP and compare the results with those from the VISION trial. Methods: Patients enrolled in the EAP at 4 institutions in the United States with available toxicity and outcome data were included. Outcome measures included OS, a prostate-specific antigen (PSA) response rate (RR) of at least 50%, and incidences of toxicity according to Common Terminology Criteria for Adverse Events version 5.0. Differences in baseline characteristics, outcome data, and toxicity between the EAP and VISION were evaluated using t testing of proportions and survival analyses. Results: In total, 117 patients with mCRPC who received [¹⁷⁷Lu]Lu-PSMA-617 within the EAP between May 2021 and March 2022 were eligible and included in this analysis. Patients enrolled in the EAP were more heavily pretreated with ARSI (≥2 ARSI regimens: 70% vs. 46%; P < 0.001) and had worse performance status at baseline (Eastern Cooperative Oncology Group score ≥ 2: 19% vs. 7%; P < 0.001) than VISION patients. EAP and VISION patients had similar levels of grade 3 or higher anemia (18% vs. 13%; P = 0.15), thrombocytopenia (13% vs. 8%; P = 0.13), and neutropenia (3% vs. 3%; P = 0.85) and similar PSA RRs (42% vs. 46%; P = 0.50) and OS (median: 15.1 vs. 15.3 mo; P > 0.05). Conclusion: Patients with PSMA-positive mCRPC who received [¹⁷⁷Lu]Lu-PSMA-617 within the EAP were later in their disease trajectory than VISION patients. Patients enrolled in the EAP achieved similar PSA RRs and OS and had a safety profile similar to that of the VISION trial patients.

[¹⁷⁷Lu]Lu-PSMA-617 was approved by the U.S. Food and Drug Administration for patients with prostate-specific membrane antigen (PSMA)–positive metastatic castration-resistant prostate cancer (mCRPC). Since the time of regulatory approval, however, real-world data have been lacking. This study investigated the efficacy, safety, and outcome predictors of [¹⁷⁷Lu]Lu-PSMA-617 at a major U.S. academic center. Methods: Patients with mCRPC who received [¹⁷⁷Lu]Lu-PSMA-617 at the Johns Hopkins Hospital outside clinical trials were screened for inclusion. Patients who underwent [¹⁷⁷Lu]Lu-PSMA-617 and had available outcome data were included in this study. Outcome data included prostate-specific antigen (PSA) response (≥50% decline), PSA progression-free survival (PFS), and overall survival (OS). Toxicity data were evaluated according to the Common Terminology Criteria for Adverse Events version 5.03. The study tested the association of baseline circulating tumor DNA mutational status in homologous recombination repair, PI3K alteration pathway, and aggressive-variant prostate cancer–associated genes with treatment outcome. Baseline PSMA PET/CT images were analyzed using SelectPSMA, an artificial intelligence algorithm, to predict treatment outcome. Associations with the observed treatment outcome were evaluated. Results: All 76 patients with PSMA-positive mCRPC who received [¹⁷⁷Lu]Lu-PSMA-617 met the inclusion criteria. A PSA response was achieved in 30 of 74 (41%) patients. The median PSA PFS was 4.1 mo (95% CI, 2.0–6.2 mo), and the median OS was 13.7 mo (95% CI, 11.3–16.1 mo). Anemia of grade 3 or greater, thrombocytopenia, and neutropenia were observed in 9 (12%), 3 (4%), and 1 (1%), respectively, of 76 patients. Transient xerostomia was observed in 23 (28%) patients. The presence of aggressive-variant prostate cancer–associated genes was associated with a shorter PSA PFS (median, 1.3 vs. 6.3 mo; P = 0.040). No other associations were observed between circulating tumor DNA mutational status and treatment outcomes. Eighteen of 71 (25%) patients classified by SelectPSMA as nonresponders had significantly lower rates of PSA response than patients classified as likely responders (6% vs. 51%; P < 0.001), a shorter PSA PFS (median, 1.3 vs. 6.3 mo; P < 0.001), and a shorter OS (median, 6.3 vs. 14.5 mo; P = 0.046). Conclusion: [¹⁷⁷Lu]Lu-PSMA-617 offered in a real-world setting after regulatory approval in the United States demonstrated antitumor activity and a favorable toxicity profile. Artificial-intelligence–based analysis of baseline PSMA PET/CT images may improve patient selection. Validation of these findings on larger cohorts is warranted.

BACKGROUND AND PURPOSE:

Recently, artificial intelligence tools have been deployed with increasing speed in educational and clinical settings. However, the use of artificial intelligence by trainees across different levels of experience has not been well-studied. This study investigates the impact of artificial intelligence assistance on the diagnostic accuracy for intracranial hemorrhage and large-vessel occlusion by medical students and resident trainees.

MATERIALS AND METHODS:

This prospective study was conducted between March 2023 and October 2023. Medical students and resident trainees were asked to identify intracranial hemorrhage and large-vessel occlusion in 100 noncontrast head CTs and 100 head CTAs, respectively. One group received diagnostic aid simulating artificial intelligence for intracranial hemorrhage only (n = 26); the other, for large-vessel occlusion only (n = 28). Primary outcomes included accuracy, sensitivity, and specificity for intracranial hemorrhage/large-vessel occlusion detection without and with aid. Study interpretation time was a secondary outcome. Individual responses were pooled and analyzed with the t test; differences in continuous variables were assessed with ANOVA.

RESULTS:

Forty-eight participants completed the study, generating 10,779 intracranial hemorrhage or large-vessel occlusion interpretations. With diagnostic aid, medical student accuracy improved 11.0 points (P < .001) and resident trainee accuracy showed no significant change. Intracranial hemorrhage interpretation time increased with diagnostic aid for both groups (P < .001), while large-vessel occlusion interpretation time decreased for medical students (P < .001). Despite worse performance in the detection of the smallest-versus-largest hemorrhages at baseline, medical students were not more likely to accept a true-positive artificial intelligence result for these more difficult tasks. Both groups were considerably less accurate when disagreeing with the artificial intelligence or when supplied with an incorrect artificial intelligence result.

CONCLUSIONS:

This study demonstrated greater improvement in diagnostic accuracy with artificial intelligence for medical students compared with resident trainees. However, medical students were less likely than resident trainees to overrule incorrect artificial intelligence interpretations and were less accurate, even with diagnostic aid, than the artificial intelligence was by itself.

The decade following passage of FDAAA has been one of easing standards for drug approvals in the US, most notably with the advent of “breakthrough” designation created by FDASIA in 2012 and the 21st Century Cures Act in 2016.

Although, as of this writing, there is no nominee for FDA Commissioner, it appears to be safe to say that the current administration intends to accelerate the pace of deregulation, mostly through further lowering of approval requirements. In fact, some of the leading contenders for the position are on record as supporting a return to pre-Kefauver-Harris days, when drug efficacy was not even considered for approval.

Build a better mouse model, and pharma will beat a path to your door - no laws needed.

In this context, it is at least refreshing to read a proposal to increase efficacy standards. This comes from two bioethicists at McGill University, who make the somewhat-startling case for a higher degree of efficacy evaluation before a drug begins any testing in humans.

We contend that a lack of emphasis on evidence for the efficacy of drug candidates is all too common in decisions about whether an experimental medicine can be tested in humans. We call for infrastructure, resources and better methods to rigorously evaluate the clinical promise of new interventions before testing them on humans for the first time.

The author propose some sort of centralized clearinghouse to evaluate efficacy more rigorously. It is unclear what they envision this new multispecialty review body’s standards for green-lighting a drug to enter human testing. Instead they propose three questions:

• What is the likelihood that the drug will prove clinically useful?
• Assume the drug works in humans. What is the likelihood of observing the preclinical results?
• Assume the drug does not work in humans. What is the likelihood of observing the preclinical results?

These seem like reasonable questions, I suppose – and are likely questions that are already being asked of preclinical data. They certainly do not rise to the level of providing a clear standard for regulatory approval, though perhaps it’s a reasonable place to start.

The most obvious counterargument here is one that the authors curiously don’t pick up on at all: if we had the ability to accurately (or even semiaccurately) predict efficacy preclinically, pharma sponsors would already be doing it. The comment notes: “More-thorough assessments of clinical potential before trials begin could lower failure rates and drug-development costs.” And it’s hard not to agree: every pharmaceutical company would love to have even an incrementally-better sense of whether their early pipeline drugs will be shown to work as hoped.

The authors note

Commercial interests cannot be trusted to ensure that human trials are launched only when the case for clinical potential is robust. We believe that many FIH studies are launched on the basis of flimsy, underscrutinized evidence.

However, they do not produce any evidence that industry is in any way deliberately underperforming their preclinical work, merely that preclinical efficacy is often difficult to reproduce and is poorly correlated with drug performance in humans.

Pharmaceutical companies have many times more candidate compounds than they can possibly afford to put into clinical trials. Figuring out how to lower failure rates – or at least the total cost of failure - is a prominent industry obsession, and efficacy remains the largest source of late-stage trial failure. This quest to “fail faster” has resulted in larger and more expensive phase 2 trials, and even to increased efficacy testing in some phase 1 trials. And we do this not because of regulatory pressure, but because of hopes that these efforts will save overall costs. So it seems beyond probable that companies would immediately invest more in preclinical efficacy testing, if such testing could be shown to have any real predictive power. But generally speaking, it does not.

As a general rule, we don’t need regulations that are firmly aligned with market incentives, we need regulations if and when we think those incentives might run counter to the general good. In this case, there are already incredibly strong market incentives to improve preclinical assessments. Where companies have attempted to do something with limited success, it would seem quixotic to think that regulatory fiat will accomplish more.

(One further point. The authors try to link the need for preclinical efficacy testing to the 2016 Bial tragedy. This seems incredibly tenuous: the authors speculate that perhaps trial participants would not have been harmed and killed if Bial had been required to produce more evidence of BIA102474’s clinical efficacy before embarking on their phase 1 trials. But that would have been entirely coincidental in this case: if the drug had in fact more evidence of therapeutic promise, the tragedy still would have happened, because it had nothing at all to do with the drug’s efficacy.

This is to some extent a minor nitpick, since the argument in favor of earlier efficacy testing does not depend on a link to Bial. However, I bring it up because a) the authors dedicate the first four paragraphs of their comment to the link, and b) there appears to be a minor trend of using the death and injuries of that trial to justify an array of otherwise-unrelated initiatives. This seems like a trend we should discourage.)

[Update 2/23: I posted this last night, not realizing that only a few hours earlier, John LaMattina had published on this same article. His take is similar to mine, in that he is suspicious of the idea that pharmaceutical companies would knowingly push ineffective drugs up their pipeline.]

Kimmelman, J., & Federico, C. (2017). Consider drug efficacy before first-in-human trials Nature, 542 (7639), 25-27 DOI: 10.1038/542025a

Neurogene’s Rett syndrome gene therapy has preliminary data supporting safety and efficacy of the one-time treatment. But a late-breaking report of a serious complication in a patient who received the high dose sent shares of the biotech downward.

The post Neurogene Gene Therapy Shows Signs of Efficacy in Small Study, But an Adverse Event Spooks Investors appeared first on MedCity News.

According to research led by the Barcelona Institute for Global Health (ISGlobal) in partnership with seven African centers, maternal antibodies transferred

The practice of medlinkyoga/medlink, encompassing physical postures, breathing exercises, and mindfulness techniques, may provide advantages for individuals

Obesity let the girl is very headache. But do you know? Bitter melon and chinese tea is the good food to weight lose. Brewing green tea with Bitter...

Vaccine also offers 65% protection against Delta variant, according to firm

Bloomington, IN : Solution Tree Press, [2023]