PP216 Indirect Treatment Comparison Assessment: An Improvement Intervention In The Scottish Medicines Consortium

2021 ◽  
Vol 37 (S1) ◽  
pp. 27-28
Author(s):  
John Scott ◽  
Moira McMurray ◽  
Rickie O'Connell ◽  
Pauline McGuire ◽  
Noreen Downes

IntroductionThe Scottish Medicines Consortium (SMC) conducts early health technology assessment (HTA) of new medicines on behalf of the National Health Service Scotland based on pharmaceutical company submissions. As the appraisals are conducted close to the point of marketing authorization, there is often a lack of direct head-to-head data. In 2019, assessment of relevant comparative efficacy was informed via indirect treatment comparisons (ITC) in 55 percent (36/66) of submissions. While the ITCs are essential to the decision-making process, they are frequently incomplete.MethodsA focus group was conducted with the clinical assessment team (n = 11) to explore problems in the submission process and to identify areas for improvement. It was agreed that providing improved guidance to companies prior to submission may prevent future inconsistencies. A working group (n = 5) was tasked with identifying and implementing potential solutions. The group reviewed the focus group findings, relevant literature, and guidance from other organizations. Draft guidance was developed that was reviewed by two pharmaceutical industry representatives (SMC subcommittee members).ResultsFindings from the focus group highlighted issues broadly related to the incomplete presentation and reporting of ITCs. The improved guidance document outlined specific requirements in a checklist format for reporting and presenting the results of different ITC data. This guidance was published in February 2020. To evaluate the impact of the updated guidance and to identify any further changes required, a follow-up focus group and survey of industry representatives is planned for March 2021.ConclusionsThe aim of the ITC guidance is to provide pharmaceutical companies with direction to improve the quality and transparency of reporting, which will in turn improve the quality of HTAs and thus strengthen the recommendations provided by the SMC. The follow-up focus groups and survey will assess the impact of the guidance. It is acknowledged that the results of this process may be limited by the small sample size and short duration of the assessment.

1992 ◽  
Vol 20 (1) ◽  
pp. 73-78
Author(s):  
Jacqueline M. Atkinson ◽  
Denise A. Coia

Using an ABA design, the impact of the unexpected delivery of Irn Bru to an out-patient clinic for depressed men was investigated using the Montgomery-Åsberg scale for depression. A significant improvement in both behaviour and affect was seen immediately, some benefit still showing at one month follow-up. The effect of the procedure on the multidisciplinary team is also discussed. Some methodological issues, including small sample size are explored. Despite the methodological problems the serious element of the study points to the important impact of unexpected, non-therapeutic elements on clinical behaviour, possibly as a result of the challenge to the therapist-patient relationship.


2019 ◽  
Vol 99 (12) ◽  
pp. 1602-1615 ◽  
Author(s):  
Malindu E Fernando ◽  
Robert G Crowther ◽  
Peter A Lazzarini ◽  
Kunwarjit S Sangla ◽  
Scott Wearing ◽  
...  

Abstract Background Abnormalities in gait have been associated with high plantar pressures and diabetes-related plantar foot ulcers. Whether these are a transient response to the ulcer or are representative of long-term lower limb biomechanical abnormalities is currently unknown. Objective The aim of this study was to examine whether 12 gait parameters identified as being associated with nonhealing diabetes-related plantar foot ulcers at baseline remained associated during a 6-month follow-up period. Design This was a longitudinal observational case-control study. Methods Gait assessments were performed at entry and twice during follow-up over a 6-month period in 12 participants with nonhealing diabetes-related plantar foot ulcers (case participants) and 62 people with diabetes and no history of foot ulcers (control participants) using a standardized protocol. Linear mixed-effects random-intercept models were used to identify gait parameters that consistently differed between case participants and control participants at all assessments after adjustment for age, sex, body mass index, presence of peripheral neuropathy, and follow-up time. Standardized mean differences (SMD) were used to measure effect sizes. Results Five of the 12 gait parameters were significantly different between case participants and control participants at all 3 time points. Case participants had a more abducted foot progression angle (SMD = 0.37), a higher pelvic obliquity at toe-off (SMD = −0.46), a greater minimum pelvic obliquity (SMD = −0.52), a lower walking speed (SMD = −0.46), and a smaller step length (SMD = −0.46) than control participants. Limitations The limitations included a small sample size, the observational nature of the study, and the inability to evaluate the impact of gait on wound healing. Conclusions This study identified abnormal gait parameters consistently associated with nonhealing diabetes-related plantar foot ulcers. Further research is needed to test the clinical importance of these gait characteristics.


2021 ◽  
Vol 8 (1) ◽  
pp. 18-28
Author(s):  
Mauricio Lema ◽  
Beatriz Elena Preciado Franco ◽  
Camila Lema ◽  
Ana María Romero Millán ◽  
Sara Paulina Mora Vélez ◽  
...  

Therapy for mCRC is based on surgery, cytotoxic chemotherapy, and biologic agents. In randomized clinical trials, median progression-free survival (PFS) and overall survival (OS) hover around 10mo and 29mo, respectively. There are no published results on survival outcomes of patients with mCRC in Colombia. Objective: Describe the survival outcomes of patients with mCRC treated by Mauricio Lema in Medellín, Colombia. Methods: This is a retrospective, case series (2008-2020) of mCRC, ≥18 years old, treated with systemic therapy for metastatic disease, and ≥3-month follow-up.  Stratification factors included: use of biologic (anti-VEGF or anti-EGFR) plus chemotherapy in 1st-Line (yes/no) and metastasectomy (yes/no). Survival analyses were evaluated using Kaplan- Meier curves. Results from the general population are described, and they are also discriminated against by the use of a biological agent in the first-line of systemic therapy. Results: 89 patients with mCRC were included. The Median follow-up was 35 months (IQR: 21-57). Median PFS and OS were 12.1mo (95%CI: 10.4-13.8) and 29.3mo (95%CI: 23.2-34.4), respectively. Median OS in patients receiving biologics was 28.8mo (95%CI: 22.1 – 35.6) vs 33.7mo (95%CI: 16.4 – 51.0) in the chemo-only group (p=0.01). Median OS in the metastasectomy and non-metastasectomy groups were 36.1 (95% CI: 26.5 - 45.7) and 25.0 months (95% CI: 15.4 - 34.5), respectively (NS). Conclusion: In this case series of patients’ survival outcomes were similar to thosereported in large phase III trials. A small sample size precludes any conclusion as to the impact of biologic agents on survival in this study.


2007 ◽  
Vol 33 (1) ◽  
pp. 13-22 ◽  
Author(s):  
Tatsuo Shiigai

Abstract This pilot study assessed the impact of implant stability criteria on implant loading time. Insertion torque (IT) and baseline implant stability quotients (ISQ) from resonance frequency analysis were recorded for 41 tapered-screw implants placed in the mandibles of 20 patients. Immediate (IL), early (EL) (6 weeks postoperative), or delayed (DL) (12 weeks postoperative) prosthetic loading was performed based on results and study criteria. Postoperative ISQ values were also recorded at 6 time intervals for the IL and EL groups and after healing for the DL group. All implants were definitively restored after 6 weeks of provisionalization. There were no failures, complications, or stability differences based on loading time. At week 12 of follow-up, ISQ values were slightly higher for IL implants compared with EL implants. IL and EL implants showed significantly higher cumulative ISQ values compared with DL implants. High IT and baseline ISQ values in all groups corresponded to high implant survival. Postoperative ISQ values in the IL and EL groups were less certain indicators of implant survival because of fluctuations relative to baseline values. Small sample size and inequitable patient distribution across groups skewed results. More research is needed before definitive results can be drawn. IL and EL were safely performed within the IT and ISQ ranges in this study, but it is unknown whether EL criteria would have also sufficed for IL.


2019 ◽  
Vol 129 (4) ◽  
pp. 127-131
Author(s):  
Agnieszka Parfin ◽  
Krystian Wdowiak ◽  
Marzena Furtak-Niczyporuk ◽  
Jolanta Herda

AbstractIntroduction. The COVID-19 is the name of an infectious disease caused by a new strain of coronavirus SARS-CoV-2 (Severe Acute Respiratory Syndrome Coronavirus 2). It was first diagnosed in December 2019 in patients in Wuhan City, Hubei Province, China. The symptoms are dominated by features of respiratory tract infections, in some patients with a very severe course leading to respiratory failure and, in extreme cases to death. Due to the spread of the infection worldwide, the WHO declared a pandemic in March 2020.Aim. An investigation of the impact of social isolation introduced due to the coronavirus pandemic on selected aspects of life. The researchers focused on observing changes in habits related to physical activity and their connections with people’s subjective well-being and emotional state.Material and methods. The study was carried out within the international project of the group „IRG on COVID and exercise”. The research tool was a standardized questionnaire.Results. Based on the data collected and the analysis of the percentage results, it can be observed that the overwhelming majority of people taking up physical activity reported a better mood during the pandemic. However, statistical tests do not confirm these relationships due to the small sample size.Conclusions. Isolation favours physical activity. Future, in-depth studies, by enlarging the population group, are necessary to confirm the above observations.


Author(s):  
Seiyeong Park ◽  
Junhye Kwon ◽  
Chiyoung Ahn ◽  
Hae-Sung Cho ◽  
Hyo Youl Moon ◽  
...  

Previous studies have identified that a behavior can occur through the strongest predictor intention, but there is a gap between intention and behavior. Dopamine receptor D2 (DRD2) is known to account for a variance in sporting behaviors in human and animal subjects. However, the relationship between DRD2 and sport participation has been poorly studied, and the limited available reports are inconsistent. The present study was performed to examine the impact of DRD2 on sport participation among Korean university students based on the integrated behavioral model (IBM). Data were collected from enrolled university students in Seoul (N = 45). Participants answered survey questions first, and then they gave investigators their hair to provide DNA information (i.e., the A1 allele of DRD2). DRD2 had a significant effect on sport participation, but only in male students. Male students who carried the A1 allele of DRD2 significantly participated in 105.10 min more sporting activities than male students who did not. Moreover, the effect of intention on sport participation was significantly decreased when considering DRD2. Despite the small sample size, the results of this study could be a preliminary case for a larger study and indicate the direction of future research. Our results suggest that DRD2 may have played an important role as the “actual skill” shown in the IBM.


2021 ◽  
Vol 12 ◽  
pp. 215013272110177
Author(s):  
Marla A. DeWitt ◽  
Ivana T. Croghan ◽  
Celine M. Vachon ◽  
Thomas D. Thacher ◽  
Marcia R. Venegas Pont ◽  
...  

Objective: The primary aim of this study was to evaluate the feasibility of collecting risk factor information and accessing digitized mammographic data in a medically marginalized population. A secondary aim was to examine the association between vitamin D status and mammographic density. Methods: Breast-screening examinations were provided for age-appropriate patients, and a referral for no-cost screening mammography was offered. Study participants were asked to undergo 25-hydroxyvitamin D testing at mammography and 1-year follow-up. Results: Of 62 women approached, 35 (56%) consented to participate. Of 32 participants who had baseline mammography, the median mammographic density measured by VolparaDensity (Volpara Solutions Limited) was 5.7%. After 1 year, 9 women obtained follow-up mammograms, with a median density of 5.7%. Vitamin D status was measured for 31 participants at baseline and 13 participants in the following year. Insufficient vitamin D status (<30 ng/mL) was noted in 77% at each time point. Mammographic density was not significantly correlated with vitamin D status ( P = .06). Conclusions: On the basis of this small pilot study, vitamin D insufficiency is common in this study population. Owing to the small sample size, an association between vitamin D insufficiency and breast density was not clear. Additional unexpected findings included substantial barriers in initial access to care and longitudinal follow-up in this population. Further study of these issues is needed.


PEDIATRICS ◽  
1993 ◽  
Vol 92 (2) ◽  
pp. 300-301
Author(s):  
DOREN FREDRICKSON

To the Editor.— I wish to comment on the study reported by Cronenwett et al,1 which was a fascinating prospective study among married white women who planned to breast-feed. Women were randomly selected to perform either exdusive breast-feeding or partial breast-feeding with bottled human milk supplements to determine the impact of infant temperament and limited bottle-feeding on breast-feeding duration. The authors admit that small sample size and lack of statistical power make a false-negative possible.


Author(s):  
Gaelle Vofo ◽  
Marrigje Aagje de Jong ◽  
Michal Kaufman ◽  
Julia Meyler ◽  
Ron Eliashar ◽  
...  

Abstract Objectives Idiopathic sudden sensorineural hearing loss (SSNHL) represents a frequently encountered otological entity, of various types and severity, with an array of associated symptoms including vertigo. This is a devastating life-changing condition with a blurry prognosis. The objective of this study was to determine the clinical association of vestibular impairment by electronystagmography (ENG) and caloric tests, and their ability to predict prognosis. Methods An observational, crossectional study was carried out amongst patients admitted with SSNHL. Each consenting patient had an audiometry test performed on admission as well as ENG and caloric tests. Treatment included oral steroids and carbogen with intratympanic steroids used only as salvage treatment. Follow-up was completed after 6 months when hearing gains were evaluated. Finally, an association was sought between the rate of recovery and ENG and caloric test results. Results Of 35 patients included, marked recovery was seen in patients without vertigo when compared to those with vertigo (p=0.003). A statistically significant association was found between the presence of vertigo and hearing deterioration (p=0.008). More so, normal electronystagmography results were associated with marked recovery (p=0.04). Conclusions The vestibular end organs are both subjectively and objectively affected in SSNHL as demonstrated by the abnormal ENG and caloric tests in our study despite the small sample size. Concomitant vestibular involvement carries poorer prognosis and routine identification may help foresee the recovery of patients with SSNHL and as such, aid in patient counseling. ENG and caloric tests are easily available and may be recommended for all patients with SSNHL.


2021 ◽  
pp. 20210414
Author(s):  
Mark Paxton ◽  
Eitan Barbalat ◽  
Nathan Perlis ◽  
Ravi J Menezes ◽  
Mark Gertner ◽  
...  

Objective: Determine the multiparametric magnetic resonance imaging (mpMRI) appearance of the prostate following focal laser ablation (FLA) for PCa and to identify imaging characteristics associated with recurrent disease. Methods: Retrospective analysis of patients who underwent FLA for low-intermediate risk PCa between 2010 and 2014 was performed. Early (median 4 months) and late mpMRI (median 49 months) follow-up were qualitatively assessed for T2-weighted, dynamic contrast enhanced (DCE) and diffusion weighted imaging (DWI) appearances and also compared to corresponding PSA values and biopsy results. Results: 55 cancers were treated in 54 men (mean age 61.0 years). Early mpMRI was performed in 30 (54.5%) patients while late follow-up mpMRI in 42 (84%). Ill-defined scarring with and without atrophy at the treatment site were the most common appearances. In patients with paired MRI and biopsy, one of four patients with clinically significant PCa on biopsy (≥GG2 or≥6 mm GG1) showed hyperenhancement or restricted diffusion at early follow-up. At late follow-up, positive biopsies were seen in 5/8 (63%) cases with hyperenhancement and 5/6 (83%) cases with restricted diffusion at the treatment site. PSA change was not associated with biopsy results at either time point. Conclusion: mpMRI is able to document the morphological and temporal changes following focal therapy. It has limited ability to detect recurrent disease in early months following treatment. Late-term mpMRI is sensitive at identifying patients with recurrent disease. Small sample size is, however, a limitation of the study. Advances in knowledge: Implementing MRI in follow-up after FT may be useful in predicting residual or recurrent PCa and therefore provide reliable outcome data.


Sign in / Sign up

Export Citation Format

Share Document