scholarly journals Modification of Asthma Clinical Trial Treatment Efficacy by Social and Environmental Exposures

2021 ◽  
Vol 2021 (1) ◽  
Author(s):  
Lizbeth Gomez ◽  
Ellen Kinnee ◽  
Joel D. Kaufman ◽  
Fernando Holguin ◽  
Michael T. Young ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Treatment Efficacy ◽  
Environmental Exposures ◽  
Trial Treatment

Out of Compassion of Out of Rights? A Story about an Amyotrophic Lateral Sclerosis (ALS) Human Clinical Trial

2021 ◽  
Vol 8 (3) ◽  
pp. 1-23
Author(s):  
Hedva Eyal
Keyword(s):  
Clinical Trial ◽  
Compassionate Use ◽  
Human Clinical Trial ◽  
Biotech Company ◽  
Patient Organisations ◽  
Informed Consent Form ◽  
Trial Treatment ◽  
Post Trial ◽  
The Right ◽  
Lateral Sclerosis

This article examines the intersection of compassion and rights, and how the two concepts are constituted and wielded in the context of human clinical trials. Doron, an ALS patient who was recruited to a clinical trial, believed that he had the right to post-trial treatment according to the wording of an informed consent form he signed before joining the trial. However, the biotech company sponsoring the trial instead offered him ‘compassionate use’ access, i.e., access at its discretion rather than as a legal obligation on its part. I argue that under a ‘bioeconomy of value’, the human clinical trial regime has been subordinated to two competing discourses: that of compassion and that of patients’ rights. Both are interpreted and deployed differently by the different stakeholders, namely the patient, the biotech company, and the medical establishment. I argue that the adoption, by bioeconomy actors, of a social value discourse of compassion is designed to preserve a hierarchy that deprives the patient of their power and their rights. Simultaneously, this practice highlights the power of the biotech industry as a moral partner and ‘saviour’ in its relationship with patient organisations and its role as a medical–scientific actor in the Israeli healthcare system.

A methodology for the extraction and analysis of real-world radiographic and electronic medical record data to reconstruct treatment arms of historical prostate cancer clinical trials.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. 5033-5033
Author(s):  
William David Lindsay ◽  
Christopher A. Ahern ◽  
Aaron Kamauu ◽  
Robert Wilder ◽  
Karen Chagin ◽  
...  
Keyword(s):  
Prostate Cancer ◽  
Clinical Trial ◽  
Clinical Trials ◽  
Real World ◽  
Castration Resistant Prostate Cancer ◽  
Efficacy Evaluation ◽  
Real World Data ◽  
Trial Treatment ◽  
Picture Archiving And Communication

5033 Background: Real-world evidence (RWE), including synthetic comparator arms created from historical real-world data (RWD), has the potential to support the safety and efficacy evaluation of new medical products. However, many available RWD sources lack the details necessary to reliably identify patients comparable to clinical trial cohorts or to assess essential oncologic efficacy endpoints. This project demonstrates the ability to extract and analyze RWD to identify patients matching eligibility criteria to four historical clinical trials in metastatic castration-resistant prostate cancer (mCRPC), and calculate outcome measures. Methods: A total of 5,741 patients treated for prostate cancer at multiple institutions (2010-2020) were analyzed in two cohorts using data extracted from the EMR, Tumor Registry, Oncology Information System, and Picture Archiving and Communication System. Of 3,486 patients with prostate cancer in Cohort 1, 422 mCRPC patients were identified: those treated with ADT who achieved castration-level testosterone ( < 50 ng/dL), had evidence of metastatic disease, and exhibited rising PSA (PCWG2). These patients were further matched to four historical clinical trial treatment arms (COU-AA-301: 49, COU-AA-302: 143, AFFIRM: 30, PREVAIL: 79), based on prior chemotherapy and receipt of Abiraterone or Enzalutamide. Overall survival (OS) and time to skeletal related events (SRE) (pathological fracture, spinal compression, surgery to bone, and radiotherapy to bone) were calculated based on diagnosis and procedure codes using the Kaplan-Meier (KM) Estimator. Of 2,255 patients with prostate cancer in Cohort 2, 101 patients received Abiraterone or Enzalutamide and 59 patients had sufficient baseline and follow-up imaging to be scored. Radiographic progression-free survival (rPFS) was calculated from the start of treatment to the time of progression (RECIST 1.1) or loss to follow-up using the KM estimator. Results: In Cohort 1, median OS was 37.7 months (95% CI: 31.5-NR), and median time to SRE was 17.9 months (13.5-22.6). Median OS per patient cohort matched to historical trial treatment arm was COU-AA-301: 23.7 months (10.7-NR), COU-AA-302: 45.9 months (34.9-NR), AFFIRM: 35.3 months (6.34-NR), PREVAIL: 41.5 months (21.9-NR). In Cohort 2, median rPFS was 37.2 months (13.3-NR). Conclusions: The methodology employed in this analysis not only successfully identified a cohort of RWD patients similar to clinical trial-defined patients, but also curated sufficiently reliable data to calculate essential endpoints (e.g., rPFS). At scale, this methodology can be used to generate RWE, including synthetic comparator arms to support clinical trials with radiographic endpoints.

scholarly journals What Are the Participants’ Perspectives of Taking Melatonin for the Treatment of Nocturia in Multiple Sclerosis? A Qualitative Study Embedded within a Double-Blind RCT

2018 ◽  
Vol 2018 ◽  
pp. 1-9
Author(s):  
Rafiyah Khan ◽  
Alan Uren ◽  
Luke Canham ◽  
David Cottrell ◽  
Marcus J. Drake ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Clinical Trial ◽  
Psychological Functioning ◽  
Qualitative Interviews ◽  
Negative Effects ◽  
Double Blind ◽  
Trial Treatment ◽  
The Central Nervous System ◽  
Qualitative Exploration ◽  
The Impact

Background. Multiple Sclerosis (MS) is a chronic neurological disorder caused by neurodegeneration within the central nervous system. It results in impaired physical, cognitive, and psychological functioning and can also lead to lower urinary tract symptoms including nocturia. While clinical trials have suggested an association between nocturia and melatonin secretion, to our knowledge, no qualitative research has been conducted on the experience of taking melatonin to treat nocturia in progressive MS within a clinical trial. Methods. 17 semistructured qualitative interviews were conducted as part of a double-blind, randomised, placebo controlled, crossover, clinical trial with consenting adults with MS. Interviews explored participants’ experiences of nocturia associated with MS and their experience of taking melatonin as a trial treatment for nocturia versus a placebo. Data was analysed using a thematic analysis. Results. Themes on the experience of nocturia revealed participants’ understandings of nocturia, the impact it had on their night, and increased daily fatigue. Themes on the intervention showed perceived improvements to nocturia, sleep, and energy and negative effects including lethargy, a lack of significant change, and physical side effects including vivid dreams. Conclusion. This qualitative exploration revealed an association between nocturia and increased levels of fatigue during the day by those with MS. However, perspectives towards the effectiveness of melatonin as a potential treatment varied as both placebo and melatonin were perceived as having very similar effects.

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