scholarly journals PRM18 - REAL-WORLD DATA (RWD) COLLECTION ALONGSIDE EARLY ACCESS TO MEDICINES SCHEMES (EAMS) - NIVOLUMAB IN GASTRIC OR GASTROESOPHAGEAL JUNCTION ADENOCARCINOMA (GC/GEJ) AFTER TWO OR MORE PRIOR THERAPIES

2018 ◽  
Vol 21 ◽  
pp. S358
Author(s):  
C Kiff ◽  
M Wang ◽  
A Amadi ◽  
D Tyas
Keyword(s):  
Real World ◽  
Gastroesophageal Junction ◽  
Access To Medicines ◽  
Real World Data ◽  
World Data ◽  
Early Access ◽  
Gastroesophageal Junction Adenocarcinoma

Early Access to Medicines Scheme: real-world data collection

2019 ◽  
Vol 24 (12) ◽  
pp. 2231-2233
Author(s):  
Hok Pang ◽  
Meng Wang ◽  
Christopher Kiff ◽  
Mira Soni ◽  
Dara Stein ◽  
...  
Keyword(s):  
Data Collection ◽  
Real World ◽  
Access To Medicines ◽  
Real World Data ◽  
World Data ◽  
Early Access

PP81 Real World Data: The Early Access To Medicines Scheme Catches The Worm

2019 ◽  
Vol 35 (S1) ◽  
pp. 53-53
Author(s):  
Adam Hall ◽  
Lok Wan Liu ◽  
Richard Macaulay ◽  
Sean Walsh
Keyword(s):  
Clinical Trial ◽  
Clinical Practice ◽  
Real World ◽  
Clinical Trial Data ◽  
Access To Medicines ◽  
Trial Data ◽  
Real World Data ◽  
World Data ◽  
Early Access ◽  
Nice Guidance

IntroductionThe Early Access to Medicines Scheme (EAMS) aims to provide access to medicines prior to market authorization for patients with severe, life-threatening diseases who do not have adequate treatment options. An EAMS designation enables the potential collection of United Kingdom-specific real world evidence (RWE) prior to health technology assessment (HTA) by the National Institute for Health and Care Excellence (NICE). This research evaluates whether RWE is being gathered through the EAMS and utilized to support HTA submissions.MethodsAll EAMS designations as of 7 November 2018 were identified from the Medicines and Healthcare products Regulatory Agency website. For products with final NICE guidance, all publicly-available NICE documentation was reviewed.ResultsSixteen product and indication pairings with an EAMS designation were identified, with 12 having received final NICE guidance (11 were recommended, 3 were recommended for temporary reimbursement via the Cancer Drugs Fund, and 2 were not recommended). Of the 11 recommended products, seven had references to the number of patients or sites with product access through the EAMS, but only one (dupilumab for atopic dermatitis) had detailed data collected during the EAMS period. The manufacturer of dupilumab reported baseline demographics and disease characteristics from a cohort of 35 patients treated under the EAMS to inform the generalizability of trial populations for clinical practice. Follow-up results from this cohort demonstrated that real-world data on dupilumab effectiveness was comparable with the clinical trial data, despite a higher proportion of patients in the real-world cohort receiving immunosuppressant therapy, which makes improvements in efficacy harder to achieve. The committee also noted that the RWE presented supported the understanding of dupilumab's long-term clinical effectiveness and informed assumptions for the economic model.ConclusionsTo date, the majority of products receiving an EAMS designation have not presented RWE at NICE reappraisal. The case of dupilumab illustrated how RWE collected through the EAMS can be used to reduce uncertainty around how clinical trial data can be translated into clinical practice. In the future, RWE may increasingly be used to help inform NICE decisions.

Neoadjuvant chemoradiation (NCRT) for esophageal (EC) or gastroesophageal junction cancer (GEJC): A Canadian single institution real-world experience using the CROSS trial regimen.

2019 ◽  
Vol 37 (27_suppl) ◽  
pp. 25-25
Author(s):  
Sidra Khalid ◽  
Wilma M. Hopman ◽  
Beatrice Preti ◽  
Anna T. Tomiak ◽  
Kiran Virik
Keyword(s):  
Clinical Trial ◽  
Real World ◽  
Gastroesophageal Junction ◽  
Trial Protocol ◽  
Single Institution ◽  
Real World Data ◽  
Trimodality Therapy ◽  
World Data ◽  
The Real ◽  
The Cross

25 Background: NCRT followed by surgery per the CROSS trial regimen is an accepted standard of care in the treatment of EC and GEJC. When treatments are used in the real-world setting, there are often patient, treatment and potential outcome differences compared to the original clinical trial. The study aim was to assess the real-world application and outcomes of the CROSS trial protocol. Methods: A retrospective chart review was undertaken of 83 patients (pts) with EC or GEJC who were treated from June 2012 to June 2018 with CRT. 65 pts were with NCRT intent to proceed to surgery. Pts’ demographics, clinical, pathological, treatment and surgical characteristics were assessed and exploratory analyses were conducted to review these factors and outcomes. Analyses included Chi-square, t-tests and Kaplan-Meier. Results: For pts who underwent NCRT (n = 65): median age was 68 yrs (range 52-80), male 79%, adenocarcinoma 82%, median (m) tumor length 5 cm, GERD 43%, clinical stage II/III 95%, and BMI > 30 in 37%. 80% completed CRT with RT ≥ 41.4 Gy; of these 88% had ≥ 50.4 Gy. Delay/interruption in chemotherapy occurred in 46% and in RT 37%. Pts who underwent surgery were younger (p = 0.04) and weighed more (p = 0.05). mOS was 37 months (M) v 14 M in those who started CRT ≤ 8 weeks (w) from diagnosis v > 8 w (p = 0.10). The median time from CRT to surgery was 8.9 w. 40 pts had surgery with a complete response in 38% and a R0 resection in 98%. Postoperative major and minor complications occurred in 67%. Those < 75 yrs v ≥ 75 yrs had a mOS of 32 M v 15 M respectively (log rank p = 0.46). 25 pts did not get surgery; 28% was due to death/progression. Pts who proceeded to surgery had a mOS of 35 M v 12 M in pts who did not go to surgery (log rank p = 0.002). Further correlative outcome data will be presented. Conclusions: Real-world data in our center showed patient, tumor and treatment differences compared to the CROSS trial protocol. Despite the broadening of eligibility and treatment criteria, survival in a single institution setting is maintained with trimodality therapy compared to NCRT alone. Real-world data is of value in the assessment of therapeutic validity of clinical trial data.

scholarly journals Ramucirumab plus paclitaxel as second-line treatment in patients with advanced gastric or gastroesophageal junction adenocarcinoma: a nationwide real-world outcomes in Korea study (KCSG-ST19-16)

2021 ◽  
Vol 13 ◽  
pp. 175883592110428
Author(s):  
Hye Sook Han ◽  
Bum Jun Kim ◽  
Hee-Jung Jee ◽  
Min-Hee Ryu ◽  
Se Hoon Park ◽  
...  
Keyword(s):  
Overall Survival ◽  
Confidence Interval ◽  
Real World ◽  
Gastroesophageal Junction ◽  
Locally Advanced ◽  
Line Treatment ◽  
Second Line ◽  
First Line ◽  
Real World Data ◽  
World Data

Background: Ramucirumab as monotherapy or in combination with paclitaxel is a second-line treatment option recommended for patients with locally advanced unresectable or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma. However, real-world data from large study cohorts focused on ramucirumab plus paclitaxel in gastric cancer are limited. Methods: The study population comprised all patients with gastric or GEJ cancer who received ramucirumab plus paclitaxel in South Korea between 1 May 2018 and 31 December 2018. We included patients with advanced gastric or GEJ adenocarcinoma and disease progression after first-line platinum and fluoropyrimidine-containing combination chemotherapy. Results: In total, 1063 patients were included in the present study. The objective response rate and disease control rate were 15.1% and 57.7%, respectively. The median progression-free survival was 4.03 months (95% confidence interval, 3.80–4.27) and the median overall survival was 10.03 months (95% confidence interval, 9.33–10.73). Grade 3 or higher treatment-related adverse events with incidence of ⩾5% were neutropenia (35.1%) and anemia (10.5%). Based on multivariable analysis, overall survival was negatively associated with Eastern Cooperative Oncology Group performance status ⩾2, weight loss ⩾10% in the previous 3 months, GEJ of primary tumor, poor or unknown histologic grade, number of metastatic sites ⩾3, presence of peritoneal metastasis, no prior gastrectomy, and time to second-line since first-line treatment <6 months. Conclusion: Our large-scale, nationwide, real-world data analysis of an unselected real-world population adds evidence for the efficacy and safety of second-line ramucirumab plus paclitaxel in patients with locally advanced unresectable or metastatic gastric or GEJ adenocarcinoma.

Real-world treatment patterns of previously treated advanced gastric and gastroesophageal junction adenocarcinoma (GC) in the United Kingdom (UK).

2015 ◽  
Vol 33 (3_suppl) ◽  
pp. 184-184 ◽  
Author(s):  
Astra M. Liepa ◽  
Jacqueline Brown ◽  
Bela Bapat ◽  
James A. Kaye
Keyword(s):  
Disease Progression ◽  
Real World ◽  
Performance Status ◽  
Gastroesophageal Junction ◽  
Real World Data ◽  
Ecog Performance Status ◽  
Gastroesophageal Junction Adenocarcinoma ◽  
Previously Treated ◽  
The Uk ◽  
Ecog Ps

184 Background: With no licensed therapies for previously treated advanced GC, little is known on how patients (pts) are managed after 1st-line chemotherapy (CTx) has failed. We present real-world data on characteristics, treatments, and resource utilization (RU) for such pts in the UK. Methods: Physicians who treat pts with advanced GC completed a web-based chart review detailing clinical and RU data for 3-4 de-identified pts each. Eligible pts were ≥18 years old, diagnosed Jan 2007-Mar 2012 with advanced GC, received 1st-line fluoropyrimidine+platinum, and had ≥3 months of follow-up after 1st-line discontinuation (DC). Data were summarized descriptively. Results: From Jun to Jul 2013, 58 physicians provided data for 200 pts. Pts’ mean age was 61 years; 69.5% were male. At advanced stage diagnosis, ECOG performance status (PS) was 21% 0, 72.5% 1, and 6.5% 2. The most common 1st-line regimens were capecitabine (cape)+oxaliplatin+epirubicin (epi) (34%), cape+cisplatin+epi (20.5%) and 5-FU+cisplatin+epi (13%). The most common reasons for 1st-line DC were completion of planned regimen (63%) and disease progression (24%). ECOG PS at 1st-line DC was 5% 0, 57.5% 1, 32% 2, 5.5% 3. 28.5% received 2nd-line, and 79% of these had PS 0/1 at start of 2nd-line. 21 unique 2nd-line regimens were reported; most common were docetaxel (28%), paclitaxel (11%), trastuzumab (9%), cape (7%) and irinotecan (7%). Among pts who received 2nd-line, 5% received 3rd-line. (See table.) The most common contributing reasons for hospitalization were palliative care and disease progression. Conclusions: In our study sample of advanced GC, the minority of pts received subsequent CTx after 1st-line CTx. There was considerable variation in 2nd-line regimens, although primarily monotherapy. Pts who received 2nd-line CTx had numerically similar or lower rates of supportive care. [Table: see text]

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