HIGHLIGHTING THE MULTIDIMENSIONAL BENEFITS OF A DRUG USING GENERALIZED PAIRWISE COMPARISONS

Abstract

Objectives: As the incidence of cancer is increasing and the concern about the sustainability of cancer treatment benefits is rising, recognizing the value of anti-PD(L)-1 inhibitors in neo-adjuvant/adjuvant settings is crucial. Research has shown that anti-PD(L)-1 inhibitors improve recurrence-free survival time in different tumor types for patients diagnosed with an early-stage cancer. We developed a health outcomes projection tool to estimate the health benefits of introducing anti-PD(L)-1 inhibitors to treat patients with an early-stage cancer across different tumor types in Belgium. Methods: A multi-tumor, multi-indication model was developed to compare the health outcomes at the population level for two scenarios: one where anti-PD(L)1s can be used for patients with early-stage cancers for the approved neoadjuvant/adjuvant indications, versus a second scenario where anti-PD(L)1s are reserved for patients who develop advanced/metastatic cancer. The model assessed patients with early-stage melanoma (stage III), renal cell carcinoma (RCC), and triple-negative breast cancer (TNBC). For each tumor/indication, a 4-health state Markov model was implemented to quantify life years (LYs) without recurrence/event, quality-adjusted life years (QALYs), number of patients treated with metastatic disease , and deaths. Inputs included clinical trial data and Belgian publicly available data (i.e. number of eligible patients, treatment uptake, etc.). Results: In Belgium, the introduction of anti-PD(L)1s to treat early-stage cancers (stage III melanoma, RCC and TNBC) is anticipated to avoid 413 recurrences (25%), while increasing in 670 the LYs without recurrence (7%), preventing 459 patients (29%) from being treated for metastatic disease, and avoiding 123 post-recurrence deaths (24%) between 2022 and 2026. Conclusions: Using anti-PD(L)1 inhibitors to treat early-stage cancers can reduce the number of recurrences, extend the time spent by patients free of cancer, and reduce the number of patients requiring metastatic treatment. This model can help inform planning and future discussions around investment in innovative treatments for early-stage cancers. Objectives: In the United States, real-world evidence on apalutamide (APA) for treatment of patients with metastatic castration-sensitive prostate cancer (mCSPC) is limited. This study describes prostate-specific antigen (PSA) response and treatment persistence among patients with mCSPC initiated on APA, with stratifications for Black/non-Black patients. Methods: Clinical data from 77 community-based urology practices were used to evaluate patients with mCSPC who received $1 APA dispensation (index date = first dispensation). Patients who previously used another next-generation antiandrogen or had ,12 months of pre-index clinical activity were excluded. Patients were followed from index date to end of clinical activity or data availability (4/1/2022). On-treatment PSA response ($90% decline from baseline PSA [PSA90]) 12-months post-index was assessed among patients with reported baseline PSA ,13 weeks prior to or on the index date. Persistence was defined as the percentage of patients with no gap in treatment .60 or .90 days of supply at 6-and 12-months post-index. Outcomes were described overall and stratified for Black/non-Black patients. Results: Overall, 589 patients with mCSPC initiated APA (mean age 75.9 years; 98 Black [16.6%], 424 non-Black [72.0%], and 67 of unknown race [11.4%]). By 12-months, PSA90 response was achieved by 72.3% overall, and 73.1% and 70.8% for Black and non-Black patients, respectively. Persistence at 6 months using .60-and .90-day gaps were 92.7% and 96.8% overall, 95.4% and 98.5% for Black patients, and 93.4% and 96.9% for non-Black patients, respectively. Persistence at 12 months using .60-and .90-day gaps were 89.1% and 94.9% overall, 93.3% and 97.8% for Black patients, and 89.3% and 94.4% for non-Black patients, respectively. Conclusions: Patients with mCSPC initiated on APA exhibited high real-world PSA response rates, and few patients had gaps in therapy. Robust real-world PSA response rates and treatment persistence were also demonstrated for both Black and non-Black patients. Objectives: In view of the publication of the draft 'Chinese Guidelines for the Application of Patient-reported Outcomes in Drug Clinical Research' (December, 2021) we examined the availability of clinical outcomes assessment (COA) instruments in Chinese language. We explored therapeutic areas where the use of COA was well established (oncology, endocrine metabolism, rheumatology, dermatology, gastroenterology, and neurology) and checked for the availability of COAs translated into traditional or simplified Chinese as well as Mandarin or Cantonese dialects. Methods: Translations available for each disease area were searched in the ePRO-VIDE database. The age group for selected COA instruments was adult population (18+). Only those COA instruments that had existing translations in Chinese (traditional or simplified, Mandarin or Cantonese dialects) were included in the final sample. Results: A total of 60 instruments were identified in the pre-specified disease areas. The largest number of available COAs translated into Chinese language were found in the following disease areas: endocrine metabolism (n=22), rheuma-tology (n=13), followed by dermatology (n=10), oncology (n=7), gastroenterology (n=5) and neurology (n=3). The vast majority of identified COAs were patient reported outcome measures. Conclusions: Several COA instruments are available for the Chinese market already, but further use in drug development and suitability for Chinese regulatory opinion is still to be determined. Most instruments have been developed into traditional or simplified Chinese, rather than Chinese dialects spoken in specific regions. The availability of validated translations of COAs in Chinese language will allow sponsors to expand their clinical investigations to this region, making new treatments more accessible to Chinese patient populations.