PNS262 GLOBAL TRENDS IN REGULATORY LEGISLATION FOR POST MARKETING SURVEILLANCE (PMS) REAL-WORLD EVIDENCE (RWE) STUDIES AND THE SECONDARY USE OF HEALTH DATA IN PMS STUDIES.

Abstract Background Rituximab is conditionally approved in Japan for use in patients with refractory nephrotic syndrome. To meet the conditions of approval, an all-case post-marketing surveillance study was conducted to confirm the real-world safety and efficacy of rituximab in patients of all ages with refractory nephrotic syndrome. Methods All patients scheduled to receive rituximab treatment for refractory nephrotic syndrome were eligible to register (registration: August 29, 2014 through April 15, 2016); the planned observation period was 2 years from the initiation of rituximab treatment (intravenous infusion, 375 mg/m2 once weekly for four doses). The study was conducted at 227 hospitals throughout Japan. Adverse drug reactions (ADRs) were collected for safety outcomes. The efficacy outcomes were relapse-free period and the degree of growth in pediatric (< 15 years) patients. Results In total, 997 (447 pediatric) patients were registered; 981 (445) were included in the safety analysis set; 852 (402) completed the 2-year observation period; and 810 (429) were included in the efficacy analysis set. Refractory nephrotic syndrome had developed in childhood for 85.0% of patients, and 54.6% were aged ≥15 years. ADRs were observed in 527 (53.7%) patients, treatment-related infection/infestation in 235 (24.0%) patients, and infusion reactions in 313 (31.9%) patients. The relapse-free period was 580 days (95% confidence interval, 511–664). There was a significant change in height standard deviation score (pediatric patients; mean change, 0.093; standard deviation, 0.637; P = 0.009). Conclusion The safety and efficacy of rituximab treatment in patients with refractory nephrotic syndrome were confirmed in the real-world setting. Clinical trial registration UMIN000014997.

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Real‐world safety of the novel, free radical scavenger edaravone for amyotrophic lateral sclerosis patients: Data from the post‐marketing surveillance SUNRISE Japan

Neurology and Clinical Neuroscience ◽

10.1111/ncn3.12490 ◽

2021 ◽

Author(s):

Kaoru Ishizaki ◽

Kenta Yoshimura ◽

Kengo Yoshida ◽

Masao Matsuda ◽

Yutaka Kawaguchi ◽

...

Keyword(s):

Amyotrophic Lateral Sclerosis ◽

Free Radical ◽

Real World ◽

Free Radical Scavenger ◽

Radical Scavenger ◽

The Novel ◽

Post Marketing Surveillance ◽

Post Marketing ◽

Lateral Sclerosis

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Real-world safety and effectiveness of nivolumab for recurrent or metastatic head and neck cancer in Japan: a post-marketing surveillance

International Journal of Clinical Oncology ◽

10.1007/s10147-021-01949-1 ◽

2021 ◽

Author(s):

Makoto Tahara ◽

Naomi Kiyota ◽

Ken-ichi Nibu ◽

Ayumi Akamatsu ◽

Tomohiro Hoshino ◽

...

Keyword(s):

Head And Neck Cancer ◽

Interstitial Lung Disease ◽

Head And Neck ◽

Neck Cancer ◽

Real World ◽

Special Interest ◽

Real World Setting ◽

Post Marketing Surveillance ◽

Post Marketing ◽

Safety Signals

Abstract Background On the basis of phase III CheckMate 141 results, nivolumab was approved for recurrent or metastatic head and neck cancer after undergoing platinum-containing chemotherapy in Japan. This post-marketing surveillance aimed to evaluate the safety and effectiveness of nivolumab for head and neck cancer in the real-world setting. Methods All patients with head and neck cancer who planned to receive nivolumab were centrally registered. This study monitored 607 patients for 6 months to assess nivolumab’s safety, especially treatment-related adverse events (TRAEs) of special interest, and effectiveness. Results TRAEs occurred in 36.1% patients, with no new safety signals. The most common TRAEs with grade ≥ 3 were interstitial lung disease (1.2%), diarrhea (0.8%), and hepatic function abnormal (0.7%). Meanwhile, thyroid dysfunction (10.2%), hepatic dysfunction (5.3%), and interstitial lung disease (4.1%) were the most common TRAE categories of special interest. Although the median time to the onset of each TRAE category of special interest was mostly 1–2 months, most of them occurred throughout the observation period; nonetheless, the majority of patients recovered or remitted. The 6-month survival rate was 55.9%. Conclusion Japanese patients with head and neck cancer treated with nivolumab in the real-world setting manifested no new safety signals. Clinical Trial Registration clinicaltrials.jp: JapicCTI-184071.

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