1 / 15

Biostatistical Analysis of Pivotal Studies Shiowjen Lee, Ph.D., Mathematical Statistician

Biostatistical Analysis of Pivotal Studies Shiowjen Lee, Ph.D., Mathematical Statistician Office of Biostatistics OPaSS, CDER, FDA. Efficacy Evaluation in Pivotal Studies. Primary: Treatment Success, is defined as % of patients with Overall

galeno
Download Presentation

Biostatistical Analysis of Pivotal Studies Shiowjen Lee, Ph.D., Mathematical Statistician

An Image/Link below is provided (as is) to download presentation Download Policy: Content on the Website is provided to you AS IS for your information and personal use and may not be sold / licensed / shared on other websites without getting consent from its author. Content is provided to you AS IS for your information and personal use only. Download presentation by click this link. While downloading, if for some reason you are not able to download a presentation, the publisher may have deleted the file from their server. During download, if you can't get a presentation, the file might be deleted by the publisher.

E N D

Presentation Transcript

  1. Biostatistical Analysis of Pivotal Studies Shiowjen Lee, Ph.D., Mathematical Statistician Office of Biostatistics OPaSS, CDER, FDA

  2. Efficacy Evaluation in Pivotal Studies Primary: Treatment Success, is defined as % of patients with Overall Lesional Assessment (OLA) score of 0 or 1 at Wk 12 Secondary: - Clinical signs/symptoms - Overall global response Others: Scalp and nail psoriasis

  3. Presentation of Efficacy Findings in Pivotal Studies • Overall efficacy findings of oral tazarotene vs. placebo • Subgroup efficacy results by gender and by baseline OLA severity • Short-term efficacy of oral tazarotene in treating scalp and nail psoriasis • Relapse rate (short-term)

  4. Baseline Demographic

  5. OLA Score and Treatment Success at Wk 12

  6. Subgroup Results of Treatment Success – Study 048

  7. Subgroup Results of Treatment Success – Study 049

  8. Scalp and Nail Psoriasis • Evaluation of short-term efficacy of tazarotene treatment (12-wk). • Severity was evaluated based on a 5-point scale (0 = none, 1 = mild, 2 = moderate, 3 = severe, and 4 = very severe). • Analysis is performed regarding the percentage of patients having severity score of 0 at Wk 12.

  9. Scalp and Nail Psoriasis – Severity Score of 0 at Wk 12

  10. Relapse Rate • Analysis is based on the patients who were treatment success at Wk 12 and had relapse during the 12-wk post-treatment. • Two definitions of relapse are considered: • Def. (a): Patients who fell back to baseline OLA score or worse. • Def. (b): Patients whose achieved maximal improvement from baseline is reduced by more than 50%.

  11. Relapse On or Prior to Wk 24

  12. Summary of Statistical Analysis in Pivotal Studies • Oral tazarotene is statistically superior to placebo regarding treatment success. Success rates are below 20% for both studies. • Female patients had higher success rates than males though male patients accounted for over 2/3 of study enrollment in each trial.

  13. Summary of Statistical Analysis in Pivotal Studies (con’t) • Treatment success decreases as baseline OLA score increases. A total of 15 patients (2%) had “very severe” OLA score at baseline. None in oral tazarotene group achieved treatment success at Wk 12. There is insufficient data to evaluate the efficacy claim of “very severe” plaque psoriasis.

  14. Summary of Statistical Analysis in Pivotal Studies (con’t) • Oral tazarotene demonstrates short-term efficacy in treating scalp psoriasis, but not nail psoriasis. Two and 1 patients in placebo group achieved severity score of 0 at Wk 12 in fingernail and toenail psoriasis, respectively. However, none was in oral tazarotene group.

  15. Summary of Statistical Analysis in Pivotal Studies (con’t) • Based on definition (a) and definition (b) of relapse, approximately 15% and 30% of patients, respectively, who were treatment success at Wk 12, had relapse at the 12-wk post-treatment period.

More Related