Current Advances in Clinical Trials for Rare Disease Populations: Spotlight on the Patient
Curr Clin Pharmacol. 2021 Mar 16. doi: 10.2174/1574884716666210316120615. Online ahead of print.ABSTRACTCharacterized by small, highly heterogeneous patient populations, rare disease trials magnify the challenges often encountered in traditional clinical trials. In recent years, there have been increased efforts by stakeholders to improve drug development in rare diseases through novel approaches to clinical trial designs and statistical analyses. We highlight and discuss some of the current and emerging approaches aimed at overcoming challenges in rare disease clinical trials, with a focus on the ultimate stakeholder, the...
Source: Current Clinical Pharmacology - March 17, 2021 Category: Drugs & Pharmacology Authors: Erica Winter Scott Schliebner Source Type: research

Novel Study Designs in Precision Medicine - Basket, Umbrella and Platform Trials
This article briefly overviews the characteristics of master protocol designs with examples of completed and ongoing clinical trials utilizing these study designs.PMID:33726655 | DOI:10.2174/1574884716666210316114157 (Source: Current Clinical Pharmacology)
Source: Current Clinical Pharmacology - March 17, 2021 Category: Drugs & Pharmacology Authors: Renju Ravi Harshad V Kesari Source Type: research

Current Advances in Clinical Trials for Rare Disease Populations: Spotlight on the Patient
Curr Clin Pharmacol. 2021 Mar 16. doi: 10.2174/1574884716666210316120615. Online ahead of print.ABSTRACTCharacterized by small, highly heterogeneous patient populations, rare disease trials magnify the challenges often encountered in traditional clinical trials. In recent years, there have been increased efforts by stakeholders to improve drug development in rare diseases through novel approaches to clinical trial designs and statistical analyses. We highlight and discuss some of the current and emerging approaches aimed at overcoming challenges in rare disease clinical trials, with a focus on the ultimate stakeholder, the...
Source: Current Clinical Pharmacology - March 17, 2021 Category: Drugs & Pharmacology Authors: Erica Winter Scott Schliebner Source Type: research

Novel Study Designs in Precision Medicine - Basket, Umbrella and Platform Trials
This article briefly overviews the characteristics of master protocol designs with examples of completed and ongoing clinical trials utilizing these study designs.PMID:33726655 | DOI:10.2174/1574884716666210316114157 (Source: Current Clinical Pharmacology)
Source: Current Clinical Pharmacology - March 17, 2021 Category: Drugs & Pharmacology Authors: Renju Ravi Harshad V Kesari Source Type: research

Current Advances in Clinical Trials for Rare Disease Populations: Spotlight on the Patient
Curr Clin Pharmacol. 2021 Mar 16. doi: 10.2174/1574884716666210316120615. Online ahead of print.ABSTRACTCharacterized by small, highly heterogeneous patient populations, rare disease trials magnify the challenges often encountered in traditional clinical trials. In recent years, there have been increased efforts by stakeholders to improve drug development in rare diseases through novel approaches to clinical trial designs and statistical analyses. We highlight and discuss some of the current and emerging approaches aimed at overcoming challenges in rare disease clinical trials, with a focus on the ultimate stakeholder, the...
Source: Current Clinical Pharmacology - March 17, 2021 Category: Drugs & Pharmacology Authors: Erica Winter Scott Schliebner Source Type: research

Novel Study Designs in Precision Medicine - Basket, Umbrella and Platform Trials
This article briefly overviews the characteristics of master protocol designs with examples of completed and ongoing clinical trials utilizing these study designs.PMID:33726655 | DOI:10.2174/1574884716666210316114157 (Source: Current Clinical Pharmacology)
Source: Current Clinical Pharmacology - March 17, 2021 Category: Drugs & Pharmacology Authors: Renju Ravi Harshad V Kesari Source Type: research

Current Advances in Clinical Trials for Rare Disease Populations: Spotlight on the Patient
Curr Clin Pharmacol. 2021 Mar 16. doi: 10.2174/1574884716666210316120615. Online ahead of print.ABSTRACTCharacterized by small, highly heterogeneous patient populations, rare disease trials magnify the challenges often encountered in traditional clinical trials. In recent years, there have been increased efforts by stakeholders to improve drug development in rare diseases through novel approaches to clinical trial designs and statistical analyses. We highlight and discuss some of the current and emerging approaches aimed at overcoming challenges in rare disease clinical trials, with a focus on the ultimate stakeholder, the...
Source: Current Clinical Pharmacology - March 17, 2021 Category: Drugs & Pharmacology Authors: Erica Winter Scott Schliebner Source Type: research

Novel Study Designs in Precision Medicine - Basket, Umbrella and Platform Trials
This article briefly overviews the characteristics of master protocol designs with examples of completed and ongoing clinical trials utilizing these study designs.PMID:33726655 | DOI:10.2174/1574884716666210316114157 (Source: Current Clinical Pharmacology)
Source: Current Clinical Pharmacology - March 17, 2021 Category: Drugs & Pharmacology Authors: Renju Ravi Harshad V Kesari Source Type: research

Current Advances in Clinical Trials for Rare Disease Populations: Spotlight on the Patient
Curr Clin Pharmacol. 2021 Mar 16. doi: 10.2174/1574884716666210316120615. Online ahead of print.ABSTRACTCharacterized by small, highly heterogeneous patient populations, rare disease trials magnify the challenges often encountered in traditional clinical trials. In recent years, there have been increased efforts by stakeholders to improve drug development in rare diseases through novel approaches to clinical trial designs and statistical analyses. We highlight and discuss some of the current and emerging approaches aimed at overcoming challenges in rare disease clinical trials, with a focus on the ultimate stakeholder, the...
Source: Current Clinical Pharmacology - March 17, 2021 Category: Drugs & Pharmacology Authors: Erica Winter Scott Schliebner Source Type: research

Novel Study Designs in Precision Medicine - Basket, Umbrella and Platform Trials
This article briefly overviews the characteristics of master protocol designs with examples of completed and ongoing clinical trials utilizing these study designs.PMID:33726655 | DOI:10.2174/1574884716666210316114157 (Source: Current Clinical Pharmacology)
Source: Current Clinical Pharmacology - March 17, 2021 Category: Drugs & Pharmacology Authors: Renju Ravi Harshad V Kesari Source Type: research

Current Advances in Clinical Trials for Rare Disease Populations: Spotlight on the Patient
Curr Clin Pharmacol. 2021 Mar 16. doi: 10.2174/1574884716666210316120615. Online ahead of print.ABSTRACTCharacterized by small, highly heterogeneous patient populations, rare disease trials magnify the challenges often encountered in traditional clinical trials. In recent years, there have been increased efforts by stakeholders to improve drug development in rare diseases through novel approaches to clinical trial designs and statistical analyses. We highlight and discuss some of the current and emerging approaches aimed at overcoming challenges in rare disease clinical trials, with a focus on the ultimate stakeholder, the...
Source: Current Clinical Pharmacology - March 17, 2021 Category: Drugs & Pharmacology Authors: Erica Winter Scott Schliebner Source Type: research

Novel Study Designs in Precision Medicine - Basket, Umbrella and Platform Trials
This article briefly overviews the characteristics of master protocol designs with examples of completed and ongoing clinical trials utilizing these study designs.PMID:33726655 | DOI:10.2174/1574884716666210316114157 (Source: Current Clinical Pharmacology)
Source: Current Clinical Pharmacology - March 17, 2021 Category: Drugs & Pharmacology Authors: Renju Ravi Harshad V Kesari Source Type: research

Current Advances in Clinical Trials for Rare Disease Populations: Spotlight on the Patient
Curr Clin Pharmacol. 2021 Mar 16. doi: 10.2174/1574884716666210316120615. Online ahead of print.ABSTRACTCharacterized by small, highly heterogeneous patient populations, rare disease trials magnify the challenges often encountered in traditional clinical trials. In recent years, there have been increased efforts by stakeholders to improve drug development in rare diseases through novel approaches to clinical trial designs and statistical analyses. We highlight and discuss some of the current and emerging approaches aimed at overcoming challenges in rare disease clinical trials, with a focus on the ultimate stakeholder, the...
Source: Current Clinical Pharmacology - March 17, 2021 Category: Drugs & Pharmacology Authors: Erica Winter Scott Schliebner Source Type: research

Novel Study Designs in Precision Medicine - Basket, Umbrella and Platform Trials
This article briefly overviews the characteristics of master protocol designs with examples of completed and ongoing clinical trials utilizing these study designs.PMID:33726655 | DOI:10.2174/1574884716666210316114157 (Source: Current Clinical Pharmacology)
Source: Current Clinical Pharmacology - March 17, 2021 Category: Drugs & Pharmacology Authors: Renju Ravi Harshad V Kesari Source Type: research

Current Advances in Clinical Trials for Rare Disease Populations: Spotlight on the Patient
Curr Clin Pharmacol. 2021 Mar 16. doi: 10.2174/1574884716666210316120615. Online ahead of print.ABSTRACTCharacterized by small, highly heterogeneous patient populations, rare disease trials magnify the challenges often encountered in traditional clinical trials. In recent years, there have been increased efforts by stakeholders to improve drug development in rare diseases through novel approaches to clinical trial designs and statistical analyses. We highlight and discuss some of the current and emerging approaches aimed at overcoming challenges in rare disease clinical trials, with a focus on the ultimate stakeholder, the...
Source: Current Clinical Pharmacology - March 17, 2021 Category: Drugs & Pharmacology Authors: Erica Winter Scott Schliebner Source Type: research