A case study of an adaptive design for a clinical trial with 2 doses and 2 endpoints in a rare disease area

Infantile choriocarcinoma is an extremely rare disease. We present a case study of a 1-month-old male with choriocarcinoma diagnosed simultaneously with his mother. On admission to hospital, the disease was very advanced and massive progression and multi-organ failure caused the death of the patient despite the implemented treatment. It was too late to save the child’s life, but early enough to save his mother. The authors believe that the serum levels of hCG should be determined in every newborn with anemia and liver tumor, especially when the mother has a positive history of miscarriage.

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Optimizing adaptive design for Phase 2 dose-finding trials incorporating long-term success and financial considerations: A case study for neuropathic pain

Contemporary Clinical Trials ◽

10.1016/j.cct.2017.03.011 ◽

2017 ◽

Vol 57 ◽

pp. 69-86

Author(s):

Jingjing Gao ◽

Narinder Nangia ◽

Jia Jia ◽

James Bolognese ◽

Jaydeep Bhattacharyya ◽

...

Keyword(s):

Neuropathic Pain ◽

Adaptive Design ◽

Dose Finding ◽

Phase 2

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Planning for an adaptive design: a case study in COPD

Pharmaceutical Statistics ◽

10.1002/pst.223 ◽

2006 ◽

Vol 5 (2) ◽

pp. 135-144 ◽

Cited By ~ 1

Author(s):

B. Jones ◽

G. Atkinson ◽

J. Ward ◽

E. Tan ◽

T. Kerbusch

Keyword(s):

Adaptive Design

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Current Advances in Clinical Trials for Rare Disease Populations: Spotlight on the Patient

Current Clinical Pharmacology ◽

10.2174/1574884716666210316120615 ◽

2021 ◽

Vol 16 ◽

Author(s):

Erica Winter ◽

Scott Schliebner

Keyword(s):

Clinical Trial ◽

Clinical Trials ◽

Drug Development ◽

Rare Disease ◽

Rare Diseases ◽

Statistical Analyses ◽

Clinical Trial Designs ◽

Novel Approaches

: Characterized 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 patient.

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Exploring the Limitations of Responsive Design Through a Case Study Approach

International Journal of Web Portals ◽

10.4018/ijwp.2021010104 ◽

2021 ◽

Vol 13 (1) ◽

pp. 62-73

Author(s):

Fernando Almeida ◽

José Augusto Monteiro

Keyword(s):

Adaptive Design ◽

Web Design ◽

Dynamic Adaptation ◽

Access Device ◽

Development Models ◽

Study Approach ◽

Responsive Design ◽

Responsive Web Design ◽

Further Development

Having an online presence is essential for any company regardless of its size and type of business. Users are currently striving to interact with companies through the web, regardless of their access device. In this sense, responsive web design emerged as a very useful technique that allows the dynamic adaptation of the design regardless of the size and resolution of the access device. Despite the unequivocal advantages associated with this technique, there are also limitations which turn this approach not feasible or advisable for all projects. This study, through the realization of five case studies, seeks to identify the main limitations of responsive design and responsive design frameworks. Additionally, this study suggests further development models that may be more effective in the dynamic adaptation of the design and contents according to the features of the access device, such as the adoption of adaptive design, use of native apps, and hybrid models.

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Architecture of an Integrated Collaboration Portal for Clinical Trial

Software Innovations in Clinical Drug Development and Safety - Advances in Medical Technologies and Clinical Practice ◽

10.4018/978-1-4666-8726-4.ch007 ◽

2015 ◽

pp. 127-162

Author(s):

Partha Chakraborty

Keyword(s):

Clinical Trial ◽

Development Cost ◽

Training Activities ◽

The Cost ◽

Key Events ◽

Document Review ◽

Patient Enrollment ◽

And Training ◽

Clinical Trial Activity

Collaboration is defined as the actions for individuals and teams to work together for a common goal. There are several bottlenecks to an efficient and effective collaborative model of clinical trial including: the lack of a centralized, consistent, globally accessible platform to manage and store essential study related documentation; inconsistent or incomplete work assignments; inefficient notification of key events requiring follow-on action; and incomplete, missing, expired, or redundant documentation and training activities and need to maintain multiple credential to access various system, Removing these barriers is an important part of establishing an environment that fosters collaboration among all constituencies involved in managing clinical trial keeping them connected, informed, and on task by providing access to everyone at any time, from anywhere.The case study below introduces need of an integrated clinical collaboration platform, addressing key functionality of such an platform and describes the architecture & design consideration to industrialize such a platform. The intended audience of this case study is the architects & designers of similar systems. The clinical trial activity for a drug in research is approximately 70% of the overall drug development cost. It is estimated that 4% of the cost of a trial is in 'rework' involving communication, regulatory issues, patient enrollment, document review and replacement of patients. The integrated clinical collaboration platform has potential to eliminate significant amount of cost of re-work, which is in order of $3.5M per trial.

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Disclosure: A Case Study of Communication about Medically Acquired Risk for a Rare Disease

The Knowledgeable Patient ◽

10.1002/9781444346855.ch7 ◽

2011 ◽

pp. 82-93

Author(s):

Rebecca E. Ryan ◽

Jessica Kaufman ◽

Sophie Hill

Keyword(s):

Rare Disease

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Transition towards Energy Efficiency: Developing the Nigerian Building Energy Efficiency Code

Sustainability ◽

10.3390/su10082620 ◽

2018 ◽

Vol 10 (8) ◽

pp. 2620 ◽

Cited By ~ 4

Author(s):

Susanne Geissler ◽

Doris Österreicher ◽

Ene Macharm

Keyword(s):

Energy Efficiency ◽

Adaptive Design ◽

Noise Pollution ◽

Building Energy ◽

Legal Framework ◽

Building Energy Efficiency ◽

To Come ◽

Energy Efficiency Policies ◽

Objective Being

In Nigeria, there is an estimated deficit of 17 million housing units. Power supply is insufficient, and the electricity supply for about 60 million Nigerians relies on private generators, causing noise, pollution, and high expenditures for mainly imported fuel. Altogether, current challenges clearly demonstrate the need for effective energy efficiency policies targeting also the building sector. The Nigerian Energy Support Program began in 2013, among others, with the objective being to support the Nigerian Government in developing the Nigerian Building Energy Efficiency Code. This paper presents two preparatory activities carried out in order to come up with suggestions for a legal framework well suited for the situation on the ground: the Case Study Building Analysis carried out in collaboration with a Nigerian developer and the Nigerian Building Energy Efficiency Guideline, elaborated together with stakeholders. The results of preparatory activities pointed out that the code must put emphasis on climate adaptive design and must define requirements and procedures in a clear and simple way to allow for effective enforcement. Only then can energy-efficient mass housing be feasible in Nigeria. The paper concludes with a description of the Nigerian Building Energy Efficiency Code (BEEC), officially approved and launched by the Federal Minister of Power, Works and Housing on 29 August 2017.

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