Patient engagement in clinical trial design and implementation: A pragmatic approach to valued insights.

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. e14084-e14084
Author(s):  
Dawn Aubel ◽  
Susan Hayes ◽  
Geoffrey Cook ◽  
Alexey Salamakha ◽  
Andrew Ciupek ◽  
...  
Keyword(s):  
Drug Development ◽  
Patient Engagement ◽  
Development Process ◽  
Development Program ◽  
Clinical Trial Design ◽  
Published Data ◽  
Drug Development Process ◽  
Plain Language ◽  
Patient Centered ◽  
Oncology Patient

e14084 Background: Evidence-based guidance on how to best facilitate patient engagement in the drug development process is lacking1,2.Well-informed, engaged patients and advocates can contribute meaningfully throughout drug development process for new therapeutics3. Regulatory agencies, including the FDA and EMA, concur4,5. However, identifying and effectively implementing meaningful, and structured patient engagements has been challenging for research organizations6. Methods: Patient advocates with deep knowledge of the special needs of patients within their respective disease areas and experience in patient-centered research and programming were identified by the patient relations’ team of the organization. Participating advocates were generally contracted for consulting support across the development lifecycle to provide timely feedback for emerging topics and agreed to provide feedback within a 3-4 week timeframe to requests for support. Called “Global Oncology Patient Insight Panel” (GOPIP), the approach facilitated fast-moving development teams to quickly engage advocates for advice while enabling projects remain on timeline. GOPIP members engaged with teams within the organization on an ad-hoc basis across the drug development process, as early as protocols’ and informed consents’ review and PRO selection, among many others patient-centered projects. Results: GOPIP engagements included 49 advocates from 19 countries, across 14 disease areas, in the program’s first 18 months. 54 projects were completed. Multiple engagements for three clinical trials, part of the development program, have been accomplished. Seven plain language summaries of published data results were reported publicly with consultants’ input. Satisfaction was high among advocates as well as internal teams. Conclusions: The Patient Insight Panel model is a valuable resource to facilitate patient engagement efforts. More research is needed to evaluate the outcomes and effectiveness of the method. References: 1. Chalasani, M., et al. 2018. Res Involv Engagem. 4,10 2. de Wit, M., et al. 2019. The Lancet, 393(10176),1095-1096 3. Kuehn, C. M. 2018. Ther. Innov. Regul. Sci., 52(5),661–668 4. FDA. 2020. https://www.fda.gov/about-fda/oncology-center-excellence/patient-focused-drug-development . Accessed February 8, 2020 5. EMA. 2016. https://www.ema.europa.eu/en/documents/other/appendix-2-guideline-evaluation-anticancer-medicinal-products-man_en.pdf Accessed February 8, 2020 6. Geissler, J., et al. 2017. Ther. Innov. Regul. Sci., 51(5),612-619.

Early Commercial Assessments: An Innovative Tactic for Small Biotechs

2015 ◽  
Vol 21 (2) ◽  
Author(s):  
Anthony Giovinazzo
Keyword(s):  
Drug Development ◽  
Development Process ◽  
Unmet Need ◽  
Development Program ◽  
Medical Community ◽  
Drug Development Process ◽  
Interesting Problem ◽  
Biotech Company ◽  
Drug Development Program

As a small biotech company embarks on a drug development program, there is a tendency for management to focus on a well-defined set of issues. Is the science behind the drug valid? Does it solve an interesting problem or unmet need? What are the prospects that the medical community and patients will embrace it as a valuable new solution? And what is the range of indications the drug is conceived as addressing? These are all valid questions, but a small biotech can do even more to prepare itself for the drug development process, which is a journey that can take many years and cost a significant amount of money. 

Intelligent Drug Development

2014 ◽  
Author(s):  
Michael Tansey
Keyword(s):  
Clinical Trials ◽  
Cost Effectiveness ◽  
Drug Development ◽  
Clinical Research ◽  
Development Process ◽  
Drug Development Process ◽  
Individual Trial ◽  
Specific Technology

Clinical research is heavily regulated and involves coordination of numerous pharmaceutical-related disciplines. Each individual trial involves contractual, regulatory, and ethics approval at each site and in each country. Clinical trials have become so complex and government requirements so stringent that researchers often approach trials too cautiously, convinced that the process is bound to be insurmountably complicated and riddled with roadblocks. A step back is needed, an objective examination of the drug development process as a whole, and recommendations made for streamlining the process at all stages. With Intelligent Drug Development, Michael Tansey systematically addresses the key elements that affect the quality, timeliness, and cost-effectiveness of the drug-development process, and identifies steps that can be adjusted and made more efficient. Tansey uses his own experiences conducting clinical trials to create a guide that provides flexible, adaptable ways of implementing the necessary processes of development. Moreover, the processes described in the book are not dependent either on a particular company structure or on any specific technology; thus, Tansey's approach can be implemented at any company, regardless of size. The book includes specific examples that illustrate some of the ways in which the principles can be applied, as well as suggestions for providing a better context in which the changes can be implemented. The protocols for drug development and clinical research have grown increasingly complex in recent years, making Intelligent Drug Development a needed examination of the pharmaceutical process.

scholarly journals Bayesian meta-analytical methods to incorporate multiple surrogate endpoints in drug development process

2015 ◽  
Vol 35 (7) ◽  
pp. 1063-1089 ◽  
Author(s):  
Sylwia Bujkiewicz ◽  
John R. Thompson ◽  
Richard D. Riley ◽  
Keith R. Abrams
Keyword(s):  
Drug Development ◽  
Analytical Methods ◽  
Development Process ◽  
Surrogate Endpoints ◽  
Drug Development Process

scholarly journals Precision Medicine: From “Omics” to Economics towards Data-Driven Healthcare - Time for European Transformation

2017 ◽  
Vol 2 (Suppl. 1) ◽  
pp. 1-10 ◽  
Author(s):  
Denis Lacombe ◽  
Lifang Liu ◽  
Françoise Meunier ◽  
Vassilis Golfinopoulos
Keyword(s):  
Drug Development ◽  
Development Process ◽  
Precision Oncology ◽  
Drug Development Process ◽  
Cancer Treatments ◽  
True Value ◽  
Efficient Access ◽  
Funding Agencies ◽  
Anti Cancer

There is room for improvement for optimally bringing the latest science to the patient while taking into account patient priorities such as quality of life. Too often, regulatory agencies, governments, and funding agencies do not stimulate the integration of research into care and vice versa. Re-engineering the drug development process is a priority, and healthcare systems are long due for transformation. On one hand, patients need efficient access to treatments, but despite precision oncology approaches, efficiently shared screening platforms for sorting patients based on the biology of their tumour for trial access are lacking and, on the other hand, the true value of cancer care is poorly addressed as central questions such as dose, scheduling, duration, and combination are not or sub-optimally addressed by registration trials. Solid evidence on those parameters could potentially lead to a rational and wiser use of anti-cancer treatments. Together, optimally targeting patient population and robust comparative effectiveness data could lead to more affordable and economically sound approaches. The drug development process and healthcare models need to be interconnected through redesigned systems taking into account the full math from drug development into affordable care.

scholarly journals Pharmacovigilance in Cameroon: Past, Present and Future Developments in Unlocking the Drug Development Process

2020 ◽  
pp. 21-44
Author(s):  
Estella Achick Tembe Fokunang ◽  
Bruna Njeba ◽  
Marie Jose Essi ◽  
Rose Ngono Abondo ◽  
Banin Andrew Nyuki ◽  
...  
Keyword(s):  
Drug Development ◽  
Development Process ◽  
Target Population ◽  
Human Consumption ◽  
Drug Development Process ◽  
Post Marketing Surveillance ◽  
Post Marketing ◽  
Development Processes ◽  
High Standards ◽  
Knowledge Attitude And Practice

The drug discovery and development processes are designed to guarantee that drugs are efficacious, nontoxic and of high standards of quality for human consumption. However, patient’s population with access to drugs at approval is only a fraction of the final target population. Therefore, a thorough understanding of the safety of medicines is generally only achieved after the marketing authorization of the drug, followed by pharmacovigilance or post marketing surveillance. Pharmacovigilance (PHV) is defined by WHO as “the science and activities that deals with the detection, assessment, understanding and prevention of the adverse drug reactions or any other possible drug-related interactions”. Health professionals, patients, drug manufacturers and drug regulatory authorities are therefore highly involved in the practice of PHV. Cameroon imports 95 % of drugs and health care products. Therefore, an effective mastery of the knowledge, attitude and practice of PHV will help to elaborate the development of our pharmacovigilance systems. This paper gives an overview of pharmacovigilance in Cameroon for unlocking the drug development process focusing on the past, present and future.

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