scholarly journals Exploring new uses for existing drugs: innovative mechanisms to fund independent clinical research

Trials ◽  
2021 ◽  
Vol 22 (1) ◽  
Author(s):  
Ciska Verbaanderd ◽  
Ilse Rooman ◽  
Isabelle Huys
Keyword(s):  
Clinical Trials ◽  
Clinical Research ◽  
Financial Support ◽  
Social Impact ◽  
Grey Literature ◽  
Economic Incentives ◽  
Phase Iii ◽  
Medical Needs ◽  
The Future ◽  
Funding Mechanisms

Abstract Background Finding new therapeutic uses for existing medicines could lead to safe, affordable and timely new treatment options for patients with high medical needs. However, due to a lack of economic incentives, pharmaceutical developers are rarely interested to invest in research with approved medicines, especially when they are out of basic patent or regulatory protection. Consequently, potential new uses for these medicines are mainly studied in independent clinical trials initiated and led by researchers from academia, research institutes, or collaborative groups. Yet, additional financial support is needed to conduct expensive phase III clinical trials to confirm the results from exploratory research. Methods In this study, scientific and grey literature was searched to identify and evaluate new mechanisms for funding clinical trials with repurposed medicines. Semi-structured interviews were conducted with 16 European stakeholders with expertise in clinical research, funding mechanisms and/or drug repurposing between November 2018 and February 2019 to consider the future perspectives of applying new funding mechanisms. Results Traditional grant funding awarded by government and philanthropic organisations or companies is well known and widely implemented in all research fields. In contrast, only little research has focused on the application potential of newer mechanisms to fund independent clinical research, such as social impact bonds, crowdfunding or public-private partnerships. Interviewees stated that there is a substantial need for additional financial support in health research, especially in areas where there is limited commercial interest. However, the implementation of new funding mechanisms is facing several practical and financial challenges, such as a lack of expertise and guidelines, high transaction costs and difficulties to measure health outcomes. Furthermore, interviewees highlighted the need for increased collaboration and centralisation at a European and international level to make clinical research more efficient and reduce the need for additional funding. Conclusions New funding mechanisms to support clinical research may become more important in the future but the unresolved issues identified in the current study warrant further exploration.

New Mechanisms to Fund Independent Clinical Trials for Repurposing Off-Patent or Generic Medicines

2020 ◽  
Author(s):  
Ciska Verbaanderd ◽  
Ilse Rooman ◽  
Isabelle Huys
Keyword(s):  
Clinical Trials ◽  
Clinical Research ◽  
Financial Support ◽  
Grey Literature ◽  
Economic Incentives ◽  
Phase Iii ◽  
Generic Medicines ◽  
Medical Needs ◽  
The Future ◽  
Funding Mechanisms

Abstract Finding new therapeutic uses for approved, off-patent or generic medicines could lead to safe, affordable and timely new treatment options for patients with high medical needs. However, due to a lack of economic incentives, pharmaceutical developers are rarely interested to invest in this type of research. Consequently, potential new uses for off-patent or generic medicines are mainly studied in independent proof-of-concept clinical trials initiated and led by researchers from academia, research institutes or collaborative groups. Yet, these researchers need additional financial support to conduct expensive phase III clinical trials to confirm the results from exploratory research. In this study, scientific and grey literature was searched to identify and evaluate new mechanisms for funding clinical trials with repurposed medicines. Semi-structured interviews were conducted with 16 European stakeholders with expertise in clinical research, funding mechanisms and/or drug repurposing between November 2018 and February 2019 to consider the future perspectives of applying new funding mechanisms. Traditional grant funding awarded by government and philanthropic organisations or companies is well known and widely implemented in all research fields. In contrast, the application potential of newer mechanisms to fund independent clinical research, such as social impact bonds or crowdfunding, is not yet known. Interviewees stated that there is a substantial need for additional financial support in health research, especially in disease domains where there is limited commercial interest. However, the implementation of new funding mechanisms is facing several practical and financial challenges, such as a lack of expertise and guidelines, high transaction costs and difficulties to measure health outcomes. Furthermore, increased collaboration and centralisation at a European and international level is recommended to make clinical research more efficient and reduce the need for additional funding. New funding mechanisms to support clinical research may become more important in the future, but the unresolved issues warrant further exploration in pilot projects.

scholarly journals Anticancer bispecific antibody R&D advances: a study focusing on research trend worldwide and in China

2021 ◽  
Vol 14 (1) ◽  
Author(s):  
Zhonghan Zhang ◽  
Fan Luo ◽  
Jiaxin Cao ◽  
Feiteng Lu ◽  
Yang Zhang ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Clinical Research ◽  
Phase I ◽  
Bispecific Antibody ◽  
Gastroesophageal Junction ◽  
Nasopharyngeal Cancer ◽  
Phase Iii ◽  
Research Trend ◽  
Distribution Analysis ◽  
The World

Abstract Background The bispecific antibody (bsAbs) research around the world has undergone great changes. We analyzed the global trend of bsAbs research and compared the differences in clinical research of bsAbs between China and worldwide. Methods BsAbs research clinical trials information was retrieved through the online open-resource clinical trial registration platform. Research information including organizations, identity numbers, locations, phases, participating centers, conditions, status, enrollment, targets, spectrums of mechanism of action (MOA), and start date was collected. Clinical trials were divided into two categories based on the attributes of pharmaceutical companies (international or China-initiated or involved). Results From 1997 to 2020, 272 clinical trials regarding bsAbs research were retrieved. Twenty-nine percent of the studies were contributed by companies from Chinese institutions, which followed the USA and ranked second. The clinical trials of bsAbs are mainly concentrated on phase I (n = 161), phase I/II (n = 54), and phase II (n = 51), and the number of phase III trials is still rare (n = 4). Tumor species distribution analysis shows that there are significantly higher focuses on gastric cancer (n = 18), esophageal/gastroesophageal junction cancer (n = 16), bladder cancer (n = 10), biliary malignant tumor (n = 8), nasopharyngeal cancer (n = 6), and thymic cancer (n = 2) in China. BsAbs target and spectrums of MOA analysis showed that international companies mainly focus on bsAbs with CD3-based (n = 63) target with MOA of T-cell redirection, while researches in China pay more attention to PD-1 (n = 9)/PD-L1 (n = 7) axises with MOA of double immune checkpoint blocking. Conclusion Global bsAbs research increased rapidly during the 1997 to 2020 period. The developed countries in America and Europe are leading the trend of bsAbs research. Anticancer bsAbs clinical research in China is booming and chasing after the world trend.

Children’s Oncology Group (COG) clinical trials as exemplars of pragmatism in clinical research.

2017 ◽  
Vol 35 (15_suppl) ◽  
pp. e18188-e18188
Author(s):  
Nupur Mittal ◽  
Anil George ◽  
Lizette Leanos ◽  
Paul Kent
Keyword(s):  
Clinical Trials ◽  
Clinical Research ◽  
Pragmatic Trial ◽  
Standard Of Care ◽  
The United States ◽  
Phase Iii ◽  
President Obama ◽  
Explanatory Trial ◽  
Phase Iii Clinical Trials ◽  
Pragmatic Clinical Trials

e18188 Background: In 2016, President Obama announced the “Moonshot Cancer Initiative” to advance cancer research. One of the key components of this initiative is promoting enrollment in pragmatic clinical trials and data sharing. As described in 2009 by Thorpe et al, using the 9-domian PRECIS-2 tool, a ‘pragmatic’ trial, as opposed to an ‘explanatory’ trial used to confirm a physiologic hypothesis, is one in which the intervention can be immediately applied into real-world clinical practice (J of Clin Epi:2009). In the US, 60%-90% children are enrolled on COG research trials. State-of-the-art treatment for a child is derived from superior therapy identified in prior trials which then serves as the standard arm of subsequent phase-III trials and becomes the de facto practice guideline and ‘standard of care’ .To accomplish this goal, COG shares its data among the 242 member institutions.Our objective is to assess “pragmatism” in COG trials. Methods: We analyzed the 158 COG phase III clinical trials between 2006-2016 for all 9 domains of the PRECIS-2 (Table 1) and graded them from 1-5 (most explanatory to most pragmatic). Results: Out of 152 phase III clinical trials on the COG website, 138 (91%) scored 5/5 for all 9 PRECIS pragmatic domains and 148 (98%) scored >4. The scoring for all 9 domains is in table 1. Conclusions: COG provides nearly all children in the United States access to pragmatic phase III research as outlined in the Moonshot Goals. Goal of merging evidence-based standard care accessible to all and conducting well- designed clinical research to identify the best therapies has been accomplished by COG and should serve as an example to other cancer organizations. 9 domains of PRECIS-2 to assess pragmatism in clinical trials. [Table: see text]

scholarly journals Discovery reliability

2019 ◽  
Vol 39 (6) ◽  
pp. 1185-1187
Author(s):  
Anders Hånell
Keyword(s):  
Clinical Trials ◽  
Clinical Research ◽  
Statistical Power ◽  
Neurological Diseases ◽  
Phase Iii ◽  
Treatment Benefit ◽  
Phase Iii Clinical Trials ◽  
Drug Studies ◽  
Clinical Drug

Whenever an experiment yields a statistically significant outcome you should ask yourself: To what extent can I trust this result? This is especially important for pre-clinical drug studies because of the frequent failures of phase III clinical trials of neurological diseases, which has put the reliability of pre-clinical research into question. Two important factors, the pre-study likelihood of treatment benefit, and statistical power, affects the reliability of the result in a quantifiable way. This can be used to assess to what extent the result of a study can be trusted (discovery reliability), and to guide the design of pre-clinical research.

Public–private joint ventures in the healthcare sector: enlarging the shadow of the future through social and economic incentives

2020 ◽  
Vol 33 (6/7) ◽  
pp. 647-662
Author(s):  
Espen Solheim-Kile ◽  
Andreas Wald
Keyword(s):  
Public Sector ◽  
Joint Ventures ◽  
Social Impact ◽  
Economic Incentives ◽  
Healthcare Sector ◽  
Goal Alignment ◽  
Content Type ◽  
Local Improvement ◽  
The Future

PurposePublic–private joint ventures (PPJVs) have a stronger partnership element than standard public–private partnerships (PPPs) but PPJVs are under-researched despite this important partnership element. This article derives knowledge of incentives and barriers to goal alignment in healthcare PPJVs.Design/methodology/approachAn in-depth case study of the UK’s Local Improvement Finance Trust (LIFT) model including three PPJVs and 34 individual projects was conducted.FindingsThe main economic incentives are future opportunities creating a strong shadow of the future. This is supplemented by social incentives such as the ability to have a social impact. Enlarging the shadow of the future can encourage both parties to think long-term, avoiding short-term opportunism.Practical implicationsPPJV is a promising model for partnership. However, complexity through fragmented public sector partners and the financial structure can create barriers for goal alignment.Originality/valueThis study challenges earlier research studies based on PPJV by providing evidence that the long-term nature of PPJV, especially the potential of new projects, enables the public sector to get more engagement from the private sector.

scholarly journals The future of phage clinical trials in Australia

2019 ◽  
Vol 40 (1) ◽  
pp. 16
Author(s):  
Keith Potent
Keyword(s):  
Clinical Trials ◽  
Clinical Research ◽  
Phage Therapy ◽  
Tax Incentives ◽  
Innovative Development ◽  
Recent Success ◽  
Mainstream Medicine ◽  
The Future ◽  
Research Organisations

Australia is well positioned to conduct clinical trials in phage-based technology. Despite challenges with translating phage therapy to mainstream medicine, our regulations are designed for safe and innovative development. Recent success indicates that Australia is ideal for conducting further phage clinical trials. There are also expert clinical research organisations and generous tax incentives.

scholarly journals Number of clinical trials of new therapeutic agents in the European Union countries of Central and Eastern Europe, 2000–2019

2021 ◽  
Author(s):  
Mikołaj Bartoszkiewicz ◽  
Joanna Kufel-Grabowska ◽  
Maria Litwiniuk
Keyword(s):  
European Union ◽  
Clinical Trials ◽  
Clinical Research ◽  
Eastern Europe ◽  
Central And Eastern Europe ◽  
Phase Iii ◽  
The European Union ◽  
Eu Countries ◽  
Phase Iii Trials ◽  
The Eu

Introduction: The clinical research market of the European Union (EU) countries of Central and Eastern Europe has been experiencing a dynamic growth of clinical trials in the last 10 years. Oncology and cardiology are the areas where the most clinical trials are conducted. Aim: This study aims to analyze the clinical research market including countries, medical fields and trial phases in the EU countries of Central and Eastern Europe. The comparative analysis of countries is divided into 5-year periods. Material and methods: Clinical research market analysis was carried out in 11 EU countries of Central and Eastern Europe: Bulgaria, Croatia, Czechia, Estonia, Hungary, Latvia, Lithuania, Poland, Romania, Slovakia, and Slovenia. In searching for the number of clinical trials, the ClinicalTrials.gov database was used. Results and discussion: From 2000 to 2019, 6497 clinical trials were conducted in the EU countries of Central and Eastern Europe. There were 1840 clinical trials registered in Poland, 1188 in Czechia, and 1005 in Hungary. The most clinical trials were registered in the field of oncology (22%), followed by cardiology (16%) and neurology (12%). Phase III trials representing as much as 60% (n = 2854) of all conducted medical experiments. The highest increase in the number of clinical trials in the last two 5-year periods (2010–2014 and 2015–2019) was recorded in Estonia, at 471%. Conclusions: There has been a significant increase in the number of clinical phase III trials in the EU countries of Central and Eastern Europe, mainly in Poland, Czechia, and Hungary.

Testing Hope

2020 ◽  
pp. 123-153
Author(s):  
Jeremy Snyder
Keyword(s):  
Clinical Trials ◽  
Clinical Research ◽  
Phase Iii ◽  
Trial Participation ◽  
Phase I Clinical Trials ◽  
Middle Income ◽  
Middle Income Countries ◽  
Experimental Intervention ◽  
Phase Iii Clinical Trials ◽  
Experimental Cancer

Chapter 5 examines human participation in phase I clinical trials for experimental cancer interventions and phase III clinical trials in low- and middle-income countries. In both cases, these participants may take part in these trials in the hope of accessing an experimental intervention that will improve their own health or that of their communities. Exploitation of hope is most likely to take place in these contexts when there is a confusion about the roles of researchers and the aims of clinical research such that participants misunderstand the likely outcomes of the trial for themselves or their communities. Researchers and their sponsors can contribute to this misunderstanding by not communicating the aims of clinical research clearly or by using the language of hope to encourage trial participation.

Perspectives in breast and ovarian cancer chemotherapy by nanomedicine approach: nanoformulations in clinical research

2020 ◽  
Vol 27 ◽  
Author(s):  
Cristina Martín-Sabroso ◽  
Ana Isabel Fraguas-Sánchez ◽  
Rafaela Raposo-González ◽  
Ana Isabel TorresSuárez
Keyword(s):  
Ovarian Cancer ◽  
Clinical Trials ◽  
Clinical Research ◽  
Maximum Tolerated Dose ◽  
Production Costs ◽  
Phase Iii ◽  
Ovarian Carcinomas ◽  
Good Tool

: Breast and ovarian carcinomas represent major health problems in women worldwide. Chemotherapy constitutes a main treatment strategy, and the use of nanocarriers a good tool to improve it. Several nanoformulations have already been approved, and others are under clinical trials for the treatment of both types of cancers. Objective: This review focuses on the analysis of the nanoformulations that are under clinical research in the treatment of these neoplasms. Results: Currently, there are 6 nanoformulations in clinical trials for breast and ovarian carcinomas, most of them in phase II and phase III. In the case of breast cancer treatment, these nanomedicines contain paclitaxel; and, for ovarian cancer, nanoformulations containing paclitaxel or camptothecin analogs are being evaluated. The nanoencapsulation of these antineoplastics facilitates their administration and reduces their systemic toxicity. Nevertheless, the final approval and commercialization of nanoformulations may be limited by other aspects like lack of correlation between the efficacy results evaluated at in vitro and in vivo levels, difficulty in producing large batches of nanoformulations in a reproducible manner and high production costs compared to conventional formulations of antineoplastics. However, these challenges are not insurmountable and the number of approved nanoformulations for cancer therapy is growing. Conclusion: Reviewed nanoformulations have shown, in general, excellent results, demonstrating a good safety profile, a higher maximum tolerated dose and a similar or even slightly better antitumor efficacy compared to the administration of free drugs, reinforcing the use of nano-chemotherapy in both breast and ovarian tumors.

Free-riding and the prisoner's dilemma: problems in funding economic analyses of phase III cancer clinical trials.

1995 ◽  
Vol 13 (9) ◽  
pp. 2457-2463 ◽  
Author(s):  
C L Bennett ◽  
T J Smith ◽  
S L George ◽  
B E Hillner ◽  
S Fleishman ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Cost Effectiveness ◽  
Financial Support ◽  
Prisoner's Dilemma ◽  
Prisoner’S Dilemma ◽  
Phase Iii ◽  
New Agents ◽  
Economic Analyses ◽  
National Cooperative

PURPOSE Both economic and clinical data on new agents are important to policy-makers who approve pharmaceuticals for widespread use. Randomized clinical trials have been used to evaluate both clinical results and total medical costs associated with new agents. With new expensive pharmaceutical agents, early assessments of economic benefit have taken on greater importance to physicians and patients. Who should provide financial support to these integrated economic and clinical analyses in clinical trials? Here we describe issues that hinder funding of economic analyses and propose potential support mechanisms. RESULTS The Cancer and Leukemia Group B (CALGB), a large, national cooperative group of academic and community hospitals in the United States, designed a non-small-cell lung cancer (NSCLC) treatment trial to compare two widely used supportive care regimens that varied 20-fold in cost. One important objective of this trial was to compare the cost-effectiveness of the two regimens. While funding for the clinical trial was supported by grants from the National Cancer Institute and the pharmaceutical companies involved in the trial, no specific funding agency was willing and/or able to provide financial support for the economic analyses. After 2 years of planning, the clinical trial was retracted when the funding for the economic analyses could not be secured. The prisoner's dilemma, individual reluctance to support a common social good, explains the lack of funding. CONCLUSION Economic theory predicts difficulties in evaluating cost-effectiveness of new pharmaceuticals and reluctance to support economic analyses of clinical trials. Economic analyses will require new sources of funds that will not take scarce resources from clinical trials groups. Options for funding include a new federal agency, coordinated work by existing agencies, or academic centers for economic analysis.

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