A relationship-based approach to improving the clinical trial experience.

2019 ◽  
Vol 37 (15_suppl) ◽  
pp. e14055-e14055
Author(s):  
Sarah MacKay Helou ◽  
Camber W. Gadberry ◽  
Nicholas Zahara ◽  
Claire Nickols ◽  
Tanya Vidhyarkorn ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Drug Development ◽  
Cancer Immunotherapy ◽  
Treatment Options ◽  
White Paper ◽  
Efficient Manner ◽  
Trial Experience ◽  
New Treatment ◽  
Early Phase Clinical Trials ◽  
New Treatments

e14055 Background: The rapidly evolving field of Cancer Immunotherapy (CIT) has mobilized biopharma to develop promising new treatments while at the same time created a need to expedite the pathway of those new therapies from bench to bedside. This race to bring innovative treatments to patients has engendered heightened levels of competition for the attention of and partnership with the best research institutions in the biopharma industry. Methods: Recognizing this reality, Roche created the immunotherapy Centers of Research Excellence (imCORE) Network: a network of leading cancer immunotherapy centers worldwide. The vision of this network was to partner with sites to streamline the operational delivery of the Roche Genentech portfolio while deepening the collective understanding of the science to prioritize and accelerate the most promising new treatment options for our patients. Creation of a network was not a novel idea, but Roche set out to build a network unique to the biopharma industry – one which was relationship-based where the site/sponsor relationship serves as the foundation for driving both collaborative scientific investigations and operational efficiencies in support of Roche’s clinical trials. While the identification and optimization of promising candidate drugs in the preclinical and translational settings are critical first steps in the drug development process, the initiation and operationalization of early phase clinical trials are equally important in the multidimensional cycle of drug development. Results: See Conclusions. Conclusions: This white paper will provide an overview of imCORE’s origins with a focus on the strategic importance of the site-sponsor relationship and the mutual value that is derived from the combined benefits of these relationships and in conducting early clinical trials more efficiently across Roche’s CIT portfolio. Further examination of communication and operational improvement to support the network will be highlighted as key drivers in creating and sustaining meaningful site/sponsor relationships that better facilitate the operationalization of clinical trials and ultimately brings novel therapies to patients in a more efficient manner than in the past.

scholarly journals Current challenges in the development of new treatments for lupus

2019 ◽  
Vol 78 (6) ◽  
pp. 729-735 ◽  
Author(s):  
Maria Dall'Era ◽  
Ian N Bruce ◽  
Caroline Gordon ◽  
Susan Manzi ◽  
Janis McCaffrey ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Drug Development ◽  
Lupus Erythematosus ◽  
Turn Of The Millennium ◽  
Eligibility Criteria ◽  
Considerable Impact ◽  
New Treatment ◽  
New Treatments ◽  
Chronic Autoimmune Disease

Systemic lupus erythematosus (SLE) is a chronic autoimmune disease with a considerable impact on patients’ quality of life. Despite the plethora of clinical trials for SLE since the turn of the millennium, only one new treatment has been approved for the condition, and the overall pace of successful drug development remains slow. Nevertheless, the myriad of clinical studies has yielded insights that have informed and refined our understanding of eligibility criteria, outcome measures and trial design in SLE. In this review, we highlight the achievements of clinical trials as well as the major pitfalls that have been identified in drug development for SLE and, in doing so, identify areas where collaboration and consensus will be important to facilitate progress.

scholarly journals Co-Clinical Trials: An Innovative Drug Development Platform for Cholangiocarcinoma

2021 ◽  
Vol 14 (1) ◽  
pp. 51
Author(s):  
Brinda Balasubramanian ◽  
Simran Venkatraman ◽  
Kyaw Zwar Myint ◽  
Tavan Janvilisri ◽  
Kanokpan Wongprasert ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Targeted Therapy ◽  
Drug Development ◽  
Surgical Resection ◽  
Treatment Options ◽  
Standard Of Care ◽  
Time Data ◽  
Current Standard ◽  
Innovative Drug ◽  
Curative Intent

Cholangiocarcinoma (CCA), a group of malignancies that originate from the biliary tract, is associated with a high mortality rate and a concerning increase in worldwide incidence. In Thailand, where the incidence of CCA is the highest, the socioeconomic burden is severe. Yet, treatment options are limited, with surgical resection being the only form of treatment with curative intent. The current standard-of-care remains adjuvant and palliative chemotherapy which is ineffective in most patients. The overall survival rate is dismal, even after surgical resection and the tumor heterogeneity further complicates treatment. Together, this makes CCA a significant burden in Southeast Asia. For effective management of CCA, treatment must be tailored to each patient, individually, for which an assortment of targeted therapies must be available. Despite the increasing numbers of clinical studies in CCA, targeted therapy drugs rarely get approved for clinical use. In this review, we discuss the shortcomings of the conventional clinical trial process and propose the implementation of a novel concept, co-clinical trials to expedite drug development for CCA patients. In co-clinical trials, the preclinical studies and clinical trials are conducted simultaneously, thus enabling real-time data integration to accurately stratify and customize treatment for patients, individually. Hence, co-clinical trials are expected to improve the outcomes of clinical trials and consequently, encourage the approval of targeted therapy drugs. The increased availability of targeted therapy drugs for treatment is expected to facilitate the application of precision medicine in CCA.

Dystrophinopathies and Limb-Girdle Muscular Dystrophies

2017 ◽  
Vol 48 (04) ◽  
pp. 262-272 ◽  
Author(s):  
Anna Sarkozy ◽  
Mariacristina Scoto ◽  
Francesco Muntoni ◽  
Joana Domingos
Keyword(s):  
Clinical Trials ◽  
Treatment Options ◽  
Shoulder Girdle ◽  
Muscle Disease ◽  
Standards Of Care ◽  
Muscular Dystrophies ◽  
New Treatment ◽  
Shoulder Girdle Muscles ◽  
Independent Ambulation ◽  
Duchenne's Muscular Dystrophy

AbstractMuscular dystrophies are a heterogeneous group of inherited diseases. The natural history of these disorders along with their management have changed mainly due to a better understanding of their pathophysiology, the evolution of standards of care, and new treatment options. Dystrophinopathies include both Duchenne's and Becker's muscular dystrophies, but in reality they are a spectrum of muscle diseases caused by mutations in the gene that encodes the protein dystrophin. Duchenne's muscular dystrophy is the most common form of inherited muscle disease of childhood. The current standards of care considerably prolong independent ambulation and survival. Several therapeutic options either aiming at substituting/correcting the primary protein defect or limiting the progression of the dystrophic process are currently being explored in clinical trials.Limb-girdle muscular dystrophies (LGMDs) are rare and heterogeneous conditions, characterized by weakness and wasting of the pelvic and shoulder girdle muscles. Originally classified into dominant and recessive, > 30 genetic forms of LGMDs are currently recognized. Further understanding of the pathogenic mechanisms of LGMD will help identifying novel therapeutic approaches that can be tested in clinical trials.

scholarly journals Clinical Trial Information As a Measure of Quality Cancer Care

2010 ◽  
Vol 6 (3) ◽  
pp. 170-171 ◽  
Author(s):  
Wei Chua ◽  
Stephen J. Clarke
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Cancer Care ◽  
Treatment Options ◽  
Small Cell ◽  
Small Cell Lung ◽  
Quality Cancer Care ◽  
Improved Outcomes ◽  
New Treatment ◽  
Clinical Trial Information

Participation in clinical trials enables patients to access new treatment options. Evidence shows improved outcomes in participants compared with nonparticipants in non–small-cell, lung, breast, colorectal, and testicular cancers.

scholarly journals Different Mechanism Involvement in the Formation of Colorectal Cancer; Mega Review

2021 ◽  
Vol 8 (9) ◽  
pp. 558-573
Author(s):  
Muhammad Ali ◽  
Yang Wang ◽  
Qi Zhang
Keyword(s):  
Colorectal Cancer ◽  
Dietary Habits ◽  
Treatment Options ◽  
Developed Countries ◽  
Term Survival ◽  
Keywords Colorectal Cancer ◽  
Cancer Tumor ◽  
Low Incidence ◽  
New Treatment ◽  
New Treatments

Colorectal cancer was infrequently diagnosed several decades ago. Nowadays, it is the world's fourth most deadly cancer with almost 900,000 deaths annually. Colorectal cancer had a low incidence several decades ago. However, it has become predominant cancer and now accounts for approximately 10% of cancer-related mortality in western countries. The ‘rise’ of colorectal cancer in developed countries can be attributed to the increasingly aging population, unfavorable modern dietary habits and an increase in risk factors such as smoking, low physical exercise and obesity. New treatments for primary and metastatic colorectal cancer have emerged, providing additional options for patients; these treatments include laparoscopic surgery for primary disease, more aggressive resection of metastatic disease (such as liver and pulmonary metastases), radiotherapy for rectal cancer and neoadjuvant and palliative chemotherapies. However, these new treatment options have had a limited impact on cure rates and long-term survival. Keywords: Colorectal cancer, Tumor marker, Hereditary colorectal cancer, Mediterranean diet.

scholarly journals The State of Lupus Clinical Trials: Minority Participation Needed

2019 ◽  
Vol 8 (8) ◽  
pp. 1245
Author(s):  
Saira Z. Sheikh ◽  
Nicole I. Wanty ◽  
Joncel Stephens ◽  
Kristen D. Holtz ◽  
Sheryl McCalla
Keyword(s):  
Clinical Trials ◽  
African Americans ◽  
Symptom Severity ◽  
Quality Care ◽  
Treatment Options ◽  
The United States ◽  
Minority Populations ◽  
Healthcare Staff ◽  
Minority Participation ◽  
New Treatment

In the United States, the reported prevalence of lupus is 100,000 to 500,000 patients. Lupus disproportionately affects minority populations, including African Americans and Latinos, and the associated health disparities are substantial. Women are at a higher risk of lupus than men and lupus prevalence is the highest in African Americans and Latinos compared to non-Hispanic whites. African Americans and Latinos also have increased disease symptom severity, experience more lupus-related complications, and have a two- to three-fold mortality rate compared to non-Hispanic Whites. Lupus clinical trials offer opportunities for quality care and can result in new treatment options, but African Americans and Latinos are underrepresented in clinical trials because of substantial patient- and provider-side barriers. In conjunction with the limited knowledge of clinical trials that potential participants may have, the healthcare staff approaching participants have limited time to adequately educate and explain the aspects of clinical trials. Indeed, ninety percent of clinical trials fail to meet their recruitment goals on time, so a multi-faceted approach is necessary to address the issue of low minority participation in clinical trials.

scholarly journals Future of risk based monitoring in clinical trials

2020 ◽  
Vol 7 (3) ◽  
pp. 221
Author(s):  
Niraj R. Vyas
Keyword(s):  
Clinical Trials ◽  
Drug Development ◽  
Treatment Options ◽  
Machine Learning Algorithms ◽  
Mitigation Strategies ◽  
Eligibility Criteria ◽  
New Ways Of Working ◽  
Trial Conduct ◽  
The Future ◽  
The Impact

<p class="abstract">Drug development is a complex and resource intensive endeavor. The average cost of developing a new drug, has been estimated to be $2 to $3 billion. However, the success rate of clinical trials is very low around and is estimated to be between 3-5%. The common reasons for failure of clinical trials include failure to demonstrate efficacy or safety, budgeting and financing, failure of subjects meeting protocol eligibility criteria, poor investigator site selection, patient withdrawals and dropouts. Considering the growing demands to get better and affordable treatment options, there needs to be fundamental shift required in drug development and specifically the clinical trials oversight processes to mitigate risks and reduce failures. The International Council for Harmonisation in the E6 R2 addendumhas now provided guidelines for adaptation of risk based approach to trial conduct and monitoring to implement mitigation strategies for potential risks which might derail the conduct of the trail. The industry is steadily gearing up to put together the required processes, systems and teams to align to the new ways of working. However with the changing landscape of drug development which includes novel therapies like gene therapy, remote/decentralized trials, growing use of wearable technologies, esource, electronic health record/electronic medical records interoperability, implementation of artificial intelligence and machine learning algorithms, the future of risk based approach towards managing clinical trials is going to be very different from what we see now. This paper explores the impact of these new developments on the future of risk based monitoring in clinical trials.</p>

scholarly journals Seeing the bigger picture: Qualitative research in the Zoom® age

2021 ◽  
Vol 8 (1) ◽  
pp. 141-144
Author(s):  
Simon Fletcher
Keyword(s):  
Clinical Trials ◽  
Focus Groups ◽  
Life Stories ◽  
Qualitative Interviews ◽  
Real Life ◽  
Know How ◽  
Life Impact ◽  
New Treatment ◽  
New Treatments

Abstract Participants in clinical trials for new haemophilia treatments are routinely asked to complete quality of life (QoL) questionnaires using validated and disease-specific instruments. Yet too often in clinical research we know very little about the life stories of individuals, making it difficult to know how they have been affected by a new therapy and what exactly has changed for the better – or for the worse. In my own research, I wanted to understand the differences that new treatments are really making to people's everyday lives. While traditional QoL instruments can be helpful, using a qualitative approach that involves speaking directly with people with haemophilia (PwH) and their family members has enabled me find out what has really been going on their lives, including impacts on the wider family. The Covid pandemic and the need to maintain social distancing changed the way in which my research has been carried out, but in fact provided an opportunity to see an even bigger picture. I believe that using videoconferencing platforms to conduct interviews and focus groups has both allowed me to see more of the world in which the participants live and has enabled participants to be more relaxed and open in their conversations, resulting in a potentially richer dataset. While this approach to qualitative QoL research should not replace interviews and focus groups, the use of videoconferencing should be considered as another methodology researchers can and should use to enable them to glean the richest data possible. Qualitative interviews offer an important complementary addition to the validated QoL measures used in clinical trials, enabling us to hear more about where improvements have occurred, where further improvements can be made, and the real-life impact of a new treatment for PwH and their families.

New treatment options for panic disorder: clinical trials from 2000 to 2010

2011 ◽  
Vol 12 (9) ◽  
pp. 1419-1428 ◽  
Author(s):  
Rafael C Freire ◽  
Jaime E Hallak ◽  
José A Crippa ◽  
Antonio E Nardi
Keyword(s):  
Clinical Trials ◽  
Panic Disorder ◽  
Treatment Options ◽  
New Treatment

BioDay Registry: Prospective, observational data collection regarding the use of new systemic treatment options in patients with atopic diseases in daily practice (Preprint)

2020 ◽  
Author(s):  
Jorien BioDay Registry ◽  
Lieneke Ariens ◽  
Lotte Spekhorst ◽  
Ischa Kummeling ◽  
Judith Thijs ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Medical Ethics ◽  
Outcome Measures ◽  
Systemic Treatment ◽  
Treatment Options ◽  
Real Life ◽  
Daily Practice ◽  
Ethics Committee ◽  
Atopic Diseases ◽  
New Treatment

BACKGROUND Dupilumab is the first antibody based treatment for atopic dermatitis (AD). It is expected that many new treatment options become available for the treatment of atopic diseases in the near future. Despite the high quality of clinical trials, results are not always generalizable to daily practice. OBJECTIVE Registry based collection of real life data can add information that is not gained in clinical trials. METHODS The BioDay Registry prospectively collects real life treatment results regarding effectiveness and safety of patients treated with new systemic treatment options for AD in a multicenter setting. Effectiveness is assessed based on both physician measured clinical eczema scores as well as patient-reported outcome measures. Long-term safety risks are monitored. The possibility for dose reduction in patients with disease control can be investigated. The treatment effect on other atopic co-morbidities will be monitored. The BioDay Registry can be easily adjusted, and new modules on new treatment options can be added. As outcome measures are in line with the core outcomes for eczema recommended by the global Harmonising Outcome Measures for Eczema (HOME) initiative, it is possible to merge data with other registries in future. RESULTS The BioDay Registry was considered as non-interventional by the local Medical Ethics Committee and the collection of data is performed according to the Helsinki Declaration. Protocol amendments will always be submitted for review to the Medical Ethics Committee. CONCLUSIONS The BioDay Registry will provide unique prospectively collected data on the use of targeted therapies in daily practice for AD patients. The results of this registry will contribute to the development of more personalized treatment strategies for patients with moderate to severe AD. CLINICALTRIAL ClinicalTrials.gov identifier: NCT03549416, retrospectively registered June 8, 2018.

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