R&D Spending & Success: Key Trends, Issues & Solutions

2017 ◽  
Vol 23 (1) ◽  
Author(s):  
Sanjay Kumar Rao
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Trial Design ◽  
Product Launch ◽  
Financial Resources ◽  
Success Rates ◽  
Trial Testing ◽  
Recent Trends ◽  
Pharmaceutical Prices ◽  
Design And Testing

Debates about rising bio/pharmaceutical prices are often predicated on the presumption that higher product prices are necessary for continuing funding of innovation. Recent trends only partially bear this out. While total spending on bio/pharmaceutical R&D has largely remained constant, costs for clinical trials have consumed a rising proportion of R&D budgets. More spending on clinical trials, however, has not yielded higher trial success rates. Prior to product launch, clinical trials take up most of the financial resources attributed to a product in development, costing on average $75-$100M. While completing a full cycle of pre-clinical and clinical trials can take an average of 7.5 years, the probability of successful filing for marketing a drug after clinical trial testing has never exceeded 13-20%, despite advances in trial design and testing processes.This article discusses key trends in R&D spending and productivity. It then lays out issues that prevent clinical trials from achieving higher success, and presents operational and strategic solutions that can be implemented to improve the effectiveness of increases in R&D spending.

Cancer

2021 ◽  
pp. 627-638
Author(s):  
Rashi Rai ◽  
Prudhvilal Bhukya ◽  
Muneesh Kumar Barman ◽  
Meenakshi Singh ◽  
Kailash Chand ◽  
...  
Keyword(s):  
Artificial Intelligence ◽  
Clinical Trial ◽  
Clinical Trials ◽  
Trial Design ◽  
Clinical Trial Design ◽  
Success Rates ◽  
Design Strategies ◽  
Safety Level ◽  
Key Steps ◽  
The Impact

Clinical trials are essential to govern the impact of a new possible treatment. It is utilized to determine the safety level and efficacy of a certain treatment. Clinical trial studies in cancer have provided successful treatment leading to longer survival span in the patients. The design of clinical trials for cancer has been done to find new ways to prevent, diagnose, treat, and manage symptoms of the disease. This chapter will provide detailed information on different aspects of clinical trials in cancer research. Protocols outlining the design and method to conduct a clinical trial in each phase will be discussed. The process and the conditions applied in each phase (I, II, and III) will be described precisely. The design of trials done in every aspect such as prevention, immunochemotherapy, diagnosis, and treatment to combat cancer will be illustrated. Also, recent innovations in clinical design strategies and principles behind it as well as the use of recent advances in artificial intelligence in reshaping key steps of clinical trial design to increase trial success rates.

scholarly journals Conceptual Framework to Support Clinical Trial Optimization and End-to-End Enrollment Workflow

2019 ◽  
pp. 1-10 ◽  
Author(s):  
Neha M. Jain ◽  
Alison Culley ◽  
Teresa Knoop ◽  
Christine Micheel ◽  
Travis Osterman ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Knowledge Representation ◽  
Conceptual Framework ◽  
Trial Design ◽  
Clinical Trial Design ◽  
Prospective Clinical Trial ◽  
Future Trends ◽  
Current State ◽  
Evaluation Strategies

In this work, we present a conceptual framework to support clinical trial optimization and enrollment workflows and review the current state, limitations, and future trends in this space. This framework includes knowledge representation of clinical trials, clinical trial optimization, clinical trial design, enrollment workflows for prospective clinical trial matching, waitlist management, and, finally, evaluation strategies for assessing improvement.

scholarly journals Edaravone in Amyotrophic Lateral Sclerosis’Lessons from the Clinical Development Program and the Importance of a Strategic Clinical Trial Design

US Neurology ◽  
2018 ◽  
Vol 14 (1) ◽  
pp. 47 ◽  
Author(s):  
Said R Beydoun ◽  
Jeffrey Rosenfeld
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Amyotrophic Lateral Sclerosis ◽  
Trial Design ◽  
Development Program ◽  
Clinical Trial Design ◽  
Clinical Development ◽  
Disease Heterogeneity ◽  
Clinical Development Program ◽  
Lateral Sclerosis

Edaravone significantly slows progression of amyotrophic lateral sclerosis (ALS), and is the first therapy to receive approval by the Food and Drug Administration (FDA) for the disease in 22 years. Approval of edaravone has marked a new chapter in pharmaceutical development since the key trial included a novel strategic clinical design involving cohort enrichment. In addition, approval was based on clinical trials that had a relatively small patient number and were performed outside of the US. Edaravone was developed through a series of clinical trials in Japan where it was determined that a well-defined subgroup of patients was required to reveal a treatment effect within the study period. Amyotrophic lateral sclerosis is associated with wide-ranging disease heterogeneity (both within the spectrum of ALS phenotypes as well as in the rate of progression). The patient cohort enrichment strategy aimed to address this heterogeneity and should now be considered as a viable, and perhaps preferred, trial design for future studies. Future research incorporating relevant biomarkers may help to better elucidate edaravone’s mechanism of action, pharmacodynamics, and subsequently ALS phenotypes that may preferentially benefit from treatment. In this review, we discuss the edaravone clinical development program, outline the strategic clinical trial design, and highlight important lessons for future trials.

Clinical Trial Design and Statistics

2018 ◽  
Author(s):  
Julie Ann Sosa
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Data Analysis ◽  
Trial Design ◽  
New Technologies ◽  
Drug Application ◽  
Clinical Trial Design ◽  
Double Blind Study ◽  
Type I ◽  
End Points

A clinical trial is a planned experiment designed to prospectively measure the efficacy or effectiveness of an intervention by comparing outcomes in a group of subjects treated with the test intervention with those observed in one or more comparable group(s) of subjects receiving another intervention.  Historically, the gold standard for a clinical trial has been a prospective, randomized, double-blind study, but it is sometimes impractical or unethical to conduct such in clinical medicine and surgery. Conventional outcomes have traditionally been clinical end points; with the rise of new technologies, however, they are increasingly being supplemented and/or replaced by surrogate end points, such as serum biomarkers. Because patients are involved, safety considerations and ethical principles must be incorporated into all phases of clinical trial design, conduct, data analysis, and presentation. This review covers the history of clinical trials, clinical trial phases, ethical issues, implementing the study, basic biostatistics for data analysis, and other resources. Figures show drug development and clinical trial process, and type I and II error. Tables list Food and Drug Administration new drug application types, and types of missing data in clinical trials. This review contains 2 highly rendered figures, 2 tables, and 38 references

A novel, hybrid, single- and multi-site clinical trial design for CLN3 disease, an ultra-rare lysosomal storage disorder

2019 ◽  
Vol 16 (5) ◽  
pp. 555-560 ◽  
Author(s):  
Heather R Adams ◽  
Sara Defendorf ◽  
Amy Vierhile ◽  
Jonathan W Mink ◽  
Frederick J Marshall ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Rare Diseases ◽  
Trial Design ◽  
Neurodegenerative Disorder ◽  
Clinical Trial Design ◽  
Trial Participation ◽  
Local Participation ◽  
Cln3 Disease ◽  
Study Participants

Background Travel burden often substantially limits the ability of individuals to participate in clinical trials. Wide geographic dispersion of individuals with rare diseases poses an additional key challenge in the conduct of clinical trials for rare diseases. Novel technologies and methods can improve access to research by connecting participants in their homes and local communities to a distant research site. For clinical trials, however, understanding of factors important for transition from traditional multi-center trial models to local participation models is limited. We sought to test a novel, hybrid, single- and multi-site clinical trial design in the context of a trial for Juvenile Neuronal Ceroid Lipofuscinosis (CLN3 disease), a very rare pediatric neurodegenerative disorder. Methods We created a “hub and spoke” model for implementing a 22-week crossover clinical trial of mycophenolate compared with placebo, with two 8-week study arms. A single central site, the “hub,” conducted screening, consent, drug dispensing, and tolerability and efficacy assessments. Each participant identified a clinician to serve as a collaborating “spoke” site to perform local safety monitoring. Study participants traveled to the hub at the beginning and end of each study arm, and to their individual spoke site in the intervening weeks. Results A total of 18 spoke sites were established for 19 enrolled study participants. One potential participant was unable to identify a collaborating local site and was thus unable to participate. Study start-up required a median 6.7 months (interquartile range = 4.6–9.2 months). Only 33.3% (n = 6 of 18) of spoke site investigators had prior clinical trial experience, thus close collaboration with respect to study startup, training, and oversight was an important requirement. All but one participant completed all study visits; no study visits were missed due to travel requirements. Conclusions This study represents a step toward local trial participation for patients with rare diseases. Even in the context of close oversight, local participation models may be best suited for studies of compounds with well-understood side-effect profiles, for those with straightforward modes of administration, or for studies requiring extended follow-up periods.

scholarly journals Registration of clinical trials submitted for publication in International Psychogeriatrics

2006 ◽  
Vol 18 (2) ◽  
pp. 191-193 ◽  
Author(s):  
David Ames
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Randomized Clinical Trial ◽  
Gold Standard ◽  
Trial Design ◽  
Treatment Guidelines ◽  
Double Blind ◽  
Double Blind Placebo ◽  
Or Management ◽  
Multifaceted Interventions

When we manage our patients both they and we would like to know that the interventions we prescribe have been tested and shown to be safe and effective for the uses to which they are put. The most powerful tool to determine the utility of specific interventions in the discipline of medicine is the double-blind placebo-controlled randomized clinical trial (RCT). Some of the complex problems encountered in psychogeriatrics do not lend themselves to straightforward yes or no outcomes, and some of the multifaceted interventions developed for the management of common psychogeriatric syndromes are difficult to test using standard RCT design, especially with regard to effective blinding and appropriate control conditions (Llewellyn-Jones et al. 1999; Haynes, 1999; Ames, 1999). Nevertheless, there are specific interventions for which RCT data have been very useful in refining treatment guidelines and advice (e.g. Doody et al., 2001) and, where this is the appropriate trial design, RCTs comprise the “gold standard” by which to assess the efficacy of a treatment or “management package”.

Metastatic colorectal cancer (mCRC) patterns of care: Implications for clinical trial design

2007 ◽  
Vol 25 (18_suppl) ◽  
pp. 4079-4079 ◽  
Author(s):  
C. S. Denlinger ◽  
M. A. Collins ◽  
Y. Wong ◽  
S. Litwin ◽  
N. J. Meropol
Keyword(s):  
Clinical Trial ◽  
Decision Making ◽  
Clinical Trials ◽  
Trial Design ◽  
Clinical Trial Design ◽  
New Drugs ◽  
Comprehensive Cancer Center ◽  
Cancer Center ◽  
Treatment Change ◽  
Therapy Regimen

4079 Background: New approaches have expanded options for patients (pts) with mCRC. To characterize current practice paradigms that might bear on clinical trial design, we analyzed decision-making and treatment patterns in pts treated at a Comprehensive Cancer Center since the introduction of cetuximab (CET), and bevacizumab (BV). Methods: A retrospective review of all pts diagnosed with mCRC between 3/1/04 and 8/28/06 treated at Fox Chase Cancer Center. Results: 160 pts were treated, with 157 pts receiving at least one therapy regimen by 10 attending oncologists. There were 350 changes in therapy with 246 (70%) including continuation of at least one prior drug (92 BV, 111 fluoropyrimidines, 43 other). The most common reasons for treatment change were toxicity (33%), progressive disease (PD) (29%), treatment breaks (15%), and metastasectomy (11%) ( Table ). PD was a more common cause for treatment discontinuation in later phases of treatment (18% initial regimen vs. 36% subsequent regimens, p=0.0002). 24% of pts treated with oxaliplatin (OX) discontinued due to neuropathy. Hypersensitivity caused discontinuation in 5% of pts with OX and 7% of pts with CET. Resection of metastases was undertaken in 38% of pts. 43% of these pts received neoadjuvant therapy, and 56% received adjuvant therapy. 30% of pts have died, 29% remain on active treatment, 28% are on a treatment break, 3% are on hospice, and 11% are lost to follow-up. Conclusions: PD is no longer the primary reason for change of therapy in pts with mCRC. Metastasectomy is common and OX neuropathy is often treatment-limiting. These findings have important implications for endpoint selection and design of clinical trials in mCRC. Future clinical trials in mCRC must recognize treatment complexities and capture key components of decision-making that may result in prolonged survival. Furthermore, treatment breaks represent a potential window for the evaluation of new drugs. [Table: see text] No significant financial relationships to disclose.

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