KIF1A.ORG is a parent-led organization established in 2017 to launch the world’s first translational research program dedicated to discovering treatment for people affected by KIF1A Associated Neurological Disorder (KAND). There is no cure or treatment for this neurodegenerative disorder. Yet.
Our organization supports researchers who engage in collaborative and translational work to rapidly discover treatment for this generation of KAND patients. We have made tangible progress over the last three years, but time is running out. 2020 is a transformational year for KIF1A with a clear path to clinical trials.
This Path to Treatment outlines our immediate therapeutic strategy with defined objectives and resources needed to bring treatment options to families affected by KAND.
Learn more and join our mission at www.kif1a.org or contact us at impact@kif1a.org.
While companies no more possesses deep and extensive product pipeline, this is the perfect time to innovate! Innovate not only in terms of product (although that is the core), but also
2018 Genetic Testing Assessment: These slides discuss issues associated with genetic testing interpretation. All who order genetic testing should be familiar with these recent publications.
KIF1A.ORG is a parent-led organization established in 2017 to launch the world’s first translational research program dedicated to discovering treatment for people affected by KIF1A Associated Neurological Disorder (KAND). There is no cure or treatment for this neurodegenerative disorder. Yet.
Our organization supports researchers who engage in collaborative and translational work to rapidly discover treatment for this generation of KAND patients. We have made tangible progress over the last three years, but time is running out. 2020 is a transformational year for KIF1A with a clear path to clinical trials.
This Path to Treatment outlines our immediate therapeutic strategy with defined objectives and resources needed to bring treatment options to families affected by KAND.
Learn more and join our mission at www.kif1a.org or contact us at impact@kif1a.org.
While companies no more possesses deep and extensive product pipeline, this is the perfect time to innovate! Innovate not only in terms of product (although that is the core), but also
2018 Genetic Testing Assessment: These slides discuss issues associated with genetic testing interpretation. All who order genetic testing should be familiar with these recent publications.
Advanced Diagnostics in the Post-PAMA EraJohn Hanna
Presentation given at the Q1 Diagnostics Summit in Boston MA December 6, 2016 discussing considerations for advanced diagnostics commercialization following the implementation of the Protecting Access to Medicare Act (PAMA) provisions for diagnostics pricing and reimbursement in the Medicare program.
Slides from the presentation on extrapolation from progression free survival to overall survival in oncology given at the 2017 HTAi Annual Meeting in Rome
Prospective identification of drug safety signalsIMSHealthRWES
At a time of growing demand for more accurate and timely
drug safety evidence, a landmark study supports the value of
electronic medical records (EmR) for detecting new adverse
reactions.
Medication nonadherence cost and noncompliance in clinical trialsSynegys
Drug development has reached over $2.6 B and is driven by a clinical trial's success rate, out-of-pocket study costs and study timescales. However, medication nonadherence is a hidden cost which heavily influences these cost drivers. We discuss how medication nonadherence introduces data variability, requiring trial managers to enrol more patients to maintain statistical power, which in turn extends trial timelines. Cost savings are described based on improving study noncompliance with a compliance tool such as Synegys' mComply. This mHealth tool reduces costs as a result of improved statistical power, lower enrollment and shorter trial duration.
The Value of Targeted Sequencing in Advanced Cancer: DCE to Elicit the Public...Office of Health Economics
This project seeks to elicit the public’s preferences for different features of a genomic test to sequence advanced solid cancer tumours. Understanding the relative preferences for various attributes of targeted testing are useful for determining the value of sequencing approaches, and informing technology adoption decisions. A discrete choice experiment (DCE) survey was designed to assess the preferences of members of the Australian general public for targeted sequencing in advanced cancer. The survey presented respondents with 12 questions in which they had to choose between two unlabelled tests (Test A and Test B). Tests were specified in terms of five attributes: time to receive the test result; cost of the test; likelihood that the test result will lead to a change in treatment; length of time health care professionals spend describing the test; and type of health care team who explains the test result. Respondents were sampled from an online panel and also completed questions related to demographic and socio-economic factors, experiences of cancer and familial history. We found that cost, timeliness, expertise/location and likeliness of changing treatment regimes were identified as attributes of genomic sequencing that are most valuable to a sample of the public. These results will ultimately be compared with the results of an ongoing DCE being conducted with patients with advanced cancer who are undergoing sequencing.
Author(s) and affiliation(s): Paula Lorgelly (OHE), Grace Hampson (OHE), James Buchanan (Oxford), Melissa Martyn (MGHA), Jayesh Desai (PeterMac), Clara Gaff (MGHA), and iPREDICT MGHA Flagship collaborators
Conference/meeting: EuHEA 2018
Location: Maastricht, the Netherlands
Date: 12/07/2018
Four strategies to upgrade clinical trial quality in this computerized world ...Pubrica
• Biostatistics Services is important for collecting, reviewing, presenting, and interpreting data in clinical research.
• Applications of clinical biostatistics services are in different areas, such as epidemiology, clinical trials, population genetics, the biology of structures, and more.
Reference : https://pubrica.com/services/research-services/biostatistics-and-statistical-programming-services/
Continue Reading: http://bit.ly/36nwtcs
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When you order our services, Plagiarism free|onTime|outstanding customer support|Unlimited Revisions support|High-quality Subject Matter Experts.
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Advanced Diagnostics in the Post-PAMA EraJohn Hanna
Presentation given at the Q1 Diagnostics Summit in Boston MA December 6, 2016 discussing considerations for advanced diagnostics commercialization following the implementation of the Protecting Access to Medicare Act (PAMA) provisions for diagnostics pricing and reimbursement in the Medicare program.
Slides from the presentation on extrapolation from progression free survival to overall survival in oncology given at the 2017 HTAi Annual Meeting in Rome
Prospective identification of drug safety signalsIMSHealthRWES
At a time of growing demand for more accurate and timely
drug safety evidence, a landmark study supports the value of
electronic medical records (EmR) for detecting new adverse
reactions.
Medication nonadherence cost and noncompliance in clinical trialsSynegys
Drug development has reached over $2.6 B and is driven by a clinical trial's success rate, out-of-pocket study costs and study timescales. However, medication nonadherence is a hidden cost which heavily influences these cost drivers. We discuss how medication nonadherence introduces data variability, requiring trial managers to enrol more patients to maintain statistical power, which in turn extends trial timelines. Cost savings are described based on improving study noncompliance with a compliance tool such as Synegys' mComply. This mHealth tool reduces costs as a result of improved statistical power, lower enrollment and shorter trial duration.
The Value of Targeted Sequencing in Advanced Cancer: DCE to Elicit the Public...Office of Health Economics
This project seeks to elicit the public’s preferences for different features of a genomic test to sequence advanced solid cancer tumours. Understanding the relative preferences for various attributes of targeted testing are useful for determining the value of sequencing approaches, and informing technology adoption decisions. A discrete choice experiment (DCE) survey was designed to assess the preferences of members of the Australian general public for targeted sequencing in advanced cancer. The survey presented respondents with 12 questions in which they had to choose between two unlabelled tests (Test A and Test B). Tests were specified in terms of five attributes: time to receive the test result; cost of the test; likelihood that the test result will lead to a change in treatment; length of time health care professionals spend describing the test; and type of health care team who explains the test result. Respondents were sampled from an online panel and also completed questions related to demographic and socio-economic factors, experiences of cancer and familial history. We found that cost, timeliness, expertise/location and likeliness of changing treatment regimes were identified as attributes of genomic sequencing that are most valuable to a sample of the public. These results will ultimately be compared with the results of an ongoing DCE being conducted with patients with advanced cancer who are undergoing sequencing.
Author(s) and affiliation(s): Paula Lorgelly (OHE), Grace Hampson (OHE), James Buchanan (Oxford), Melissa Martyn (MGHA), Jayesh Desai (PeterMac), Clara Gaff (MGHA), and iPREDICT MGHA Flagship collaborators
Conference/meeting: EuHEA 2018
Location: Maastricht, the Netherlands
Date: 12/07/2018
Four strategies to upgrade clinical trial quality in this computerized world ...Pubrica
• Biostatistics Services is important for collecting, reviewing, presenting, and interpreting data in clinical research.
• Applications of clinical biostatistics services are in different areas, such as epidemiology, clinical trials, population genetics, the biology of structures, and more.
Reference : https://pubrica.com/services/research-services/biostatistics-and-statistical-programming-services/
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Decision makers in the healthcare field like doctors, patients and policy makers need access to clinical evidence to address issues that have bearing on the health of the population and the treatment prescribed and thereby on the financials implications of the healthcare industry.
BUSINESS83www.AHDBonline.com l American Health & Drug Ben.docxjasoninnes20
BUSINESS
83www.AHDBonline.com l American Health & Drug Benefits lVol 5, No 2 l March/April 2012
Rheumatoid arthritis (RA) is a chronic systemicautoimmune disorder and the most common formof inflammatory arthritis.1 RA affects 1% of the
population, most often adults aged 40 to 70 years.2
Recent epidemiologic data indicate that the incidence of
RA in women has risen in the past 10 years.3 Because RA
affects many individuals who are of working age and
remains a major cause of disability, the economic burden
of RA adds a significant cost not only to patients and
their families, but also to society as a whole.1,4 In addi-
tion, reduced quality of life, loss of work productivity,
and substantial healthcare utilization are factors that
must be considered in RA management.4,5
Because complications of RA may begin to develop
within months of disease onset, early and aggressive
treatment is considered clinically necessary to manage
immediate symptoms of pain associated with inflamma-
tion, but also to slow disease progression to prevent long-
term disability.1,6,7 Historically, estimates of work disabili-
Ms Greenapple is President, Reimbursement Intelligence,
LLC, Madison, NJ.
Trends in Biologic Therapies for
Rheumatoid Arthritis: Results from a
Survey of Payers and Providers
Rhonda Greenapple, MSPH
Background: Advances in therapies for rheumatoid arthritis (RA), particularly biologics,
have transformed the treatment paradigm for RA. However, the associated costs of these
therapies result in a significant economic burden on the healthcare system. As a chronic
disease requiring lifelong treatment, most health plans now position RA drugs as a high-
priority therapeutic category.
Objective: To identify provider and payer practices and perceptions regarding coverage
of RA biologics in the current marketplace, as well as emerging trends in reimbursement
practices.
Method: In November 2011, Reimbursement Intelligence, a healthcare research company,
collected and analyzed quantitative and qualitative data via parallel-structure online surveys
of 100 rheumatologists and 50 health plan payers (medical and pharmacy directors) who
represent more than 80 million covered lives. The surveys included approximately 150 ques-
tions, and the surveys were designed to force a response for each question.
Results: Payers reported using tier placement, prior authorization, and contracting in
determining coverage strategies for RA biologics. Among providers, experience with older
RA agents remains the key driver for the choice of a biologic agent. A majority of payers
and providers (68% and 54%, respectively) reported that they did not anticipate a change
in the way their plans would manage biologics over the next 2 to 4 years. Payers’ re -
sponses indicated uncertainty about how therapeutic positioning of newer, small-molecule
drugs at price parity to biologics would affect the current reimbursement landscape.
Survey responses show that approval of an indication f ...
Traditionally, physicians recruited clinical trial subjects, but pharmaceutical companies have become ever more involved through centralized campaigns. Physicians are vital to a trial and the pharmaceutical effort helps shift some of the recruitment demands away from the site to allow them to focus on the subjects. Thus, it is practical to understand if different recruitment methods could change or skew the study population. This study determines if differences or similarities occurred between subjects recruited by physicians and pharmaceutical companies. It discovered that some of both occurred. The pharmaceutical company efforts helped recruit potential subjects from the general population that were similar to subjects recruited by the physicians, but this particular campaign was limited by language which affected recruitment of Hispanic subjects. The social impact of this study provides insight about pharmaceutical company recruitment. Since the National Library of Medicine has indicated that clinical trials should reflect the broader diseased population, the efforts of the pharmaceutical company can help support the physicians’ efforts by recruiting from the broader population. Together, both efforts can create a global good by allowing the trial to reflect the population of post-approval use. These findings still raise a question about the proper balance between the two recruitment groups so that the intended characteristics of the diseased population are maintained. Because differences between physician and pharmaceutical recruited subjects can exist, the potential of one group to bias the trial results exist. As such, some analysis by recruitment method can help ensure that variations in the study population are minimal without skewing the data to create positive study results.
Cancer Clinical Trials_ USA Scenario and Study Designs.pdfProRelix Research
Clinical trials in oncology are vital for the advancement of cancer treatments and
care. The US is at the forefront of these clinical trials, with many different study
designs being used to assess the efficacy and safety of new treatments. This article
will explore the current state of oncology clinical trial services in the US, as well as
discuss different types of study designs that are commonly used. It will provide
insight into how these trials are conducted, what data is collected, and how this
information can be used to improve patient care.
The United States Food and Drug Administration (FDA) has released
several guidance documents over the years through the Oncology Center
of Excellence to support the development of oncologic treatments and
diagnoses. Furthermore, information on the clinical trials for the treatment
of different types of cancer or specific interventions can be found on the
National Cancer Institute (NCI) website and Clinical Trials. Currently,
ClinicalTrials.gov, a website maintained by the National Library of
Medicine (NLM) and the National Institutes of Health (NIH) contains
listings of publicly and privately sponsored trials and includes information
on 91,937 studies related to cancer indicating the high volume of
research being conducted in this field.According to the World Health Organization (WHO), cancer is the leading
cause of death worldwide, with a death rate of one in six in 2020 (1).
Aside from the high mortality rate and morbidity associated with cancer, it
also negatively impacts the quality of life and poses a significant financial
burden on patients and payers making it imperative to develop effective
treatments for the disease. According to Global Cancer Observatory
(GLOBACAN), the United States accounted for 13.3% of all estimated
new cases of cancer in 2020 (2). In 2020, the single leading type of
cancer in the United States was breast cancer (11.1%) followed by lung
cancer (10%), prostrate (9,2%), colorectum (6.8%), and melanoma of the
skin (4.2%). Despite the significant prevalence of cancer and numerous
clinical trials conducted for oncology treatments, data have shown an
almost 95% attrition rate for anticancer drugs from Phase I trials until
marketing authorization. Various factors such as inaccurate preclinical
models, lack of suitable biomarkers in clinical trials, and a disconnect
between industry, academia, and regulators are responsible for the high
attrition rate (3). Therefore, it is vital to develop suitable study designs
and protocols for candidate molecules such that they obtain regulatory
approval and can be marketed. In addition to these challenges, the
development of anti-cancer agents comes at a monumental cost of an
estimated $2.8 billion. Several factors such as the choice of relevant
endpoints, the choice of appropriate biomarkers that are guided by tumor
biology, and careful patient selection are expected to improve the overall
fate of oncologic agents in the clinical trial phase
Movers & shakers interview with vivek trikha head diagnostics onc quest ...oncquest
OncQuest Laboratories Limited (formerly Dabur OncQuest) formally announced its existence as an independent entity, moving away from being a part of Dabur Pharma Limited, on December 31, 2007. OncQuest Laboratories Limited was incorporated in January 2008.
Effective strategies to monitor clinical risks using biostatistics - Pubrica.pdfPubrica
In clinical science, biostatistics services are essential for data collection, analysis, presentation, and interpretation. Epidemiology, clinical trials, population genetics, systems biology, and other disciplines all benefit from it. It aids in the evaluation of a drug's effectiveness and safety in clinical trials.
Continue Reading: https://bit.ly/3tRRxkW
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Why Pubrica:
When you order our services, We promise you the following – Plagiarism free | always on Time | 24*7 customer support | Written to international Standard | Unlimited Revisions support | Medical writing Expert | Publication Support | Biostatistical experts | High-quality Subject Matter Experts.
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Leading the Way in Nephrology: Dr. David Greene's Work with Stem Cells for Ki...Dr. David Greene Arizona
As we watch Dr. Greene's continued efforts and research in Arizona, it's clear that stem cell therapy holds a promising key to unlocking new doors in the treatment of kidney disease. With each study and trial, we step closer to a world where kidney disease is no longer a life sentence but a treatable condition, thanks to pioneers like Dr. David Greene.
Navigating the Health Insurance Market_ Understanding Trends and Options.pdfEnterprise Wired
From navigating policy options to staying informed about industry trends, this comprehensive guide explores everything you need to know about the health insurance market.
Global launch of the Healthy Ageing and Prevention Index 2nd wave – alongside...ILC- UK
The Healthy Ageing and Prevention Index is an online tool created by ILC that ranks countries on six metrics including, life span, health span, work span, income, environmental performance, and happiness. The Index helps us understand how well countries have adapted to longevity and inform decision makers on what must be done to maximise the economic benefits that comes with living well for longer.
Alongside the 77th World Health Assembly in Geneva on 28 May 2024, we launched the second version of our Index, allowing us to track progress and give new insights into what needs to be done to keep populations healthier for longer.
The speakers included:
Professor Orazio Schillaci, Minister of Health, Italy
Dr Hans Groth, Chairman of the Board, World Demographic & Ageing Forum
Professor Ilona Kickbusch, Founder and Chair, Global Health Centre, Geneva Graduate Institute and co-chair, World Health Summit Council
Dr Natasha Azzopardi Muscat, Director, Country Health Policies and Systems Division, World Health Organisation EURO
Dr Marta Lomazzi, Executive Manager, World Federation of Public Health Associations
Dr Shyam Bishen, Head, Centre for Health and Healthcare and Member of the Executive Committee, World Economic Forum
Dr Karin Tegmark Wisell, Director General, Public Health Agency of Sweden
One of the most developed cities of India, the city of Chennai is the capital of Tamilnadu and many people from different parts of India come here to earn their bread and butter. Being a metropolitan, the city is filled with towering building and beaches but the sad part as with almost every Indian city
Medical Technology Tackles New Health Care Demand - Research Report - March 2...pchutichetpong
M Capital Group (“MCG”) predicts that with, against, despite, and even without the global pandemic, the medical technology (MedTech) industry shows signs of continuous healthy growth, driven by smaller, faster, and cheaper devices, growing demand for home-based applications, technological innovation, strategic acquisitions, investments, and SPAC listings. MCG predicts that this should reflects itself in annual growth of over 6%, well beyond 2028.
According to Chris Mouchabhani, Managing Partner at M Capital Group, “Despite all economic scenarios that one may consider, beyond overall economic shocks, medical technology should remain one of the most promising and robust sectors over the short to medium term and well beyond 2028.”
There is a movement towards home-based care for the elderly, next generation scanning and MRI devices, wearable technology, artificial intelligence incorporation, and online connectivity. Experts also see a focus on predictive, preventive, personalized, participatory, and precision medicine, with rising levels of integration of home care and technological innovation.
The average cost of treatment has been rising across the board, creating additional financial burdens to governments, healthcare providers and insurance companies. According to MCG, cost-per-inpatient-stay in the United States alone rose on average annually by over 13% between 2014 to 2021, leading MedTech to focus research efforts on optimized medical equipment at lower price points, whilst emphasizing portability and ease of use. Namely, 46% of the 1,008 medical technology companies in the 2021 MedTech Innovator (“MTI”) database are focusing on prevention, wellness, detection, or diagnosis, signaling a clear push for preventive care to also tackle costs.
In addition, there has also been a lasting impact on consumer and medical demand for home care, supported by the pandemic. Lockdowns, closure of care facilities, and healthcare systems subjected to capacity pressure, accelerated demand away from traditional inpatient care. Now, outpatient care solutions are driving industry production, with nearly 70% of recent diagnostics start-up companies producing products in areas such as ambulatory clinics, at-home care, and self-administered diagnostics.
R3 Stem Cells and Kidney Repair A New Horizon in Nephrology.pptxR3 Stem Cell
R3 Stem Cells and Kidney Repair: A New Horizon in Nephrology" explores groundbreaking advancements in the use of R3 stem cells for kidney disease treatment. This insightful piece delves into the potential of these cells to regenerate damaged kidney tissue, offering new hope for patients and reshaping the future of nephrology.
Defecation
Normal defecation begins with movement in the left colon, moving stool toward the anus. When stool reaches the rectum, the distention causes relaxation of the internal sphincter and an awareness of the need to defecate. At the time of defecation, the external sphincter relaxes, and abdominal muscles contract, increasing intrarectal pressure and forcing the stool out
The Valsalva maneuver exerts pressure to expel faeces through a voluntary contraction of the abdominal muscles while maintaining forced expiration against a closed airway. Patients with cardiovascular disease, glaucoma, increased intracranial pressure, or a new surgical wound are at greater risk for cardiac dysrhythmias and elevated blood pressure with the Valsalva maneuver and need to avoid straining to pass the stool.
Normal defecation is painless, resulting in passage of soft, formed stool
CONSTIPATION
Constipation is a symptom, not a disease. Improper diet, reduced fluid intake, lack of exercise, and certain medications can cause constipation. For example, patients receiving opiates for pain after surgery often require a stool softener or laxative to prevent constipation. The signs of constipation include infrequent bowel movements (less than every 3 days), difficulty passing stools, excessive straining, inability to defecate at will, and hard feaces
IMPACTION
Fecal impaction results from unrelieved constipation. It is a collection of hardened feces wedged in the rectum that a person cannot expel. In cases of severe impaction the mass extends up into the sigmoid colon.
DIARRHEA
Diarrhea is an increase in the number of stools and the passage of liquid, unformed feces. It is associated with disorders affecting digestion, absorption, and secretion in the GI tract. Intestinal contents pass through the small and large intestine too quickly to allow for the usual absorption of fluid and nutrients. Irritation within the colon results in increased mucus secretion. As a result, feces become watery, and the patient is unable to control the urge to defecate. Normally an anal bag is safe and effective in long-term treatment of patients with fecal incontinence at home, in hospice, or in the hospital. Fecal incontinence is expensive and a potentially dangerous condition in terms of contamination and risk of skin ulceration
HEMORRHOIDS
Hemorrhoids are dilated, engorged veins in the lining of the rectum. They are either external or internal.
FLATULENCE
As gas accumulates in the lumen of the intestines, the bowel wall stretches and distends (flatulence). It is a common cause of abdominal fullness, pain, and cramping. Normally intestinal gas escapes through the mouth (belching) or the anus (passing of flatus)
FECAL INCONTINENCE
Fecal incontinence is the inability to control passage of feces and gas from the anus. Incontinence harms a patient’s body image
PREPARATION AND GIVING OF LAXATIVESACCORDING TO POTTER AND PERRY,
An enema is the instillation of a solution into the rectum and sig
1. Clinical Trials Intelligence
Biomarker Roles within Clinical Trials
An Analysis of Clinical Trials from 2007-2011 and 2012-2016.
By Gavin Coney, Head of Clinical Products, Clarivate Analytics
April 2017
2. 2
There is a lot of discussion across biopharma
about the increasing complexity in the design
and management of clinical trials. Recent
estimates suggest the cost of bringing a drug
to market is as high as $3 billion, and only
one in 10 drugs successfully navigate the
process and make it to market. These trends
are not sustainable, and the use of targeted
clinical strategies is being seen as a critical
step towards improving success rates.
3. Clarivate Analytics | Biomarker Types and Roles within Clinical Trials 3
In this paper we examine the application of specific
biomarker roles (therapeutic effect marker, toxic
effect marker, disease marker). In this regard we
analyzed the growth in the application of different
biomarker roles across multiple indications with
some interesting trends that will be relevant to
clinical development professionals. While we note
the overall increase in the use of biomarkers, that
growth is not uniform across all biomarkers with
important implications given the continued high
level of unproven efficacy across late-stage trials.
The increase in complexity has triggered a
considerable extension in the average duration
of clinical trials and a reduction in the differential
duration of trial phases.1 (See Figure 1.)
Any increase in trial duration leads to increasing
costs, delays to launch and the potential for
a drug to miss the vital slot as first-in-market,
holding back potentially lifesaving treatments
for patients. Development strategies need
to ensure that the launched compound can
demonstrate sufficient efficacy while avoiding
excessive recruitment demands, undesired
toxicity and costly trial amendments.
While timelines are extending sharply, notably in
oncology, we also note a significant rise in the volume
of trials. Analyzing the number of trials within Cortellis
Clinical Trials Intelligence with a “Start Date” each year
between 2007 and 2016 we see an ongoing rise in the
number of trials. During our two comparison periods,
January 1, 2007 - December 31, 2011 (2007-2011) against
January 1, 2012 - December 31, 2016 (2012-2016), we see
a 33% increase in the number of trials.2
(See Figure 2.)
Source: 1 Journal of Health Economics, DiMasi, Joseph A, Grabowski Henry G & Hansen, Ronald W.
“Innovation in the Pharmaceutical Industry: New Estimates of R&D Cost.” 47, 20-33 (2016).
2
Cortellis Clinical Trials Intelligence, Clarivate Analytics, March 15, 2017.c
Figure 2: Trials Commenced by Year 2007-2016
Figure 1: Mean Phase Length in Months
25
15
20
10
5
0
30
35
45
40
Phase I Phase II Phase III
2007
12007
2008
13124
2009
14568
2010
15467
2011
16622
2012
18657
2013
17806
2014
18320
2015
20243
2016
20678
4. Cortellis | Powering Life Sciences Innovation4
We believe the increasing volume of trials
has implications for patient recruitment and
competition, but also specifically relevant to this
analysis, a heightened need to ensure that the
clinical strategy is based on the best available
intelligence relating to disease mechanisms,
target actions and patient segmentation.
To understand whether the fundamentals of the
underlying biological models are keeping pace
with the complexity of trials, we consulted a recent
published analysis. This demonstrated that the
overwhelming majority of reasons given for failure
are not due to strategic reasons, but are due to
unproven efficacy (52%) and safety issues (24%)
highlighting the importance of a solid biological
rationale, suggesting scientific inadequacies
in the understanding of the underlying disease
mechanism, understanding of the drug action and
the selection of patient cohorts.3
(See Figure 3.)
Figure 3: Trial Failures
Patient populations are diverse and a treatment that
is effective and safe in one sub-population may not
be similarly beneficial and may introduce additional
toxicity in other groups. Patient segmentation
utilizes molecular sub-types for the design of
trials with the aim of increasing the likelihood of
proving the efficacy of the targeted therapies under
development, while reducing or eliminating the
impact of known actions that may increase the
frequency of non-responders or serious adverse
events. The effective applications of biomarkers are
critical in the definition of a patient segmentation
strategy and trial inclusion criteria. A patient
segment that is too broad may lead to unproven
efficacy; conversely a model based on incorrect
assumptions may have implications for both efficacy
and safety and an extremely tight patient segment
may lead to challenges in patient recruitment.
New drugs employing such a targeted approach have
transformed our ability to treat specific diseases,
but also highlighted the potential for therapeutic
development and patient care when a strong
biomarker strategy is employed. Within Chronic
Myeloid Leukemia (CML) and Acute Myeloid Leukemia
(AML) patients who are Philadelphia Chromosome-
positive, Imatinib has validated the clinical strategy
by providing a significantly improved five-year
survival rate for CML patients of 81%, up from 31%.4
This improvement in patient care is also reflected in
other examples where the confidence and validity of
the clinical strategy has been used to define a specific
patient population, reduce risk and ultimately shorten
time to market, particularly when it enables the granting
of a Facilitated Regulatory Pathway. Historically,
drug approvals sought to maximize the potential
market opportunity both in the initial indication and
later line extensions. The rising average duration of
trials suggests that a change in strategic approach is
valuable, particularly after the recent successes with
drugs like Imatinib and Keytruda. Keytruda developed
by Merck is indicated for patients with metastatic or
advanced non-small cell lung cancer who test positive
for TPS>1% PD-L1 expression and negative for EGFR
and ALK mutations. This new drug has a mechanism
of action that is so tightly understood and critically
linked to a clear biomarker strategy that development
professionals had the confidence to carefully select
patients from specific sub-groups from the earliest
trials and demonstrate efficacy from the earliest
opportunity, leading to a uniquely accelerated approval
and ultimately benefiting both patients and Merck
with the granting of a Marketing Authorization.
To determine whether these celebrated successes
reflect the beginning of a new stage of therapeutic
evolution, we wish to understand how deep the shift
has occurred towards personalized therapies and
how widespread such an approach is across the
therapeutic areas within clinical development. By
comparing the two periods, 2007-2011 vs. 2012-2016,
we can identify a number of trends in the application of
biomarkers within active clinical trials that demonstrate
an increase in both the application of specific
biomarker types and roles, also highlighting some
areas where there are opportunities to increase our
knowledge of the underlying biological mechanisms.
Source: 3
Nature Reviews Drug Discovery, Harrison RK. “Phase II and Phase III Failures 2013-2015.” 15, 817–818 (2016)
Commercial
Efficacy
Operational
Safety
Strategy
15%
24%
6%
52%
3%
5. Clarivate Analytics | Biomarker Types and Roles within Clinical Trials 5
We analyzed a total database of 263,210 clinical
trial records within Cortellis Clinical Trials
Intelligence, each record representing a single
clinical trial, populated from intelligence gathered
from 30 global trial registries and additional
published source references. (See Figure 4.)
Figure 4: Number of Trials Using Specific Biomarker Roles, 2007-2011 - 2012-2016
Source: Cortellis Clinical Trials Intelligence, Clarivate Analytics, March 2017.c. 4
New England Journal of Medicine, Druker
BJ, et al. “Five-Year Follow-up of Patients Receiving Imatinib for Chronic Myleloid Leukemia.” 355, 2408-2417 (2006)
Trials Using Biomarkers for Therapeutic Effect
25719
41512
Trials Using Biomarkers for Toxic Effect
2287
4542
Trials Using Biomarkers for Disease Risk
6004
13204
2007 - 2011
2012 - 2016
Trials Using Biomarkers for Therapeutic Effect
25719
Trials Using Biomarkers for Toxic Effect
2287
4542
Trials Using Biomarkers for Disease Risk
6004
13204
2007 - 2011
2012 - 2016
Trials Using Biomarkers
for Therapeutic Effect
Trials Using Biomarkers
for Toxic Effect
Trials Using Biomarkers
for Disease Risk
43%
5%
14%
61%
99%
120%
0%
20%
40%
60%
80%
100%
120%
140%
0%
5%
10%
15%
20%
25%
30%
35%
40%
45%
50%
All indications % of all trials 2012 - 2016 All indications % increase
As expected, our analysis confirmed that the use
of biomarkers is increasing. Examining trials from
2007 to 2016, we see a rise in the number of trials
commenced. From 2007-2011, there were 71,735
while from 2012-2016 we found 95,552. This is
an increase of 33%, with a significantly higher
corresponding jump in the application of biomarkers
to assess therapeutic effect, toxicity and disease.
When analyzing the role of all biomarkers we see
that markers of disease increased by 120% over the
time period, followed by markers for toxic effect,
99%, and therapeutic effect markers at 61%.
To focus on our analysis of growth in the application
of specific biomarker types, we examined the
relationship between growth in the utilization
of specific biomarker roles against the current
level of use of each role. (See Figure 5.)
Here we can see that while therapeutic effect markers
are the most commonly employed in 43% of trials, at a
61% growth rate, they are not growing in utilization as
fast as toxic effect, 99%, or disease at 120%. Given the
relatively low historical application of toxic effect and
disease markers, this investment in clinical programs
will shape future therapies available to patients.
Figure 5: % of Total Trials Using Specific Biomarker Roles Against
Growth in the Application of Biomarker Usage
6. Cortellis | Powering Life Sciences Innovation6
Given the overall strength of growth across all
biomarker roles across all indications, an examination
of individual therapeutic groups will highlight those
areas experiencing the highest level of growth and
currently undergoing the most dramatic transformation.
We will examine therapeutic effect markers, toxic effect
markers and disease markers within therapy areas (TAs)
that demonstrated the highest levels of transformation.
Looking at the growth in application of therapeutic
effect markers, nutritional disorders display a rapid
growth rate against a high base level. Neurological
disease is clearly experiencing a period of rapid
growth in the application of therapeutic effect
markers against a lower relative base, along with
cardiovascular disease and musculoskeletal disease.
We can also observe the appearance of gynecology
and obstetrics as high growth areas for therapeutic
effect markers as being notable for not appearing as
highly in any of the other categories. (See Figure 6.)
Figure 6: Growth in the Application of Therapeutic Effect Biomarkers
Against Current % Share of Trials Within the Same Therapy Area (TA)
While the overall adoption of toxic effect markers is
lower than other roles, there is strong evidence of
growth in both established and emerging therapeutic
areas. Despite the relatively strong presence of
oncology in this list in respect of total volumes as a
percentage of all TA trials, oncology does not appear
on our list of highest growth areas for application of
toxic effect markers. Similarly, metabolic disorders
have a high rate of current utilization, but a lower
level of increase. The highest growth therapy areas
within the high volume groups are neurological
disease at 177%, cardiovascular disease at 144% and
infectious diseases at 114%. Within the lower volume
groups, nutritional is again an area of clear growth at
152%, along with musculoskeletal disease at 107%
and metabolic disorder: 103%. (See Figure 7.)
We will examine therapeutic effect
markers, toxic effect markers and
disease markers within therapy areas that
demonstrated highest levels of transformation.
25%
58%
39% 41%
80%
53%
158%
30%
51%
62%
40% 46% 64%
88%
49% 104%
32%
47%
59%
73%
52%
46%
34%
42% 41%
47%
42%
36% 37%
79%
0%
20%
40%
60%
80%
100%
0%
50%
100%
150%
200%
Neoplasm Gastrointestinal
disease
Endocrine
disease
Metabolic
disorder
Cardiovascular
disease
Inflammatory
disease
Neurological
disease
Immune
disorder
Respiratory
disease
Genitourinary
disease
Hematological
disease
Infectious
disease
Gynecology
and obstetrics
Musculoskeletal
disease
Nutritional
disorder
% increase % Total Trials in TA 2012 - 2016
Gastrointestinal
disease
Endocrine
disease
Metabolic
disorder
Cardiovascular
disease
Inflammatory
disease
Neurological
disease
Immune
disorder
Respiratory
disease
Genitourinary
disease
Hematological
disease
Infectious
disease
Gynecology
and obstetrics
Musculoskeletal
disease
Nutritional
disorder
% increase % Total Trials in TA 2012 - 2016
0%
20%
40%
60%
80%
100%
120%
140%
160%
180%
200%
0%
1%
2%
3%
4%
5%
6%
7%
8%
40%
76%
61%
103%
144%
96%
177%
71%
79%
96%
75%
114%
65%
107%
152%
6%
5%
5%
5%
4%
5%
4%
6%
6%
5%
7%
6%
5%
4%
3%
Neoplasm
Figure 7: Growth in the Application of Toxic Effect Biomarkers
Against Current % Share of Trials Within the Same Therapy Area
7. Clarivate Analytics | Biomarker Types and Roles within Clinical Trials 7
Confirming our analysis at the global level earlier, we
see some areas of very strong growth in the utilization
of disease markers (>100%). Within the utilization
of disease markers, we see that gynecology and
obstetrics uses disease markers at a higher level than
any of the other TAs represented here; conversely, it is
also displaying the lowest level of growth in utilization.
Neurological disease, starting from a low base, is
experiencing a huge rise in disease marker investment
as it has in the other categories. (See Figure 8.)
Source: Cortellis Clinical Trials Intelligence, Clarivate Analytics, March 2017.c.
Gastrointestinal
disease
Endocrine
disease
Metabolic
disorder
Cardiovascular
disease
Inflammatory
disease
Neurological
disease
Immune
disorder
Respiratory
disease
Genitourinary
disease
Hematological
disease
Infectious
disease
Gynecology
and obstetrics
Musculoskeletal
disease
Nutritional
disorder
% increase % Total Trials in TA 2012 - 2016
0%
5%
10%
15%
20%
25%
30%
35%
0%
50%
100%
150%
200%
250%
Neoplasm
75%
121%
80%
131%
201%
137%
237%
100%
171%
119%
78%
123%
73%
193%
114%
29%
15%
23%
12%
14%
10%
9%
14%
20%
15%
23%
9%
32%
9% 10%
Figure 8: Growth in the Application of Disease Biomarkers Against
Current % Share of Trials Within the Same Therapy Area
The analysis we have shown here provides a macro
view of an industry which has many individual
successes and many ongoing challenges. Combining
our awareness of the different biomarker roles and
therapy areas, we can clearly confirm that applications
of biomarkers for therapeutic effect, toxic effect and
disease are all rising. However, the level of current use
and investment varies considerably across different
therapy areas reflecting different clinical strategies and
confidence in the underlying biological fundamentals.
The importance of confidence in the underlying
biological rationale is critical given the increasing
duration and cost of conducting clinical trials.
Biomarkers play a key role in the selection of patients
and the ability to sufficiently demonstrate efficacy.
In this analysis, we have demonstrated some strong
and some weaker areas of growth. Neurological
disease appears in the highest growth position in
all three biomarker roles that we examined. Given
that neurological disease is a relatively mature
area, this represents a continued and increasing
confidence in the role of biomarkers for therapeutic
effect, toxic effect and disease. Nutritional disorder,
despite having a much lower base of trial volume,
is demonstrating our second highest growth in all
categories except disease markers. Overall use of
biomarkers within musculoskeletal disease trials
also performs well across the tables. Despite the
much celebrated successes that have propelled the
potential appreciation in the role of biomarkers in
neoplasm/oncology, growth is slowing and it does not
appear near the top of any of the growth metrics.