Critical Path Institute
Encyclopedia
Critical Path Institute (C-Path) is an independent, non-profit organization committed to transformational improvement of the drug development
process. An international leader in forming collaborations around this mission, C-Path has established first-of-its-kind, global consortia that currently include over 1,000 scientists from government regulatory and research agencies, academia
, patient advocacy organizations
, and thirty major pharmaceutical companies.
To address the declining productivity of the pharmaceutical industry, the U.S. Food and Drug Administration (FDA) launched the Critical Path Initiative (CPI) in 2004, its national strategy for forming collaborations to transform the way FDA-regulated medical products are developed, evaluated, and manufactured. C-Path was created, with full support from the FDA and initial funding from the state of Arizona
, to fill an essential role as a neutral third party that enables scientists from the regulated industry and international regulatory agencies to work together in establishing a faster, safer, and more efficient path through the drug development process.
Since its launch in 2005, five C-Path consortia—made up of participants from industry, academia, regulatory agencies, and patient advocacy groups—have focused on identifying the best methods for testing drug safety and efficacy, and sharing their data and knowledge about major diseases (clinical trial
data, quantitative disease
progression models, and biomarkers). In order to maintain its status as a neutral, trusted third-party, C-Path receives no funding from companies that develop products regulated by the FDA, and as such, relies on in-kind contributions, philanthropic support, foundation and federal grants, a volunteer board of directors, and community engagement to accomplish its mission.
, the Bill & Melinda Gates Foundation, and others. C-Path focuses on work that has been identified as high priority by the FDA and is in the interest of national and global public health
.
on how a biomarker can be used to support specific decisions to be made by the industry and/or the FDA, it can be designated as qualified for that use. Based on the work with C-Path consortia, the FDA recently released a Draft Guidance for Qualification of Drug Development Tools. This is the process being used by C-Path consortia and others to obtain regulatory decisions on the acceptability of new testing methods in drug development.
that are revolutionizing the drug development process. Readers Digest
selected C-Path as one of 18 Ideas to Reform Health Care, citing its ability to “bring together the FDA and drug, biotech, and diagnostic companies (many of them fierce competitors) to talk more openly so patients can get safer drugs more quickly and inexpensively.”
, with offices in Phoenix, Arizona
, and Rockville
, Maryland
. Raymond L. Woosley, M.D., Ph.D. is the founding President & CEO.
Drug development
Drug development is a blanket term used to define the process of bringing a new drug to the market once a lead compound has been identified through the process of drug discovery...
process. An international leader in forming collaborations around this mission, C-Path has established first-of-its-kind, global consortia that currently include over 1,000 scientists from government regulatory and research agencies, academia
Academia
Academia is the community of students and scholars engaged in higher education and research.-Etymology:The word comes from the akademeia in ancient Greece. Outside the city walls of Athens, the gymnasium was made famous by Plato as a center of learning...
, patient advocacy organizations
Patient advocacy
A Patient Advocate acts as a support structure and if legally contracted to do so may act as a liaison between a patient and their Health Care Provider. Most health care professionals see themselves as advocates for their patients, however their time and scope are limited by their job function...
, and thirty major pharmaceutical companies.
Background
New drug development can cost nearly $1 billion and take, on average, 15 years to get from laboratory testing to regulatory approval; only five- to 15-percent of new medicines that enter human testing reach the market. In 2010, only 21 new drugs were approved for marketing.To address the declining productivity of the pharmaceutical industry, the U.S. Food and Drug Administration (FDA) launched the Critical Path Initiative (CPI) in 2004, its national strategy for forming collaborations to transform the way FDA-regulated medical products are developed, evaluated, and manufactured. C-Path was created, with full support from the FDA and initial funding from the state of Arizona
Arizona
Arizona ; is a state located in the southwestern region of the United States. It is also part of the western United States and the mountain west. The capital and largest city is Phoenix...
, to fill an essential role as a neutral third party that enables scientists from the regulated industry and international regulatory agencies to work together in establishing a faster, safer, and more efficient path through the drug development process.
Since its launch in 2005, five C-Path consortia—made up of participants from industry, academia, regulatory agencies, and patient advocacy groups—have focused on identifying the best methods for testing drug safety and efficacy, and sharing their data and knowledge about major diseases (clinical trial
Clinical trial
Clinical trials are a set of procedures in medical research and drug development that are conducted to allow safety and efficacy data to be collected for health interventions...
data, quantitative disease
Disease
A disease is an abnormal condition affecting the body of an organism. It is often construed to be a medical condition associated with specific symptoms and signs. It may be caused by external factors, such as infectious disease, or it may be caused by internal dysfunctions, such as autoimmune...
progression models, and biomarkers). In order to maintain its status as a neutral, trusted third-party, C-Path receives no funding from companies that develop products regulated by the FDA, and as such, relies on in-kind contributions, philanthropic support, foundation and federal grants, a volunteer board of directors, and community engagement to accomplish its mission.
Approach
C-Path works with over 1,000 scientists from over 30 major pharmaceutical companies around the world under a common legal agreement; this agreement allows the sharing of data to generate new scientific knowledge and tools that will enable the design of more efficient clinical trials leading to rapid approval of therapies. They also work with scientists from academia, the National Institutes of Health, the Centers for Disease Control, the World Health OrganizationWorld Health Organization
The World Health Organization is a specialized agency of the United Nations that acts as a coordinating authority on international public health. Established on 7 April 1948, with headquarters in Geneva, Switzerland, the agency inherited the mandate and resources of its predecessor, the Health...
, the Bill & Melinda Gates Foundation, and others. C-Path focuses on work that has been identified as high priority by the FDA and is in the interest of national and global public health
Public health
Public health is "the science and art of preventing disease, prolonging life and promoting health through the organized efforts and informed choices of society, organizations, public and private, communities and individuals" . It is concerned with threats to health based on population health...
.
Qualification Process
C-Path and FDA scientists have worked together to identify the guiding principles for how biomarkers can be scientifically reviewed in order to bring greater efficiency to the drug development process. Instead of describing biomarkers with general terms such as “validated” or “surrogate endpoints” as is generally done, C-Path prefers the designation qualified for a specific use or fit for use. By specifying the intended “use,” much greater clarity is provided on the context and intent for using a biomarker. Once a scientific consensusScientific consensus
Scientific consensus is the collective judgment, position, and opinion of the community of scientists in a particular field of study. Consensus implies general agreement, though not necessarily unanimity. Scientific consensus is not by itself a scientific argument, and it is not part of the...
on how a biomarker can be used to support specific decisions to be made by the industry and/or the FDA, it can be designated as qualified for that use. Based on the work with C-Path consortia, the FDA recently released a Draft Guidance for Qualification of Drug Development Tools. This is the process being used by C-Path consortia and others to obtain regulatory decisions on the acceptability of new testing methods in drug development.
C-Path Consortia
C-Path consortia are public/private partnershipsPublic-private partnership
Public–private partnership describes a government service or private business venture which is funded and operated through a partnership of government and one or more private sector companies...
that are revolutionizing the drug development process. Readers Digest
Reader's Digest
Reader's Digest is a general interest family magazine, published ten times annually. Formerly based in Chappaqua, New York, its headquarters is now in New York City. It was founded in 1922, by DeWitt Wallace and Lila Bell Wallace...
selected C-Path as one of 18 Ideas to Reform Health Care, citing its ability to “bring together the FDA and drug, biotech, and diagnostic companies (many of them fierce competitors) to talk more openly so patients can get safer drugs more quickly and inexpensively.”
- The Predictive Safety Testing Consortium (PSTC) was the first of C-Path’s consortia, and brings together pharmaceutical companies to share and qualify one another's safety testing methods. The process included advisors from the FDA and its European counterpart, the European Medicines Agency (EMA)European Medicines AgencyThe European Medicines Agency is a European agency for the evaluation of medicinal products. From 1995 to 2004, the European Medicines Agency was known as European Agency for the Evaluation of Medicinal Products.Roughly parallel to the U.S...
. The industry members of the consortium share experience with pre-clinical and clinical safety biomarkers in six workgroups: carcinogenicity, kidneyKidneyThe kidneys, organs with several functions, serve essential regulatory roles in most animals, including vertebrates and some invertebrates. They are essential in the urinary system and also serve homeostatic functions such as the regulation of electrolytes, maintenance of acid–base balance, and...
, liverLiverThe liver is a vital organ present in vertebrates and some other animals. It has a wide range of functions, including detoxification, protein synthesis, and production of biochemicals necessary for digestion...
, muscleMuscleMuscle is a contractile tissue of animals and is derived from the mesodermal layer of embryonic germ cells. Muscle cells contain contractile filaments that move past each other and change the size of the cell. They are classified as skeletal, cardiac, or smooth muscles. Their function is to...
, vascularVascularVascular in zoology and medicine means "related to blood vessels", which are part of the circulatory system. An organ or tissue that is vascularized is heavily endowed with blood vessels and thus richly supplied with blood....
injury, and cardiac hypertrophyHypertrophyHypertrophy is the increase in the volume of an organ or tissue due to the enlargement of its component cells. It should be distinguished from hyperplasia, in which the cells remain approximately the same size but increase in number...
. All biomarker research programs have a strong clinical and translationalTranslational medicineTranslational medicine is a medical practice based on interventional epidemiology. It is regarded by its proponents as a natural progression from Evidence-Based Medicine. It integrates research from the basic sciences, social sciences and political sciences with the aim of optimising patient care...
focus to select new safety tools that are applicable across the drug development spectrum. - The Patient-Reported Outcome (PRO) Consortium was formed by C-Path in cooperation with the FDA and the medical products industry. Its purpose is to develop, evaluate, and qualify PRO instruments (e.g., questionnaires) for use in clinical trials designed to assess the safety and effectiveness of medical products. PRO instruments offer a means for capturing how a patient feels or functions with respect to her/his health or condition. Instruments are currently being developed to support label claims for efficacy in asthmaAsthmaAsthma is the common chronic inflammatory disease of the airways characterized by variable and recurring symptoms, reversible airflow obstruction, and bronchospasm. Symptoms include wheezing, coughing, chest tightness, and shortness of breath...
, depression, irritable bowel syndromeIrritable bowel syndromeIrritable bowel syndrome is a diagnosis of exclusion. It is a functional bowel disorder characterized by chronic abdominal pain, discomfort, bloating, and alteration of bowel habits in the absence of any detectable organic cause. In some cases, the symptoms are relieved by bowel movements...
, diabetes, mild cognitive impairment, and cancerCancerCancer , known medically as a malignant neoplasm, is a large group of different diseases, all involving unregulated cell growth. In cancer, cells divide and grow uncontrollably, forming malignant tumors, and invade nearby parts of the body. The cancer may also spread to more distant parts of the...
(breastBreast cancerBreast cancer is cancer originating from breast tissue, most commonly from the inner lining of milk ducts or the lobules that supply the ducts with milk. Cancers originating from ducts are known as ductal carcinomas; those originating from lobules are known as lobular carcinomas...
and lungLung cancerLung cancer is a disease characterized by uncontrolled cell growth in tissues of the lung. If left untreated, this growth can spread beyond the lung in a process called metastasis into nearby tissue and, eventually, into other parts of the body. Most cancers that start in lung, known as primary...
). - The Coalition Against Major Diseases (CAMD) includes patient advocacyPatient advocacyA Patient Advocate acts as a support structure and if legally contracted to do so may act as a liaison between a patient and their Health Care Provider. Most health care professionals see themselves as advocates for their patients, however their time and scope are limited by their job function...
groups, biopharmaceuticalBiopharmaceuticalBiopharmaceuticals are medical drugs produced using biotechnology. They include proteins , nucleic acids and living microorganisms like virus and bacteria where the virulence of viruses and bacteria is reduced by the process of attenuation, they can be used for therapeutic or in vivo diagnostic...
companies, foundations, and advisors from the FDA, EMA, the National Institute of Neurological Disorders and StrokeNational Institute of Neurological Disorders and StrokeThe National Institute of Neurological Disorders and Stroke is a part of the U.S. National Institutes of Health . It conducts and funds research on brain and nervous system disorders and has a budget of just over US$1.5 billion...
(NINDS), and the National Institute on AgingNational Institute on AgingThe National Institute on Aging ' is a division of the U.S. National Institutes of Health , located in Baltimore, Maryland.The NIA leads a broad scientific effort to understand the nature of aging and to extend the healthy, active years of life...
(NIA). Members share pre-competitive data and knowledge that will more efficiently and safely speed development of treatments and preventions for Alzheimer’s, Parkinson’s, Huntington’s, and other debilitating neuro-degenerative diseasesDegenerative diseaseA degenerative disease, also called neurodegenerative disease, is a disease in which the function or structure of the affected tissues or organs will progressively deteriorate over time, whether due to normal bodily wear or lifestyle choices such as exercise or eating habits...
. CAMD’s overall objective is to help scientists identify clinical and laboratory characteristics (biomarkers) of patients destined to develop Alzheimer’s but before they are symptomatic and therefore more likely to benefit from new therapies. CAMD gathers and submits the evidence for the FDA and EMA to officially designate such tools as qualified for use in drug development.
-
- CAMD developed and launched the largest standardized databaseDatabaseA database is an organized collection of data for one or more purposes, usually in digital form. The data are typically organized to model relevant aspects of reality , in a way that supports processes requiring this information...
of combined clinical trialClinical trialClinical trials are a set of procedures in medical research and drug development that are conducted to allow safety and efficacy data to be collected for health interventions...
s to be openly shared by pharmaceutical companies and made available to qualified researchers around the world. This database—involving anonymous data from approximately 4,000 Alzheimer's patients—provides a platform from which to design more efficient clinical trials to test new treatments. This will allow the industry to more rapidly identify potential therapies that prevent the disease or halt its progression.- The Critical Path to TB Drug Regimens (CPTR) is a collaboration of pharmaceutical companies, government, regulatory, and multilateral agencies, donors, academia, advocates, and NGOs that aims to accelerate the development of new, safe, and highly effective tuberculosis treatmentTuberculosis treatmentTuberculosis treatment refers to the medical treatment of the infectious disease tuberculosis .The standard "short" course treatment for TB is isoniazid, rifampicin , pyrazinamide, and ethambutol for two months, then isoniazid and rifampicin alone for a further four months...
regimens with shortened durations of therapy. Joining the Bill & Melinda Gates Foundation and the Global Alliance for TB Drug Development (TB Alliance) as CPTR founding organizations, C-Path provides overall program management for this initiative and leads one component, the CPTR Regulatory Science Consortium. - The Polycystic Kidney DiseasePolycystic kidney diseaseAutosomal dominant polycystic kidney disease is an inherited systemic disorder that predominantly affects the kidneys, but may affect other organs including the liver, pancreas, brain, and arterial blood vessels...
(PKD) Consortium was created and is managed by C-Path with funding from the PKD Foundation, and includes three academic medical centers (University of Colorado, Emory University, and Mayo Clinic). Its mission is to evaluate the evidence supporting total kidney volumeRenal functionRenal function, in nephrology, is an indication of the state of the kidney and its role in renal physiology. Glomerular filtration rate describes the flow rate of filtered fluid through the kidney...
(TKV) as a biomarker for assessing the progression of autosomal dominant PKD. If the data support the conclusion that TKV is an early and reliable predictor of subsequent renal failure, C-path will submit a request to the FDA that TKV be qualified as a measure of disease progression that can be used in drug development to test the efficacy of new drugs.
- The Critical Path to TB Drug Regimens (CPTR) is a collaboration of pharmaceutical companies, government, regulatory, and multilateral agencies, donors, academia, advocates, and NGOs that aims to accelerate the development of new, safe, and highly effective tuberculosis treatment
- CAMD developed and launched the largest standardized database
Location
C-Path is headquartered in Tucson, ArizonaTucson, Arizona
Tucson is a city in and the county seat of Pima County, Arizona, United States. The city is located 118 miles southeast of Phoenix and 60 miles north of the U.S.-Mexico border. The 2010 United States Census puts the city's population at 520,116 with a metropolitan area population at 1,020,200...
, with offices in Phoenix, Arizona
Phoenix, Arizona
Phoenix is the capital, and largest city, of the U.S. state of Arizona, as well as the sixth most populated city in the United States. Phoenix is home to 1,445,632 people according to the official 2010 U.S. Census Bureau data...
, and Rockville
Rockville, Maryland
Rockville is the county seat of Montgomery County, Maryland, United States. It is a major incorporated city in the central part of Montgomery County and forms part of the Baltimore-Washington Metropolitan Area. The 2010 U.S...
, Maryland
Maryland
Maryland is a U.S. state located in the Mid Atlantic region of the United States, bordering Virginia, West Virginia, and the District of Columbia to its south and west; Pennsylvania to its north; and Delaware to its east...
. Raymond L. Woosley, M.D., Ph.D. is the founding President & CEO.