June 13, 2017

 

TUCSON, Ariz., and AUSTIN, Texas – June 13, 2017 – The Clinical Data Interchange Standards Consortium (CDISC) and Critical Path Institute (C-Path) announce the availability of a Duchenne muscular dystrophy therapeutic area user guide (TAUG-DMD v1.0) for public review. The review period for the TAUG-DMD v1.0 began on May 8, 2017, and runs through July 6, 2017. Qualified researchers and clinicians are encouraged to review TAUG-DMD v1.0 and offer feedback.

To review the user guide, please visit: https://www.cdisc.org/public-review/duchenne-muscular-dystrophy-v10-public-review.

TAUG-DMD v1.0 describes the most common clinical concepts relevant to Duchenne studies using the CDISC standard format. This format allows datasets from different sources to be compared or combined for analysis. The US Food and Drug Administration (FDA) Binding Guidance requires that sponsors whose studies start after December 17, 2016, must submit data in FDA-supported CDISC formats listed in the FDA Data Standards Catalog.

Duchenne is a rare, fatal, genetic neuromuscular disorder that is diagnosed in childhood and primarily affects boys. It causes progressive loss of muscle, resulting in loss of the ability to walk, loss of upper body strength, progressive breathing issues, cardiomyopathy, and premature death. There are treatments for Duchenne that slow down disease progression to some extent, but there is currently no cure. Further research is needed to develop better therapies to respond to this urgent unmet medical need.

C-Path launched the Duchenne Regulatory Sciences Consortium (D-RSC) in partnership with Parent Project Muscular Dystrophy (PPMD) to establish drug development tools that can accelerate clinical progress of new therapies for the disease.

“The Duchenne therapeutic area user guide allows users to structure data from existing studies so that they can be compared and aggregated in a scientifically meaningful way,” said Jane Larkindale, DPhil, Executive Director of D-RSC. “Our consortium will use TAUG-DMD v1.0 to develop an aggregated database to extract new knowledge from existing studies, which will inform development of future clinical trials, allowing more efficient testing of future therapies.”

This first version (v1.0) of TAUG-DMD focuses on the representation of data using the CDISC Study Data Tabulation Model (SDTM). Types of data covered include genetic information and imaging data, as well as results from a variety of disease measures including cardiac, musculoskeletal, and pulmonary function assessments. Data standards are also being developed for a variety of functional tests.

TAUG-DMD v1.0 was developed under the Coalition for Accelerating Standards and Therapies (CFAST), with clinical advice and input from D-RSC members. CFAST, a joint initiative of CDISC and C-Path, was formed to accelerate clinical research and medical product development by creating and maintaining data standards, tools, and methods for conducting research in therapeutic areas important to public health, with invaluable support and advice from such organizations as the National Cancer Institute, Innovative Medicines Initiative, TransCelerate, and regulatory agencies including the FDA, Japan’s Pharmaceutical and Medical Devices Agency, and the European Medicines Agency.

CDISC standards have been adopted and used in more than 90 countries, and are required by regulatory authorities in the US and Japan. To date, therapeutic area standards have been developed for more than 25 different disease areas, with most being developed under the CFAST program. Use of these standards from the start of clinical research programs has proven capable of saving both time and resources. Researchers in Duchenne are encouraged to implement these standards into their processes.

About the organizations:

Critical Path Institute (C-Path) is an independent, nonprofit organization established in 2005 with public and private philanthropic support from the Arizona community, Science Foundation Arizona, and the US Food and Drug Administration (FDA). C-Path’s mission is to catalyze the development of new approaches that advance medical innovation and regulatory science, accelerating the path to a healthier world. An international leader in forming collaborations, C-Path has established 14 global, public-private partnerships that currently include over 1,450 scientists from government and regulatory agencies, academia, patient advocacy organizations, and dozens of major pharmaceutical companies. C-Path is headquartered in Tucson, Arizona. For more information, visit www.c-path.org.

About CDISC – CDISC is a 501(c)(3) global nonprofit organization that develops data standards to foster smarter research and enable connections to healthcare. CDISC standards allow data to speak the same language, by providing common formats for data collection, data sharing and data analyses to make the most of the valuable information offered by patients participating in research studies around the globe, enabling researchers to discover new treatments, find breakthroughs, and unlock cures. CDISC standards are required for regulatory submissions to the US FDA and Japan PMDA, are endorsed by the China CFDA, and are requested for use by the European Innovative Medicines Initiative (IMI). The suite of CDISC standards is freely available on the CDISC website.

CDISC is funded through the generous support of over 400 member organizations from pharmaceutical, biotech, clinical research organizations, regulatory agencies, academia, and healthcare, as well as through grants, authorized CDISC Education courses, events, and charitable contributions. To find out more about how to support CDISC and get involved, please visit www.cdisc.org and www.unlockcures.org.

C-Path Contact:
Kissy Black
+1.615.298.1144
[email protected]

May 1, 2017

 

Critical Path Institute and TGen Forge Partnership to Advance TB Research

TUCSON and FLAGSTAFF, May 1, 2017 – The Critical Path to TB Drug Regimens (CPTR), an initiative of the Critical Path Institute (C-Path), has received a $1.1 million grant from the Bill & Melinda Gates Foundation to significantly enrich its Relational Sequencing TB Data Platform (ReSeqTB). To this end, C-Path and the Translational Genomics Research Institute (TGen) are excited to announce a new partnership to sequence at least 12,000 tuberculosis (TB) bacteria isolates from around the world.

ReSeqTB standardizes, curates, and aggregates genomic, phenotypic, and clinical outcome data, to better understand the genetic basis of drug resistance in Mycobacterium tuberculosis, cause of one of the world’s most deadly diseases. The new data are intended to augment the ReSeqTB data-sharing platform, which provides key data on TB drug resistance to researchers worldwide.

“This critical partnership will allow us to bridge knowledge gaps essential for identifying TB drug-resistance patterns, and ultimately help us to better treat patients,” said Debra Hanna, PhD, Executive Director of CPTR. “With TGen’s expertise in DNA sequencing, we at C-Path are able to extract valuable data from existing clinical samples and make them available globally via the ReSeqTB data-sharing platform.”

TGen’s Pathogen Genomics Division, or TGen North, plans to sequence the DNA of the isolates using state-of-the-art, next-generation sequencing technology over the next three years, in an effort to build a database that can be used to pinpoint genetic markers that indicate how best to treat each individual, said Dr. David Engelthaler, Associate Professor and Co-Director of TGen North. Dr. Engelthaler went on to say that identifying DNA markers associated with TB drug resistance will also enable clinicians to more rapidly diagnose the disease and direct patients toward the best available treatments for their particular strain of bacteria.

“TB can quickly develop resistance to treatments, even multiple-drug resistance. And the degree and type of drug resistance varies from place to place,” Dr. Engelthaler said. “We need better ways to determine which patients should receive which specific drugs to address their infection. The ultimate goal is a more personalized, and effective, approach to treating this disease.”

Dr. Marco Schito, Scientific Director of CPTR, also emphasized how the addition of these important data—which will increase the number of isolates represented on ReSeqTB to twenty thousand—aids in the goal of personalized medicine.

“ReSeqTB is at the forefront of the genomic revolution,” he said. “The integration of clinical outcome data will be key toward having personalized medicine options available to patients who are infected with drug-resistant tuberculosis bacteria. This would have a significant impact on global health.”

As a sub-award from C-Path, and working with multiple collaborators, TGen North will sequence samples derived from as many as 35 countries around the world, including developing nations in Africa, Asia, Eastern Europe, and South America, where drug-resistant TB continues to present a significant threat. The genome samples will come from public health laboratories, clinical drug trials, and biobanks. The sources of samples and the number of countries involved could grow over time, providing an expanding base of evidence for researchers to draw on.

“ReSeqTB is a critical endeavor if we hope to drive the development of new diagnostics in drug-susceptibility tests to support TB patients and their diagnoses, and to prescribe appropriate therapy,” said Dr. Hanna. “The vision for ReSeqTB is to better understand TB resistance across the globe by bringing together comprehensive, whole-genome sequencing data, robust phenotypic data, and patient-outcome data related to those isolates wherever and whenever possible. Our partnership with TGen North is another step toward realizing this vision.”

 

About the Organizations:

Critical Path Institute (C-Path) is an independent, nonprofit organization established in 2005 with public and private philanthropic support from the Arizona community, Science Foundation Arizona, and the US Food and Drug Administration (FDA). C-Path’s mission is to catalyze the development of new approaches that advance medical innovation and regulatory science, accelerating the path to a healthier world. An international leader in forming collaborations, C-Path has established 14 global, public-private partnerships that currently include over 1,450 scientists from government and regulatory agencies, academia, patient advocacy organizations, and dozens of major pharmaceutical companies. C-Path is headquartered in Tucson, Arizona. For more information, visit www.c-path.org.

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Translational Genomics Research Institute (TGen) is a Phoenix, Arizona-based nonprofit organization dedicated to conducting groundbreaking research with life changing results. TGen is focused on helping patients with neurological disorders, cancer, and diabetes, through cutting edge translational research (the process of rapidly moving research toward patient benefit). TGen physicians and scientists work to unravel the genetic components of both common and rare complex diseases in adults and children. Working with collaborators in the scientific and medical communities literally worldwide, TGen makes a substantial contribution to help our patients through efficiency and effectiveness of the translational process. TGen is allied with City of Hope, a world-renowned independent research and cancer and diabetes treatment center. This precision medicine alliance enables both institutes to complement each other in research and patient care, with City of Hope providing a significant clinical setting to advance scientific discoveries made by TGen. For more information, visit: www.tgen.org. Follow TGen on Facebook, LinkedIn and Twitter @TGen.

 

C-Path Contact:
Kissy Black
+1.615.298.1144
[email protected]

April 26, 2017

Critical Path Institute Launches Transplant Therapeutics Consortium

Tucson, Ariz., April 26, 2017 –Critical Path Institute (C-Path) is pleased to announce the launch of the Transplant Therapeutics Consortium (TTC), co-founded by the two leading US organizations in organ transplantation: the American Society of Transplantation (AST) and the American Society of Transplant Surgeons (ASTS). The TTC is a collaboration within the transplant community including clinicians, surgeons, industry scientists, and others. The overall objective of the TTC is to collaborate on the development and regulatory endorsement of new drug development tools applicable in kidney transplant, with the hope of expanding to other solid organ transplantation in the future.

In the United States, there are currently 600,000 patients receiving dialysis every year, with more than 105,000 patients on the kidney transplant waiting list. In 2016, there were 19,000 kidney transplants in the US, and the failure rates for living donor kidney transplants at 1, 5, and 10 years are approximately 3%, 15%, and 40%, respectively. Deceased donor kidney transplant failure rates are even higher, with 8%, 27%, and 53% of these transplants failing at 1, 5, and 10 years, respectively (https://www.usrds.org/2016/view/v2_07.aspx). Developing new immunosuppressive therapies that can reduce long-term kidney transplant failure will ensure that fewer patients receive multiple transplants, and that more patients on the waiting list can receive a kidney.

“As demonstrated in other therapeutic areas of unmet need, the use of standardized datasets from a broad number of stakeholders informs the development of biomarkers, outcome assessment measures, and other novel tools that regulatory authorities may endorse. Ultimately, the incorporation of these new methods and tools into drug development programs will increase the likelihood of success in getting safe and effective therapies to transplantation patients,” said C-Path President and CEO Dr. Martha Brumfield.

The three areas of focus for TTC include: (1) translating immunosuppressive therapies from other therapeutic areas to transplantation, (2) evaluating a number of candidate biomarkers for kidney transplant failure, with the objective of identifying those most likely to inform innovative clinical trial design and seeking regulatory endorsement, and (3) exploring methodologies that are capable of effectively incorporating the voice of the patient into clinical trial design. The sharing of knowledge, expertise, and data will be critical to the success of each of these TTC goals.

“Due to the number of patients waiting for a kidney, the initial goal of the TTC will be to focus on kidney transplantation. The TTC will aim to improve outcomes for kidney transplant recipients, lower kidney transplant failure risks, and reduce the need for multiple transplants. The hope is to expand to other solid organ transplantation in the future,” said AST President Anil Chandraker, MD.

“The challenge of introducing new immunosuppressive therapies for the transplant population is becoming increasingly difficult. Severity of illnesses and underlying comorbidities, and limited numbers of patients with endpoints that are in continual evolution, makes the design, administration, and regulation of clinical trials increasingly difficult. It is our hope that the TTC will address these issues at their core and reduce barriers for the effective and safe use of new agents that benefit our patients,” said Timothy L. Pruett, MD, President of ASTS.

About the Organizations:

Critical Path Institute (C-Path) ) is an independent, nonprofit organization established in 2005 with public and private philanthropic support from the Arizona community, Science Foundation Arizona, and the US Food and Drug Administration (FDA). C-Path’s mission is to catalyze the development of new approaches that advance medical innovation and regulatory science, accelerating the path to a healthier world. An international leader in forming collaborations, C-Path has established 14 global, public-private partnerships that currently include over 1,450 scientists from government and regulatory agencies, academia, patient advocacy organizations, and dozens of major pharmaceutical companies. C-Path is headquartered in Tucson, Arizona. For more information, visit www.c-path.org.

The American Society of Transplantation (AST) is an organization of more than 3,500 professionals dedicated to advancing the field of transplantation and improving patient care by promoting research, education, advocacy, and organ donation. The society is the largest organization of transplant professionals in North America. AST members are sought out as transplant experts and advocates. Other transplant organizations, policymakers, regulatory agencies, payors, academic institutions, and the general public look to the AST for guidance, research, and resources related to transplantation.

The American Society of Transplant Surgeons (ASTS) represents approximately 1,800 professionals dedicated to excellence in transplantation surgery. Our mission is to advance the art and science of transplant surgery through leadership, advocacy, education, and training.

C-Path Contact:
Kissy Black
+1.615.298.1144
[email protected]

April 18, 2017

 

CDISC Announces New Standard for Major Depressive Disorder to Foster Development of New Treatments

Austin, TX – 18 April 2017 – The Clinical Data Interchange Standards Consortium (CDISC) today announced the release of Major Depressive Disorder (MDD) Therapeutic Area User Guide v1.0. The MDD standard is freely available on the CDISC website. Therapeutic Area User Guides provide research professionals with guidance on how to represent patient data in clinical research to improve quality, efficiency and cost effectiveness, enabling smarter research to unlock cures.

According to the World Health Organization, an estimated 350 million people of all ages suffer from depression globally. Depression is the leading cause of disability worldwide, and is a major contributor to the overall global burden of disease.

“We congratulate the team on the release of the Major Depressive Disorder (MDD) Therapeutic Area User Guide! Including MDD, a record number of 10 Therapeutic Area User Guides were released in 2016. We would like to recognize the tremendous effort put forth by all CDISC standards development teams and thank them for their dedication throughout the year. We are making great strides in increasing the breadth of clinical data standards available to support research,” said Brooke Hinkson, Head, Global Clinical Data Standards at Merck and lead of the TransCelerate BioPharma (“TransCelerate”) Clinical Data Standards initiative.

This CDISC Therapeutic Area standard was developed through the Coalition For Accelerating Standards and Therapies (CFAST) initiative, a partnership of CDISC and the Critical Path Institute (C-Path), with participation from the U.S. NIH National Cancer Institute Enterprise Vocabulary Services (NCI-EVS), the U.S. Food and Drug Administration (FDA), TransCelerate, the Japan Pharmaceutical and Medical Devices Agency (PMDA) and other stakeholders to accelerate clinical research and medical product development by creating and maintaining data standards, tools and methods for conducting research in therapeutic areas that are important to public health.

CDISC standards have been adopted and used in more than 90 countries, and are required for submissions to the U.S. FDA and Japan PMDA. To date, Therapeutic Area standards have been developed for over 25 disease areas. CDISC standards make it possible for data to speak the same language, empowering data collection and sharing that makes the most of the valuable information offered by patients participating in research studies around the globe.

About CDISC

CDISC is a 501(c)(3) global, non-profit organization that develops data standards to foster smarter research and enable connections to healthcare. CDISC standards allow data to speak the same language, by providing common formats for data collection, data sharing and data analyses to make the most of the valuable information offered by patients participating in research studies around the globe, enabling researchers to discover new treatments, find breakthroughs, and unlock cures. CDISC standards are required for regulatory submissions to the U.S. FDA and Japan PMDA, are endorsed by the China CFDA, and are requested for use by the European Innovative Medicines Initiative (IMI). The suite of CDISC standards is freely available on the CDISC website.

CDISC is funded through the generous support of over 400 member organizations from pharmaceutical, biotech, clinical research organizations, regulatory agencies, academia, and healthcare, as well as through grants, authorized CDISC Education courses, events and charitable contributions. To find out more about how to support CDISC and get involved, please visit www.cdisc.org and www.unlockcures.org.

CDISC Contact:
Andrea Vadakin
+1.316.558.0160
[email protected]

April  11, 2017

On March 24, the world will recognize the 35th annual observance of World Tuberculosis (TB) Day. The leading cause of death by an infectious disease, TB kills 1.8 million people per year, according to the World Health Organization (WHO). Even more worrying is the increase in the number of strains of the bacteria Mycobacterium tuberculosis that are resistant to the highly potent drugs used to treat TB. For the past 25 years, the WHO has warned of the rise in drug-resistant TB, and reports of multidrug resistance are now found in nearly every country along with the emergence of extremely drug resistant (XDR) TB.

For most of us, the word “tuberculosis” is often forgotten, since the number of cases is minimized due to public health and surveillance efforts afforded to living in the developed world. TB, which was also known as “consumption,” conjures up images of pre-depression hospital wards, featuring endless rows of beds filled with sick and forlorn patients staring out windows toward gray skies. These preconceptions are understandable for those who have lived their whole lives with access to the luxury of modern medicine, making it easy to forget that tuberculosis is still one of the leading causes of death worldwide.

One Arizona organization, however, is championing a worldwide partnership that seeks to address the complex challenges in treating TB and ending the global epidemic. The Critical Path to TB Drug Regimens (CPTR)–a consortium within the larger Tucson-based nonprofit the Critical Path Institute (C-Path)– is waging the global fight against tuberculosis through collaboration with TB research organizations, academia, and the pharmaceutical industry. CPTR uses global data aggregation and standardization to understand the genetic basis of drug resistance, and advanced modeling and simulation approaches to develop tools that lead to better drugs and drug regimens to treat TB.

But C-Path is not the first Arizona-based organization to be at the forefront of TB diagnosis and treatment. Just down the road lies the Tucson Medical Center, whose roots extend back nearly a century, to a time when experimental TB treatment was shaping the future of the American Southwest.

At the turn of the 20th century, tuberculosis was killing one out of seven people in North America and Europe. Cramped, heavily populated cities along the Eastern seaboard served as an ideal environment for the deadly and highly infectious bacteria to spread from person to person, with no regard for age, race, or social status. Though it would be years until the first successful antibiotic was developed, physicians of the time had noted the benefits of dry, fresh air and a warm, sunny climate.

By the 1920s, those who were fortunate enough to do so flocked to the “premier destination” for tuberculosis patients. That destination was Southern Arizona. The resort-like sanatoriums of sunny Tucson offered both isolation from the general public, as well as the best care that could be offered to those facing the grim reality of living with a TB diagnosis. The sharp influx in population resulting from the surge of ailing immigrants shaped the city itself, heavily influencing the original layout of the settlement and its building designs. At one point there were more than 30 sanatoriums in use within the city of Tucson alone.

One such destination for TB patients was the internationally known Desert Sanatorium. The historic “Desert San” provided an ideal environment for those suffering from tuberculosis to have the best chance at recovery in an age when the likelihood of survival wasn’t much better than a flip of a coin. Time passed, and by the early 1940s the world was engulfed in war. The Desert Sanatorium found itself stripped of critical staff and resources, eventually closing its doors in 1943. They wouldn’t remain closed for long, however. The Tucson community rallied to raise the necessary funds to convert the local landmark into a brand-new hospital — the Tucson Medical Center.

“In the 1920s a tuberculosis treatment facility was established in Tucson to provide comfort, therapy and healing,” says Mimi Warwick Coomler, Vice President and Chief Nursing Officer of Tucson Medical Center. “This site and these efforts would soon become Tucson Medical Center. Some of the original buildings remain in service today, as a testament to the importance and impact of a community’s strength and commitment to eradicating a disease that continues to this day.”

Today, the locally governed nonprofit hospital has been providing first-rate medical care for its community members for over 70 years. Though the Tucson Medical Center now provides a multitude of health care programs, from maternity to hospice services, the landmark hospital has not forgotten its origins as a leading name in tuberculosis therapy so many years ago.

Although TB is far from the disease it once was, a rise in drug resistance is worrying public health officials on a global scale. Reports within the last 10 years have shown some bacteria to be resistant to all anti-TB drugs, which raises the real possibility of untreatable TB disease, bringing back the haunting image of an overcrowded hospital ward. With stronger and more deadly drug-resistant strains killing thousands each day, an entirely modern, global solution is necessary. Thankfully, organizations such as C-Path are continuing the work that was started so long ago by the Tucson Medical Center. Leading the way in global TB diagnostics and drug development, C-Path’s CPTR initiative is working with a global coalition of leading international pharmaceutical companies, public health experts, and regulatory authorities to bring the best TB care to those who need it most in the developing world.

Tuberculosis is not a disease of the past, but rather a leading cause of death across the globe. Lessons from the past, highlighted by the impactful history of Tucson Medical Center, should be a reminder of the consequences of inaction. Though there is still much to be done to prevent the loss of life resulting from the devastating disease, by looking to the past successes achieved by institutions such as the Tucson Medical Center, initiatives such as C-Path’s CPTR can hopefully, one day, celebrate a global victory over tuberculosis on the 24th of March.

C-Path Contact:
Kissy Black
615.298.1144
[email protected]