Parkinson’s UK Announces Major Pharma Companies Sign Up to CPP

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Major pharma companies sign up to groundbreaking Parkinson’s consortium

Seven of the world’s largest pharmaceutical companies have signed up to a groundbreaking consortium aimed at accelerating the development of safe and effective therapies for Parkinson’s.

The Critical Path for Parkinson’s consortium will bring together leading academics; industry members AbbVie, AstraZeneca, Biogen, Eli Lilly and Company, Merck Sharp and Dohme (known as Merck & Co. Inc. in the United States and Canada), Pfizer, and UCB; and founders the Critical Path Institute (C-Path) and Parkinson’s UK, to share data, expertise and resources to promote and develop new treatments for Parkinson’s.

The consortium, which was launched in October 2015, was formed by Parkinson’s UK and C-Path to increase investment into research and development of new Parkinson’s treatments.

Dr Arthur Roach, Director of Research at Parkinson’s UK, the principal funder of the consortium, says:

“Despite significant advances in our understanding of the genetics, biochemistry and pathology of Parkinson’s, the development of new treatments has not kept pace. New treatments are desperately needed to deal with the devastating effects of this progressive condition.

“Investing in clinical trials for brain disorders currently carries a high cost and high risk of failure. As the world’s largest patient-led Parkinson’s charity, we know that people living with conditions such as Parkinson’s have often been disappointed when drugs that showed significant promise early on failed in late stage testing.

“We see the consortium as a crucial part of strategies to develop new treatments that work at the earliest stage of the condition, with the goal of slowing its progression, and eventually finding a cure.”

Parkinson’s affects 127,000 people in the United Kingdom, and approximately 7 million people worldwide. In 2012/13, the National Health Service (NHS) spent more than £212 million on caring for people with Parkinson’s in England (1), with the cost of the condition in the United States approximately US$25 billion per year (2). With the baby boomer generation becoming older, the number of people with neurodegenerative conditions, including Parkinson’s, is likely to increase and will become one of the biggest issues faced by healthcare.
The International Federation of Pharmaceutical Manufacturers & Associations (IFPMA) estimated that the cost of developing a single drug was US$1.3 billion, rising from US$138 million in 1978 (3), a tenfold increase attributed to a multiplicity of factors faced by drug research.

Diane Stephenson, PhD, Executive Director, Critical Path for Parkinson’s consortium, C- Path, says:

“With the increase in the costs of getting a drug to market, the design of a clinical trial is a crucial part of a drug’s success. There is a strong realisation from the industry that collaboration among industry, academia, and worldwide regulatory agencies, along with the sharing of data, has the potential to create a more efficient development process. This recognition is evidenced by the fast pace at which members of this new consortium have joined.”

Parkinson’s UK has committed over £1million to the Critical Path for Parkinson’s consortium.

For more information, and to donate to Parkinson’s UK research and the work with the Critical Path Institute, visit http://parkinsons.org.uk/research


 

Media enquiries

Please contact: Hanna Kilpin, Senior Media & PR Officer on 020 7963 9311 or email: hkilpin@parkinsons.org.uk

Out of hours: 07961 460248 Notes to editors

  1. Parkinson’s and the NHS in England: the cost of poor care. Parkinson’s UK. October 2013.
  2. Statistics on Parkinson’s. Parkinson’s Disease Foundation.
  3. The Pharmaceutical Industry and Global Health: Facts and Figures 2015.
    International Federation of Pharmaceutical Manufacturers & Associations.

 

About Parkinson’s UK

Every hour, someone in the UK is told they have Parkinson’s.

It affects 127,000 people in the UK – which is around one in 500 of the population.

Parkinson’s is a degenerative neurological condition, for which there currently is no cure. The main symptoms of the condition are tremor, slowness of movement and rigidity.

Parkinson’s UK is the UK’s leading charity supporting those with the condition. Its mission is to find a cure and improve life for everyone affected by Parkinson’s through cutting edge research, information, support and campaigning.
For advice, information and support, visit http://www.parkinsons.org.uk or call our free, confidential helpline on 0808 800 0303.

C-Path’s Polycystic Kidney Disease Outcomes Consortium Secures EMA Qualification Opinion for Enrichment Biomarker in ADPKD

November 13, 2015

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C-Path’s Polycystic Kidney Disease Outcomes Consortium Secures EMA Qualification Opinion for Enrichment Biomarker in ADPKD

TUCSON, Ariz., November 13, 2015 – The Critical Path Institute (C-Path) announced today that the European Medicines Agency (EMA) rendered a positive qualification opinion to C-Path’s Polycystic Kidney Disease Outcomes Consortium (PKDOC) for total kidney volume (TKV) as a prognostic biomarker to select patients for clinical trials of new therapies for Autosomal Dominant Polycystic Kidney Disease (ADPKD).

ADPKD is a debilitating genetic disease affecting approximately 600,000 Americans and 12 million people worldwide. There is only one medication developed to treat ADPKD, called tolvaptan, which has been approved in Europe, Japan, and Canada, but has not yet been approved in the United States.

TKV is a measurement of the impact of ADPKD on the size of the kidneys and is considered to be predictive of a future decline in kidney function. The EMA opinion states, “CHMP [Committee for Medicinal Products for Human Use] supports baseline total kidney volume, in combination with patient age and eGFR [estimated glomerular filtration rate] as a prognostic biomarker to identify patients likely to experience a progressive decline in renal function, as characterized by a decline in eGFR or progression to end-stage renal disease.

“From the data provided it is reasonable to expect that baseline TKV can predict disease progression and is a biomarker valuable for risk stratification.”

”This qualification, along with a similar one from FDA, confirms the relationship between TKV and ADPKD disease progression, and will help in the design of clinical trials for new therapies for ADPKD” says C-Path Chief Operating Officer and PKDOC Co-Director Steve Broadbent.

The PKDOC created a Clinical Data Interchange Standards Consortium (CDISC) data standard for ADPKD and used it to remap the data from several patient registries and observational studies. The database was then used to develop a joint model linking baseline TKV with clinical outcomes.

About PKDOC:
The PKDOC is a successful collaboration between C-Path, the PKD Foundation, CDISC, four leading academic medical centers (Tufts University, University of Colorado Denver, Emory University, and Mayo Clinic), and three pharmaceutical companies. Its mission is to develop tools and promote research that will lead to the discovery of treatments for PKD and improve the lives of all it affects. The consortium is led by C-Path and funded through a grant from the PKD Foundation and philanthropic donations.

About the organizations:

C-Path (Critical Path Institute) 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 12 global, public-private partnerships that currently include over 1,300 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 PKD Foundation is the only organization in the United States solely dedicated to finding treatments and a cure for polycystic kidney disease (PKD) to improve the lives of those it affects. This is done through promoting programs of research, education, advocacy, support, and awareness on a national level, along with direct services in local communities across the country. Their vision is that one day no one will suffer the full effects of PKD. Visit pkdcure.org to learn more about PKD and the Foundation.

C-Path Contact:
 Kissy Black
+1.615.298.1144
kissyblack@lotosnile.com
PKD Foundation Contact:
 Angela Connelly
816.931.2600 ext. 212
angelac@pkdcure.org

 

Parkinson’s UK Receives CAMD Nonprofit Organization Award

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Dr. Arthur Roach, Director of Research at Parkinson’s UK accepts the first Nonprofit Organization Award from CAMD presented by Dr. Diane Stephenson, Critical Path Institute, at the annual meeting held at FDA on Oct 20th, 2015. Parkinson’s UK is recognized for their leadership in supporting the launch of a fully dedicated consortium aimed at integrating patient level data from Parkinson’s disease patients around the world.

Parkinson’s UK and Critical Path Institute Launch the Critical Path for Parkinson’s (CPP) Consortium

October 14, 2015
CPP

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Parkinson’s UK and Critical Path Institute Launch the Critical Path for Parkinson’s (CPP) Consortium

Data from past Parkinson’s trials will be collected, standardized, and integrated, in order to inform new approaches to designing clinical trials focused on early-stage Parkinson’s.

London and Tucson – October 14, 2015 – Parkinson’s UK, one of the world’s largest charity funders of Parkinson’s research, and Critical Path Institute (C-Path), a pioneering nonprofit organization dedicated to accelerating the pace and reducing the costs of medical product development, announced the formation of the Critical Path for Parkinson’s Consortium (CPP).

The consortium will bring together pharmaceutical companies and academic partners working toward a common goal of establishing best practices and more efficient protocols for planning and designing clinical trials in early Parkinson’s—ultimately improving the efficiency and success rates of much-needed treatments for people with Parkinson’s.

Parkinson’s UK has been a leader in bringing together many participants in the Parkinson’s research enterprise to find ways to share data and work collaboratively. Together, Parkinson’s UK and C-Path can maximize the benefit of data-sharing, which is critical for a condition such as Parkinson’s, for which there are no effective treatments.

“In the era of electronic knowledge exchange, only when data-sharing becomes the norm can we derive its full benefits,” says Sue Dubman, UCSF Cancer Bio-informatics Director, patient advocate, and Parkinson’s patient.

Parkinson’s UK Chief Executive Steve Ford says, “Despite huge scientific progress, there have been no major advances in the treatments available for Parkinson’s in the last decade.

“At Parkinson’s UK, we’re adopting a new approach: tackling the critical road blocks standing in the way of the scientific breakthroughs that could have a huge impact on the lives of people with Parkinson’s.

“It’s our goal that the partnership with the Critical Path Institute will improve the clinical trial process and deliver new and better treatments, faster, to the people who urgently need them.”

“The importance of the role of patient advocacy organizations, such as Parkinson’s UK, in making new innovations possible for patients has never been more critical. The partnership between C-Path and Parkinson’s UK will complement each organization’s strengths. With the extensive reach and data available from Parkinson’s UK, and C-Path’s scientifically rigorous approach to data standardization and clinical trial modeling and simulation, the potential value of this effort, both to those who develop treatments for Parkinson’s, and those who live with the condition, cannot be underestimated,” says Martha A. Brumfield, President and Chief Executive Officer of C-Path.

The new consortium will be co-directed by Dr. Arthur Roach (Director of Research, Parkinson’s UK) and Dr. Diane Stephenson (Executive Director, Critical Path for Parkinson’s Consortium , C-Path).

 

ABOUT PARKINSON’S:

Parkinson’s is a progressive neurological condition. Traditionally, Parkinson’s has been viewed as a disorder in which individuals don’t have enough of a chemical called dopamine because specific nerve cells inside their brain have died. Low levels of dopamine slow the body’s movement, which makes everyday activities, such as eating, getting dressed, or using a phone or computer difficult or frustrating. It is not known why these cells die, but current research indicates that the processes that lead to dopamine deficit start much earlier (decades), making it a pressing need to increase the understanding of the condition’s progression. The main symptoms of Parkinson’s are tremor, muscle stiffness, and slowness of movement. But not everyone will experience all of these. As well as the symptoms that affect movement, other issues, such as tiredness, pain, and depression impact daily life. Parkinson’s doesn’t directly cause people to die, but symptoms do get worse over time, which greatly impacts the ability of individuals to function, leading to a severe decrease in quality of life.

ABOUT PARKINSON’S UK:

Every hour, someone in the UK is told they have Parkinson’s.

It affects 127,000 people in the UK, which is around one in 500 of the population.

Parkinson’s is a degenerative neurological condition, for which there currently is no cure. The main symptoms of the condition are tremor, slowness of movement and rigidity.

Parkinson’s UK is the UK’s leading charity supporting those with the condition. Its mission is to find a cure and improve life for everyone affected by Parkinson’s through cutting edge research, information, support and campaigning.

For advice, information and support, visit www.parkinsons.org.uk.

ABOUT CRITICAL PATH INSTITUTE (C-Path):

Established in 2005 as a nonprofit organization, C-Path was formed to deliver on the vision of FDA’s Critical Path Initiative. C-Path is committed to improving human health and well-being by developing new technologies and methods to accelerate the development and review of medical products. An international leader in forming collaborations around this mission, C-Path has established twelve global, public-private partnerships that include more than 1,000 scientists from regulatory and other government agencies, academia, patient advocacy organizations, and 41 major pharmaceutical companies. C-Path has headquarters in Tucson, AZ, and has been funded by public and private philanthropic support from the University of Arizona, Science Foundation Arizona (SFAz), the US Food and Drug Administration (FDA), and the Tucson community. For more information, visit www.c-path.org.

C-Path Contact:
 Kissy Black
+1.615.298.1144
kissyblack@lotosnile.com

 

PD Imaging Biomarker Team at European Medicines Agency (EMA)

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Experts of the Parkinson’s disease imaging biomarker team participated in a Scientific Advice meeting at the European Medicines Agency on October 7th 2015. The purpose of this meeting was to review the evidence provided to the Agency that supports the use of Molecular Imaging of the Dopamine Transporter as an enrichment biomarker in clinical trials targeting subjects at first signs and symptoms of PD. Team members from left: Steve Arneric, C-Path; Derek Hill, Ixico; Klaus Romero, C-Path; Patricia Cole, Takeda; Arthur Roach, Parkinson’s UK; Ken Marek, MNI; Diane Stephenson, C-Path; Dawn Matthews, ADm Diagnostics; Mark Gordon, Boerhinger Ingelheim.

Critical Path Institute Secures Regulatory Support For Skeletal Muscle Safety Biomarkers

April 13, 2015
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Critical Path Institute Secures Regulatory Support For Skeletal Muscle Safety Biomarkers

FDA and EMA Letters of Support Suggest Valuable Research Potential

TUCSON, Ariz., April 8, 2015 – The Critical Path Institute (C-Path) announced today that both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have issued Biomarker Letters of Support for four skeletal muscle safety biomarkers identified and evaluated by C-Path’s Predictive Safety Testing Consortium (PSTC)’s Skeletal Myopathy Working Group. This is the second time the two agencies have provided a Biomarker Letter of Support to PSTC in the past six months, following FDA and EMA support for the kidney safety biomarkers osteopontin (OPN) and neutrophil gelatinase-associated lipocalin (NGAL).

“This Letter of Support intends to encourage scientists to collect data from exploratory studies, which may lead to qualification of these types of biomarkers,” said Janet Woodcock, M.D., director of the FDA’s Center for Drug Evaluation and Research. “We are optimistic about how this effort will further advance biomarker development.”

The skeletal safety biomarkers – myosin light chain (Myl3), skeletal muscle troponin I (sTnI), fatty acid binding protein 3 (FABP3), and creatine kinase muscle type (CK-M, the homodimer CK-MM) – are proteins that can be measured in plasma or serum. Higher levels of these biomarkers could indicate that skeletal muscle injury is occurring.

“The development of accurate, reliable biomarkers continues to be one of the most productive methods of aligning and streamlining the research and regulatory processes,” says C-Path President and CEO Martha A. Brumfield, PhD. “The support of these skeletal muscle safety indicators by the FDA and EMA is an encouraging development in the PSTC’s larger mission of facilitating scientific validation and regulatory qualification of novel safety biomarkers.”

Each agency’s Letter of Support for Myl3, sTnI, FABP3, and CK-M is intended to encourage the biomarkers’ use in both nonclinical and exploratory clinical studies as markers of skeletal muscle injury. Combined FDA and EMA support indicates that these biomarkers have strong potential for use in humans and warrants additional exploration and gathering of data. With this milestone, work will continue in earnest on the qualification of Myl3, sTnI, FABP3, and CK-M for use in clinical trials.

Letters of Support for these skeletal muscle biomarkers are posted on the FDA DDT website and the EMA website, and can also be accessed via the C-Path PSTC website, along with a summary data package describing the studies that support the use of these kidney safety biomarkers.



About the Critical Path Institute

The Critical Path Institute (C-Path) is an independent, non-profit organization established in 2005 with public and private philanthropic support from the Arizona community, Science Foundation Arizona, and the U.S. 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 seven global, public-private partnerships that currently include over 1,000 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 http://www.c-path.org.

C-Path Contact:
 Kissy Black
+1.615.298.1144
kissyblack@lotosnile.com

 

Critical Path Institute Secures Additional Regulatory Support For Kidney Safety Biomarkers

January 12, 2015

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Critical Path Institute Secures Additional Regulatory Support For Kidney Safety Biomarkers

EMA Letter of Support Opens Door for Clinical Qualification

TUCSON, Ariz., January 12, 2015 – The Critical Path Institute (C-Path) announced today that the European Medicines Agency (EMA) issued a first-of-its kind Biomarker Letter of Support for two essential kidney safety biomarkers identified and evaluated by the Predictive Safety Testing Consortium (PSTC)’s Nephrotoxicity Working Group.

The kidney safety biomarkers, osteopontin (OPN) and neutrophil gelatinase-associated lipocalin (NGAL), are proteins that can be measured in urine. Higher levels of OPN and NGAL could indicate that the kidneys are being damaged, which may result in a loss of kidney function. The Letter of Support intends to encourage scientists to collect data from nonclinical and exploratory clinical studies, which may lead to qualification of these biomarkers.

“We are pleased to continue working collaboratively with the EMA, academia, and industry to identify tools, processes, and methods to improve the drug development process,” said Martha Brumfield, Ph.D., president and chief executive officer of C-Path. “With this Letter of Support, the EMA has opened doors that encourage the generation of necessary, rigorous clinical data to determine if these biomarkers hold clinical utility.”

The EMA’s Letter of Support indicates that these biomarkers have strong potential for use in humans and warrant additional exploration and data gathering. The EMA granted the Letter of Support for OPN and NGAL to encourage their use in both nonclinical and exploratory clinical studies as markers of proximal renal tubule degeneration/necrosis. With this milestone, work will continue in earnest on the qualification of OPN and NGAL for use in clinical trials.

“We are all incredibly excited about our collaborative interactions with EMA and the creation of the Letter of Support, enabling us to further define the path to clinical biomarker qualification,” said John Michael Sauer, Ph.D., Executive Director, Predictive Safety Testing Consortium, Critical Path Institute.

The Letter of Support is posted on the EMA website and can also be accessed via the C-Path PSTC website under the Regulatory Successes tab, along with a summary data package describing the studies that support the use of these kidney safety biomarkers.

About the Critical Path Institute

The Critical Path Institute (C-Path) is an independent, non-profit organization established in 2005 with public and private philanthropic support from the Arizona community, Science Foundation Arizona, and the U.S. 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 seven global, public-private partnerships that currently include over 1,000 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 http://www.c-path.org.

C-Path and IMI 2nd Annual Meeting

critical_path_institute_no_tag  IMI-logo

Accelerating the Development of Drugs, Diagnostics, and Devices: Partnerships to Expand the Precompetitive Space 

Wednesday, December 3, 2014
Bethesda, MD, USA

On December 3, 2014, the Critical Path Institute (C-Path) and Innovative Medicines Initiative (IMI) convened an international group of thought leaders to identify new ways to collaborate and achieve a common goal of a robust regulatory science infrastructure that better supports efficient and productive medical product development.

The meeting, Accelerating the Development of Drugs, Diagnostics and Devices: Partnerships to Expand the Precompetitive Space was the second annual meeting of C-Path and IMI, and explored key topics around public-private partnerships (PPPs) to advance regulatory science, data-sharing and patient safety biomarkers.

The organizations explored lessons learned from the PPP model and discussed the importance of leveraging resources and avoiding duplication of efforts. IMI and C-Path have been working together and sharing best practices for many years now, and, as IMI Executive Director Professor Michel Goldman characterized it, the event was “an excellent opportunity to discuss how open collaboration models can contribute to solving medicine’s greatest challenges.”

The ideas, goals, and progress discussed during the conference were subsequently featured in the January 15, 2015 issue of Biocentury Innovations in an article entitled “Consortium Crosstalk.”

Agenda Event videos
8:45-9:00 am
Welcome with Martha Brumfield (C-­Path) and Michel Goldman (IMI)

Martha Brumfield
(C-Path)

Michel Goldman
(IMI)

9:00-11:00 am
Session 1: Partnerships to Advance Regulatory Science and Leverage Global Biopharmaceutical DevelopmentCo-chair/Moderators: Martha Brumfield (C-­Path) and Michel Goldman (IMI)Panelist: Janet Woodcock (FDA), Dalvir Gill (TransCelerate BioPharma Inc), David Wholley (FNIH), William Chin (PhRMA)Panel Discussion Topics:

  • What have partnerships produced that could not have been accomplished by a single organization?
  • What metrics should be applied to evaluating partnerships?
  • What are the key challenges facing today’s partnerships, and how can those challenges be optimally addressed?
  • What factors should be considered when partners prioritize projects?
  • How can newly formed partnerships leverage ongoing efforts of established partnerships?
  • How to ensure coordination?

Session 1 –
Introduction

Janet Woodcock
(FDA)

Dalvir Gill
(TransCelerate
BioPharma Inc)

David Wholley
(FNIH)

William Chin
(PhRMA)

Session 1 –
Panel Discussion

1:30-1:00 pm
Session 2: Safety Biomarkers: The PSTC and SAFE-­T CollaborationCo-chair/Moderators: John-­Michael Sauer (C-­Path) and Michael Lawton (Pfizer)Panelist: Denise Robinson-­Gravatt (formerly Pfizer), Douglas Keller (Sanofi), John-­Michael Sauer (C-­Path), Michael Lawton (Pfizer), Ameeta Parekh (FDA), Maria Teresa De Magistris (IMI SAFE-­T), Frank D. Sistare (Merck), Thorsten Vetter (EMA)Panel Presentations:

  • The past: Key lessons learned from the SAFE-­T/PSTC collaboration Denise Robinson-­Gravatt (Pfizer)
  • The present: benefits from the ongoing collaboration; preclinical and clinical qualification of markers for BSEP inhibition Douglas Keller (Sanofi)
  • The future: How to build on a successful collaboration John-­Michael Sauer (C-­Path) and Michael Lawton (Pfizer)

Expert Opinion: ShaAvhrée Buckman-­Garner (FDA)

Panel Discussion Topics:

  • How has the SAFE-­T/PSTC collaboration been set up?
  • What has been achieved through this collaboration (e.g. Strategic benefits: capitalizing on synergies and improving regulatory interactions, a real-­life example: preclinical and clinical qualification approaches of markers for BSEP inhibition)
  • What are the key lessons learned from the SAFE-­T/PSTC collaboration?
  • What were major obstacles in setting up the collaborative agreement?
  • What is needed to be more efficient in the future?
  • How to build on a successful collaboration and what areas would benefit from more collaboration

Session 2 –
Introduction

Denise
Robinson-­Gravatt

(formerly Pfizer)

Douglas Keller
(Sanofi)

John-­Michael Sauer
(C-­Path)

Michael Lawton
(Pfizer)

ShaAvhrée
Buckman-­Garner

(FDA)

Session 2 –
Panel Discussion

2:00-4:00 pm
Session 3: Maximizing the value of data shared by multiple organizationsCo-chair/Moderators: Enrique Avilés (C-­Path) and Ann Martin (IMI)Panelist: Sharon Hesterlee (PPMD), Kald Abdallah (Project DataSphere), Ed Bowen (Pfizer, TransCelerate BioPharma Inc.), Keith Elliston (tranSMART Foundation), Bron Kisler (CDISC), Mary Ann Slack (FDA)Panel Discussion Topics:

  • What are the best methods to maximize the research utility of data contributed by multiple organizations to a collaborative effort?
  • Success stories in analyzing & pooling data to yield new insights & tools
  • Harmonization of approaches to data sharing/aggregating of data
  • How to increase collaboration across existing data sharing initiatives
  • Integrating Electronic Health Records into Clinical Trial databases

Session 3 –
Introduction

Sharon Hesterlee
(PPMD)

Kald Abdallah
(Project DataSphere)

Ed Bowen
(Pfizer,
TransCelerate
BioPharma Inc.)

Keith Elliston
(tranSMART
Foundation)

Bron Kisler
(CDISC)

Mary Ann Slack
(FDA)

Session 3 –
Panel Discussion

4:00-4:30 pm
Closing Remarks: Identification of key next steps Michel Goldman and Martha Brumfield

Martha Brumfield
(C-Path)

Michel Goldman
(IMI)

Conference Highlights Public Private Partnership Role in Medical Product Development

December 3, 2014 | Download PDF

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Conference Highlights Public Private Partnership Role in Medical Product Development

Global organizations convene cross-sector thought leaders to discuss public-private partnerships


BETHESDA, MD, December 3, 2014—Today, the Critical Path Institute (C-Path) and Innovative Medicines Initiative (IMI) are convening an international group of thought leaders to identify new ways to collaborate and achieve a common goal of a robust regulatory science infrastructure that better supports efficient and productive medical product development. Accelerating the Development of Drugs, Diagnostics and Devices: Partnerships to Expand the Precompetitive Space is the second annual meeting of C-Path and IMI, and will explore key topics around partnerships to advance regulatory science, data-sharing and patient safety biomarkers.

“We are proud to partner with our colleagues at IMI to host a discussion on the role of public-private partnerships,” said Martha Brumfield, Ph.D., president and chief executive officer of C-Path. “No single entity has adequate resources, experience and data to tackle the complex issues in disease areas such as Alzheimer’s, Parkinson’s, schizophrenia, and diabetes. Global, collaborative efforts, along with the early engagement of regulatory authorities, advance innovation in these and other areas of unmet need.”

Public-private partnerships (PPPs) are at the forefront of a cross-sector effort to transform the expensive, time-consuming, high-risk and complicated process that delivers new treatment options. In its second annual conference, the two organizations will explore lessons learned with the PPP model and identify additional ways to leverage resources and avoid duplication of efforts.

“Collaboration is essential if we are to solve the biggest challenges in medicines development and to ensure patients gain rapid access to innovative therapies,” said Michel Goldman, M.D., Ph.D., executive director of IMI. “IMI and C-Path have been working together and sharing best practices for many years now, and this event represents an excellent opportunity to discuss how open collaboration models can contribute to solving medicine’s greatest challenges.”

Three separate panel discussions, moderated by C-Path and IMI, will draw on the perspective of government officials, researchers, regulators, and pharmaceutical industry representatives. The sessions include:

  • Partnerships to Advance Regulatory Science and Leverage Global Biopharmaceutical Development will feature a discussion between Janet Woodcock of the U.S. Food and Drug Administration (FDA), Dalvir Gill of TransCelerate BioPharma, William Chin of Pharmaceutical Research and Manufacturers of America (PhRMA) and David Wholley of the Foundation for the National Institutes of Health (FNIH).
  • In session two, Safety Biomarkers: The Predictive Safety Testing Consortium (PSTC) and Safer and Faster Evidence-based Translation (SAFE-T) Collaboration conversation, John-Michael Sauer of C-Path and Michael Merz of Novartis will moderate a discussion with representatives from Merck, Sanofi, the FDA and European Medicines Agency.
  • The third panel, Maximizing the Value of Data Shared by Multiple Organizations, brings together representatives from non-profit organizations and government to discuss ways to maximize the research utility of data.

PPPs have been successfully used to create new data standards and to identify biomarkers in several therapeutic areas, including pressing public health concerns such as Alzheimer’s disease, tuberculosis and kidney safety. “These models are the future of drug development and while significant progress has been made, today’s conversations will certainly push us forward,” said Brumfield.

Michel Goldman added, “Public-private partnerships are now an established feature of the drug development landscape. Today’s meeting will allow us to review what PPPs can do and how we can do even more to contribute to the development of the medicines of the future, for the benefit of patients and society.”



Notes to Editors

The event will be held at the Hyatt Regency Bethesda, One Bethesda Metro Center, 7400 Wisconsin Avenue, Bethesda, MD 20814. It will also be possible to view the slides and hear the event audio via WebEx: https://pstcevent.webex.com/pstcevent/onstage/g.php?MTID=e6c6c0956270c0c4b7c3cc2d0f8ee970e

Password: Cpath

About the Critical Path Institute

The Critical Path Institute (C-Path) is an independent, non-profit organization established in 2005 with public and private philanthropic support from the Arizona community, Science Foundation Arizona, and the U.S. 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 seven global, public-private partnerships that currently include over 1,300 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 and at @cpathinstitute.

About the Innovative Medicines Initiative

The Innovative Medicines Initiative (IMI) is working to improve health by speeding up the development of, and patient access to, innovative medicines, particularly in areas where there is an unmet medical or social need. It does this by facilitating collaboration between the key players involved in healthcare research, including universities, the pharmaceutical and other industries, small and medium-sized enterprises (SMEs), patient organisations, and medicines regulators.

IMI is a partnership between the European Union and the European pharmaceutical industry, represented by the European Federation of Pharmaceutical Industries and Associations (EFPIA). IMI has a budget of €3.3 billion for the period 2014-2024. Half of this comes from the EU’s research and innovation programme, Horizon 2020. The other half comes from large companies, mostly from the pharmaceutical sector; these do not receive any EU funding, but contribute to the projects ‘in kind’, for example by donating their researchers’ time or providing access to research facilities or resources. For more information, visit www.imi.europa.eu and at @IMI_JU.

Contact
C-Path: Catherine Brady at (617) 945-9316
IMI:  Alessandra Paccamiccio at (32) 2 221 8183