• A Collaborative Event

  • Establishing Best Practices

  • Integrating Expanded Access Into Clinical Drug Development

WIDENING THE REACH OF DRUG DEVELOPMENT

WideTrial™ is the leading source of regulatory and business knowledge relating to group-level Expanded Access programs in the U.S.

 

WideTrial™ is the world’s first non-profit platform for sponsoring FDA-authorized Expanded Access programs.

ABOUT

WideTrial is a non-profit company built to make well-designed Expanded Access programs more feasible for those involved in the development of new treatments for serious unsolved diseases.  Our platform offers centralized expertise and advisory services to drug companies, clinical research centers, and disease foundations.  It also offers direct support by way of multi-party collaborations and sponsorship of large-group Expanded Access trials.  WideTrial was founded in 2012 as ALS Emergency Treatment Fund, a 501c3 organization, originally focusing on Expanded Access in neurodegenerative diseases.

WHY EXPANDED ACCESS

Group-level Expanded Access clinical trials (not to be confused with single-patient “compassionate use”) allow meaningful numbers of patients and their physicians to explore a new investigational treatment with a well designed protocol and supply-chain to work in harmony with the continued clinical development of the particular treatment.

When integrated into the drug development cycle, Expanded Access trials bring many benefits to the drug company, including wider patient engagement, increased chances of discovering response-predictive biomarkers, and information that leads to more-highly targeted pivotal trials in historically difficult diseases.

HOW IT WORKS

In each disease area, WideTrial works with leading organizations and pharmaceutical companies to identify trial-stage therapeutics that:

  1. meet the regulatory criteria for Expanded Access and
  2. have disease modifying potential in the opinion of key medical leaders

Qualified trial sites are invited to join WideTrial’s network, to be named as investigators on upcoming access trials.

Under collaboration agreement with each drug company, WideTrial conducts some or all components of the Expanded Access trial, including sponsorship of the regulatory filing, protocol, site coordination, monitoring, data collection, and reporting. Operational and patient assistance funds are provided by charitable sources. Product supply is provided by the drug company.

WideTrial has an experienced, knowledgeable team of Expanded Access experts to guide each program.

“Collaborating with clinics and non-profits to sponsor expanded access trials. Brilliant!”

Nicholas Boulis, MD, Emory University Medical Center, Neurological Surgery

“It's great to see WideTrial emerging to tackle such a critical issue with such a comprehensive solution.”

John Carney, Center for Practical Bioethics

“After an ALS diagnosis, there is no disappointment greater that the news that you don't meet the “inclusion” criteria for a clinical trial, one that just maybe will stall the disease. Access trials are for patients like me!”

Ed Tessaro

“Traditional clinical trials are expensive, but wide access trials may allow us to bring more patients into the drug development process at very little cost. That helps everyone.”

Jonathan Katz, MD, Forbes Norris ALS/MND Research Center

“WideTrial offers a possible way to avoid many of the ethical issues that come with individual patient compassionate use. By offering Expanded Access via a standardized route with the capability of collecting high quality data, WideTrial seems poised to meet the needs of both patients and companies.”

Alison Bateman House, PhD, Division of Medical Ethics, NYU Langone Medical Center

THERAPEUTIC AREAS

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NEURODEGENERATIVE DISEASES

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CANCER

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RARE & GENETIC DISORDERS

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INFECTIOUS DISEASES

GET IN TOUCH

CONTACT US

Email: info@widetrial.org

Phone: 415-637-4774

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NEURODEGENERATIVE DISEASES

Unrelenting, progressively debilitating, and usually fatal.   That’s the hallmark of diseases like Alzheimers, Huntingtons, ALS / MND, and Muscular Dystrophy.   These conditions not only rob patients of their independence, but –due to the lack of accessible treatment options- also rob patients of hope.

That is why neurodegenerative diseases are the initial focus of WideTrial. In fact, we were founded by members of the ALS/MND community, and our original name was ALS Emergency Treatment Fund.

There is tremendous opportunity to use wide access trials to help de-risk drug development in these poorly characterized disorders. By enabling the collection of treatment outcomes data along with consented biosamples from much larger and more diverse sets of patients, single-arm access trials may help identify subpopulations of patients who perform relatively well with the particular drug.

A treatment that appears to have “failed” in a placebo-controlled population-based trial may succeed in a more targeted trial that focuses on patients who have the molecular fingerprints associated with response.   Wide access trials can help find those fingerprints.

If you are working in neurodegenerative diseases and you would like to suggest a particular treatment for an Expanded Access trial, please speak with us.

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CANCER

In the late 1990s and early 2000s a golden age of cancer therapy brought the first selective receptor inhibitor drugs to a landscape of grim medical tradeoffs, especially for chemo-resistant and chemo-sensitive patients.

But, even after the potential of these new drugs was evident, vast sections of the indicated patient populations were excluded from participating in the research trials. Enrollment criteria for these studies, as they should be, were very restrictive in order to ensure the greatest chance of clear efficacy data and a swift FDA approval. Most dying patients did not meet the study criteria.

This is what Expanded Access was made for.

Herceptin, Gleevec, Tykerb, Iressa, Tarceva, Nexavar, and Sutent were each breakthrough cancer therapies, and each enrolled hundreds or thousands of patients through large-group Expanded Access trials. The outcomes of theses access trials provided supporting evidence of safety and efficacy, and also identified high-response subgroups of the population.

WideTrial has spent years studying these cases and has the personnel from some of these programs on its team right now.

Today’s new antibody and immunotherapy treatments offer similar breakthrough opportunity in deadly unsolved cancers. With its partners in molecular diagnostics, WideTrial can use biosamples and endpoint data from its access trials to inspect miRNA, mRNA, and oncogene markers for correlation with treatment response, a dramatic improvement to the drug development process that can only be achieved through large-n programs like ours.

To learn more, please contact us here.

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RARE OR GENETIC DISORDERS

Rare and ultra rare serious disorders impact over 3 million people in the U.S. today. Altogether, rare diseases constitute a not-so-rare unmet need, but individual families can feel like they are all alone fighting for treatment options.

New therapies for enzyme replacement, gene silencing, and cell regeneration may bring tremendous promise for these families, but many can’t access them. A suffering child may be too far progressed in his disease to be eligible for a research trial; or he may be too young or too old or have too many other health issues to be an acceptable research candidate.   But, in cases in which the doctor and family believes exploratory treatment is appropriate, we want to make sure it’s possible.

WideTrial welcomes engagements with drug companies working in difficult rare diseases. We understand the expense of manufacturing product at small scale. We appreciate the challenges of research in small populations and we are committed to supporting cost-sharing partnerships among industry, advocacy, and the medical community.

If you are working in this space and you would like to suggest a particular treatment for Expanded Access, please speak with us.

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INFECTIOUS DISEASES

WideTrial is in position to play huge role in the medical response to infectious disease outbreaks, such as Ebola, Zika, and avian influenza.

Authorities such as the U.S. Department of Health & Human Services, and U.S. Department of Defense, and World Health Organization have tasked innovators to come up with new ways to bring treatment to populations in need in times of emergency. We intend for WideTrial to enable more-complete access to new therapeutics and vaccines without interfering with ongoing research trials.

If you would like to contribute or participate in this humanitarian effort, please contact us.