Clinical Research: Phase 1 - Phase 4

Clinical Research: Phase 1 - Phase 4

Understanding New FDA Guidance for Pediatric Oncology Studies: A Sponsor’s Guide to the FDA RACE for Children Act

Summary The Research to Accelerate Cures and Equity (RACE) for Children Act The Research to Accelerate Cures and Equity (RACE) for Children Act aims to improve and expand treatment options for pediatric cancer patients by mandating that all new adult oncology drugs also be tested in children when the molecular targets are relevant to a...

Clinical Research: Phase 1 - Phase 4

Contract Pharma – Realizing the Full Potential of Precision Medicine in Oncology

Innovation in oncology drug development is being driven by “precision medicine.” Precision medicine promises a new paradigm in oncology where every patient receives truly personalized treatment. This approach to disease diagnosis, treatment and prevention utilizes a holistic view of the patient—from their genes and their environment to their lifestyle—to make more accurate decisions. Growing at...

Clinical Research: Phase 1 - Phase 4

Applied Clinical Trials – Patient Engagement and Advocacy: Advancing the Cause of Clinical Drug Research

Duchenne muscular dystrophy (DMD) patients almost universally lose the ability to walk as the disease progresses. Patients generally understand that loss of ambulation is to be expected, and as was learned from a patient preference study, they tend to put greater priority on maintaining use of their hands. Yet for many years, DMD drug trials...

Clinical Research: Phase 1 - Phase 4

High Potential: Precision Cancer Drugs

Unlocking the Full Potential of Precision Medicine in Oncology Precision oncology promises a new standard of care where therapies are tailored to the molecular profile of a specific tumor. For the full potential of precision medicine to be realized, regulatory, technical, clinical, and economic frameworks will need to evolve to the nuances of these novel...

Clinical Research: Phase 1 - Phase 4

PharmaVoice – Small Pharma Driving Big Pharma Innovation

“Big pharma’s new role in this is to partner and fund the innovation that it so sorely needs to bulk up its dwindling pipelines. This is a cost-effective move for larger drug companies to leverage outside scientific talent to gain access to breakthrough discoveries. “‘This is a dominant trend in the industry,’ says Nach Davé,...

Clinical Research: Phase 1 - Phase 4

Considerations for Operationalizing Pediatric Oncology Studies

Remarkable progress has been made in our understanding of the genomics of pediatric cancers, and these advancements have led to the recognition that products being studied for use in adult cancer indications may have health benefits for pediatric patients. By closing the orphan drug exemption loophole and enabling earlier discussions with the FDA, the Research...

Clinical Research: Phase 1 - Phase 4

The Challenges in Fulfilling the Promise of Precision Oncology

Precision oncology promises a new model of cancer care where medical decisions are based on a holistic view of the patient, including their genes, environment, and lifestyle, and tailored to the molecular profile of their tumor. To date, great strides toward the paradigm of precision oncology have been made in the area of cancer immunotherapy,...

Clinical Research: Phase 1 - Phase 4

Understanding Recent Regulatory Changes for Pediatric Oncology Trials

Of the 1.7 million new cases of cancer in the U.S. in 2018, 10,590 involved children aged 14 or younger.[1] While children typically have a better cure rate for cancer, biopharma companies have traditionally shied away from pediatric oncology trials, resulting in fewer treatment options. However, the regulatory landscape is evolving, and the demand for...

Clinical Research: Phase 1 - Phase 4

Considerations In Conducting Pediatric Clinical Trials

There are many challenges inherent in pediatric clinical studies, where the traditional paradigms for evaluating pharmaceutical agents in adults often do not apply. These occur on ethical, physiological, pharmacometrics and economic grounds, among many others. It’s important to recognize that while these efforts are challenging, they are not impossible — and they may be better suited to creative solutions...

Clinical Research: Phase 1 - Phase 4

Neurodegenerative Disease Research: Steps to a Successful Clinical Trial

Neurodegenerative disorders present some of the biggest challenges in planning and conducting clinical drug trials. Identifying strategies to proactively address or mitigate these challenges can help to ensure a successful trial. Since the first drugs for neurodegenerative diseases (NDDs) were approved in the 1990s, an increasing number of disease- modifying therapies have been approved for...