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Regulator and Funder? FDA’s Orphan Products Grants Program awards significant funding to help move promising treatments through clinical development

FDA Law Blog: Drug Discovery

Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. Each year, FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.

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The rising impact of biomarkers in early clinical development

Drug Target Review

As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. Safety biomarkers account for adverse effects of a therapy under study. There are several types of biomarkers to consider.

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The Growing Trend of Patient-Led Clinical Research in Rare Disease

Conversations in Drug Development Trends

By: Juliane Mills, Senior Director, Therapeutic Strategy Lead, Rare Disease The rise of patient-led clinical research, particularly in rare disease, represents a significant shift in the clinical trial landscape. Why Is There an Increase in Patient-Led Rare Disease Research?

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Redefining Acceleration of the Drug Development Journey

PPD

As a result, biopharma and biotech companies working to bring their drug pipelines to the market require deep expertise from trusted partners to help deliver critical therapies for their patients. However, getting essential treatments to patients quickly and safely requires more than just technological innovation. billion in 2023 to 1.2

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Obesity care gets personalised: tailoring therapies with Phenomix

Drug Target Review

Obesity treatment is undergoing a major shift, much like the advances seen in cancer care. Phenomix Sciences , led by CEO Mark Bagnall, is using precision medicine to make treatments more targeted and effective. Phenomix Sciences, built on over a decade of clinical research at the Mayo Clinic, is disrupting this outdated approach.

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Key Trends Drug Developers Need to Know to Succeed

PPD

In response, the PPD clinical research business of Thermo Fisher Scientific conducted its third global survey of 150 drug developers to capture a comprehensive view of these evolving trends. Furthermore, the development of innovative therapies often necessitates intricate protocols, adding layers of complexity to trial management.

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Overcoming Orphan Drug Development Challenges with Real-World Data and Evidence

PPD

Developing treatments for individuals living with rare diseases is critical, but orphan drug development is laden with unique obstacles that necessitate innovative, multifaceted approaches. This limited pool poses significant barriers to conducting statistically significant studies and validating the efficacy and safety of new treatments.