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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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Shifting Paradigms in PAH Clinical Trials: 7 Key Takeaways for Success

PPD

Food and Drug Administration (FDA)-approved therapies for treating PAH were primarily vasodilators, designed to overcome the imbalance between vasoactive and vasodilator mediators and to restore endothelial cell function. Historically, the available drugs and U.S. None offers a cure for PAH.

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Relieve Caregiver Burden, Improve Neurodegenerative Clinical Trials

PPD

Yet, four out of five care partners of people with neurodegenerative disorders have never been informed of clinical trial opportunities , according to a survey of more than 250 caregivers conducted by PPD, the clinical research business of Thermo Fisher Scientific. There’s little time to lose.

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Improving Diversity: Increased Patient Access to Clinical Trials

Vial

The success of clinical trials hinges on increasing access to participation by all eligible patients, including populations that have been underrepresented due to the barriers highlighted below. Specifically, U.S. Patients underlined the need for transparency by pharma and other related companies to build trust and partnership.

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Navigating the Challenges in Clinical Research for Hepatitis C Therapies

Vial

In the decades since its identification in 1989, advances in clinical research for new Hepatitis C therapies and the introduction of direct-acting antiviral agents (DAAs) have led to viral eradication in more than 98% of patients, resulting in HCV crossing from manageable to being the first curable , chronic viral infection.

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Type 2 Diabetes: Challenges & Emergent Strategies for Clinical Research

Conversations in Drug Development Trends

Partnering with a CRO that emphasizes the complexity of the patient’s voice and harnesses the nuances of clinical trial design is crucial for executing successful clinical research. These data emphasize the need for precise and meaningful body composition endpoints in T2D and obesity clinical trials.

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What types of clinical research are there?

Antidote

Before any new therapy can be put on the market, it must be tested to ensure its safety and effectiveness, and the research for this process is done through clinical trials.