A New Era For Patient Recruitment And Retention In Rare Disease

By Janelle Hart, Managing Editor, Custom Content & Andrea Charles, Head of Custom Content, Norstella

Pharmaceutical and biotech companies are increasingly diversifying their pipelines to address high and rare unmet needs, although R&D in this area presents unique challenges. Norstella, a company specializing in supporting drug development, is helping organizations navigate these complexities. Major pharmaceutical companies, including Amgen, Biogen, Novartis, and AstraZeneca, are investing heavily in rare disease research, despite the challenges in achieving profitable returns.

In response, regulators and payers are implementing new policies and payment models to support these initiatives. The FDA's START Pilot Program aims to expedite the advancement of therapies for rare diseases by offering guidance and interaction with FDA experts. The pharmaceutical industry is also leveraging Real-World Data (RWD) and machine learning (ML) techniques to enhance patient recruitment and retention in clinical trials.

Meanwhile, the life sciences industry is utilizing digital tools and innovative trial designs to improve the patient experience and reach eligible individuals globally. However, global efforts to improve patient access and reimbursement for rare disease therapies have shown mixed results.

Looking to the future, experts anticipate advances in gene therapy, gene-editing technologies like CRISPR-Cas9, RNA-based therapeutics, and precision medicine. Uncover how Norstella's insights publications, data analytics, consulting, and market research services are paving the way for these life-saving treatments to reach patients with rare diseases by accessing the full article below.