Using Data To Improve Rare Disease Diagnosis And Treatment
A deeper understanding of rare diseases – how they differ across geographies and ethnicities, or which genes and biomarkers are most useful targets for new treatments – can help to accelerate and de-risk clinical trials. On the commercial front, patient data and insights could mean the difference between a category leader and a failure.
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Options, Expertise And Risk Tolerance Needed For Successful Value-Based Contracts
Biohaven Pharmaceuticals developed six different value-based contracting options to support the launch of acute migraine therapy, Nurtec ODT. Despite contract complexity and lingering regulatory risk, payers are aggressively pursuing value-based contracts, leading even small biopharmas to invest in strong health economics capabilities.
Expanding The Tent: Improving Trial Participation Among Under-Represented Patient Populations
The biopharma industry has struggled to recruit patients into clinical trials that adequately reflect the diverse patient populations they hope to reach with new products. Failure to improve minority subgroup participation now will cost trial sponsors later.
Cystic Fibrosis: Successes, Opportunities And Challenges
What is next for the treatment of cystic fibrosis, a rare disease market that has boomed in recent years on the back of new drug approvals with hefty price tags? Pipelines are full, but securing reimbursement is a challenge.