Help your client overcome the challenges of patient recruitment, study design and market access with patient-centric solutions for rare disease and orphan drug development – find new approaches in this issue of Consultant Connection.
CONSULTANT CONNECTION – OVERCOMING THE UNIQUE CHALLENGES FOR RARE DISEASE AND ORPHAN DRUG DEVELOPMENT
Give your client insightful solutions to accelerate their orphan drug development program
The development of orphan drugs to target rare diseases is growing due to recent financial and regulatory incentives. But even with these positive changes, developing treatments for rare disease presents many unique challenges beyond the scope of standard drug development.
Here are some of the issues that your client may face:
Identifying and retaining hard-to-find patients. Recruitment is an inherent challenge for studies of rare diseases given sparse populations. But now it’s possible to more precisely pinpoint relevant populations for your client’s study. Follow a proven methodology to increase the visibility of your client’s study and help boost enrollment with the Covance Xcellerate® Clinical Trial Optimization® Suite, an extensive database of self-registered LabCorp patients and strong relationships between Covance and leading advocacy groups.
Creating an efficient study design. Direct your client to more relevant trial results by creating a comprehensive study design that considers the desired outcomes and builds in validated endpoints. You and your client can gain a deeper understanding of the disease and incorporate essential study design points during the protocol design process by accessing our robust data that captures co-morbidities and key lab values. In addition, you can evaluate if surrogate markets or imaging technologies can improve predictability and accelerate your client’s results.
Incorporating patient-centric practices. To increase the level of patient engagement, consider embracing and incorporating patient-centric practices into your client’s program. Through voice of the patient research, you’ll easily understand the complete patient ecosystem – inclusive of advocacy groups, treating physicians, investigators and patients. With a deeper understanding of patient perceptions, your client can differentiate their study, increase enrollment and create a unique reach spanning several patient touch points.
Maximizing commercial potential. Receiving regulatory approval doesn’t necessarily translate to market success for your client. It is also critical to understand their treatment’s value through patient reported outcomes (PROs) and other health-related quality-of-life instruments. Market research can be hugely beneficial. From the payer perspective, the expense of orphan drugs can present payment hurdles and price sensitivity. You can help your client understand the best route to market with a value based strategy that considers potential roadblocks and maximizes their investment.
Case Study: Assessing Study Feasibility with LabCorp Data
A small virtual biotechnology company based in the US needed to launch a clinical program for a new treatment for cold agglutinin disease (CAD), a difficult-to-diagnose, rare autoimmune hematologic condition found only in about 1 in 300,000 people in the U.S.
To extend their limited resources, the client needed comprehensive support on issues ranging from site selection to regulatory submissions. They requested a feasibility survey from Covance, who used LabCorp data to confirm the prevalence of the disease and identify potential sites for their clinical trials.
Lecia Johnson and her team are standing by to provide consultants with seamless access to Covance global solutions – preclinical through clinical, to registration and market access. Contact us today – we look forward to helping you succeed.Contact the team >
From early development to post-market studies, you can apply our experience gained from supporting 56 indications across 103 studies in the last six years. Together, let’s help your client overcome key development challenges and make a lasting impact on unmet medical needs.
Rare & Orphan Disease Resources
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