Rare disease

Championing innovation in rare disease by partnering with patient communities

Leverage our specialist networks who will draw on their combined expertise in medical strategy, market access and regulatory documents to make your limited resources count.

In rare disease, patient populations are small, tightly knit and often underserved. Though products may be approved quickly, patients can struggle to access the treatments they need.

From centres of excellence, patient advocacy groups to digital influencers, a partnership approach in rare disease is critical.  We help you better understand rare disease communities and authentically engage with them.

By combining specialist rare disease knowledge with tech-enabled insights, we provide guidance on which stakeholders you most need to partner with and where you can bring most value to them.

Deep rare disease expertise

Our multidisciplinary network of rare disease specialists use their skills across strategy, engagement, communications, market access and regulatory writing to support with critical activities, including: 

Partnering with patient advocacy groups and communities 

Preparing clinical trial protocols, including for gene therapies 

Developing integrated evidence plans and real-world evidence strategies 

Communicating key clinical and real-world evidence to clinicians and patient communities 

Generating value dossiers in ultra-rare diseases

Planning omnichannel engagement and education initiatives

Your specialist network of advisers

More than 15 years’ partnering with rare disease communities

>75% PHDs, MScs and higher scientific qualifications

Multidisciplinary insight engine

Supporting in haemophilia and rare bleeding disorders, we mapped patient pathways and identified centres of excellence, as well as developing symposia with multidisciplinary healthcare professionals and patients. We supported a webinar aiming to improve diagnosis through targeting clinical specialties not usually involved in the care of rare bleeding disorders:

Thank you team, for making this [symposium] possible and facilitating the positive ‘vibe’ in the team. Your high demands on quality go hand in hand with creating and sustaining a very good spirit. It was a pleasure and an honour to be able to add my story to what feels like the gate to a new era in which the treatment of haemophilia will hopefully enter a new phase.

Patient Advocacy Lead

I appreciate the flexibility of the team to quickly start a new project and the responsiveness and quick follow-ups – excellent collaboration and team within Bioscript!

Senior Manager

International Medical Information

A breadth of capabilities

By combining global cross-functional working, specialist knowledge and tech-enabled insights, we help our clients streamline their scientific strategy and enhance patient access.

Find out more

Bioscript Data Insights

Strategic planning

Medical communications

Market access consulting

Regulatory writing