Developing First-in-Class Gene Therapy for an Ultra-Rare Disease

The Client

A Leader in Healthcare Technology Transformations

As an industry thought leader, this healthcare consulting firm partners with customers to re-engineer value chains that improve clinical, financial, and operational outcomes that drive business transformations. By offering unmatched depth and breadth of experience in the healthcare technology space, end clients are empowered to deliver exceptional value that enables better care and patient outcomes.

The Situation

A New Innovation Comes to Market

Innovations in the life sciences industry continue to improve patient outcomes daily. The speed at which innovations come to market has changed as a result of COVID-19, which accelerated the pace in how vaccines and therapies are developed and delivered.

Such was the case for this healthcare consulting firm’s long-term client — a leading drug manufacturer spearheading the launch of a new first-in-class gene therapy for an ultra-rare disease.

Ahead of the launch, the client wanted to conduct patient targeting, segmentation, and stratification analyses to help determine the launch strategy of this pivotal and highly anticipated treatment.

But the firm had one problem. Given the expedited turnaround time of the project and the workload to pull this new product launch off, it realized it would need to bring outside expertise to supplement the team. An expert that could:

  • Understand and analyze patient data to make strategic recommendations
  • Conduct patient-based analyses with existing data for ultra-orphan disease populations
  • Analyze patient-based data using SQL server
  • Collaborate with cross-functional partners
  • Lead a small team
  • Interface with the end-client

It was important for the expert to have technical knowledge of SHYFT NOVA and HIPAA guidelines.

The Solution

An Expert With Proven Patient Data Analytics Experience

Having worked with Graphite on various engagements, the healthcare consulting firm reached out to its account executive (AE) to kick off the search for an independent strategic patient services consultant.

With the scope of work on hand, the AE used their knowledge of the platform to invite six candidates out of 1,622 experts on the platform to apply for the project.

Of the six candidates, one particular expert was flagged for the client due to their deep technical knowledge and strategic thinking at large healthcare organizations, including Walgreens. Because the AE had previously worked with the candidate, they could personally speak to the candidate’s exceptional results.

After engaging with each candidate via the platform— connecting via Graphite’s in-app meeting rooms — the firm decided to move forward with the AE’s suggested expert. The candidate’s experience in patient data analytics, which included IQVIA/IMS, LexisNexis, and Symphony, won the firm over.

Not to mention the candidate’s additional background in running data visualizations using tools like SFDC reporting and Tableau.


Exceptional Results Leads to Better Patient Outcomes

Once onboarded, the expert set out to understand the product that the leading drug manufacturer was launching. Doing so provided the expert context around the data they would be reviewing in order to provide strategic recommendations.

To arrive at their conclusion, the expert conducted patient-based analyses using tools such as Jira andSQL, diving into the data to unearth key insights to drive a successful product launch. They also worked closely with leadership, patient services, and strategic planning to devise the best product launch for the new first-in-class gene therapy drug.

Within six months of interacting with the end client and working as part of the Biotech team (the in-house, corporate organization), the expert had become a trusted partner. Not only were they able to lead the project, but the firm retained the expert to facilitate the execution as well due to the end-client’s positive feedback.

Graphite’s AE and customer success team connected with both the firm and the expert to keep the project moving forward and ensure its success. Because of the platform’s ease of use — with features that made tracking hours, invoices, and payments easy — and frequent communication from the team, both the expert and client reported being pleased with their Graphite experience.

Back to Case Studies

Developing First-in-Class Gene Therapy for an Ultra-Rare Disease

Learn how an independent expert helps a leading drug manufacturer spearhead the launch of a new first-in-class gene therapy for an ultra-rare disease.

Skills Onboarded
About the Client
Project Duration
Independent Experts Hired
Project Booking Value


Key roles identified and onboarded

Eager to onboard a team that could achieve results within six months, the client and their dedicated Graphite account manager partnered to determinze the specifications for each of the four roles. They were:

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Gabriela Ziffermann

Digital Messaging Program Manager
  • Provide expertise in creating digital two-way communications compliant with federal government regulations (via text, email, and online portal)
  • Working with internal developers and product managers to implement the messaging rollout.
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