cell + gene
therapy

Great advances are happening in the treatment of rare diseases and oncology indications. Yet, along with their promise come unique challenges. Dispersed patient populations. Constantly evolving regulatory guidance. Long-term safety concerns. One-time potentially curative therapies with staggering costs. The path toward a cure can be unchartered and uncertain. With Evidera, you have a partner with the experience and expertise to put your cell and gene therapy on a clear path to changing lives.

ADAPT at the Speed of Insight

Optimizing Drug Development Plans for Cell and Gene Therapies

The cell and gene therapy landscape is evolving before our eyes. To realize the life-changing potential of these advancements, we must adapt to the shifting landscape as our scientific knowledge increases, new discoveries are made, and expectations and requirements progress.

How we help you quickly adapt to change:

• Industry-leading experts in early development planning and asset optimization. We generate robust plans that are relevant to all stakeholders — plans that maximize trial efficiency, leverage real-world evidence, and satisfy regulators and payers.
• Engagement with regulators and health technology assessment (HTA) bodies. Through our integrated scientific advice offering, we ensure alignment on evidence needs, identify challenges, and seek regulatory and HTA guidance — allowing us to move your programs forward with speed and agility.
• Identify critical evidence needs to stay ahead of changing global payer/HTA demands. From health economic models to support cost-effectiveness to pricing and reimbursement strategies and innovative value demonstration and delivery, our market access experts provide actionable insights to demonstrate product value and optimize reimbursement.

WHITE PAPER

Can Early Collaboration Between Multiple European Stakeholders
Increase Access for Patients Living with Rare Diseases?

PUSH Beyond Traditional

Novel Therapies Often Require Novel Study Designs

Small sample sizes. Pediatric populations. Unique symptom clusters. We understand the world in which cell and gene therapies for rare disease thrive. A one-size-fits-all trial design and data collection approach is not the answer. Instead, we consider every option, even those that push traditional thinking.

How we help you drive progress forward:

• We consider novel, traditional and every design option in between. What are your unique needs? Is there value in surrogate endpoints? How can we ensure data are robust? Once the challenges, limitations, and merits of various design options are assessed, we recommend a path that advances your product in the right direction.
• Skilled in natural history studies to fill knowledge gaps. From defining the appropriate study population to informing the development of external control arms, our experience with natural history studies ensures your study drives meaningful outcomes forward.
• Masters in long-term follow-up and safety studies. Whether gathering data for five or 15 years in the future, we anticipate and address regulatory requirements and stakeholder questions — ensuring your cell and gene therapy is poised to demonstrate long-term effectiveness, safety , and value.

WHITE PAPER

Natural History Studies in Rare Diseases and Genetic Biomarkers

REACH the Right Patients

Optimal Patient Recruitment and Retention Strategies

Are recruitment and retention challenges keeping you up at night? Need access to trained investigational sites across multiple countries? By putting patients at the epicenter of every program, we remove barriers, drive participation, and enhance their overall experience.

How we help you reach and retain your greatest stakeholder:

• Leverage patient preferences for improved recruitment and retention. With patient input on protocol designs and operational implementation, we can identify potential barriers to participation to ensure patients start and stay engaged in your programs.
• Expertise in patient selection and enrollment strategies. Whether using insight from the patient journey or relying on our established relationships with experienced cell and gene investigator sites, we have the strategies in place to efficiently recruit patients and reduce start-up times.
• Established virtual/decentralized studies to facilitate patient participation. We can eliminate logistical burdens and help you drive recruitment, engagement, retention, and data quality through our proven digital enablement solutions.

WHITE PAPER

Patient Engagement in Clinical Trial Protocol Design and Recruitment Strategies:
What Does It Mean for Orphan Drug Manufacturers?

Ready To Move Your
Therapy Forward?