Innovation for Rare & Orphan Drugs

Advancing the Commercialization of Rare & Orphan Drugs

Innovative Therapies Deserve Innovative Strategies

With more than 7,000 distinct types of rare and genetic diseases and 400+ million individuals suffering from a rare disease, regenerative medicine holds the hope for a cure – transforming healthcare by revolutionizing patient care from conventional treatment models to curative therapy models. With the advancement of new technologies coupled with the creation of new companies offering a range of innovative products and treatments, regenerative medicine constitutes one of the fastest-growing fields of research and the promise of commercial success for all patients.

Creating better patient experiences and bringing effective therapies to patients around the world

In the value-driven healthcare environment, rare and orphan drug manufacturers are becoming increasingly aware that new models and new approaches are needed for a successful product launch. At EVERSANA, we understand the complexities of developing products for small populations that require global reach. That’s why we are focused on an end-to-end process that’s connected and assures that patients are identified, diagnosed and onboarded and that they stay adherent to their therapies.

Top Issues We Address

  • Upcoming innovative changes in research, clinical development, manufacturing, registrational paths and commercial models impacting the rare disease community
  • Developing strong economic models in demonstrating the value of subsidizing patient healthcare costs
  • Finding the right patients and designing a collaborative clinical trial
  • Establishing a well-informed sales and marketing plan
  • Accurate forecasting to ensure successful market access
  • Optimizing a supply chain management system

Leveraging AI to Advance Rare Disease Patient Identification & Commercialization

Today we have access to more data from more sources than we could ever dream possible. The need for data is elevated as diagnosis becomes a race against time and the pipeline for innovative new therapies continues to grow. In 2019, the FDA approved 22 drugs and biologics with orphan drug designation. In 2020, they approved 31. As this pipeline expands, manufacturers have a responsibility to the patient to ask themselves – How do we cut down that time to diagnosis?

Delays in rare disease diagnosis can be an understandable result of provider expertise. Providers may see one or two rare cases in a year — or in a career, which is not enough to refer to or look for diagnostic criteria to find patients who may be hiding in HIPAA-compliant data.

Harness the Power of Data

With machine learning that enables the synthesis of disparate data sets to provide greater insight into the patient journey and the disease progression, manufacturers can help bridge the gap between data aggregation and execution. When manufacturers leverage RWD to generate RWE (from electronic medical records, claims, physician and nonclinical data, etc.), they can inform the start of the patient journey – not just the end. From this, a data-driven pull-through for patient care will be possible. With the power of aggregated data, manufacturers can give providers the data they need for a broader, more efficient diagnosis experience.

Examples of How to Leverage the Power of Data

  • Build an AI-compatible data ecosystem that incorporates healthcare and non-healthcare data sources
  • Extract actionable insights, such as identifying undiagnosed patients, from data through AI
  • Determine what constitutes an AI-driven insight, and how to translate these insights to drive the next best action for commercial activities

Featured Content

A Data-Driven Commercialization Pathway to Expedite Rare Disease Diagnosis and Adherence

“We often think about big data as this big, cold, robotic thing; and I think it reveals, in real time to us daily, that there are opportunities for many small, important moments and many impactful, emotional things to happen for patients and caregivers.” —Amy Hutnik, General Manager, Agency, Advisory & Evidence Services When the life […]

The Power of Working Together With the Rare Disease Community

“Alone, we can do so little; together, we can do so much.” – Helen Keller This sentiment addresses the power of working together to improve health in rare disease. At EVERSANA, we have developed a unique working relationship with rare disease communities. Successfully working with these communities, especially in gene therapy, requires a demonstrated long-term […]

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