Slide Rare Disease Rare Diseases Are Not That Rare!

Rare diseases, also known as orphan diseases, affect a small percentage of the patient population. With about 7,000 known rare diseases1 collectively affecting around 1 in 15 people worldwide2, the life sciences industry is significantly shifting its focus towards tackling them. Although, there are many challenges involved.

Given the limited understanding of most rare diseases, patients struggle with getting the right diagnosis and the proper treatment – an FDA-approved treatment is available for only 10% of rare diseases. Also, the commercialization process of orphan drugs differs from traditional medicines with a lot more involvement from multiple stakeholders. To succeed in this environment, companies must focus on creating awareness and building trust among patients while fostering a culture of patient-centricity.

Axtria is committed to a healthier world and recognizes the attention that Rare Diseases demand. Here is a collection of our thought leadership exploring the details of this topic and how life sciences companies can embark on demystifying this “unknown.”

Rare Diseases Orphan drugs

White Paper

Challenges And Opportunities To Commercialize Orphan Drugs For Rare Diseases In The US

Orphan drugs (ODs) for rare diseases (RDs) present pharma companies with the opportunity to address a substantial unmet medical need, with approximately 7,000 RDs, and only about 5% having effective treatments.

Case Study

Orphan Drug Commercialization For An SMB Pharma Organization Enabled By Axtria DataMAx™

The orphan drug market is historically dominated by the big pharmaceutical company or biotech corporations; however, Small-Medium Businesses (SMBs) also attempt to form their presence felt.

Orphan Drug Commercialization
Rare Diseases

White Paper

Challenges And Opportunities To Commercialize Orphan Drugs For Rare Diseases In The US

Orphan drugs (ODs) for rare diseases (RDs) present pharma companies with the opportunity to address a substantial unmet medical need, with approximately 7,000 RDs, and only about 5% having effective treatments.

Rare Disease

Case Study

Orphan Drug Commercialization For An SMB Pharma Organization Enabled By Axtria DataMAx™

The orphan drug market is historically dominated by the big pharmaceutical company or biotech corporations; however, Small-Medium Businesses (SMBs) also attempt to form their presence felt.

Rare Disease Artificial Intelligence and Machine Learning

Blog

Challenges Of Accelerating Diagnostic Journies For Rare Diseases

Traditionally, pharma companies have not had enough focus on patient identification, especially during the early stages of a rare disease product cycle. This has often led to missed opportunities in terms of reaching out to the patients as early as possible in their diagnosis journey.

Rare Disease Diagnostic

Blog

Rare Disease Diagnoses Opportunities For AI And Machine Learning

Over the past few years, life sciences companies have made commendable advancements in the realm of rare diseases, but the dominance of the associated challenges still exists. These challenges can range from ‘rare disease awareness’ to the ‘holistic treatment of patients.’

Sources:

  1. Genetic and Rare Diseases Information Center – FAQs About Rare Diseases
  2. World Health Organization – BP6_19Rare.pdf, 6.19 Rare Diseases

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