News & Events Rare Disease Day 2020

Rare Disease Day 2020

February 28, 2020

Each year on the last day of February, the rare disease community comes together to raise awareness for the hundreds of millions around the world impacted by rare diseases. We at CTI are proud to have been a part of Rare Disease Day for the past 7 years, and will continue to work passionately to help drugs and devices for rare diseases around the world reach the patients that need them. 
What is a rare disease?
A rare disease is defined in the United States as any disease, disorder, illness or condition affecting fewer than 200,000 people at any given time. In Europe, a rare disease is defined as a disease or disorder affecting fewer than 1 in 2000 people.

You can learn more about rare diseases by visiting the National Organization for Rare Disorders (NORD) website.

What are some challenges to expect with rare disease programs?
Risk, rarity, and complexity all play a factor in our partners' decisions to work on programs in a rare disease. Concerns about risk can be mitigated by exploring regulatory incentives and working with a CRO who knows how to navigate those regulatory pathways. Concerns about recruiting rare patients and planning complex programs can be addressed with careful planning and by working with an experienced team who has encountered and overcome similar challenges before.

Click here to read more about the challenges of running a rare disease program and how to overcome them.
Why face these challenges?
In short, there are millions of patients around the world without approved treatments for their rare disorders and they are relying on the scientific and pharmaceutical communities to give them hope.

Changes in policy by the FDA and regulatory agencies around the world have resulted in a significant increase in rare disease drug approvals in recent years. The Orphan Drug Act in the United States and similar policies around the world offer tax breaks, expedited reviews, or market exclusivity to incentivize the development of rare disease treatments that may not otherwise be considered financially viable. Over the past two years, more than half of all new drug approvals from the FDA have been for rare diseases, historically high numbers.

However, despite these encouraging numbers, according to the National Organization for Rare Disorders (NORD), more than 90% of rare diseases are without an approved treatment.
CTI's commitment to patients with rare diseases
There are 300 million people living with a rare disease around the world, not including the number of family and community members also impacted. This number includes members of our own team who have children, friends, or family members directly impacted by the rare diseases on which we work. We care about our team members and our patients, and we believe that caring fuels a different level of commitment that sets us apart from others in the industry. CTI is committed to working with our partners to help find treatments, and even cures, for these patients.

As illustrated in the sections above, the CTI staff is made up of experts in rare disease research. CTI has been working in rare disease research for 20 years, and the vast majority of our active prospective studies are in rare diseases. We know how to work with our partners and regulatory agencies and be proactive in our program planning to overcome many of the challenges inherent to rare disease research. From IT to Safety, to Project Management to Monitoring, our team members are experts in the challenges inherent to these types of trials, and are committed to finding the solutions necessary to bring new treatments to patients in need.

To find out more about our work expertise in rare disease research, visit our website or fill out our contact form to start a conversation with one of our team members.

The Year of Hope

CTI Cares - Providing Hope to our Communities
The CTI Cares program was established in response to the great interest of our employees who want to give back to society, and in part due to the prior success we’ve had in charitable giving programs.  Each month, an employee-nominated charity is spotlighted and supported by our employees and the company as a whole. This month, our highlighted cause is the ALS Association.
The ALS Association is a national non-profit organization fighting ALS.  By leading the way in global research, providing assistance for people with ALS through a nationwide network of chapters, coordinating multidisciplinary care through certified clinical care centers, and fostering government partnerships, the Association builds hope and enhances quality of life while aggressively searching for new treatments and a cure. To learn more or to donate, visit

Additional Highlights

Upcoming Meetings We'll be Attending
AST: Cutting Edge of
Phoenix, AZ
March 5-7
EASL International Liver Conference
March 15-19
DIA Europe
March 16-19
EBMT Annual Meeting of the European Society for Blood and Marrow Transplantation
March 22-25
New Hires & Promotions
CTI is thrilled to welcome all of our new employees, and to congratulate our recently promoted employees! 
Join Our Team!

We're looking to build our team in following new cities:
  • Atlanta, GA
  • Boston, MA
  • Chicago, IL
  • Dallas, TX
  • Houston, TX
  • Indianapolis, IN
  • San Diego, CA
  • Washington DC

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