As many as 30 million Americans may be living with a “rare disease,” officially defined as a condition affecting fewer than 200,000 people.

The Genetic and Rare Diseases Information Center says there are some 7,000 known rare diseases. Most are genetic. Only about 10% of all rare diseases have an approved treatment.

To hasten the development of drugs and therapies, the Food and Drug Administration in 2019 began an initiative to “to facilitate a cooperative approach and common standardized platforms to better characterize rare diseases, incorporate the patient’s perspective in clinical outcome assessment measures, and build clinical trial readiness in the pre-competitive space.”

Dubbed the Rare Disease Cures Accelerator, the initial focus of the program is building “an integrated database and analytics hub designed to promote the secure sharing of existing patient-level data and encourage the standardization of new data collection.”

A second key part was to award grants “develop standard core sets of clinical outcome assessments (COAs) and endpoints for specific disease indications.”

In May, the FDA asked for input from industry, rare disease organizations, patients and others regarding the “implementation and sustainment of global clinical trials networks.”

“As drug development for rare diseases can be challenging due to the small number of patients and limited understanding of the variability and progression of the diseases, the FDA is committed to developing bold new approaches to harness the infrastructure of global clinical trial networks,” said Anand Shah, FDA deputy commissioner for medical and scientific affairs.

A recent report on Regulatory Focus, the website of the Regulatory Affairs Professionals Society, said the stakeholders who commented “called for regulatory clarity, smart use of existing resources, and a move toward harmonized trial standards and assessments.”

Several comments addressed the need to ensure the quality of the data collected as well as reducing and eliminating some of the challenges unique to rare disease trials.

On behalf of the Biotechnology Innovation Organization, Danielle Friend, senior director of science and regulatory affairs, said the networks need to support “the collection of high-quality data that are endorsed by the agency for regulatory decision-making.” She said a rare disease network must recognize and address “heterogeneity in rare diseases, lack of harmonization among global regulators in rare disease regulatory policy, and current inconsistencies in clinical trial network operation.” The FDA plan also should consider combining rare diseases into a single trial when possible.

The National Organization for Rare Diseases echoed those comments, and, according to Regulatory Focus, said “Increased collaboration and a focus on increasing the speed and success of clinical trials can have the effect of ‘breaking down the silos of activity currently taking place in rare disease research.’”

Now the FDA will analyze the comments, incorporating them into a development plan for its trials networks program. No timeline was given for this phase of the program.

Photo by CDC on Unsplash


author avatar
Green Key

#WeAreGreenKey: Spotlight on Kayla Jones

Kayla has been with Green Key for six years. Originally a member of the Pharma team, she transitioned to the Marketing team in 2020. Kayla let us in on her opportunity to switch teams, her efforts to enhance training programs at Green Key, and the importance of a strong marketing strategy at the company.

Jun 6, 2023

You Don’t Have to Be a Scientist to Have a Life Sciences Career

Curious about a career in the life sciences industry, but you’re not a scientist? Don’t let that discourage you. Pharmaceutical companies have thousands of jobs in a variety of areas that don’t require biology or other science background.

The challenge for new entrants is that recruiters look for great talent with relevant experience. Whether you’re a recent or upcoming grad or a mid-career professional wanting to change industries, it’s up to you to show how your background and the experience you have is relevant. That means showing how your skills are transferable.

For example, an accountant who helped develop sales projections for a new product or territory may be able to demonstrate how the research and analysis that went into the report applies in the pharmaceutical world. A marketer with lead generation experience should explain how that can help the company expand sales.

Biospace has a primer on using skills and experience in other jobs to open career opportunities in the life sciences sector. The advice is basic, yet what it lacks in specifics it makes up for in providing direction.

For starters, Biospace counsels to “Get clear on what your transferable skills are.” As commonsense as that is, so many job seekers will start by simply updating an existing resume.


First become knowledgeable about the skills important to life sciences and pharmaceutical firms. Inventory the skills you’ve developed and your experience, listing those most relevant and transferable.

“Some suggestions to consider,” says the article, ”Are research, analysis, data analysis, problem solving, communication, time management, communication (written and verbal), planning, strategizing, team management, project management, presenting, conflict resolution, collaboration and training.”

Then “Come up with examples from your past job roles and duties.” Finally, “Include accomplishments that emphasize the transferable skills.”

Now you’re ready to revise your resume to highlight those skills and show how they will benefit the company.


author avatar
Green Key