The Impact of Single Indication Orphan Drugs on the Future of Rare Diseases
According to a new study by FDA and HHS researchers published in the January 2024 issue of Health Affairs , 65% of 491 novel drugs (NMEs)...
What is the impact of rare diseases on individuals and health care systems?
A new NIH study on fourteen rare disease populations revealed the cost of rare disease healthcare to be three to five times higher than...
Congressionally funded report details diagnostic challenges and costs of rare diseases in the US
On October 18, 2021, the Government Accountability Office (GAO) published a report entitled, Rare Diseases: Although Limited, Available...
Diagnostics: Moving to the forefront of Global Healthcare
Diligentia Strategy is pleased to participate at the Southeast Life Sciences conference October 27-29 in Charleston, SC. Joff Masukawa...
Economic Burden of Rare Diseases Nearly $1 Trillion
While there are estimated to be more than 7,000 rare diseases, The National Economic Burden of Rare Disease Study includes 379 rare...
CBO Comparison of Brand-Name Drug Prices Among Selected Federal Programs
On February 18, 2021, the Congressional Budget Office (CBO) published a report entitled, A Comparison of Brand-Name Drug Prices Among...
Orphan drugs account for more than half of all FDA drug approvals in 2020
In 2020, more than half (31 of 53, or 58%), of all of novel approvals rendered by the FDA were for drugs to treat patients with rare...
Rare Disease Stakeholders: Do mRNA COV19 Vaccines Affect Eligibility to Receive Future Gene Therapy?
The American Society of Gene and Cell Therapy issued FAQs on December 30, 2020 to explain how mRNA vaccines for COVID19 work and why they...
How do we identify and measure value for patients with rare diseases?
Rare diseases impact 1 in 10 Americans, and over 400 million people worldwide. The current development and FDA approval process can cost...
National Burden of Rare Disease Survey
National Burden of Rare Disease Survey – We Need YOU: What’s the cost of living with a rare disease to YOU? If you or a member of your...
