Pharmaceutical Technology

APRIL 27, 2023

Pompe Disease, also known as glycogen storage type II, is a rare inherited lysosomal disorder caused by a deficiency of enzyme acid alpha-glucosidase (GAA) leading to the accumulation of glycogen in skeletal and cardiac muscles. Sanofi markets avalglucosidase alfa as Lumizyme in the US to treat LOPD since its approval in 2010.

Food and Drug Administration 52

Food and Drug Administration Food Marketing Medicine 52

PM360

OCTOBER 28, 2022

Shepherding a new drug to market is a long, expensive, and uncertain process. billion dollars to bring a new drug from discovery to regulatory approval and market launch. Approval success rates of drug candidates are between 10%–20% in the United States, the European Union, and Japan. in 2010 to only 1.9%

Pharma 105

Pharma Food and Drug Administration Government Medicine 105

Clarivate

MAY 21, 2024

The resulting complexity feeds a vicious cycle: a lack of standardized methodologies for evidence generation leads to insufficient evidence to quantify the long-term benefits of non-OS endpoints, deterring HTA bodies from giving due consideration to these outcomes in decision making [6]. Oncologist, 2010. Am J Cancer Res, 2021.

Food and Drug Administration 52

Food and Drug Administration Patients Consulting Marketing 52

World of DTC Marketing

APRIL 7, 2022

The seventh leading cause of death in the United States, diabetes costs $327 billion in medical costs and lost work and wages. Launch prices of new cancer drugs in the U.S. Food and Drug Administration (FDA). billion estimated cumulative spending on the new oral targeted cancer drugs by the end of 2018.

Healthcare 215

Healthcare Healthcare Food and Drug Administration Government 215

Pharmaceutical Technology

MAY 18, 2023

Other processes include direct-to-patient (DtP) shipments of drugs and other supplies to a patient’s home, as well as in-home care with mobile nurses. Further analysis shows that low accrual rates have been the most common trial termination reason from 2010 to 2021, comprising 25.4% of all terminated trials.

Recruitment 52

Recruitment Food and Drug Administration Patients Media 52