Restricting orphan drug approvals could save the US billions of dollars, study from University of California’s Center for Health Policy and Economics suggests.

Emer Cooke seems set to take over as head of the European Medicines Agency – one of the world's most powerful regulatory bodies – before the end of the year. We report on the reaction of a range of pharmaceutical sector stakeholders to her nomination.

Pharma has won pricing battles during the pandemic, but doesn’t seem to be winning war as senators on both sides of the aisle question how some drug companies were able to skirt controversial intellectual property ownership issues in COVID-19 drug and vaccine agreements with the US government. IP experts say the biggest harm to the government from the contracting language may not be the pricing concerns but the government’s ability to share leanings from the partnerships with other coronavirus development programs.

An agreement between Vertex and NHS England secures rapid access to Kaftrio, the company’s triple therapy for cystic fibrosis, including for patients with rare mutations not covered by the soon-to-be-granted European marketing authorization. The final price will depend on an assessment by health technology appraisal body NICE.

PhRMA sues to block implementation of the law, saying it could allow states to compel manufacturers to dispense other medications without compensation.

Gene and cellular therapies that can replace decades of expensive therapy do not come cheap. Even the initial remedy of payment by installments could be irrelevant when not all patients respond in the same way.

Novartis says that while the comparators used in the evidence it submitted to NICE were the right ones, it will work to address the health technology assessment body’s outstanding data requirements to secure access to its treatment for secondary progressive multiple sclerosis.

The expected launch of BioMarin's Roctavian for hemophilia A is expected to heighten the profile of expensive gene therapies on payers' radar. Specialty drug costs are expected to continue to grow in 2021. 

Value and outcomes-based purchasing agreements could see a boost under proposed Medicaid rule that CMS says will make it easier for manufacturers to experiment different contracting structures in the commercial health insurance market. CMS says the intent of these agreements is to lower drug prices for commercial payers, but the government acknowledges this is not guaranteed, and health policy experts worry both private payers and Medicaid could end up paying higher prices if the proposal is finalized.