In update of 2011 draft guidance, FDA gives more detail on documentation needed for orphan sponsors to receive a program fee waiver; Guidance also suggests the agency is no longer working with SBA to make small business determinations.

FDA’s most recent accelerated approvals for Merck’s Keytruda call for confirmatory trials in a broader patient population than the approved indications, in the latest example of FDA’s growing comfort with multi-purpose confirmatory trials in the PD-1/PD-L1 inhibitor space.

Promising non-traditional antibiotic therapies need a clear path to market to entice investment, US Biomedical Advanced Research and Development Authority official says.

PRIMA, the new service the National Institute for Health and Care Excellence offers to review economic models, must not be affected by confidentiality and conflict of interest issues, warns UK the pharmaceutical industry group, ABPI.

Promising non-traditional antibiotic therapies need a clear path to market to entice investment, US Biomedical Advanced Research and Development Authority official says.

With Flatiron, Roche is at the forefront of the emerging field of RWE. Roche Pharma President O’Day described how the company is using such data in the real world of regulation and reimbursement.

The four latest products to be accepted onto the European Medicines Agency’s popular priority medicines scheme target mantle cell lymphoma, chikungunya, hematopoietic stem cell transplant patients, and the rare monogenic disease, X-linked myotubular myopathy.

Third-party value assessment organization says AbbVie/Neurocrine's elagolix would be cost effective for endometriosis and associated pain, using a placeholder price of $7,000 per year. But the cost would be substantial for health budgets given the broad prevalence of the condition. 

Bills allowing pharmacies to substitute biosimilars for reference biologics have been enacted in all but six states. Interactive map notes state-by-state provisions.

FDA alerted Mylan to expect a complete response letter related to its ANDA for generic Advair. The company said it still could receive an approval prior to the standard 90-day time period after responding to the letter, because the application has a priority designation.

US FDA touts new evidence that high performance of pharmaceutical manufacturing sites on 10 quality maturity metrics correlates with robust pharmaceutical quality systems, operational excellence and good business. An FDA-funded study by St. Gallen also finds that low performance, on the other hand, correlates with manufacturing problems.

Generic industry officials ask that information requests and discipline review letters be issued during ANDA amendment assessments, but the US agency says they are not allowed in GDUFA II.

"We can say we are OTC but the botanicals are doing a lot of the heavy lifting," says Kelli Rodriquez, the firm's sales and marketing chief. Salicylic acid supports the acne treatment claim under an OTC monograph; leaving out benzoyl peroxide, an allergen for some consumers, also allows labeled use more frequently with a claim of faster symptom reduction.

Energy & Commerce's Republican and Democrat leadership ask FDA Commissioner Scott Gottlieb about work by the agency's Office of Criminal Investigations focused on opioid drugs, the office's staffing and its priorities. They note FDA's FY2018 appropriation included $94m for its work in international mail facilities.