In this week’s news, a proposed US FDA budget hike, CAR-T may be covered on Medicare, deals in the early phase CRO space and more…
US FDA set for funding boost under proposed budget
First up is a potential US FDA funding increase in a new budget proposal.
Bills passed last week would give the agency $3.068 billion in discretionary funding, up $269 million on 2018, if US President Donald Trump signs them into law later this month, as he has promised.
An explanatory note says the FDA funding increase will support a range of activities, from drug reviews and inspections through to combating the opioid epidemic and promoting US drug and device production.
Funds are also earmarked for various data and digital health projects, as well as to support cancer and rare disease research.
Pressure group the Alliance for a Stronger FDA welcomed the proposed increase, writing it is “extremely pleased with the conference bill and hopeful that it will become law shortly.”
US considering covering CAR-T on Medicare
CAR-T cancer therapies would be covered by Medicare under a plan put forward by the agency that runs the US Government’s health programme.
Centers for Medicare and Medicade Services (CMS) suggested the costly meds would be covered if offered by an approved registry or clinical study, in which patients are monitored for at least two years post-treatment.
The agency said, “Evidence from the registries and studies would help CMS identify the types of patients that benefit from CAR T-cell therapy, informing a future decision by the agency regarding the types of cases in which Medicare would cover the treatment with no registry or trial requirement.”
The CMS called for feedback on the proposal.
CAR-T, or chimeric antigen receptor T-cell, therapies are immune system cells that have been engineered to target tumours more effectively. At present there are only two CAR-T therapies approved in the US: Novartis’ Kymriah, which costs $475,000; and Gilead’s Yescarta, which costs $373,000.
Reuters spoke to several clinics which welcomed the proposal on the basis the current lack of a payment pathway has left them with unpaid insurance claims in the tens of millions of dollars for CAR-T therapies.
Other FDA news; CGT designation explained; creates bulk API list
The FDA has issued draft guidelines clarifying when and how drug firms can seek competitive generic therapy (CGT) designation for their products.
The CGT designation was introduced in 2017 to encourage industry to make generic versions of drugs without competition. To date 100 candidates products have been awarded CGT status.
Last week also saw the FDA issue a list of bulk drug substances compounders, including those registered as outsourcing facilities, can work with.
Agency Commissioner Scott Gottlieb said the aim was to balance the need for access to medicines with patient safety. See his full statement here.
Italy suggests WHO should ask pharma for drug cost and price data
The Italian Government has suggested the World Health Organisation (WHO) should play a role in drug and vaccine price transparency efforts.
Earlier this month Giulia Grillo, Italy’s Minister of Health, wrote to WHO Director General Tedros Adhanom Ghebreyesus suggesting the Swiss body should require drug makers to disclose R&D and production costs, as well as prices charged for medicines and vaccines.
She said, “International action is required to improve the transparency in the reporting of prices, R&D costs and production costs of medicines and vaccines including public sources.”
EMA outlines big plans for big data
The EMA has shared its thoughts on how ‘big data’ will be used in drug development to help firms understand the information they should provide.
The agency issued recommendations in a report last week, explaining the aim is to look at how data should be sourced, standardised and submitted.
“Massive amounts of data are generated on a daily basis through wearable devices, electronic health records, social media, clinical trials or spontaneous adverse reaction reports.
“There is no doubt that insights derived from this data will increasingly be used by regulators to assess the benefit-risk of medicines across their whole lifecycle” it wrote.
Developments in the CRO space
Last week saw some notable developments in the early phase development services space.
Welsh CRO Simbec Orion was acquired by a private equity investment group called CBPE Capital. Private equity funds typically hold investments for five to seven years before seeking a buyer or taking the asset public.
Meanwhile, Charles River acquired early-stage focused CRO Citoxlab. The French contractor has capabilities in preclinical drug safety testing.
Also in the news
The Medicine maker provided coverage of pharma’s plans for Brexit.
The Hill reported details of an investigation of the role anti-vaccine campaigners have played in recent measles outbreaks in the US. Reuters looked at how low vaccination rates may have contributed to the problem. And, according to Bloomberg, beleaguered social media network Facebook says it may remove anti-vaccine adverts.
Industry group PhRMA has urged US trade officials to address what it calls “serious and growing market access and intellectual property barriers” that are preventing its members from accessing “top overseas markets.”
STAT looked at what happens to biotech start-ups after the initial funding rounds when the hard work begins.
GEN looked at trends in the evolution of filtration technologies used in bioprocessing.
Israeli drug firm Teva is to sell 11 manufacturing sites this year according to Biopharmadive.