FDA – Last Week in Review
Last week, the US Food and Drug Administration (FDA) made key decisions regarding the approval of multiple medications and accepted submissions of a few others. First, the agency approved the use of semaglutide as a first-line diabetes therapy. In addition, it accepted the application for brexpiprazole in treating agitation related to Alzheimer’s disease. It wasn’t the only application for the treatment of the otherwise intractable disease.
APAC Therapeutic Access and Financing Trends to Watch in 2023
The market access and reimbursement landscape in APAC is set to shift in 2023, according to a new interview with Jeff Weisel, APAC managing director for PRMA Consulting. Weisel notes that global economic forces will play a significant role in payer decision-making in APAC, with inflation and the strengthening dollar being counterbalanced by the availability of biosimilars.
Indian Pharma Industry Growth May Hit Drug Pricing and Regulatory Overhang Roadblocks
A report covered in Express Pharma predicts that growth in the Indian pharma market may be capped just shy of last year’s numbers. In its prediction, the report notes this may occur in response to pricing negotiations, ongoing supply chain disruptions, inflation, and regulatory pressure. However, the report is overall neutral-to-positive for the industry
FDA Approves Alzheimer’s Treatment by Biogen and Eisai
The US Food and Drug Administration (FDA) has approved Biogen and Eisai’s new Alzheimer’s drug, lecanemab. The drug is the first of its kind because it slowed cognitive decline in patients, a sticking point with the ill-fated Aduhelm that was pulled from the market last year. However, it is unclear whether payers will cover the therapy, which looks to be expensive.
Interview with Director of FDA’s CBER, Peter Marks
Ben Comer, Chief Editor of Life Science Leader, spoke with Peter Marks, director of the US Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research (CBER) last year to discuss the Center’s goals for the coming year. The first priority is working through a backlog of vaccine-related submissions and reviewing evidence from vaccines approved via Emergency Use Authorizations (EUAs).
2022 FDA New Drug Approvals Lowest in Six Years
The US Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) gave the nod to 37 new drugs for clinical use last year, a low after the past five years. However, this number was buffered by a slate of approvals by the Center for Biologics Evaluation and Research (CBER), reflecting a growing push for biologics.
Achieving EU Medical Device Regulation Compliance
Medical device companies must apply a coordinated approach to achieve compliance with the EU Medical Device Regulation (MDR). In a recent Life Science Leader article, learn key strategies for planning for MDR compliance. The first such strategy is the establishment of an effective leadership team that can synchronize activities company-wide.
FDA Accelerated Approval Reforms Makes the Cut on US Congress Government Spending Bill
The US Food and Drug Administration (FDA) got a win in the just-passed $1.7 trillion government spending bill. The bill includes provisions that would allow the agency to ask applicants start confirmatory studies before they are granted accelerated approval. In addition, companies receiving accelerated approval would need to provide data from confirmatory studies every six months.
Spanish Agency for Medicines and Health Products Expedites Drug National Code Application Process
The Spanish Agency for Medicines and Health Products (AEMPS) has implemented changes to the application process for national code (NC) of centrally authorized medicinal products. Applicants may now send their NC application once they and the relevant regulatory agencies finalized texts. This and other changes are intended to speed up the application process.
Aligning Cancer Therapeutic Evidence Between EMA and EU HTAs
Many of the health technology assessment agencies (HTAs) of EU member nations have evidence requirements for cancer drugs that are different than those of the European Medicines Agency (EMA). This disparity often delays patient access. In a new article in Value in Health, authors break down these different requirements to highlight the need for alignment between EMA and EU HTA requirements.
NICE Calls for Real-World Evidence on Parkinson’s Disease Medical Devices
The UK’s National Institute for Health and Care Excellence (NICE) recently recommended that the National Health Service (NHS) collect real-world evidence (RWE) regarding a series of medical devices used in the management of Parkinson’s disease. The devices all serve to record information about the wearer’s Parkinson’s-related motor symptoms.
EMA Leans into Push to Improve Real-World Data Sources for Regulatory Decision-Making
Officials from the European Medicines Agency (EMA) and member state national health technology assessment (HTA) agencies recently announced they were going forward with plans to improve the quality of real-world data (RWD) resources for use in regulatory decision-making. The EMA and HTA bodies established a Big Data Steering Group, which has already endorsed documents that will go through a phase of public consultation.
How Will FDA Real-World Data Guidances Affect Patient Advocacy Groups?
With the US Food and Drug Administration’s (FDA) growing focus on the use of real-world data (RWD) and real-world evidence (RWE) in decision-making, patient advocacy groups must adapt to the new regulatory landscape. Fortunately, the guidances contain information to guide advocacy organizations in setting up and managing their patient registries.
PTC Therapeutics Nets Brazilian Approval for Familial Partial Lipodystrophy Drug
ANVISA, Brazil’s health regulatory body, has approved PTC Therapeutics’ Waylivra for the treatment of family partial lipodystrophy (FPL). The approval marks the first of its kind worldwide for the disease. The drug is already approved in Brazil and the EU for the treatment of patients living with Familial Chylomicronemia Syndrome.
The Latest Shifts in the FDA Accelerated Approval Pathway
The US Food and Drug Administration (FDA) is actively retooling its accelerated approval pathway, with or without actions by Congress. GSK announced yesterday, November 22nd, that the agency asked it to pull a multiple myeloma drug based on negative post-confirmatory trial data. Unlike previous situations of the sort, the agency moved in just over two weeks.
Podcast Q&A: Streamlining Health Technology Assessments by Automating Literature Reviews
This podcast segment features the Q&A from DistillerSR's November 17, 2022 webinar and features a panel of experts, including Chris [...]
What’s the State of Market Access in Denmark After Five Years with the Danish Medicines Council?
Denmark established the Danish Medicines Council (DMC) back in 2017 to help make assessment of medicines faster, transparent, and more consistent. In a recent Amerisource Bergen article, Flemming Axelsen, MSC, looks back at the past five years of the DMC, explaining its successes, failures, and the lessons that can be gleaned from both.
NICE Makes New Round of COVID-19 Drug Approval Recommendations
The UK’s NICE has announced its recommendations of approval for three COVID-19 drugs and the rejection of five others based on value and efficacy data. Paxlovid, RoActemra, and Olumiant from Pfizer, Roche, and Eli Lilly, respectively, made the cut. Uncertain efficacy data and pricing issues sank the applications of drugs from Merck, Gillead, GSK, Regeneron, and AstraZeneca
Report Puts Forward Four Steps to Accelerate Drug Access in Canada
A recent report from the Conference Board of Canada posits four steps to speed up access to cancer therapeutics in Canada. First, they recommend changing the current health technology assessment (HTA) practices to provide additional pathways that will allow patients to access drugs while payers, pharma, and regulatory bodies work out reimbursement strategies.
Pfizer is a Fan of FDA’s Real-Time Oncology Application Review Process
Pfizer has expressed positivity towards the US Food and Drug Administration’s (FDA’s) Real-Time Oncology Review (RTOR) program. The RTOR will allow data submissions earlier in the review process in order to accelerate future decision-making and market access. Although the Alliance for Regenerative medicine has expressed concerns about the program, Pfizer is in support.
Endpoint Outcomes Adopts Lumanity Branding After Acquisition
Lumanity’s most recent acquisition, Endpoint Outcomes, will be folded into the company’s overall brand. The move fully integrates Endpoint’s health economics and outcomes research (HEOR) expertise into Lumanity, expanding its reach into the HEOR field, with an emphasis on the US market. In addition, the Lumanity’s HEOR team will broaden its services in Europe to help meet HTA processes and payer decision-making.
Flatiron ResearchX Session – What have we learned: Adapting to RWE regulatory guidance and experience – October 18th
Countries are increasingly releasing guidance after guidance in the use of real-world evidence (RWE) in regulatory decision-making. With the growing demand for RWE from industry and government alike, preparing for regulatory applications supported by RWE is more important than ever. On October 18th, Flatiron is hosting its next ResearchX Session.
Webinar: Fit for Local Context? Establishing or Improving Deliberative Processes for HTA
Pharma, biotech, and other stakeholders are increasingly being involved in deliberative processes for health technology assessment decisions, but navigating these interactions is not always clear. ISPOR and Health Technology Assessment International (HTAi) recently released a guidance for deliberative processes. This guidance and the thought processes behind it will be laid out in an upcoming webinar on September 30th.
Gilead Science’s Long-Acting HIV Drug Secures Win With EU Approval
Gilead Science has received approval from the European Commission for its HIV drug lenacapavir. The medication is used to treat multi-drug resistant HIV and is injected once every six months. This approval marks a win for Gilead, as the drug was placed on a temporary clinical by the FDA over the risk of contamination from glass shards.
New MDCG Guidance on Conformity Assessment and Notified Bodies
The Medical Device Coordination Group (MDCG) has released a new guidance pertaining to conformity assessment and notified bodies. In part 1 of the guidance, the MDCG seeks to support the process of designating notified bodies in NANDO. In the second part, the guidance goes over the notification process in NANDO and the objection period.
