DIA: Driving Insights to Action

EFPIA Shares Industry View of EMA Regulatory Framework

Scientific advancements in pharmaceutical R&D are simultaneously benefiting patients and challenging industry and regulatory frameworks throughout Europe. “The pharmaceutical R&D of today is driven by groundbreaking innovation which has the potential to transform patient treatments and improve patient outcomes, as we move more and more from treating symptoms to really treating the underlying cause of diseases,” explains Dr. Nathalie Moll, Director General of the European Federation of Pharmaceutical Industries and Associations (EFPIA). “Developing this new generation of treatments raises new and challenging questions of our regulatory system, underlying the need for evolution to adapt to such advanced technologies.”

Time to Press the Pause Button on Speedy Drug Approvals?

New drugs are getting through the FDA approval process faster. According to a study published in January 2020, the review time for standard drug applications has decreased from more than 36 months in 1983 to approximately ten months in 2018. “It can be a good thing for drugs to reach the market faster, if those drugs offer large benefits. But often drugs offer only very small benefits over existing treatments,” says Dr. Jonathan Darrow, pharmaceutical policy expert and Assistant Professor at Harvard Medical School in Boston. “The system we have is a patchwork of various policies,” explains Dr. Joshua Sharfstein, former FDA Principal Deputy Commissioner, now Vice Dean and Professor at Johns Hopkins Bloomberg School of Public Health in Baltimore. “We should take a step back and say, ‘Are there ways to align incentives more so we have more money to spend for treatments that make a huge difference for people who need them?’”

Reach More Patients While Improving Data Quality?

Digital tools that revolutionize information and communication technology are increasingly employed to transform the quality and quantity of clinical trial data. “The ability for us to reach patients in geographic locations and in demographic groups that the current system is missing is arguably the most important role of these technologies,” suggests Lauren Oliva, Global Regulatory Policy Lead for New Technologies, Biogen. “With the increased quantity of data, you do increase the quality to some extent in terms of your reach and the types of things that you could measure now that you wouldn't be able to measure in the past,” explains Josh Cosman, Principal Engineer, Digital & Quantitative Medicine, Biogen. Lauren will Chair the Drug Development Tools in a Digital Era session at DIA Europe 2020.

ATMPs May Deliver on Promise of Individualized Medicine

FDA’s Center for Biologics Evaluation and Research (CBER) is responsible for assuring the safety and efficacy of biological products including vaccines, allergenic products, blood and blood products, and cellular tissue and gene therapies. “This area of advanced therapy medicinal products (ATMPs) is just an incredibly exciting one to be working in right now,” explains Center Director Dr. Peter Marks. “We have our work cut out for us here, but to me it's wonderful work to have to do because it shows how fast the science is advancing, how fast hopefully the technology will advance, to bring these medical benefits to patients.”

Relevance Will Put Europe on Investment Fast Track

The DIA Europe 2020 Keynote Address will focus on the intersections of science and business as explored by a scientist turned investor, Mr. Joep Muijrers, who currently serves as CFO of PureTech, a listed biopharmaceutical company that focuses on developing medicines for devastating diseases including cancer, CNS disease, and immunological disorders. “What is the value of a product that, quite often against the odds in this sector, makes it and gets to patients and then doesn't make a dent whatsoever – it's not being used. What's the relevance of that?” he asks. “Put it this way: In this industry, we tell the end user what's good for them. Isn't that crazy?”