CER Daily Newsfeed

FDA Explains When it Will Rescind Breakthrough Designations

(6/23, Michael Mezher, Regulatory Focus) reports “...The [FDA] provides examples of three scenarios in which it might rescind a product’s BTD, including when another drug is approved for an unmet need that was cited in the rationale for granting BTD. FDA notes that other drugs intended for the same unmet medical need that are granted accelerated approval will not typically be considered as reason for rescinding BTD. The other two scenarios in which FDA says it might rescind BTD include when ‘emerging data for the designated drug no longer support a finding that “preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies,”’ and when the drug’s sponsor halts a drug development program for the use the BTD was granted for.” Full

 

RWD in Clinical Trials: External Control Arms Take the Lead

(6/23, Mary Ellen Schneider, Regulatory Focus) reports “...Real-world data is ‘messy’ because it was created for a different purpose, explained Nancy Dreyer, PhD, chief scientific officer and senior vice president for IQVIA Real World Solutions. This means that using an external control arm will introduce some error, she said, but if the effect size is large enough the benefit or the harm will still show up. This makes external control arms useful options for new cancer treatments but not a good option for treatments likely to show small incremental benefit. ‘The smaller the expected benefit from the treatment, the riskier it is to use an external comparator because it is less likely to be informative,’ Dreyer said.” Full

 

COTA’s Miruna Sasu on Real-World Evidence: “The Ecosystem Has Changed”

(6/23, Mary Caffrey, Evidence-Based Oncology) reports “...[COTA Healthcare CEO Miruna Sasu, PhD, MBA:] One thing [the FDA] did that I really respect was ensuring the enablement of EMRs in general; that was a big deal. A second thing was the guidance on how RWD should be used for clinical trials and regulatory approvals; that was a big deal as well. That guidance being issued said, ‘We’re doing this work, and we are open to receiving external controls and databases, and so on.’ The third thing they can do, and should do, goes along with the EMR piece, now that we’re going into using EMRs for clinical trials and augmenting clinical trials. To ease the burden on both patients and providers, one area of expansion would be to capture the data at the provider level in such a way that it looks more like a clinical trial.” Full

 

Bringing Innovation to Value-Based Care at Scale, With an Assist From Technology

(6/23, Mary Caffrey, Evidence-Based Oncology) reports “...By some estimates, low-value care represents 30% of all cancer care spending, [Ravi Parikh, MD, MPP] said, but measuring it, pinpointing it, and eliminating it are tough to do. High-profile efforts, such as the Choosing Wisely campaign, and Medicare’s crackdown on low-value care have had mixed results. In the months before COVID-19 vaccines were available, the scenario that arose from the pandemic was one in which many experts thought low-value cancer care would be revealed: Providers and patients would weigh the risk of being in medical facility—and halting cancer’s progression—against the chances of contracting COVID-19. But, as Parikh explained, it’s not that simple. ‘We could just decrease utilization across the board,’ he said. ‘That might decrease low-value care, but it also might decrease some high-value cancer care.’” Full

 

Bringing Evidence Back into Fashion in European Drug Rules

(6/23, Peter O'Donnell, Applied Clinical Trials) reports “...[The European Confederation of Pharmaceutical Entrepreneurs (EUCOPE)] points out that orphan drugs and advanced therapies in particular, due to their smaller patient populations, rely on less conventional methodological approaches. The HTA discussions chaired by Dominique Le Guludec, the French official quoted above as a champion of comparative trials, provoked an instant response from supporters of a more liberal approach to evidence-generation. RWE4Decisions, a consortium of organizations with an interest in exploring the potential of RWE more extensively, publicly queried what guidance will emerge for the use of RWE in the upcoming joint HTA system in Europe, ‘since oncology and advanced therapies are priority areas, and often come with little evidence insofar as randomized controlled trials are not always possible.’” Full

 

UK Medicines Regulator Specs out Independent Future

(6/24, The Pharma Letter) reports “...One such strategic initiative is the new Innovative Licensing and Access Pathway (ILAP), an important new route designed to reduce the time to market for innovative medicines. Drugmakers who avail themselves of this route will benefit from enhanced input from the MHRA and other stakeholders, including the UK’s health technology watchdog, the National Institute for Health and Care Excellence (NICE).” Paid Subscription Required