Sue Sutter

<p><span>Regulatory uncertainty and the biopharma industry’s longstanding aversion to risk are hindering adoption of artificial intelligence and machine learning in drug and biologic development, panelists said at a recent US FDA/CTTI workshop.</span></p> <p><span>&nbsp;</span></p>

<p><span>Covis’ preterm birth prevention drug was withdrawn in April 2023. The agency now&nbsp;is considering whether hydroxyprogesterone caproate should be added to the list of products withdrawn or removed from the market after being found to be unsafe or not effective.</span></p>

<p><span>Determining what falls within the statutory definition of ‘biological product’ is an interpretative question that courts, rather than the agency, must resolve, Lilly said in a lawsuit repeatedly citing the US Supreme Court’s June decision in <em>Loper Bright</em>. </span></p>

<p><span>A real-world evidence study also does not meet the regulatory standard for an adequate and well-controlled clinical investigation, the agency said. The FDA should allow the accelerated approval drug to remain available like other treatments that failed their confirmatory trials, Intercept said.</span></p> <p><span>&nbsp;</span></p>

<p><span>Obeticholic acid has not confirmed clinical benefit and the benefit-risk profile is not favorable in primary biliary cholangitis, the FDA advisory committee said. The agency must now decide whether to keep Ocaliva on the market with new study requirements or seek withdrawal.</span></p>

<p><span>Studies should enroll a representative subgroup of US patients based on the incidence or prevalence of cancer in the US, and the comparator arm should use the US standard of care when possible, the FDA says in draft guidance.</span></p>

<p><span>As amended, the Give Kids A Chance Act would renew the rare pediatric disease voucher program for five years and give the FDA new authority to bring enforcement action when pediatric studies are overdue. However, it would not remove the exemption from pediatric study requirements for non-cancer orphan drugs, which Democrats had sought.&nbsp;</span></p>

<p><span>Draft recommendation for investigators to maintain a task log of activities that local health care providers perform was removed from final guidance on decentralized clinical trials. Other changes include more detail on US FDA oversight and what HCPs can and cannot do in such studies.</span></p>

<p><span>Draft guidance recommendations on setting enrollment goals lead PhRMA and BIO to seek assurances of FDA flexibility absent reliable disease prevalence data. </span></p>

<p><span>The Cardiovascular and Renal Drugs Advisory Committee voted 10-6 that efficacy had been shown for the ultra-rare disease, but even panelists in the majority questioned whether the product satisfied the threshold requirement for an adequate and well-controlled study.</span></p>