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Real-World Evidence Increasingly Influences FDA Drug Approvals, Study Finds

Real World EvidenceDrug Approval
  • A systematic review of FDA approval documents from 2019-2021 found that 85% of new drug and biologic approvals incorporated real-world evidence (RWE) in some form, with the proportion increasing from 75% in 2019 to 96% in the first half of 2021.
  • Among 88 approvals that included RWE studies intended to support safety or effectiveness, 74% influenced FDA's benefit-risk assessment, with 9% serving as substantial or primary evidence and 65% providing supportive evidence.
  • FDA's documented feedback on RWE studies highlighted common issues including methodological problems, sample size concerns, and omission of patient-level data, emphasizing the importance of fit-for-purpose data selection and rigorous study design.

FDA Approves Breakthrough Twice-Yearly HIV Prevention Drug Lenacapavir with Nearly 100% Efficacy

Drug Approval
  • The FDA has approved lenacapavir, a breakthrough preventative treatment for HIV that offers nearly 100 percent protection from the virus and could change the course of the AIDS epidemic.
  • The new drug represents a significant advancement in HIV prevention with its twice-yearly dosing schedule, potentially improving patient adherence compared to daily prevention medications.
  • Despite the promising therapeutic breakthrough, deep cuts to health initiatives could hinder the rollout of this potentially game-changing HIV prevention treatment.

FDA Approves Zanubrutinib for Waldenström's Macroglobulinemia Treatment

Drug ApprovalRare Disease
  • The FDA has approved zanubrutinib (Brukinsa) for treating adult patients with Waldenström's macroglobulinemia, marking the second therapy specifically approved for this rare lymphoma.
  • The approval was based on the phase 3 ASPEN trial comparing zanubrutinib to ibrutinib, showing a higher very good partial response rate of 28% versus 19% respectively.
  • Zanubrutinib demonstrated improved tolerability compared to the first-generation BTK inhibitor ibrutinib across several clinically important side effects.
  • The drug is administered orally at either 160 mg twice daily or 320 mg once daily, offering patients a convenient single-agent treatment option.

A Study Comparing BGB-3111 and Ibrutinib in Participants With Waldenström's Macroglobulinemia (WM)

NCT03053440CompletedPhase 3
BeiGene
Posted 1/25/2017

UK Approves First COVID-19 Monoclonal Antibody Treatment as AstraZeneca Reports 77% Efficacy in Prevention Trial

Monoclonal AntibodyDrug Approval
  • The UK's MHRA approved Ronapreve, the first monoclonal antibody therapy specifically designed to treat and prevent COVID-19, developed by Regeneron and Roche.
  • The treatment combines two monoclonal antibodies administered by injection or infusion, working by binding to the virus and preventing respiratory system entry.
  • AstraZeneca simultaneously announced that its AZD7442 antibody combination reduced COVID-19 symptom risk by 77% in a trial of 5,172 participants, with over three-quarters having comorbidities affecting vaccine response.
  • Both treatments represent significant advances for vulnerable populations who may not respond adequately to COVID-19 vaccines alone.

FDA Approves Aduhelm: First Disease-Modifying Alzheimer's Treatment in Nearly Two Decades

Drug ApprovalMonoclonal AntibodyNeurology
  • The FDA granted accelerated approval to Biogen's aducanumab (Aduhelm) as the first disease-modifying Alzheimer's treatment in nearly two decades, despite divided expert opinions on its clinical efficacy.
  • Aduhelm, priced at $56,000 annually, works by reducing amyloid plaques in the brain and will require monthly intravenous infusions with comprehensive clinical and MRI monitoring for potential adverse effects.
  • As part of the accelerated approval, Biogen must conduct a Phase 4 confirmatory trial, while experts view this approval as a potential catalyst for developing more effective Alzheimer's treatments targeting multiple pathways.

A Study to Evaluate Safety and Tolerability of Aducanumab in Participants With Alzheimer's Disease Who Had Previously Participated in the Aducanumab Studies 221AD103, 221AD301, 221AD302 and 221AD205

NCT04241068TerminatedPhase 3
Biogen
Posted 3/2/2020

Multiple Dose Study of Aducanumab (BIIB037) (Recombinant, Fully Human Anti-Aβ IgG1 mAb) in Participants With Prodromal or Mild Alzheimer's Disease

NCT01677572TerminatedPhase 1
Biogen
Posted 10/5/2012

A Study of Aducanumab in Participants With Mild Cognitive Impairment Due to Alzheimer's Disease or With Mild Alzheimer's Disease Dementia to Evaluate the Safety of Continued Dosing in Participants With Asymptomatic Amyloid-Related Imaging Abnormalities

NCT03639987TerminatedPhase 2
Biogen
Posted 12/20/2018

221AD302 Phase 3 Study of Aducanumab (BIIB037) in Early Alzheimer's Disease

NCT02484547TerminatedPhase 3
Biogen
Posted 9/15/2015

221AD301 Phase 3 Study of Aducanumab (BIIB037) in Early Alzheimer's Disease

NCT02477800TerminatedPhase 3
Biogen
Posted 8/13/2015

FDA Approves Monjuvi in Combination with Lenalidomide for Relapsed/Refractory DLBCL

Drug ApprovalMonoclonal AntibodyOncology
  • The FDA has approved Monjuvi (tafasitamab-cxix) in combination with lenalidomide as the first second-line treatment for adult patients with relapsed or refractory diffuse large B-cell lymphoma who are not eligible for autologous stem cell transplant.
  • The approval was based on the Phase 2 L-MIND study, which demonstrated a 55% overall response rate with a 37% complete response rate and median duration of response of 21.7 months.
  • Approximately 10,000 patients are diagnosed annually in the United States with relapsed or refractory DLBCL who are not eligible for stem cell transplant, representing a significant unmet medical need.
  • Monjuvi is a humanized CD19-targeting monoclonal antibody that received accelerated approval and will be co-commercialized by MorphoSys and Incyte in the United States.

Open Label Study to Evaluate the Safety and Efficacy of Lenalidomide With MOR00208 in Patients With R-R DLBCL

NCT02399085CompletedPhase 2
MorphoSys AG
Posted 3/29/2016

Pertuzumab Regimen Shows Modest Survival Benefit in HER2-Positive Early Breast Cancer

Monoclonal AntibodyOncologyDrug ApprovalTargeted Therapy
• The APHINITY trial's 6-year analysis reveals that adjuvant pertuzumab with trastuzumab plus chemotherapy improved overall survival by 0.9% in HER2-positive early breast cancer patients, though not reaching statistical significance.
• Patients with node-positive disease showed the most significant benefit from the pertuzumab regimen, with a 4.5% absolute improvement in invasive disease-free survival at 6 years compared to placebo.
• The pertuzumab combination maintained a favorable cardiac safety profile with severe cardiac events occurring in less than 1% of patients, supporting its continued use in high-risk HER2-positive early breast cancer.

European Regulators Approve Lenacapavir, First Twice-Yearly HIV Prevention Injection

Drug Approval
  • The European Medicines Agency's advisory committee issued a positive opinion on lenacapavir for pre-exposure prophylaxis, marking the final regulatory hurdle before formal approval by the European Commission.
  • Lenacapavir demonstrated 100% efficacy in preventing HIV transmission during clinical trials, offering a twice-yearly injection alternative to daily prevention pills.
  • The approval comes as HIV cases rose 11.8% in Europe in 2023, with over 24,700 new diagnoses across the EU, Iceland, Liechtenstein, and Norway.
  • Gilead Sciences has committed to making generic versions available in 120 low-income countries, though concerns remain about equitable global access.

China Grants Conditional Approval to Seaweed-Derived Alzheimer's Drug Oligomannate

Drug Approval
  • Chinese regulators have granted conditional approval to Oligomannate, a seaweed-derived drug for mild to moderate Alzheimer's disease, marking the first new Alzheimer's therapy approval in nearly two decades.
  • The drug, developed by Shanghai Green Valley Pharmaceuticals, showed cognitive improvements in a Phase 3 trial of 818 patients with benefits observed as early as four weeks.
  • Unlike traditional approaches targeting brain protein buildup, Oligomannate works by modulating the gut microbiome to reduce neuroinflammation associated with Alzheimer's disease.
  • The company plans to launch a global Phase 3 trial in 2020 with sites in the United States, Europe, and Asia to seek international regulatory approval.

FDA Expands Real-World Evidence Framework to Accelerate Drug Approvals and Support Regulatory Decisions

Real World EvidenceDrug Approval
  • The FDA launched a comprehensive Real-World Evidence Program in December 2018 under the 21st Century Cures Act, allowing RWE to support drug approvals for new indications and population expansions.
  • Real-world evidence studies cost significantly less than randomized controlled trials, with postmarket cardiovascular outcomes trials for diabetes drugs costing around $250 million compared to much lower RWE study costs.
  • Electronic health record adoption has dramatically increased from 31% of hospitals in 2003 to 99% currently, and from 11% of office-based doctors in 2006 to 90% in 2017, creating vast new data sources for RWE generation.
  • The biopharmaceutical industry must establish rigorous standards for regulatory-grade data quality, standardized definitions, and new statistical models to ensure broader RWE implementation and regulatory acceptance.

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