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The Rising Importance of Real-World Data and Evidence in Pharmaceutical R&D

Real World Evidence
  • Real-World Data (RWD) and Real-World Evidence (RWE) are transforming pharmaceutical R&D by complementing traditional randomized clinical trials with data collected from routine healthcare settings.
  • Regulatory agencies worldwide, including the FDA and EMA, are increasingly accepting RWE for drug approvals, label expansions, and post-marketing surveillance, potentially accelerating patient access to treatments.
  • RWE offers advantages including larger sample sizes, greater external validity, and insights into real-world effectiveness, though challenges remain in data quality, standardization, and privacy protection.

FDA Approves Leqvio as First-Line Monotherapy for Cholesterol Management

Drug ApprovalReal World Evidence
  • The FDA has approved Novartis' Leqvio (inclisiran) for use as monotherapy alongside diet and exercise to reduce LDL cholesterol in adults with hypercholesterolemia, removing the previous requirement for concurrent statin therapy.
  • The twice-yearly injectable treatment addresses a critical unmet need, as up to 80% of atherosclerotic cardiovascular disease patients fail to reach guideline-recommended LDL cholesterol targets below 70 mg/dL.
  • Real-world data from the V-INITIATE trial demonstrated that early addition of Leqvio to statin therapy achieved 60% LDL cholesterol reduction compared to 7% with usual care, with 81.8% of patients reaching target levels versus 22.2% in the control group.

Trial to Assess the Effect of Long Term Dosing of Inclisiran in Subjects With High CV Risk and Elevated LDL-C

NCT03814187CompletedPhase 3
Novartis Pharmaceuticals
Posted 4/16/2019

A Randomized Study to Evaluate the Effect of an "Inclisiran First" Implementation Strategy Compared to Usual Care in Patients With Atherosclerotic Cardiovascular Disease and Elevated LDL-C Despite Receiving Maximally Tolerated Statin Therapy (VICTORION-INITIATE)

NCT04929249CompletedPhase 3
Novartis Pharmaceuticals
Posted 6/25/2021

Almirall Launches First Dermatology Study to Assess Patient Wellbeing as Primary Endpoint in Psoriasis Treatment

Monoclonal AntibodyReal World Evidence
  • Almirall has launched the POSITIVE study, the first dermatological research to assess patient wellbeing as a primary endpoint using the WHO-5 questionnaire in 780 adults with moderate-to-severe psoriasis.
  • The 24-month real-world evidence study across nine European countries evaluates tildrakizumab treatment impact on overall wellbeing, family environment, and physician satisfaction.
  • Nearly 77% of psoriasis patients report the disease negatively affects their daily activities and wellbeing, with up to 25% experiencing depression, highlighting the need for holistic treatment approaches.
  • The study represents a paradigm shift from traditional disease burden assessment to positive treatment goals that promote good health and wellbeing in dermatology care.

Real-World Study Shows Axicabtagene Ciloleucel Outperforms Tisagenlecleucel in Relapsed/Refractory DLBCL

CAR-TReal World Evidence
  • A large French real-world study of 809 patients with relapsed/refractory DLBCL found axicabtagene ciloleucel (axi-cel) demonstrated superior efficacy compared to tisagenlecleucel (tisa-cel) with significantly higher response rates and improved survival outcomes.
  • After propensity score matching of 418 patients, axi-cel achieved 80.4% overall response rate and 60.3% complete response rate versus 66.0% and 42.1% respectively for tisa-cel, with one-year progression-free survival of 46.6% versus 33.2%.
  • The superior efficacy of axi-cel came with increased toxicity, including significantly higher rates of severe immune cell-associated neurotoxicity syndrome (13.9% vs 2.9%) and prolonged hematological toxicity.
  • This comprehensive analysis from the DESCAR-T registry provides the first real-world evidence of overall survival advantage for axi-cel over tisa-cel in third-line or later treatment of DLBCL.

French Register Of Patients With Hemopathy Eligible For CAR-T Cell Treatment (DESCAR-T)

NCT04328298Recruiting
The Lymphoma Academic Research Organisation
Posted 12/19/2019

Real-World Evidence Shows Immune Checkpoint Inhibitors Benefit NSCLC Patients with Poor Performance Status

Real World Evidence
  • Real-world data from 1,395 NSCLC patients with ECOG performance status ≥2 demonstrates significant overall survival benefit with immune checkpoint inhibitor treatment compared to no treatment, regardless of PD-L1 expression levels.
  • For patients with PD-L1 expression ≥50%, pembrolizumab monotherapy achieved median survival of 7.1 months versus 2.7 months with no treatment, with adjusted hazard ratio of 0.39.
  • In patients with low PD-L1 expression (<50%), pembrolizumab monotherapy showed similar overall survival and progression-free survival outcomes compared to combination pembrolizumab plus chemotherapy.
  • Chinese real-world study of 351 NSCLC patients found no significant efficacy differences between different PD-1 inhibitors, with overall median progression-free survival of 9.5 months and objective response rate of 47.3%.

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.

Lorlatinib Shows Promising Efficacy in ALK-Rearranged NSCLC with 21.8 Month Median PFS

Real World EvidenceOncologyTargeted Therapy
  • A real-world study of lorlatinib in ALK-rearranged non-small cell lung cancer demonstrated a median progression-free survival of 21.8 months, significantly higher than in previous trials.
  • The objective response rate was 43% with disease control achieved in 94% of patients, while 81% of patients with brain metastases showed objective response for intracranial lesions.
  • Patients experiencing adverse events, particularly hypercholesterolemia and edema, had significantly better outcomes than those without side effects, suggesting a potential correlation between drug exposure and efficacy.

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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