INSIGHTS ON CELL & GENE CLINICAL TRIALS

• Real World Evidence (RWE) In Regulatory Decision Making: Establishing Study Credibility And Totality Of Evidence

  This blog shares some of the learnings from the recent Duke-Margolis webinar entitled, "Bolstering RWE Study Credibility and Its Role in a Totality of Evidence Approach".

• Keeping A Focus On Rare Disease

  Every last day of February is Rare Disease Day, the biggest day of the year for the rare disease community. Read some highlights from this year's event attended by researchers, physicians, regulators, and patient advocates.

• REMS Technology Of Today And Tomorrow - Transformative Solutions To Help Reduce Burden And Improve Safety

  During the 12^th REMS Summit in Virginia, UBC’s Natalie O’Donnell, Corporate Vice President, Safety, REMS, and Strategic Engagement and Justin Wilson, Director, Software Development and Technology, presented a session on the impact technology has had on REMS and the potential effect it can have in the future. 

• 6 Reasons Why Early Oncology Drug Trials Fail (And How To Avoid Them)

  Unfortunately, not all oncology trials succeed. In fact, the phase success and likelihood of approval (LOA) rates for oncology are the lowest across major therapeutic areas. Although there are many reasons for these relatively poor success rates, issues determining dose, schedule, and regimen in early phase trials are among the most prominent.

• Key Considerations When Designing A Phase 1 Oncology Trial

  Selecting a safe starting dose must be balanced against the proportion of patients treated at sub-therapeutic doses. This is especially important for agents that demonstrate minimal toxicity in preclinical testing or for drugs that are unlikely to ever reach maximum tolerated dose. This approach has the potential to reduce the number of dose escalations while preventing patients from being treated at overly toxic doses that lack incremental biological activity.

• 5 Reasons To Consider APAC For Clinical Trials

  Small and mid-size biopharma companies in the U.S. and Europe are under intense pressure to find more efficient and cost-effective ways to commercialize their products. In an increasingly competitive clinical trial environment, sponsors have begun to shift their focus to the Asia-Pacific (APAC) region for their studies.Here are five reasons why the APAC region may be a good fit for your global clinical trial.

• Non-Hodgkin’s Lymphoma CAR T-Cell Therapy: Where Do We Go From Here?

  Following the success of Kite/Gilead’s Yescarta and Novartis’ Kymriah in treating relapsed or refractory diffuse large B-cell lymphoma, key players are pursuing therapies in earlier treatment settings and in mantle cell lymphoma, chronic lymphocytic leukemia, and follicular lymphoma.

• The Critical Role Registries Play In Rare Diseases

  UBC's Dr. Don Gabriel, Medical Oncology & Scientific Strategy and Colleen Valenzuela, Clinical Operations recently appeared in a trade publication speaking to the importance of registries in rare diseases.

• More On SPEAR T-Cell Platform, Initial Responses In Four Solid Tumor Indications

  At JPM Healthcare Conference, Adaptimmune reported two confirmed Partial Responses (PRs) — one in a patient with liver cancer and one in a patient with melanoma. The company also reported two unconfirmed PRs — one in a patient with gastro‑esophageal junction cancer and one in a patient with head and neck cancer. These data further confirm the potential of Adaptimmune’s SPEAR T-cell platform for patients with multiple solid tumors. Data were previously reported showing compelling efficacy with ADP-A2M4 in synovial sarcoma.

• Establishing Risk-Based Monitoring Within A Quality-Based System As “Best Practice” For Clinical Studies

  This report based on a survey of ACRO members reveals that Risk-Based Monitoring (RBM) makes clinical trial quality review more efficient and effective. It found that when a company reviews data through a centralized system using the RBM model, CROs and technology companies are better able to detect quality issues earlier and make rapid corrections at the site level. This type of approach is now central to ensuring the safety of patients in clinical trials, and is expected to continue to grow in importance as clinical trials becomes more numerous and complex.