INSIGHTS ON CELL & GENE CLINICAL TRIALS

• 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

  With statistical simulations have shown that as few as one in three trials using the 3+3 design succeed in identifying the maximum tolerated dose and concerns that this method of dose escalation may result in a high percentage of patients being treated at subtherapeutic doses their is new focus on innovative phase 1 trials.

• 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.

• CAR T-Cell Therapy In Non-Hodgkin’s Lymphoma: 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, many key players are now pursuing development of therapies in earlier treatment settings and in other non-Hodgkin’s lymphoma subtypes, such as 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.

• The Journey To Get An Ovarian Cancer T Cell Therapy Into Clinical Trials

  Daniel J. Powell Jr., Ph.D., and his research team at the University of Pennsylvania has launched a new clinical trial to treat ovarian cancer with a pioneering therapy involving genetically modified T cells. The trial, which has treated its first patient, almost didn’t happen.

• Takeaways from LSPA’s CRO & CMO Summit

  Last week I attended Life Sciences Pennsylvania’s (LSPA) CRO & CMO Summit, which was held at West Pharmaceutical Services. For those of you unfamiliar with it, LSPA is the trade association for the Commonwealth’s life sciences community. LSPA represents biotechnology, medical device, diagnostic, pharmaceutical, research, and investment entities, along with many service providers that support the industry.

• Regulatory Oversight On Gene Therapy In The U.S. And EU

  Decades of painstaking research have recently begun to yield gene therapy products that are delivering meaningful benefits to human health. With the rapid evolution of the gene therapy field, regulatory agencies have been working to keep pace with these scientific and clinical breakthroughs. An understanding of the regulations and guidance documents reflecting regulator current thinking surrounding gene therapies is essential to success.