ASCO 2015 Highlights

On April 25, 2016, an FDA advisory committee voted not to recommend the approval of eteplirsen, an experimental drug that targets one of many genetic mutations causing Duchenne muscular dystrophy (DMD), a deadly degenerative disease that has no cure. After agreeing to study the real-world effects of eteplir­sen, the FDA advisory committee rejected findings that patients who have been taking eteplirsen since 2011 were still able to walk because the clinical data did not meet the FDA requirements for a well-controlled study.

In the United States, the standard of care for locally advanced bladder cancer after radical cystectomy is to “consider” adjuvant chemotherapy and ad­juvant radiation. Results of a 3-arm randomized clinical trial showed that adjuvant radiation therapy alone or combined with chemotherapy (ie, chemoradiotherapy) did not significantly improve disease-free survival compared with adjuvant chemotherapy alone. However, the findings hint at benefits for chemoradiotherapy that should be studied further. Brian Baumann, MD, a radiation oncology resident at the University of Pennsylvania, Philadelphia, presented the findings at the 2016 Genitourinary Cancers Symposium.

High-risk patients with metastatic renal-cell carcinoma (RCC) and venous tumor thrombus derived no benefit from cytoreductive nephrectomy and should be evaluated for clinical trials of systemic therapy, suggested a retrospective multicenter review.

The Individual Patient Expanded Access Application, which is designed to streamline the application procedure for individual patients who apply for expanded access to investigational therapeutics, including expanded access to drugs that are not in clinical trials, was recently updated by the FDA. Form FDA 3926 authorizes expanded access to investigational drugs for patients with serious or life-threatening conditions who have no therapeutic options.

Opposition to proposed changes to the Medicare Part B payment for prescription drugs continues to grow since the Centers for Medicare & Medicaid Services (CMS) announced its plan in March 2016.

Early intervention utilizing a team approach is important to successful palliative care, agreed a multidisciplinary panel convened at the 2016 National Comprehensive Cancer Network (NCCN) annual conference.

“Value” in healthcare has been defined as outcomes relative to cost. Determining the value of a healthcare benefit remains difficult and complex for many stakeholders. With the increasing cost of cancer therapies, value has become an important topic of conversation for patients, healthcare providers, self-funded plan sponsors (employers), and payers.

Daratumumab in combination with lenalidomide and dexamethasone achieved a 63% reduction in progression-free survival (PFS) compared with lenalidomide and dexamethasone alone in patients with multiple myeloma who had received at least 1 previous line of therapy, according to study results presented at the 2016 European Hematology Association Annual Congress meeting.