Author Interviews, Breast Cancer, Cancer Research, Genetic Research, Novartis / 03.06.2019

MedicalResearch.com Interview with: [caption id="attachment_49521" align="alignleft" width="170"]Fabrice André, MD, PhD Research director and head of INSERM Unit U981 Professor in the Department of Medical Oncology Institut Gustave Roussy in Villejuif, France Global SOLAR-1 Principal Investigator. Dr. Fabrice André[/caption] Fabrice André, MD, PhD Research director and head of INSERM Unit U981 Professor in the Department of Medical Oncology Institut Gustave Roussy in Villejuif, France Global SOLAR-1 Principal Investigator. MedicalResearch.com: What is the background for this study? How does Piqray®  differ from other treatments for this type of advanced breast cancer? 
  • The US Food and Drug Administration (FDA) approved Piqray® (alpelisib, formerly BYL719) in combination with fulvestrant for the treatment of postmenopausal women, and men, with hormone receptor positive, human epidermal growth factor receptor-2 negative (HR+/HER2-), PIK3CA-mutated, advanced or metastatic breast cancer, as detected by an FDA-approved test after disease progression following an endocrine-based regimen.
  • Piqray is the first and only combination treatment with fulvestrant specifically for postmenopausal women, and men, with HR+/HER2- advanced or metastatic breast cancer with a PIK3CA mutation following progression on or after an endocrine-based regimen, bringing a biomarker-driven therapy option to this population for the first time.
  • Advanced breast cancer is incurable, and patients with all types need more treatment options. With this approval, physicians can now use an FDA-approved test to determine if their patients’ HR+/HER2- advanced breast cancer has a PIK3CA mutation and may be eligible for treatment with Piqray plus fulvestrant combination therapy. 
ASCO, Author Interviews, Cancer Research, J&J-Janssen, NEJM, Prostate Cancer / 01.06.2019

MedicalResearch.com Interview with: [caption id="attachment_49474" align="alignleft" width="142"]Dr. Kim Chi. MDProfessor of MedicineMedical Oncologist and Medical Director at BC Cancer – VancouverUniversity of British Columbia,Principal Investigator of the TITAN Study. Dr. Kim Chi[/caption] Dr. Kim Chi. MD Professor of Medicine Medical Oncologist and Medical Director at BC Cancer – Vancouver University of British Columbia, Principal Investigator of the TITAN Study. MedicalResearch.com: What is the background for this study? Response: For more than 70 years, androgen deprivation therapy (ADT) has been the standard of care therapy for patients with metastatic prostate cancer. The Phase 3 TITAN study looked at adding apalutamide (®®®®) to ADT compared with placebo plus ADT in a broad group of patients with metastatic castration-sensitive prostate cancer (mCSPC), regardless of disease volume or prior docetaxel treatment history. Metastatic castration-sensitive prostate cancer is prostate cancer that still responds to androgen deprivation therapy and has spread to other parts of the body. Patients with mCSPC tend to have a poor prognosis, with a median overall survival (OS) of less than five years, underscoring the need for new treatment options. The dual primary endpoints of this study were overall survival and radiographic progression-free survival (rPFS). 
Author Interviews, Pharmaceutical Companies, Prostate, Urology / 31.05.2019

MedicalResearch.com Interview with: [caption id="attachment_49467" align="alignleft" width="160"]Steven A. Kaplan, M.D., FACS Professor of Urology Director, The Men's Health Program Icahn School of Medicine at Mount Sinai Dr. Kaplan[/caption] Steven A. Kaplan, M.D., FACS Professor of Urology Director, The Men's Health Program Icahn School of Medicine at Mount Sinai MedicalResearch.com: What is the background for this study? Response: PLUS is the first large-scale trial conducted in North America and Europe specifically designed to study the effects of mirabegron in controlling residual symptoms of urinary urgency and frequency in men with benign prostatic hyperplasia (BPH) using common agents such as tamsulosin (Flomax). We explored whether mirabegron (Myrbetriq), an agent approved for the treatment of overactive bladder (OAB), improved patient outcomes when added to tamsulosin. This was a randomized, double-blind, placebo-controlled, multi-center study enrolling 715 male patients 40 years of age and older.
Author Interviews, Boehringer Ingelheim, NEJM, Pharmaceutical Companies, Pulmonary Disease / 30.05.2019

MedicalResearch.com Interview with: [caption id="attachment_49455" align="alignleft" width="200"]Donald Zoz, M.D.,Senior associate directorClinical Development & Medical AffairsBoehringer Ingelheim Pharmaceuticals, Inc. Dr. Zoz[/caption] Donald Zoz, M.D., Senior associate director Clinical Development & Medical Affairs Boehringer Ingelheim Pharmaceuticals, Inc. MedicalResearch.com: What is the background for this study? How does nintedanib differ from other treatments for SSc-ILD? What are the main findings?  Response: SENSCIS is a Phase III double-blind, randomized placebo-controlled trial that included 576 patients in 32 countries. It is the largest trial to have been conducted in patients with systemic sclerosis associated interstitial lung disease (SSc-ILD). The primary endpoint was the annual rate of decline in forced vital capacity (FVC) over 52 weeks. At the end of the 52-week trial, patients receiving nintedanib had an adjusted annual rate of decline in FVC (mL/year) of -52.4 with nintedanib versus -93.3 with placebo (absolute difference 41.0mL/year [95% CI 2.9, 79.0]; p=0.04). This corresponds to a relative difference of 44% reduction in lung function decline. There are currently no approved treatments for SSc-ILD., BI conducted the SENSCIS study to evaluate in SSc-ILD patients the impact of nintedanib. Nintedanib, a selective tyrosine kinase inhibitor, is an antifibrotic agent. Results of the study, which were published in The New England Journal of Medicine and presented at the American Thoracic Society (ATS) International Conference, showed that nintedanib slowed the loss of pulmonary function by 44% in patients with SSc-ILD relative to placebo, as measured by FVC over 52 weeks. 
Allergan, Author Interviews, Bipolar Disorder, Mental Health Research / 23.05.2019

MedicalResearch.com Interview with: [caption id="attachment_49298" align="alignleft" width="200"]Dr. Stephen Stahl MD PhDProfessor of Psychiatry University of California San Diego Dr. Stahl[/caption] Dr. Stephen Stahl MD PhD Professor of Psychiatry University of California San Diego  MedicalResearch.com: What is the background for this study? How does cariprazine differ from other medications for bipolar depression?  MedicalResearch.com: It is important to note that cariprazine, a dopamine D3-preferring D3/D2 receptor and serotonin 5-HT1A receptor partial agonist, is approved for the treatment of schizophrenia (1.5-6 mg/d) and bipolar mania (3-6 mg/d) in adults. It is not yet approved for depressive episodes related to bipolar I disorder (bipolar depression). In these data that focus on the investigational use for the treatment of bipolar depression, cariprazine has demonstrated efficacy vs placebo (PBO) in 3 phase 2/3 studies of patients with bipolar depression (NCT01396447, NCT02670538, NCT02670551). These analyses investigated the efficacy of cariprazine in patients with bipolar depression and concurrent manic symptoms (mixed features). 
Author Interviews, Diabetes, Ophthalmology, Regeneron / 17.05.2019

MedicalResearch.com Interview with: [caption id="attachment_38910" align="alignleft" width="300"]Blausen.com staff (2014). "Medical gallery of Blausen Medical 2014". WikiJournal of Medicine 1 (2). DOI:10.15347/wjm/2014.010. ISSN 2002-4436. Illustration depicting diabetic retinopathy Illustration depicting diabetic retinopathy[/caption] Robert L. Vitti, MD, MBA Vice President and Head, Ophthalmology Regeneron Pharmaceuticals Dr. Vitti discusses the recent announcement that the FDA has approved EYLEA to treat all stages of diabetic retinopathy. MedicalResearch.com: Can you provide additional background on this approval? Would you briefly explain diabetic retinopathy and it's impact on patients? Response: The FDA has approved EYLEA (aflibercept) Injection to treat all stages of diabetic retinopathy (DR). DR is the leading cause of blindness among working-aged American adults. Approximately 8 million people live with DR, a complication of diabetes characterized by damage to the blood vessels in the retina (per 2010 data). The disease generally starts as non-proliferative diabetic retinopathy (NPDR) and often has no warning signs or symptoms. Over time, NPDR often progresses to proliferative diabetic retinopathy (PDR), a stage in which abnormal blood vessels grow on the surface of the retina and into the vitreous cavity, potentially causing severe vision loss.
Author Interviews, Kidney Disease, Pharmaceutical Companies / 26.04.2019

MedicalResearch.com Interview with: [caption id="attachment_48873" align="alignleft" width="133"]Dr. Jay Venkatesan MDPresident and CEO of Angion Dr. Venkatesan[/caption] Dr. Jay Venkatesan MD President and CEO of Angion Dr. Venkatesan discusses the recent announcement that ANGION, has received DOD funding for the study of ANG-3070, in treatment of CKD caused by focal segmental glomerulosclerosis,  MedicalResearch.com: What is the background for this announcement? Would you tell us a little about focal segmental glomerulosclerosis (FSGS)? How does ANG-3070 work to prevent kidney scarring? Response: Angion has received a follow-on grant from the Department of Defense (DoD) for $4.76 million in support of the development of ANG-3070, our drug candidate for a form of chronic kidney disease known as focal segmental glomerulosclerosis (FSGS). This funding will allow us to expand our proof-of-concept data for ANG-3070 as a potential anti-fibrotic agent for slowing the progression of FSGS. FSGS is a serious kidney disorder characterized by progressive scarring of the glomeruli, the filtering units of the kidney. There are approximately 80,000 cases of FSGS in the U.S. and Europe, involving both children and young adults. If uncontrolled, FSGS can lead to kidney failure, which may lead to the need for dialysis or a kidney transplant. No therapies exist that treat the underlying cause of FSGS. Therapies such as corticosteroids, immunosuppressants or diuretics  are used, but are mainly supportive and a large proportion of patients progress to end-stage renal disease over a 5-10 year period of time. ANG-3070 is an oral small molecule that selectively inhibits molecular pathways associated with scarring or fibrosis in the kidney and other organs. Our current preclinical study in collaboration with NEPTUNE aims to identify the “signalosome,” or human disease and drug response profile based on the genes, networks and pathways that correlate with the therapeutic activity of ANG-3070 in FSGS. Ultimately, this collaboration will allow us to  develop a precision medicine approach to identify and treat patients in whom ANG-3070 is most likely to block the pathways causing FSGS. 
Author Interviews, Hospital Acquired, Infections, Merck / 16.04.2019

MedicalResearch.com Interview with: [caption id="attachment_41611" align="alignleft" width="167"]Dr. Elizabeth Rhee MD Director, Infectious Disease Clinical Research at Merck Dr. Rhee[/caption] Elizabeth Rhee, MD Executive Director, Infectious Disease Clinical Research Merck Research Laboratories MedicalResearch.com: What is the background for this study? Would you briefly explain the condition of ventilated nosocomial pneumonias? Dr. Rhee: Nosocomial pneumonia (NP) is a lung infection that occurs during a hospital stay. NP is often serious, and is associated with high mortality. It is one of the most common health-care associated infections in both the U.S. and Europe, accounting for over 20% of such cases. Gram-negative bacteria, mainly Pseudomonas aeruginosa (PSA) and Enterobacteriaceae, are frequent causes of nosocomial pneumonia. Limited options currently exist for the management of NP caused by Gram-negative pathogens. This is concerning because rates of resistance to Gram-negative bacteria are growing, and they are becoming increasingly difficult to treat. Forms of nosocomial pneumonia include hospital-acquired pneumonia (HAP), ventilator-associated pneumonia (VAP), and ventilated HAP. High rates of death (ranging from 20% to more than 50%) are especially associated with ventilated HAP. Pseudomonas aeruginosa, a Gram-negative bacterium, is the most common cause of HAP/VAP in both the U.S. and Europe. Patients with NP are often critically ill, requiring ventilator support and time in intensive care, and it was important to look at this population as we explore new options for the treatment of NP. Ceftolozane/tazobactam (C/T) is an antipseudomonal cephalosporin/beta-lactamase inhibitor combination with broad in vitro activity against Gram-negative pathogens, including multi-drug resistant (MDR) P. aeruginosa and many extended-spectrum beta-lactamase (ESBL) producers. It is FDA approved for complicated intra-abdominal and urinary tract infections in adults at 1.5g (1g ceftolozane/0.5g tazobactam) q8h. C/T is currently being studied at an investigational new dose of 3g (2g/1g) q8h, for the treatment of ventilated nosocomial pneumonia, in the ASPECT-NP Phase 3 trial.
Alzheimer's - Dementia, Author Interviews, Merck, NEJM / 10.04.2019

MedicalResearch.com Interview with: Michael F. Egan, MDVice President,  NeuroscienceGlobal Clinical DevelopmentMerck Research LaboratoriesNorth Wales, PAMichael F. Egan, MD Vice President,  Neuroscience Global Clinical Development Merck Research Laboratories North Wales, PA  MedicalResearch.com: What is the background for this study?   Response: Alzheimer’s disease (AD) appears to be due to the gradual accumulation of amyloid over many years (the “amyloid hypothesis”). At some point, it is thought that amyloid triggers abnormalities in tau, which then forms deposits within neurons and leads to progressive neurodegeneration. Amyloid is made up of  a small, sticky peptide, Abeta, which is produced when the enzyme BACE cleaves a large protein called APP.  In our trial, we tested whether a potent BACE inhibitor, verubecestat, could slow disease progression in subjects with early AD (or prodromal AD) by blocking formation of Abeta.  A previous trial in subjects with dementia due to AD failed to find evidence of efficacy. One possible reason for this failure is that subjects had too much amyloid in their brain already.
Author Interviews, Cancer Research, Genentech, Leukemia / 25.03.2019

MedicalResearch.com Interview with: [caption id="attachment_48117" align="alignleft" width="200"]Nancy Valente, M.D.VP of Global Hematology DevelopmentGenentech Dr. Valente[/caption] Nancy Valente, M.D. VP of Global Hematology Development Genentech Dr. Valenta discusses the announcement of the submission by Genentech of a supplemental New Drug Application to the FDA for Venclexta plus Gazyva for people with previously untreated chronic lymphocytic leukemia  with co-existing medical conditions. MedicalResearch.com: What is the background for this study? What are the main findings of the Phase III CLL14 study?  Response: We completed the submission of a supplemental New Drug Application (sNDA) to the FDA for Venclexta® (venetoclax) in combination with Gazyva® (obinutuzumab) in people with previously untreated chronic lymphocytic leukemia (CLL) and co-existing medical conditions. CLL is the most common form of adult leukemia and more than 20,000 new cases will be diagnosed in the U.S. this year. The sNDA is based on data from the Phase III CLL14 study, which evaluated fixed-duration Venclexta in combination with Gazyva in people with previously untreated CLL. Results showed this chemotherapy-free combination can help people with previously untreated CLL live significantly longer without their disease worsening (progression-free survival; PFS) compared to standard-of-care Gazyva plus chlorambucil. The FDA is reviewing our application under the Real-Time Oncology Review (RTOR) pilot program, which is exploring a more efficient review process to ensure safe and effective treatments are available to patients as early as possible. 
Author Interviews, Diabetes, Pharmaceutical Companies, Weight Research / 12.03.2019

WeightControl.com Interview with: [caption id="attachment_47883" align="alignleft" width="142"]Dr. Lynn Kramer, MD FAANVP and Chief Clinical Officer & Chief Medical OfficeEisai Co., Ltd Dr. Kramer[/caption] Dr. Lynn Kramer, MD FAAN VP and Chief Clinical Officer & Chief Medical Office Eisai Co., Ltd WeightControl.com: What is the background for this announcement? Response: On February 25th, Eisai announced that the U.S. Food and Drug Administration (FDA) accepted its supplemental New Drug Application to potentially update the label for BELVIQ® (lorcaserin HCI) CIV 10 mg twice-daily/BELVIQ XR (lorcaserin HCI) CIV once daily to include long-term efficacy and safety data from CAMELLIA-TIMI 61, a clinical trial of BELVIQ in 12,000 overweight and obese patients with cardiovascular (CV) disease and/or multiple CV risk factors such as type 2 diabetes mellitus (T2DM).
Abbvie, Author Interviews, Dermatology, Immunotherapy / 11.03.2019

MedicalResearch.com Interview with: [caption id="attachment_47874" align="alignleft" width="200"]Anne Robinson, Pharm DExecutive Scientific DirectorAbbVie Dr. Robinson[/caption] Anne Robinson, Pharm D Executive Scientific Director AbbVie MedicalResearch.com: What is the background for the risankizumab data presented at the American Academy of Dermatology 2019 Annual Meeting? Response: Abstracts presented by AbbVie at the American Academy of Dermatology (AAD) 2019 Annual Meeting highlight additional data from the Phase 3 clinical trial program evaluating the safety and efficacy of risankizumab, an investigational interleukin-23 (IL-23) inhibitor. The registrational program for risankizumab evaluated more than 2,000 adult patients with moderate to severe plaque psoriasis across four pivotal studies.
Author Interviews, Dermatology, Pharmaceutical Companies / 05.03.2019

MedicalResearch.com Interview with: [caption id="attachment_47805" align="alignleft" width="155"]Dr. James Q. Del Rosso, D.O., FAOCD, DermatologistResearch Director and Principal InvestigatorDel Rosso Dermatology Research Center, Las Vegas, NVGalderma Consultant Dr. Del Rosso[/caption] Dr. James Q. Del Rosso, D.O., FAOCD, Dermatologist Research Director and Principal Investigator Del Rosso Dermatology Research Center, Las Vegas, NV Galderma Consultant MedicalResearch.com: What is the background for this study? What are the main findings?
  • The ANSWER study, a 12-week, randomized, multicenter, Phase 4, Phase 3b in Canada and Europe clinical trial, is the first study of its kind to compare the efficacy and safety of combination therapy with Oracea® (doxycycline, USP) 40 mg Capsules + Soolantra® (ivermectin) Cream, 1% versus Soolantra® (ivermectin) Cream, 1% monotherapy in 273 adults with severe papulopustular rosacea (IGA 4) at clinical trial sites in the United States, Canada and Europe (Czech Republic, Poland, Hungary and Germany).
  • Results showed the combination therapy with Oracea Capsules + Soolantra Cream was well tolerated and effective with a faster onset of action than Soolantra Cream given as monotherapy. Key highlights of the study include:
  • The mean reduction in percentage of inflammatory lesions from baseline to Week 12 was significant with combination therapy compared to monotherapy (80.29% vs. 73.56%, respectively; p=0.032).
  • 5 times as many patients taking combination therapy achieved 100% clearance of inflammatory lesions by Week 12 compared with monotherapy (17.8% vs. 7.2%, respectively; p=0.006).
  • Over 2 times as many patients taking combination therapy achieved 100% clear (IGA 0) by Week 12 compared with monotherapy (11.9% vs. 5.1%, respectively; p=0.043).
  • Combination therapy was generally well tolerated and no discontinuation of treatments due to side effects.
Author Interviews, Dermatology, Eli Lilly / 16.02.2019

MedicalResearch.com Interview with: [caption id="attachment_47533" align="alignleft" width="200"]Lotus Mallbris, MD PhD Vice President, Head of Global Immunology Drug Development Platform Team Leader at Lilly Dr. Mallbris[/caption] Lotus Mallbris, MD PhD Dermatologist and Vice President, Head of Global Immunology Drug Development Platform Team Leader at Lilly MedicalResearch.com: What is the background for this study? Would you briefly explain what is meant by atopic dermatitis? How common is this condition?  Response:The BREEZE-AD1 and BREEZE-AD2 clinical trials are multicenter, randomized, double-blind, placebo-controlled, Phase 3 studies to evaluate the efficacy and safety of baricitinib monotherapy in adult patients with moderate to severe atopic dermatitis. These are two of five studies that will be part of the placebo-controlled data program intended to support global registrations. Atopic dermatitis, a serious form of eczema, is a chronic, relapsing skin disease characterized by intense itching, dry skin and inflammation that can be present on any part of the body. It affects approximately 1-3 percent of adults worldwide.
Author Interviews, Cost of Health Care, Dartmouth, JAMA, Pharmaceutical Companies / 09.01.2019

MedicalResearch.com Interview with: Steven Woloshin, MD, MS Professor Co-director of the Center for Medicine and Media The Dartmouth InstituteSteven Woloshin, MD, MS Professor Co-director of the Center for Medicine and Media The Dartmouth Institute MedicalResearch.com: What is the background for this study? What are the main findings? What influence does medical marketing have on medical care and drug prices? Response: There are published studies looking at promotional spending mostly for drugs (DTC and professional).  This paper is unique because it is such a broad look including not just drugs but also marketing of disease (in "awareness campaigns"), health services and laboratory tests. What is new here is the size and scope of marketing.  For context, $29.9 billion spent on promoting prescription drugs, disease awareness campaigns, health services, and laboratory tests corresponds approximately to $1000 per American.    For context, FDA's total budget is around $5 billion – and NIH's total budget is about $30 billion. This figure is up from $17.7 billion in 1997, with the most rapid increase in DTC promotion of prescription drugs and health services.   Pharmaceutical marketing to professionals (detailing visits and samples) accounted for most spending and remained high despite policies to limit industry influence. $30 billion is of an underestimate (egg, we did not include monies spent on professional marketing (detailing) of laboratory tests, health services or devices, the value of drug coupons/discounts/rebates, company marketing budgets, lobbying or campaign contributions). Further it is just the tip of the iceberg – marketing works so promotional spending is an important driver of why medical care is so expensive:  it leads to more – and more expensive - tests and treatments.
Author Interviews, Clots - Coagulation, J&J-Janssen / 05.12.2018

MedicalResearch.com Interview with: [caption id="attachment_46370" align="alignleft" width="200"]Paul Burton MD, PhD, FACC Vice President, Medical Affairs Internal Medicine Janssen Scientific Affairs, LLC. Dr. Burton[/caption] Paul Burton MD, PhD, FACC Vice President, Medical Affairs Internal Medicine Janssen Scientific Affairs, LLC. MedicalResearch.com: What is the background for this study? What are the main findings? Response: More than 900,000 Americans experience a venous thromboembolism (VTE) each year, with about one-third of these occurrences being fatal. Once a person experiences a VTE, they are at increased risk of a repeat occurrence. Guidelines currently recommend standard anticoagulant therapy with a Factor Xa inhibitor, like XARELTO® (rivaroxaban), for three months or longer. For those people who have had a VTE and stop anticoagulant therapy, as many as 10 percent of them will experience another VTE within one year and 20 percent within three years. This study examined extended use of XARELTO® after the recommended three-month treatment period in patients who experienced an initial VTE, showing XARELTO® was associated with a decreased risk of recurrent VTE with no increase in major bleeding during this time period. 
Author Interviews, Cost of Health Care, Hematology, J&J-Janssen, Thromboembolism / 04.12.2018

MedicalResearch.com Interview with: [caption id="attachment_46370" align="alignleft" width="200"]Paul Burton MD, PhD, FACC Vice President, Medical Affairs Internal Medicine Janssen Scientific Affairs, LLC. Dr. Burton[/caption] Paul Burton MD, PhD, FACC Vice President, Medical Affairs Internal Medicine Janssen Scientific Affairs, LLC. MedicalResearch.com: What is the background for this study? What are the main findings? Response: Despite being largely preventable, venous thromboembolism (VTE) is the second leading cause of death in people with cancer. The risk of VTE is five times greater in people with cancer than those without cancer, and that risk is magnified in those receiving certain types of chemotherapy, in the newly diagnosed and in those with more advanced, metastatic disease. This 6,194-patient study examined economic burden associated with VTE, and found patients newly diagnosed with cancer who are at a higher risk of a VTE had significantly higher all-cause and VTE-related health care costs compared to patients with a lower risk of VTE.
Author Interviews, Clots - Coagulation, Hematology, J&J-Janssen / 04.12.2018

MedicalResearch.com Interview with: [caption id="attachment_46370" align="alignleft" width="200"]Paul Burton MD, PhD, FACC Vice President, Medical Affairs Internal Medicine Janssen Scientific Affairs, LLC. Dr. Burton[/caption] Paul Burton MD, PhD, FACC Vice President, Medical Affairs Internal Medicine Janssen Scientific Affairs, LLC. MedicalResearch.com: What is the background for this study? What are the main findings? Response: Treatment of venous thromboembolism (VTE) is complicated among morbidly obese patients. Current guidelines do not recommend use of Factor Xa inhibitors in these patients due to limited clinical data available. That's why Janssen undertook this study to examine XARELTO® (rivaroxaban) in these patients. In this 5,780-patient retrospective study, results found patients treated with XARELTO® had a similar risk of recurrent VTE and major bleeding compared to those taking warfarin. However, treatment with XARELTO® was associated with less all-cause health care resource utilization (HCRU) (e.g., inpatient hospitalizations and outpatient visits) and reduced total medical costs compared to warfarin. Of note, patients taking XARELTO® had an average $2,829 lower total medical costs per patient per year (PPPY) than those taking warfarin, which was mainly driven by lower hospitalization costs.
Author Interviews, Cancer Research, Hematology, J&J-Janssen / 04.12.2018

MedicalResearch.com Interview with: [caption id="attachment_46272" align="alignleft" width="160"]Caroline McKay, PhD Real World Value & Evidence, Oncology Janssen Scientific Affairs Dr. McKay[/caption] Caroline McKay, PhD Real World Value & Evidence, Oncology Janssen Scientific Affairs MedicalResearch.com: What is the background for this study? What are the main findings? Response: Considering patient preferences in treatment decision-making in oncology is growing in importance. While recent introduction of new treatments for multiple myeloma have improved survival and the possibility of sustaining longer remission periods, regimen options still vary with respect to efficacy, safety, and dosing. Therefore, patients and providers must consider tradeoffs inherent in making treatment decisions that are growing in complexity. Despite this, there is a lack of research describing patient preferences within the context of currently available treatment regimens. To address this gap, this study examined how multiple myeloma patients evaluate, or weigh, treatment options. Key findings from the research are that treatment preferences do not appear to be static, but instead suggest that the relative importance of treatment attributes may change over time and treatment history. Further, patients place higher importance on overall survival and progression-free survival than other treatment attributes, and may be willing to accept an increase in the risk of serious side effects and reduced convenience in exchange for greater efficacy; however, when efficacy is comparable, patients appear to place greater weight on dosing frequency than on the duration of treatment administration, i.e., more frequent dosing appears to be less preferable to patients than longer administration/infusion time. 
Author Interviews, Cancer Research, Hematology, J&J-Janssen / 02.12.2018

MedicalResearch.com Interview with: [caption id="attachment_46315" align="alignleft" width="129"]Peter Voorhees, MD Plasma Cell Disorders Program Department of Hematologic Oncology and Blood Disorders Levine Cancer Institute Atrium Health Dr. Vorhees[/caption] Peter Voorhees, MD Plasma Cell Disorders Program Department of Hematologic Oncology and Blood Disorders Levine Cancer Institute Atrium Health MedicalResearch.com: What is the background for this study? Response: All multiple myeloma arises from its precursor conditions, monoclonal gammopathy of undetermined significance (MGUS) and smoldering multiple myeloma (SMM). Although the rate of progression to multiple myeloma for patients with MGUS is low (~5% over 5 years), patients with SMM have a ~50% likelihood of requiring therapy for their multiple myeloma within the first 5 years of diagnosis. For those at intermediate to high risk of disease progression, early intervention to delay progression of disease, thereby averting disease-related morbidities related and potentially changing the natural course of the disease, is highly desirable. On the other hand, given the fact that these patients are by definition asymptomatic and would otherwise be monitored off treatment, it is critical that any intervention applied in this group of patients is well tolerated. Daratumumab is a highly attractive candidate in this particular space, because it has single agent activity in heavily-pretreated relapsed/refractory multiple myeloma and a favorable side effect profile relative to many other myeloma therapeutics. Additionally, given the importance of impaired immune surveillance in multiple myeloma, the immuno-stimulatory effects of daratumumab in the bone marrow microenvironment could potentially reawaken robust T cell responses to the disease.
Author Interviews, Cost of Health Care, Hematology, J&J-Janssen, Lymphoma / 02.12.2018

MedicalResearch.com Interview with: [caption id="attachment_46320" align="alignleft" width="133"] Dr. Sundaram[/caption] Murali Sundaram, MBA, Ph.D. Director of Real World Value and Evidence Oncology, Janssen MedicalResearch.com: What is the background for this study? Response: Ibrutinib is a novel Bruton's tyrosine kinase (BTK) inhibitor approved for the treatment of patients with newly diagnosed chronic lymphocytic leukemia (CLL). Ibrutinib is administered orally while standard of care (CD20 monoclonal antibody-based chemoimmunotherapy [CIT]) is administered intravenously. This difference in route of administration impacts what type of benefit covers these treatments (i.e., pharmacy benefit for oral ibrutinib and medical benefit for intravenous CIT). Previous studies evaluating the costs burden of patients treated with ibrutinib versus CIT did not include the full spectrum of real-world healthcare costs.
Allergies, AstraZeneca, Author Interviews / 01.12.2018

MedicalResearch.com Interview with: [caption id="attachment_46299" align="alignleft" width="125"]Mr. Tosh Butt Vice President, Respiratory AstraZeneca Mr. Butt[/caption] Mr. Tosh Butt Vice President, Respiratory AstraZeneca Mr. Butt discusses the recent announcement that the FDA has granted Orphan Drug Designation for Fasenra for the treatment of Eosinophilic Granulomatosis with Polyangiitis.  MedicalResearch.com: What is the background for this announcement? Can you tell us a little more about Eosinophilic Granulomatosis with Polyangiitis/Churg Strauss? How does it differ/resemble severe eosinophilic asthma?
  • The US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for FASENRA™ (benralizumab) for the treatment of Eosinophilic Granulomatosis with Polyangiitis (EGPA). The ODD application was based on epidemiology demonstrating the rarity of the disease (<200k US patients) and a scientific rationale that FASENRA may benefit patients with this condition. The core role of eosinophilia in EGPA and FASENRA’s demonstrated eosinophil-depleting properties provided this rationale and suggest it may deliver benefit to patients with EGPA.
  • EGPA is a rare autoimmune disease that can cause damage to multiple organs and tissues. EGPA is characterized by inflammation of blood vessels and the presence of elevated levels of eosinophils, a type of white blood cell. All patients with EGPA have very high levels of eosinophils at some point in their disease. FASENRA induces rapid and near-complete depletion of eosinophils in the blood and has proven efficacy in severe eosinophilic asthma, which suggest it may deliver benefit to patients with EGPA. 
Asthma, AstraZeneca, Author Interviews, Pulmonary Disease / 22.11.2018

MedicalResearch.com Interview with: “Asthma Inhaler” by NIAID is licensed under CC BY 2.0Sean O'Quinn MPH Director, Patient Reported Outcomes AstraZeneca  MedicalResearch.com: What is the background for this study? How does benralizumab differ from traditional medications for asthma? Response:  FASENRA™ (benralizumab 30mg for subcutaneous injection as add-on maintenance therapy in severe eosinophilic asthma for patients 12 years and older) has a strong clinical profile, including powerful efficacy against exacerbations and the ability to improve lung function. Benralizumab is a respiratory biologic that binds directly to the IL-5α receptor on eosinophils and attracts natural killer cells to induce rapid and near-complete depletion of eosinophils via apoptosis. (NOTE: The mechanism of action of FASENRA in asthma has not been definitively established.) Benralizumab is not indicated for treatment of other eosinophilic conditions or for relief of acute bronchospasm or status asthmaticus. The most common adverse reactions include headache and pharyngitis. Dependence on rescue medications is indicative of poor asthma control. In the Phase III SIROCCO/CALIMA trials, patients with severe eosinophilic asthma had significantly reduced exacerbation frequency and improved lung function when treated with benralizumab 30mg Q8W (first three doses Q4W) vs. placebo. Less was known about the effects of benralizumab on rescue medication usage—specifically daily total rescue medication use, daytime and nighttime rescue medication use, and nighttime awakenings requiring rescue medication use. The aim of this analysis was to understand the potential treatment effects of benralizumab on these parameters. 
AstraZeneca, Author Interviews, Kidney Disease / 05.11.2018

MedicalResearch.com Interview with: [caption id="attachment_45705" align="alignleft" width="125"]Dr-Danilo Verge.png Dr. Verge[/caption] Danilo Verge MD MBA Vice President, CVRM Global Medical Affairs AstraZeneca MedicalResearch.com: What is the background for this study? Response: Dapagliflozin, an SGLT2 inhibitor (sodium-glucose co-transporter 2), has been shown to improve glycemic control by decreasing glucose reabsorption in the kidneys and inducing urinary glucose clearance. SGLT2 inhibitors have also been shown to be effective in lowering albuminuria and stabilizing eGFR (estimated glomerular filtration rate). The effect of dapagliflozin on UACR (urine albumin-to-creatinine ratio) has been shown to vary among patients. The objective of this post-hoc analysis, based on the pooled data from 11 randomized, placebo-controlled clinical trials, was to assess baseline characteristics and concurrent changes in cardiovascular (CV) risk markers associated with UACR response to dapagliflozin.
AstraZeneca, Author Interviews, Blood Pressure - Hypertension, Kidney Disease / 05.11.2018

MedicalResearch.com Interview with: [caption id="attachment_45702" align="alignleft" width="125"]Lei Qin Lei Qin[/caption] Lei Qin MS Director, Health Economics and Payer Analytics AstraZeneca MedicalResearch.com: What is the background for this study? Response: Renin-angiotensin-aldosterone system inhibitors (RAASi) are guideline-recommended therapies for patients with chronic kidney disease (CKD), but are commonly prescribed at suboptimal doses, which has been associated with worsening clinical outcomes. The objective of our study was to estimate the real-world associations between RAASi dose and adverse clinical outcomes in patients prescribed RAASi therapies with new-onset CKD in the UK.
AstraZeneca, Author Interviews, Kidney Disease, Mineral Metabolism / 05.11.2018

MedicalResearch.com Interview with: [caption id="attachment_45698" align="alignleft" width="125"]Rahul Agrawal MD PhD VP, Global Medicines Leader AstraZeneca Dr. Agrawal[/caption] Rahul Agrawal MD PhD VP, Global Medicines Leader AstraZeneca MedicalResearch.com: What is the background for this study?   About the study: HARMONIZE Global is a Phase III, randomized, multicenter, double-blind, placebo-controlled trial involving 267 patients with hyperkalemia (mean potassium levels greater than 5.0 mEq/L) in 47 study locations across the Asia Pacific region, which will support registration in Japan, Taiwan, Korea and Russia. Study design: The trial design of HARMONIZE Global is similar to HARMONIZE (NCT02088073) but evaluated two doses of LOKELMATM (sodium zirconium cyclosilicate) instead of three, as well as patients in different geographical regions.
Asthma, Author Interviews, FDA, Regeneron / 31.10.2018

MedicalResearch.com Interview with: RegeneronNeil Graham,  M.B.B.S., M.D., M.P.H VP of Immunology & Inflammation Regeneron MedicalResearch.com: What is the background for this announcement?  Response: Patients with moderate-to-severe asthma often have uncontrolled, persistent symptoms despite standard-of-care therapy that may make them suitable for treatment with a biologic therapy. They live with coughing, wheezing and difficulty breathing, and are at risk of severe asthma attacks that may require emergency room visits or hospitalizations. [i],[ii] Oral corticosteroids can provide relief for severe, short-term symptoms. However, their chronic use is limited to the most severe patients due to the potential for serious side effects. [iii],[iv] A particular type of inflammation contributes to the cause of uncontrolled symptoms in multiple inflammatory diseases such as asthma and atopic dermatitis.[v] Dupixent is a medicine that inhibits the overactive signaling of interleukin-4 (IL-4) and interleukin-13 (IL-13), two key proteins that contribute to this type of inflammation. This inhibits cytokine-induced inflammatory responses, including the release of proinflammatory cytokines, chemokines, nitric oxide, and IgE; however, the mechanism of action of Dupixent in asthma has not been definitively established.
Author Interviews, Flu - Influenza, Genentech / 30.10.2018

MedicalResearch.com Interview with: [caption id="attachment_45220" align="alignleft" width="200"]Mark D. Eisner, MD, MPH Vice President, Product Development Immunology Infectious Disease and Ophthalmology Genentech  Dr. Mark Eisner[/caption] Mark D. Eisner, MD, MPH Vice PresidentProduct Development Immunology, Infectious Disease and Ophthalmology Genentech Dr. Eisner discusses the announcement that the FDA has approved XOFLUZA™ (baloxavir marboxil) for the treatment of acute, uncomplicated influenza. MedicalResearch.com: What is the background for this announcement? Response: Each year, an estimated 3-11 percent of the U.S. population gets the flu, and it can be very serious, resulting in hospitalization or even death. Since 2010, the Centers for Disease Control and Prevention (CDC) estimates that the flu has resulted annually in 9.2 to 35.6 million illnesses, 140,000 to 900,000 hospitalizations and 12,000 to 80,000 deaths. The severity of last year’s flu season underscores the need for new medical options beyond currently available antivirals. XOFLUZA was granted Priority Review in June 2018 based on results from the Phase III CAPSTONE-1 study of a single dose of XOFLUZA compared with placebo or oseltamivir 75 mg, twice daily for five days, in otherwise healthy people with the flu, as well as results from a placebo-controlled Phase II study in otherwise healthy people with the flu.