Marketers Spend At Least Six Times FDA Budget on Promotion of Medical Services

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.

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XARELTO® Associated With a Decreased Risk of Recurrent VTE

MedicalResearch.com Interview with:

Paul Burton MD, PhD, FACC Vice President, Medical Affairs Internal Medicine Janssen Scientific Affairs, LLC.

Dr. Burton

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.  Continue reading

Healthcare Costs in Patients with Cancer Rise with Increasing Risk of Venous Thromboembolism

MedicalResearch.com Interview with:

Paul Burton MD, PhD, FACC Vice President, Medical Affairs Internal Medicine Janssen Scientific Affairs, LLC.

Dr. Burton

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. Continue reading

Lower Overall Costs with Rivaroxaban (XARELTO® ) vs Warfarin Among Morbidly Obese Patients with Venous Thromboembolism

MedicalResearch.com Interview with:

Paul Burton MD, PhD, FACC Vice President, Medical Affairs Internal Medicine Janssen Scientific Affairs, LLC.

Dr. Burton

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. Continue reading

How Do Patients With Multiple Myeloma Weight Treatment Options?

MedicalResearch.com Interview with:

Caroline McKay, PhD Real World Value & Evidence, Oncology Janssen Scientific Affairs

Dr. McKay

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.  Continue reading

ASH18: CENTAURUS Study Evaluates Dosing Schedule of DARZALEX® (daratumumab) for High Risk Smoldering Multiple Myeloma

MedicalResearch.com Interview with:

Peter Voorhees, MD Plasma Cell Disorders Program Department of Hematologic Oncology and Blood Disorders Levine Cancer Institute Atrium Health

Dr. Vorhees

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.

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CLL: Overall Treatment Savings With Ibrutinib (Imbruvica) Despite Higher Prescription Costs

MedicalResearch.com Interview with:

Dr. Sundaram

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.

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FDA Grants Orphan Drug Designation For Eosinophilic Granulomatosis with Polyangiitis

MedicalResearch.com Interview with:

Mr. Tosh Butt Vice President, Respiratory AstraZeneca

Mr. Butt


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. 

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Asthma: Biologic Benralizumab (FASENRA) Reduced Need For Rescue Medication

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.  Continue reading

Dapagliflozin (FARXIGA): Reduction in Albuminuria Cannot Be Predicted by Clinical Characteristics

MedicalResearch.com Interview with:

Dr-Danilo Verge.png

Dr. Verge

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.

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Real-World Dosing of RAASi are Associated With Risk of Adverse Events in CKD

MedicalResearch.com Interview with:

Lei Qin

Lei Qin

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.

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LOKELMA (Sodium zirconium cyclosilicate) for Elevated Potassium: Results of the HARMONIZE GLOBAL Study

MedicalResearch.com Interview with:

Rahul Agrawal MD PhD VP, Global Medicines Leader AstraZeneca

Dr. Agrawal

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. Continue reading

FDA Approves Dupixent: Only Self-Administered Biologic for Moderate-to-Severe Asthma

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. Continue reading

FDA Approves Single Dose XOFLUZA™ For Uncomplicated Flu

MedicalResearch.com Interview with:

Mark D. Eisner, MD, MPH Vice President, Product Development Immunology Infectious Disease and Ophthalmology Genentech 

Dr. Mark Eisner

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.

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Less Smoking Linked to Lower Antibody Levels in Modern Rheumatoid Arthritis Patients

MedicalResearch.com Interview with:

Pr Gilles Boire, M.D., M. ScService de rhumatologie Département de médecine Faculté de médecine et des sciences de la santé Université de Sherbrooke

Prof. Boire

Pr Gilles Boire, M.D., M. ScService de rhumatologie
Département de médecine
Faculté de médecine et des sciences de la santé
Université de Sherbrooke

MedicalResearch.com: What is the background for this study?

Response: Rheumatoid arthritis (RA) patients are heterogeneous at initial presentation, in response to treatments and according to their outcomes. No clinical features and very few biomarkers, except autoantibodies such as anti-Cyclic Citrullinated Peptides/Proteins (CCP), identify patients with divergent prognostic trajectories.

To help improve early prognostic classification, we initiated 20 years ago the single center longitudinal observational Early Undifferentiated PolyArthritis (EUPA) study of consecutive patients presenting with recent-onset inflammatory polyarthritis, 90% of which fulfill classification criteria for RA at baseline. Our registry includes 739 very early RA patients (median symptom duration 3.6 months), rapidly treated to joint remission (i.e. 0/66 swollen joint) and followed over 5 years. Each patient visit is linked to biosamples and to sequential radiographs scored according to the modified Sharp/van der Heijde method. As we had the clinical impression that clinical features of recruited patients were evolving, we compared patients from 3 periods (1998-2004; 2005-2010; 2011-2017).  Continue reading

Single Dose Baloxavir marboxil (Xofluza) Has Potential To Improve Treatment of High-Risk Flu

MedicalResearch.com Interview with:

Mark D. Eisner, MD, MPH Vice President, Product Development Immunology Infectious Disease and Ophthalmology Genentech 

Dr. Eisner

Mark D. Eisner, MD, MPH
Vice President, Product Development Immunology
Infectious Disease and Ophthalmology
Genentech 

MedicalResearch.com: What is the background for this study?

Response: CAPSTONE-2 is a Phase III multicenter, randomized, double-blind study that evaluated the efficacy and safety of a single dose of baloxavir marboxil compared with placebo and oseltamivir in people 12 years and older who are at a high risk of complications from the flu. The Centers for Disease Control and Prevention (CDC) defines people at high risk for serious flu complications to include adults 65 years of age or older, or those who have conditions such as asthma, chronic lung disease, morbid obesity or heart disease.

A total of 2,184 participants enrolled in the study and were randomly assigned to receive a single, oral dose of 40 mg or 80 mg of baloxavir marboxil (according to body weight), placebo or 75 mg of oseltamivir twice daily for five days. The primary objective of the study evaluated the efficacy of a single dose of baloxavir marboxil compared with placebo by measuring the time to improvement of influenza symptoms. Key secondary endpoints compared outcomes in baloxavir marboxil versus placebo or oseltamivir – these included time to resolution of fever, time to cessation of viral shedding, infectious virus detection in swabs of the nose and throat, prescription of antibiotics and influenza-related complications.

Genentech announced initial results from the study on July 16, 2018 but the full data was presented for the first time during a late-breaking oral presentation at the annual IDWeek meeting in San Francisco, CA on October 6, 2018.

Baloxavir marboxil is a first-in-class, single-dose investigational oral medicine with a novel proposed mechanism of action designed to target the influenza A and B viruses, including oseltamivir-resistant strains and avian strains (e.g. H7N9, H5N1). Baloxavir marboxil is the first potential influenza treatment to demonstrate a clinically meaningful benefit for people highly vulnerable to serious influenza complications in clinical trials.

The FDA accepted a New Drug Application (NDA) and granted Priority Review to baloxavir marboxil as a single-dose, oral treatment for acute, uncomplicated influenza in people 12 years and older. The NDA was based on results from the Phase III CAPSTONE-1 study of a single dose of baloxavir marboxil compared with placebo or oseltamivir 75 mg, twice daily for five days, in otherwise healthy people with the flu. Results from a placebo-controlled Phase II study in otherwise healthy people with the flu were included as supporting data in the NDA. The FDA is expected to make a decision on approval by December 24, 2018. Continue reading

Topical Minocycline Foam for Moderate-to-Severe Acne Meets Phase 3 Study Endpoints

MedicalResearch.com Interview with:
foamixDavid Domzalski
CEO

Foamix Pharmaceuticals

MedicalResearch.com: What is the background for this study?  How does FMX101 differ from other treatment for acne, ie benzoyl peroxide, topical clindamycin etc? 

Response: This study measures the safety and efficacy of a topical foam formulation of the antibiotic minocycline, for the treatment of moderate-to-severe acne.

Minocycline is one of the most commonly used products for the treatment of acne, but is currently only available in an oral dosage form.

Significant side effects are associated with oral minocycline, including GI upset, photosensitivity, headaches, dizziness, and other potential effects on the CNS.  In addition to the side effects associated with oral minocycline, many currently available topical acne medications contain ingredients which can be drying and irritating to the skin.  These side effects can be frustrating to patients and potentially impact overall compliance to their treatment regimen.  The study addresses important unmet needs in dermatology to determine whether providing patients with a topical dosage form of minocycline may have potential advantages over existing products.

In our first two Phase 3 clinical studies, >95% of facial local tolerability signs and symptoms were classified as “none” or “mild,” including dryness, erythema and itching.  Also, our topical minocycline foam, FMX101, is a natural triglyceride-based vehicle that does not contain ingredients that serve as  primary irritants or surfactants.  We believe that FMX101, if approved, would be the first topical minocycline available for the treatment of acne and provide a novel and much needed treatment option for patients who suffer from the physical and psycho-social effects of acne. Continue reading

New Antibiotic Combination IMI/REL Can Treat Resistant Infection With Less Kidney Toxicity

MedicalResearch.com Interview with:

Michelle Hoffman Brown Associate Principal Scientist at Merck Merck

Michelle Brown

Michelle Hoffman Brown
Associate Principal Scientist
Merck

MedicalResearch.com: What is the background for this study? What are the kidney risks of using colistin to treat carbapenem-resistant bacterial infections?

Response: Gram-negative pathogens are responsible for half of all healthcare-associated infections and their ability to resist traditional antibiotics makes them more dangerous for seriously ill patients in a healthcare setting. The need for new approaches to treat these pathogens is essential and this trial aimed to evaluate the efficacy and safety of imipenem/relebactam (IMI/REL) for the treatment of these challenging infections.

Nephrotoxicity is a common complication of colistin-based therapy and is the potential adverse experience of greatest concern to prescribing clinicians, limiting its use to treat carbapenem-resistant bacterial infections. Relebactam is a novel β-lactamase inhibitor that restores imipenem activity against many imipenem-non-susceptible strains of Gram-negative pathogens. In the Phase 3 RESTORE-IMI 1 study (NCT02452047), IMI/REL was shown to be as effective as, but better tolerated than, colistin plus imipenem, including as demonstrated by a lower incidence of treatment-emergent nephrotoxicity (prespecified secondary endpoint). This analysis looked at additional renal safety data from the RESTORE-IMI 1 trial.  Continue reading

Retrotope Expands Compassionate Use Access to RT001 for Amyotrophic Lateral Sclerosis

MedicalResearch.com Interview with:
RetrotopeRobert J Molinari, Ph.D.

President and CEO
Retrotope, Inc.

 

MedicalResearch.com: What is the background for this study?
How does RT001 differ from other treatments for neurodegenerative diseases? 

Response: Lipid peroxidation in critical cell and mitochondrial membranes represents a common pathway of cell death in many neurodegenerative diseases, regardless of the initiating trigger of original damage, e.g. aberrant gene expression, misfolded proteins such as tau and amyloid, environmental insults, etc. This hypothesis was supported by work showing that stabilizing lipids (using virtually indistinguishable isotopic variants of the original dietary molecules) was able to mitigate disease-related cell dysfunction, and reverse disease in a variety of animal models, and more recently, in human patients enrolled in clinical trials.

It has been known for decades that lipid peroxidation detritus of oxidized membrane fats are present in most all diseases of degeneration and aging, including Alzheimer’s disease, Parkinson’s disease, atherosclerosis, ALS, Huntington’s, and fatal inborn genetic errors resulting in neurodegenerative diseases (among others). The hypothesis that controlling such oxidation could provide therapeutic benefit was abandoned when numerous formal trials of classical antioxidants, e.g. Co-enzyme Q, Vitamin E, and others failed to provide meaningful benefit. We believed that the antioxidant mechanism used to attempt control of the membrane oxidation was flawed, but that the target itself was correct. Indeed, by using a new class of oral drugs that are lipids fortified against damage at the key susceptible bonds, we observed reduction in lipid peroxidation damage that halted, and even reversed neurodegenerative disease progression. Dosed in amounts and forms similar to omega 3 and 6 supplements, these drugs exhibited profound disease modification across a broad range of diseases in animal models, placebo controlled- and open label- human trials. Continue reading

How Much Do Clinical Trials For New Medications Really Cost?

MedicalResearch.com Interview with:

Thomas J Moore Senior Scientist Institute for Safe Medication Practices Lecturer, Department of Epidemiology and Biostatistics The George Washington University Milken Institute of Public Health Alexandria, VA 22314

Thomas J Moore

Thomas J Moore AB
Senior Scientist Institute for Safe Medication Practices
Lecturer, Department of Epidemiology and Biostatistics
The George Washington University
Milken Institute of Public Health
Alexandria, VA 22314

MedicalResearch.com: What is the background for this study? What are the main findings?

  • The study provides realistic cost estimates of pivotal clinical trials that establish drug benefits to support FDA approval of 59 new drugs released for marketing in 2015-2016.
  • The median estimated cost was just $19 million, with half of the 138 trials studied clustered between $12 million and $33 million.
  • The highest cost trials–with estimates up to $345 million–were for new drugs that were similar to drugs already available and already proven in treating serious illnesses. 

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IXORA-S Study Suggest Taltz May Provide Significantly Greater Clearance of Nail Psoriasis

MedicalResearch.com Interview with:

Lotus Mallbris, M.D., Ph.D., Vice president, Immunology Development Lilly Bio-Medicines 

Dr. Mallbris

Lotus Mallbris, M.D., Ph.D.,
Vice president, Immunology Development
Lilly Bio-Medicines 

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: By exploring creative clinical approaches and patient-centric pathways to more thoroughly address the key aspects of treating these complex conditions, Lilly is bringing innovation forward in hopes of reducing the burden of dermatologic disease for people around the world.

The results of the IXORA-S study suggest that Taltz may provide significantly greater clearance of nail psoriasis than ustekinumab. This is significant because nail lesions are a common feature of psoriasis. It’s often associated with discomfort, which can lead to functional impairment and distress, further supporting the importance of complete clearance.   Continue reading

Asthma: Add-on Maintenance Treatment with FASENRA (benralizumab) Can Reduce Exacerbations

MedicalResearch.com Interview with:

Tosh Butt, MBA VP Respiratory AstraZeneca

Tosh Butt

Tosh Butt, MBA
VP Respiratory
AstraZeneca

MedicalResearch.com: What is the background for this study? How is benralizumab different from more traditional treatments for asthma?

    • BORA is a randomized, double-blind, parallel-group, Phase III extension, and is one of six Phase III trials in the WINDWARD program in asthma. The current analysis includes results for 1,926 patients from the two placebo controlled exacerbation trials, SIROCCO (48 week) and CALIMA (56 weeks). BORA provides evidence that add on maintenance treatment with FASENRA (benralizumab) resulted in a consistent safety profile over a second year of treatment, with no increase in the frequencies of overall or serious adverse events, and sustained efficacy in terms of reducing asthma exacerbations, and improving lung function and asthma symptoms. The BORA trial results could provide confidence to patients with severe eosinophilic asthma and physicians that the positive outcomes they may be seeing with benralizumab can be maintained over a second year of treatment.
  • FASENRA, a different kind of respiratory biologic, has a strong clinical profile which includes the ability to show lung function improvement after the first dose, the potential to reduce – or even stop – oral steroid use, and the convenience of 8-week dosing (no other respiratory biologic offers this dosing). FASENRA is approved for add-on maintenance treatment of patients with severe asthma ages 12 years and older, and with an eosinophilic phenotype. FASENRA binds directly to the IL-5a receptor on an eosinophil and uniquely attracts natural killer cells to induce apoptosis, or cell death. Other biologics currently available are anti-IL5s – a passive approach that primarily acts to block differentiation and survival of the eosinophil.

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FDA Approves Poteligeo® (mogamulizumab-kpkc) for T-Cell Lymphoma of the Skin

MedicalResearch.com Interview with:

Jeffrey S. Humphrey, MD President of Kyowa Kirin Pharmaceutical Development, Inc

Dr. Humphrey

Jeffrey S. Humphrey, MD
President of Kyowa Kirin Pharmaceutical Development, Inc

MedicalResearch.com: What is the background for this announcement? Would you briefly explain what is meant by Mycosis Fungoides and Sézary Syndrome?

Response: Kyowya Kirin has received FDA approval for Poteligeo (mogamulizumab), based on findings from the MAVORIC trial. Mogamulizumab is a humanized immunoglobulin G1 (IgG1) monoclonal antibody (mAb) that targets CC chemokine receptor 4 (CCR4), for the treatment of the most common subtypes of cutaneous T-cell lymphoma (CTCL), mycosis fungoides (MF) and Sézary syndrome (SS).

MF and SS may have a profound and severe impact on quality of life, including a patient’s functional, emotional and social well-being, as symptoms may include a scaly red rash or light or dark patches in areas of the body that are not usually exposed to the sun; thin, reddened, eczema-like rash; thickened scaly, red skin (or plaques) or psoriasis-like rash; more advanced disease can include tumors (with significant thickness) on the skin, which may develop ulcers and become infected. Because CTCL manifests in skin lesions, it is often mistaken for other skin conditions (early stage MF and SS can be diagnosed as other skin conditions), which can delay conclusive diagnosis and treatment options.

MF is the most common subtype of CTCL, affecting 50-70% of individuals. In most patients diagnosed with early stage MF, the skin involvement does not progress, but in some patients, it will slowly progress. SS accounts for approximately 3% of CTCL cases and is a more aggressive, leukemic form of CTCL, affecting the blood, skin, lymph nodes and visceral organs

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For Resistant HIV: Phase III Trial of Trogarzo Demonstrates Safety and Efficacy

MedicalResearch.com Interview with:
TaiMed BiologicsStanley Lewis, M.D.

TaiMed Biologics
Irvine, CA 92614


MedicalResearch.com: What is the background for this study?

Response: The phase III clinical trial was conducted to assess the efficacy and safety of Trogarzo™ (ibalizumab-uiyk) injection in patients with multidrug resistant HIV-1. The study design was approved by the FDA. Results obtained were included in the New Drug Application submitted to the FDA which approved Trogarzo™ on March 6, 2018.

The phase III, open-label study, enrolled 40 patients with multidrug-resistant (MDR) HIV-1 in whom multiple antiretroviral therapies had failed. All patients at baseline were experiencing viral failure. After a seven-day control period, patients received an intravenous 2000 mg loading dose of Trogarzo™ which was the only change made to their antiretroviral regimen. Through the 24-week treatment period of the study, patients were given a maintenance dose of 800 mg of Trogarzo™ every two weeks along with an optimized background regimen that included at least one additional fully active agent.

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Crohn’s Disease: Positive Findings from Phase III Study of Antibiotic Combination

MedicalResearch.com Interview with:
RedHill Biopharma LtdMr. Gilead Raday, MPhil, MSc
Chief Operating Officer
RedHill Biopharma Ltd

MedicalResearch.com: What is the background for this study? Would you briefly explain what is meant by Crohn’s disease?  How common is it and whom does it affect? 

Response: Crohn’s disease (CD) is a chronic, relapsing inflammatory gastrointestinal disorder characterized by a variety of symptoms, including severe abdominal pain, diarrhea, bleeding, bowel obstruction, fever and weight loss. The underlying cause of Crohn’s is unknown; however, CD is believed to arise secondary to genetic and environmental stimuli. More than 1.5 million people suffer from CD globally and it is prevalent in the U.S., affecting more than 200 people per 100,000.

The current standard of care for Crohn’s disease is limited to anti-inflammatories, immuno-suppressants and biologics that treat auto-immune disorders. These therapies target symptomatic improvement in the inflammation associated with CD, are widely considered to be of limited efficacy in the long term, and are associated with numerous side effects. This speaks to the great unmet need for an effective therapy for this debilitating disease.

Additionally, there is no current therapy that treats the suspected underlying cause of Crohn’s disease. We have developed RHB-104 with the MAP hypothesis in mind, which posits that Crohn’s disease is caused by infection by a bacteria, Mycobacterium avium subspecies paratuberculosis (MAP). This is similar to peptic ulcer disease, a condition that was initially associated with stress, smoking, NSAIDs and other behavioral factors, yet was found to be caused by H. pylori bacterial infection in the 1980s, revolutionizing the field of ulcer treatment. Validation of this theory would revolutionize how Crohn’s disease is viewed and treated by the medical community and RHB-104 is the only therapy in development targeting MAP infection.  Continue reading