Pharmaceutical Technology

JULY 14, 2022

Historically, the pharmaceutical industry has failed to meet the needs of this patient population. Most famously, the US passed its Orphan Drug Act in 1983, providing innovators with financial motivation to develop orphan drugs and meet the needs of these forgotten patients. Efforts have been made to change this.

Drugs 147

Drugs Medicine Marketing Development 147

Rethinking Clinical Trials

JULY 22, 2022

Associate Vice President, Research Partnerships. Since the protocols have launched, 20,000+ patients have enrolled at more than 620 sites across numerous networks, 11 protocols were developed, 10 launched, with 26 publications across 13 journals. View the Summary of NIH-Funded ACTIV/ACTIV-Associated Clinical Trials. Key Points.

Vaccination 130

Vaccination Vaccine Clinical Trials Clinical Trials 130

Rethinking Clinical Trials

OCTOBER 18, 2023

Speaker Keith Marsolo, PhD Associate Professor Department of Population Health Sciences Duke University School of Medicine Slides Keywords PCORnet, Common Data Model, EHR, Social Determinants of Health Key Points There are many different definitions of social determinants of health. It can be complicated to tease out by the insurance name.

Clinical Trials 117

Clinical Trials Clinical Trials Clinical Research Research 117

Rethinking Clinical Trials

JULY 13, 2023

In modeling exercises, optimal implementation of these therapies, such as an ARNI, a beta blocker, an MRA, and an SGLT2 inhibitor, compared to conventional therapy (an ACEi or ARB plus a beta blocker) is estimated to afford more than 6 years of survival in a typical 55-year-old patient.    Speaker Ankeet S.

Clinical Trials 130

Clinical Trials Clinical Trials Scientist Research 130

World of DTC Marketing

NOVEMBER 3, 2020

teaspoons of salt a day, which translates into 3,400 milligrams (mg) of sodium That’s 50% more than the government’s recommended 2,300 mg for the general public and more than twice the 1,500 mg recommended for all adults by the more-precautionary American Heart Association. According to STAT News “Americans consume an average of 1.5

Pharma Companies 190

Pharma Companies Scientist Medicine Research 190

Rethinking Clinical Trials

MARCH 28, 2023

                                           Speakers Erin Holve, PhD, MPH, MPP Chief Research Infrastructure Officer, Patient-Centered Outcomes Research Institute (PCORI) Russell Rothman. Patients and caregivers are integrated into all phases of PCORnet®.-enabled enabled research.

Clinical Trials 130

Clinical Trials Clinical Trials Trials Research 130

Rethinking Clinical Trials

NOVEMBER 16, 2022

Associate Director, Center for Bioethics & Social Sciences in Medicine. But if PCTs use a waiver/alteration of consent, the trial cannot assume sharing data is consistent with preferences of patient-subjects and cannot rely on informed consent to fulfill the ethical obligation of respect. Stephanie Morain, PhD, MPH.

Clinical Trials 246

Clinical Trials Clinical Trials Trials Research 246

Rethinking Clinical Trials

MARCH 21, 2023

Goldberg Distinguished Professor and Chief of Oncology Physician-in-Chief, North Carolina Cancer Hospital Director, Cancer Outcomes Research Program University of North Carolina Slides Keywords Patient-reported outcomes, Oncology, Clinical Trials Key Points Symptoms are common in cancer care. Quality of life (QOL) was assessed at 6 months.

Clinical Trials 130

Clinical Trials Clinical Trials Trials Research 130

Rethinking Clinical Trials

JULY 9, 2023

BeatPain Utah, an NIH Pragmatic Trials Collaboratory Demonstration Project, is an embedded pragmatic clinical trial comparing the effectiveness of nonpharmacologic intervention strategies for patients with back pain seeking care in federally qualified health centers in Utah.

Clinical Trials 130

Clinical Trials Clinical Trials Trials Research 130

Rethinking Clinical Trials

MARCH 6, 2024

Food insecurity is associated with diabetes. The dietician met with the patient to share education about nutrition, portion size, recipes to make food taste good, and information via an optional diabetes self-management program. 9% of the U.S. Food-as-Medicine observational studies have had a large correlation with improved health.

Clinical Trials 141

Clinical Trials Clinical Trials Medicine Trials 141

Rethinking Clinical Trials

JULY 27, 2022

It is a decentralized, fully remote trial, and data are largely patient reported. There were no differences observed in the relief of mild-to-moderate COVID-19 symptoms between patients taking Ivermectin-400 and the placebo. Were there concerns about having patients not associated with a site? Learn more about ACTIV-6.

Trials 169

Trials Pharmacy Medicine Clinical Trials 169

XTalks

JUNE 28, 2023

Elevidys was granted FDA accelerated approval for the treatment of pediatric patients four to five years of age with DMD who have a confirmed mutation in the DMD gene. It is indicated for patients who do not have a pre-existing medical reason preventing treatment with the therapy.

Gene Therapy 98

Gene Therapy Gene Protein FDA Approval 98

Rethinking Clinical Trials

APRIL 27, 2023

The reasoning for this temperature is unclear, though it likely represents what was considered the average body temperature of most patients. In a recent international survey of more than 270 centers, nearly half now use a cooler temperature dialysate in patient care of less than or equal to 36.0

Trials 130

Trials Clinical Trials Clinical Trials Medicine 130

Rethinking Clinical Trials

MARCH 1, 2023

S1800A was a Phase II randomized study of ramucirumab plus pembrolizumab versus standard of care for NSCLC patients previously treated with immunotherapy performed within the Lung-MAP platform. The study does not include tissue specimen collection, image submission, or patient-reported outcome instruments.

Clinical Trials 130

Clinical Trials Clinical Trials Trials Packaging 130

Rethinking Clinical Trials

JULY 19, 2023

About 6-12 months after enrollment, patients were assessed for the primary outcome, which was the prescription of all 3 recommended therapies. of the patients ended up being prescribed all 3 therapies, and 37.9% of patients were prescribed all 3 therapies across the intervention sites. In the usual care arm sites, 14.5%

Trials 130

Trials Cardiology Medicine Nurses 130

Pharmaceutical Technology

JUNE 29, 2022

Others judge its success based on the fact that 95% of rare diseases still have no available therapies and patient needs remain unmet. For example, while orphan drug designations have more than doubled in the last decade compared to the previous one, only 16% of therapies with orphan tags have managed to gain FDA approval in some indications.

Drugs 246

Drugs FDA Approval Marketing Development 246

Rethinking Clinical Trials

AUGUST 28, 2023

The American College of Physicians guideline states that “physicians and patients should treat acute, sub-acute and chronic LBP with non-drug therapies.” Patients in the usual care arm clinics are then scheduled to see a PCP. Presenters also shared challenges regarding scheduling and patient insurance coverage.

Trials 130

Trials Research Medicine Doctors 130

XTalks

AUGUST 19, 2022

Bluebird bio’s gene therapy Zynteglo (betibeglogene autotemcel, beti-cel) has been awarded a much anticipated approval from the US Food and Drug Administration (FDA) for the treatment of adult and pediatric patients with beta thalassemia who need regular blood transfusions. Zynteglo’s hefty price tag of $2.8

Gene Therapy 95

Gene Therapy FDA Approval Gene Clinical Trials 95

pharmaphorum

JANUARY 11, 2022

A prescription digital therapeutic (DTx) for leukaemia patients developed by Blue Note Therapeutics has been awarded breakthrough device status by the FDA. Breakthrough designations can speed up the development of new therapies, making them available to patients sooner.

Development 63

Development Production Drugs 63

Rethinking Clinical Trials

OCTOBER 19, 2022

Core Faculty & Associate Director for Global Programs. These data increasingly include info that might signal physical, mental health or behavioral health risks to patient-subjects (e.g. What do patients and other stakeholders desire? ? ? ? ? ? ? ? ? ? ? ? ?. Speakers. Joseph Ali, JD. Assistant Professor, Dept. Crofford, MD.

Clinical Trials 130

Clinical Trials Clinical Trials Trials Medicine 130

Rethinking Clinical Trials

AUGUST 23, 2022

Associate Professor with Tenure, Medicine. Associate Professor, Global Health. Participating organizations included FDA, NIH, academic institutions, pharmaceutical and device companies, patient advocacy groups, community groups, and data groups, and representatives from each organization had opportunity to extend invitation to others.

Clinical Trials 130

Clinical Trials Clinical Trials Trials Medicine 130

Rethinking Clinical Trials

AUGUST 10, 2023

For example, if the method of patient reported outcomes is gamified, patients will be more likely to continue reporting their outcomes on a regular basis rather than giving up or forgetting after the first time, which may increase the quality and quantity of data for the trial overall.

Clinical Trials 130

Clinical Trials Clinical Trials Trials Medicine 130

pharmaphorum

DECEMBER 23, 2021

The designation – which is awarded to therapies that could represent a significant improvement over standard of care in either efficacy, safety, or both – has been awarded on the strength of phase 1 results in patients with metastatic or locally advanced EGFR-mutated non-small cell lung cancer (NSCLC).

Drugs 71

Drugs Antibody Clinical Trials Clinical Trials 71

XTalks

NOVEMBER 24, 2022

Hemgenix is approved for patients who currently use factor IX prophylaxis therapy or have serious hemorrhage or spontaneous bleeding episodes. The prevalence of Hemophilia B in the population is around one in 40,000 and 15 percent of patients with hemophilia have the B type. Hemophilia B primarily affects men.

Gene Therapy 52

Gene Therapy Gene FDA Approval Genetics 52

XTalks

JANUARY 28, 2022

Up to 50 percent of patients will develop metastatic disease, mainly spreading to the liver, making treatment difficult and limited. Kimmtrak has a price tag of $18,760 per vial, which amounts to a weekly dose. months), so the average cost per patient will be approximately $400,000.

FDA Approval 98

FDA Approval Development Licensing Licensing 98

pharmaphorum

JUNE 12, 2022

The panel voted 13 to 0 on the question of whether beti-cel’s benefits outweighed the risks of the gene therapy in patients with beta thalassaemia who are dependent on blood transfusions, in a dream result for the biotech which just a few weeks ago was expressing doubts about its ability to continue as an operating concern.

Gene Therapy 58

Gene Therapy Gene Marketing Trials 58

Rethinking Clinical Trials

DECEMBER 14, 2022

Associate Dean for Research. There are two main types of clinical trials: patient randomized trial and cluster randomized trial (CRT). Raffin Professor of Medicine and Biomedical Ethics and Professor of Pediatrics, Medicine, and by courtesy of Bioengineering. Director, Stanford Center for Biomedical Ethics. Key Points.

Trials 130

Trials Clinical Trials Clinical Trials Medicine 130

XTalks

JULY 12, 2021

The updated prescribing information states that the drug should be initiated in patients with mild cognitive impairment due to Alzheimer’s disease or mild Alzheimer’s dementia. This is because the clinical trials only included patients with early or mild Alzheimer’s and not patients with later stages of the disease.

Drugs 98

Drugs Clinical Trials Clinical Trials Trials 98

Intouch Solutions

APRIL 11, 2024

Pre-Clinical : Deeper comprehension of the impacts and outcomes associated with a particular drug. Commercial : Improved experience and communication between patients and doctors. Clinical : Optimized efficiency, inventory management, and service quality of clinical trials. It uses customer insights to deliver personalized experiences.

Compliance 52

Compliance Marketing Drugs Pharma Companies 52

The Pharma Data

NOVEMBER 29, 2021

1.1.529 a variant of concern, named Omicron, on the advice of WHO’s Technical Advisory Group on Virus Evolution (TAG-VE). There is currently no information to suggest that symptoms associated with Omicron are different from those from other variants. On 26 November 2021, WHO designated the variant B.1.1.529 Studies underway.

Vaccination 52

Vaccination Vaccine Genome Genomics 52

XTalks

JUNE 8, 2021

Novartis’ Zolgensma (onasemnogene abeparvovec) gene therapy has been making significant strides as of late, including dosing of the first Spinal Muscular Atrophy (SMA) patient with the treatment in the UK last week. With a price tag of over $2.5 Baby Arthur: First UK Patient.

Gene Therapy 110

Gene Therapy Gene Protein Trials 110

pharmaphorum

NOVEMBER 19, 2021

The agreement has been hailed by patient associations, as it adds to current treatment options for the progressive muscle wasting disease – Biogen’s injectable Spinraza (nusinersen) and one-shot gene therapy with Novartis’ Zolgensma (onasemnogene abeparvovec) – which are also backed by NICE.

Drugs 98

Drugs Gene Therapy Genetic Disease Gene 98

pharmaphorum

JUNE 8, 2021

The FDA “has failed in its responsibility to protect patients and families from unproven treatments with known harms” in approving Biogen’s Alzheimer’s disease drug Aduhelm. ICER points to brain swelling and potential bleeding seen in approximately 30% of patients treated with aducanumab. Broad label questioned.

Drugs 98

Drugs Marketing FDA Approval Trials 98

XTalks

SEPTEMBER 27, 2022

About half of patients who do not receive any treatment die within five years of symptom onset. The record-breaking price tag of Skysona is not surprising for one-time gene therapies like it. Patients were also required to have a neurologic function score (NFS) of less than one, indicating limited changes in neurologic function.

FDA Approval 52

FDA Approval Gene Therapy Drugs Gene 52

XTalks

FEBRUARY 27, 2024

Skyclarys (omobaloxone) was approved in 2023 for the treatment of adults and pediatric patients 16 years of age and older with Friedreich’s ataxia, a rare, neurological disease​​. Sarepta hopes to clarify Elvidys’ effectiveness in older children in a confirmatory trial.

Research 59

Research Drugs Gene Therapy Clinical Trials 59

XTalks

OCTOBER 26, 2021

The Phase III trial results show that Givlaari offers sustainable benefit to AIP patients and confirm its favorable safety profile. The treatment came with an initial price tag of $39,000 per vial, which amounts to a total of $575,00 each year. This is a huge development for patients.”. AHP affects 1 in 100,000 people in Europe.

Gene Silencing 98

Gene Silencing Gene Clinical Trials Clinical Trials 98

pharmaphorum

JULY 25, 2022

J&J filed for approval of the bispecific in the US in December based on the results of the phase 1b MajesTEC-1 study in myeloma patients who had been treated with a median of five lines of prior therapy. Most patients diagnosed with myeloma will relapse over the course of their disease, so the disease is still considered incurable.

Antibody 52

Antibody Medicine Drugs Trials 52