Orphan Drug Credits Make It onto Short-Term US Government Spending Bill
As a government shutdown looms large in US politics, certain pharma favorites have made it into a short-term continuing resolution. Key among these is the DeLauro bill, which includes nearly $7 million in funding for rare diseases and orphan drugs research. This program is a long-standing provision, funding work in rare diseases since 1983.
AstraZeneca’s Newest Push in Oncology RWE
The pharma giant AstraZeneca has expanded its presence in the chronic lymphocytic leukemia (CLL) space in recent years. Now, according to its VP and global medical franchise head of Hematology Carlos Doti, the company will help support these oncology therapeutic development efforts with a renewed focus using real-world evidence (RWE).
Enzyvant Merges with Altavant to Advance Treatments for Rare Diseases
Enzyvant and Altavant have announced plans for a merger, which will take on the name of the former. The new company will have a facilities to manufacture regenerative medicines for rare diseases, particularly in immunology and cardiopulmonology. The combined Envyzant intends to build on its track record in the rare disease therapeutics space.
Engaging Patients and Improving Outcomes through Self-Driven Healthcare
The UK agency Innovate UK established the idea of self-driven healthcare (SDH), a broad term covering strategies to help engage patients in their own healthcare. In a newly published paper in the Journal of the Royal Society of Medicine, authors lay out a framework for SDH that would use digital health technologies like medical devices and apps to deliver healthcare information to patients and their providers.
Mathematical Modeling of HIV/AIDs – Accounting for Social Determinants of Health
While research has established that social determinants of health (SDoH) play a major role in outcomes for people living with HIV/AIDS, most mathematical models fail to account for them in a meaningful capacity. In a newly published article in Nature, authors describe the development and application of a mathematical model that incorporates SDoH into its analysis.
Using Real-World Evidence to Investigate COVID-19 Breakthrough Infections
Real-world evidence (RWE) has been essential for the public health, pharma, and biotech response to the COVID-19 pandemic. In a new interview with Med Ad News, Matthew W. Reynolds, IQVIA’s vice president of scientific affairs and real world solutions, speaks about the use of RWE in studying COVID-19 breakthrough infections to prepare for the next outbreak.
Medical Sciences Liaison Society – Current & Future Medical Affairs Competencies Survey
The Medical Sciences Liaison Society (MSLS) is seeking responses for its Current & Future Medical Affairs Competencies Survey. The survey will cover core competencies currently held by the rapidly growing medical affairs community as well as those that will be needed going forward. Participants will receive a free copy of the report as it is released later this year.
Call For Reviewers: ISPOR 2023 – Opens November 18th
ISPOR will shortly open up applications for peer-reviewers for its upcoming ISPOR 2023 conference. Nominees will review research abstract submissions to ensure that research presented at the conference will meet ISPOR’s high standards. Reviewers are required to be non-student members of ISPOR and must have time to review abstracts from January 20th to February 3rd, 2023. Submissions open November 18th and close December 6th.
Verily Announces Plans for IBD Registry in Collaboration with Chrohn’s & Colitis Foundation
Late last month, the Alphabet subsidiary Verily announced it will expand its pilot program with the Chrohn’s & Colitis Foundation to build a data registry of patients with irritable bowel disorder (IBD). The updated registry will include symptoms and patient concerns, and will be used to further research into the underlying mechanisms and etiology of the disease.
US Could Save Nearly $4 Trillion by Addressing Health Equity
New data released by the Partnership to Fight Chronic Disease (PFCD) shows that the US could save $3.8 trillion by addressing health equity in people with chronic health issues. On a more granular level, the study found that the nation would cut healthcare costs by $2.7 trillion and recoup another $1.1 due to lost work hours in 10 years.
ICER Publishes Revised Hemophilia Gene Therapy Evidence Report
A revised evidence report has been published by the Institute for Clinical and Economic Review (ICER) detailing the effectiveness and value of two gene therapies for hemophilia A and B. The gene therapies offer nearly complete remission of bleeds for a period of time without the need for prophylactic factor replacement.
Supporting the Social Determinants of Health Infrastructure with Federal Funding
The growing public focus on social determinants of health (SDoH) spurred by the COVID-19 pandemic has outstripped the infrastructure in place to address issues of health equity. According to several stakeholders, increased federal funding is key to advancing these goals and developing a tech infrastructure that can support SDoH-related efforts
Norstella and Citeline Announce Complete Merger
Norstella, a biotech that provides end-to-end services for therapeutic development has merged with Citeline, a leader in pharma intelligence. The acquisition brings the value of Norstella up to $5 billion, ranking among the largest in the industry. Norstella will use Citeline’s range of services and assets to help improve the company’s drug development support services.
AJMC “What We’re Reading” – Uninsured Children, HIV and Monkeypox, and Health Outcomes by US State
In the latest edition of “What We’re Reading,” the AJMC staff cover several breaking stories. A recent study found that people living in conservative US states have lower life expectancies than more liberal ones. The researchers’ anaysis shows that over 170,000 lives could be saved if certain liberal policies were enacted nationwide. In other news, new data shows a striking comorbidity between monkeypox and HIV.
Clinical Leader Live: Are Decentralized Clinical Trials Cutting Costs at Clinical Sites?
The logistics of the COVID-19 pandemic directly impacted the ability of many pharma and biotech companies to perform research at traditional clinical trial sites. As a result, the use of decentralized clinical trials (DCTs) and hybrid format trials boomed. An episode from earlier this year covers how this has impacted traditional clinical sites and their staff.
FDA Issues Final Guidance on Clinical Trials with Multiple Endpoints
Last week, the US Food and Drug Administration (FDA) released its final guidance on multiple endpoints in clinical trials. The goal of the guidance is to provide standardized strategies for clinical trials with multiple endpoints to avoid ambiguity in study findings that might have bearing on the regulatory approval process and patient outcomes.
Call for Abstracts: ISPOR 2023
ISPOR has opened abstract submissions for issue panels, workshops, and breakout sessions at its upcoming ISPOR 2023, the leading conference for health economics and outcomes research (HEOR) professionals. ISPOR 2023 takes place in Boston, MA, from May 7th-May 10th, 2023. Abstract submissions for issue panels, workshops, and breakout sessions close by December 15th, 2022. Abstracts for research and case studies open November 2nd and closes January 12th, 20223.
Effectiveness and Safety of Psoriasis Treatment Validated by Real-World Data Study
A new real-world data (RWD) of guselkumab, Janssen’s therapeutic for plaque psoriasis, confirmed the safety and efficacy that was demonstrated in randomized controlled trials (RCTs). Data from 79 people with moderate to severe plaque psoriasis. The majority of participants had a 90% or higher reduction in the plaque area and intensity index (PASI).
NICE: NHS Should Gather Real-World Evidence from Parkinson’s Disease Digital Health Tech
The UK’s National Institute for Health and Care Excellence (NICE) has released a recommendation regarding wearable devices for people with Parkinson’s disease. These devices collect mountains of information that could be useful for research and patient monitoring. In a new article by NICE, learn how wearables could advance Parkinson’s care and find out about 5 promising upcoming devices.
FDA Opens Applications for PDUFA VII Real-World Evidence Programs
The US Food and Drug Administration (FDA) will start accepting applications for two programs that are part of the Prescription Drug User Fee Act (PDUFA VII). These programs are intended to help improve the use of real-world evidence (RWE) in approvals for new therapeutics. In addition, the program is set to promote novel clinical trial designs.
Target RWE Teams Up with AASLD to Improve Outcomes for Cirrhosis Patients
Target RWE, a leader in real-world evidence (RWD) and observational research, is starting a new collaboration with the American Association for the Study of Liver Diseases (AASLD). Through this partnership, Target RWE will help the organization with its Cirrhosis Quality Collaborative (CQC), which aims to improve healthcare quality and outcomes for patients with cirrhosis through data analytics.
Electronic Health Records Show Hospital Room Layout Affects Post-Surgical Outcomes
A recent study of patients undergoing high-risk surgeries at the University of Michigan hospital found that the layout of a hospital room and surrounding areas affects clinical outcomes. The study, which pulled real-world data (RWD) from the electronic health records of nearly 4,000 patients found that patients in rooms that are within visual range of nurses fare better than those who were not. In addition, having a window was tied to lower mortality rates.
Digestive Diseases Cause a Huge Financial Burden in Europe
A newly published report in the United European Gastroenterology Journal finds that gastrointestinal diseases exact an outsized financial burden on European countries. The study identified disparities between central and western Europe in terms of gastrointestinal disease spending, due in part to tobacco smoking. Altogether, digestive disease spending in the EU totaled around $20 billion in 2021 alone.
Renalytics to Present Real-World Evidence for KidneyIntelX Platform
Renalytics has announced that its speakers will give four presentations at this years American Society of Nephrology Kidney Week early this coming November. The talks will focus on the company’s real-world evidence (RWE) nephrology platform KidneyIntelX. One talk will elaborate on the platform’s use to determine kidney disease progression in Black Americans, while another will detail how the platform helped identify kidney health inequities
ISPOR Releases Good Practices Report on Improving Reproducibility of Real-World Evidence Studies
Although real-world evidence (RWE) studies are increasing in prominence, reproducibility issues hold back research. To address this pressing issue in the field, ISPOR has published a new paper in Value in Health describing the HARmonized Protocol Template to Enhance Reproducibility (HARPER). This framework is intended to help promote three goals the organization has established.
