Advancing Rare Disease Drugs Through Real-World Evidence
Patients with rare diseases often suffer with few effective treatment options. Therapeutic development for these drugs targeting rare diseases increasingly depends on real-world evidence (RWE). In a new IQVIA blog, learn more about efforts in the UK to advance the development of drugs for rare diseases through the careful use of RWE.
FDA Pumps the Brakes on Fulcrum Sickle Cell Drug Trial
The US Food and Drug Administration (FDA) has put a damper on Fulcrum Therapeutics’ ambitions by placing a full hold on a clinical trial for the company’s new therapeutic, FTX-6058. The company says the FDA made the decision based on preclinical data, although the specific reason for the hold has yet to be reported.
Clinical Trials Must Include Diversity Plan in FDA Applications
Many patients from rural areas and marginalized backgrounds are severely underrepresented in many clinical trials. To address this problem, the US Food and Drug Administration (FDA) has announced that in the near future, all clinical trials registered through the agency must contain a plan to ensure diversity in the participant population. The plan will take time, however, as the agency must issue a final guidance and wait for public input.
Avidity Biosciences’ Treatment for Rare Form of Muscular Dystrophy Deemed Orphan Drug
Avidity Biosciences’ treatment for a rare muscular dystrophy called facioscapulohumeral muscular dystrophy (FSHD) has been given orphan drug status by the US Food and Drug Administration (FDA). The drug, if approved, would be the first for the disease, which results in deterioration of muscle function beginning in the upper extremities.
US Govt Accountability Office Recommends FDA and HHS Improve IRB Inspection Oversight
The US Food and Drug Administration (FDA) and Department of Health and Human Services (HHS) received a rebuff from the Government Accountability Office (GAO) over their oversight over institutional review boards (IRBs) for clinical trials. IRBs, which help oversee ethics in research on the local level, are supposed to be overseen by the agencies, but in practice this rarely happens.
Labcorp Reveals Name for New CRO – Fortrea
Labcorp has announced that its upcoming clinical development spin-off will be named Fortrea. Fortrea will be independent and publicly traded, providing drug and health tech development and commercialization services to pharma and biotechs. Once spun off, Fortrea will begin with a workforce of 19,000 employees.
Food Insecurity and Health Outcomes in Sub-Saharan African Nations
Several Sub-Saharan African (SSA) nations face significant levels of food insecurity, which is known to worsen health outcomes. A new study published in BMC Public health analyzes analyzed the effects of food insecurity on life expectancy and infant mortality in SSA nations and found that both were negatively impacted by food insecurity.
Children with Asthma Don’t Get Enough GP Visits
A set of studies published in the Archives of Disease in Childhood and the Journal of Asthma found that patients with childhood asthma benefit from regular general practitioner (GP) visits but unfortunately do not get enough of them. Only 20% saw their GP for a majority of their visits, with less than 40% seeing their GP in the week following a hospital discharge for asthma-related reasons.
US GSA Launches AI Healthcare Challenge
The US General Services Administration (GSA) has announced the launch of a new competition to develop artificial intelligence (AI) solutions to improve health outcomes. The challenge, launched by the Centers of Excellence (CoE) and Challenge.gov, will focus on accessibility, oncology, logistics and safety, mental healthcare, and substance use disorders.
Operation Warp Speed for Rare Diseases is on the Way
Director Peter Marks of the US Food and Drug Administration’s (FDA’s) Center for Biologics Evaluation and Research has announced the next stage of Operation Warp Speed, a program intended to find cures and treatments for rare diseases. The program will support drugmakers developing drugs for rare diseases and will establish close lines of communication between companies and the agency to expedite the process.
BSQ’s Abecma CAR-T Multiple Myeloma Therapy Cuts Progression or Death in Half
Bristol Myers Squibb has released positive new data from its KarMMa-3 trial for Abecma, its CAR-T therapy for multiple myeloma. The results show that patients taking Abecma instead of the current standard of care have 51% reduced risk of death or disease progression. Data also indicates that patients in the Abecma group had a median time without disease progression of 13.3 months.
ISPOR Task Force – Good Practices for Patient Preference Studies
Healthcare decision-makers have increasingly recognized the importance of patient health preferences, incorporating results from patient preference studies into a wide range of deliberative processes. ISPOR has published a set of good practices to enhance the impact of these studies in a newly published paper in the organization’s Value in Health journal.
CMS Leaders Propose Universal Foundation for Quality Measures
The US Centers for Medicare and Medicaid Services (CMS) has several varying sets of quality measures with some degree of [...]
Boehringer Ingelheim Reveals Positive Trial Results for Rare Psoriasis Therapeutic
Boehringer Ingelheim revealed new results from its Phase IIb EFFIOSAYIL 2 trial that shows its new antibody-based therapeutic Spevigo can prevent flare-ups in a rare and severe form of psoriasis, generalized postular psoriasis (GPP). The drug is the first treatment to be approved for the disabling and sometimes life-threatening condition.
Bristol Myers Reveals Positive Phase II Data for Leukemia CAR-T Cell Therapy Breyanzi
Bristol Myers Squibb has announced new clinical trial data for its leukemia CAR-T cell therapy Breyanzi. The data shows that the therapy has met its primary endpoint in the treatment of patients with relapsed or refractory chronic lymphocytic leukemia (CLL), beating standard care in complete response rate. Bristol Meyer intends to present the data at an upcoming conference.
Verana Health Publishes Real-World Data Ophthalmology Study Replicating Phase III Trials
Verana Health has announced the publication of its research in Ophthalmic Surgery, Lasers and Imaging (OSLI) Retina. The study used de-identified real-world data (RWD) and replicated two phase III trials for the use of Regeneron in wet age-related macular degeneration, making it the first of its kind in the field.
Ipsen CEO Talks Rare Disease Therapeutic Development and the Risks of Gene Therapy
David Loew, CEO of Ipsen, spoke at the JP Morgan Healthcare Conference about his outlook on the rare disease field. Loew noted that Ipsen was leaning into the market full-steam, regardless of pressure from Big Pharma companies attempting to crowd the space. That said, the CEO was more cautious on the gene therapy and neuroscience fronts.
Nature Medicine Perspective: Next-Gen Evidence-Based Medicine
The recent wave of digital health solutions, data analytics, and artificial intelligence (AI) is driving the newest generation of evidence-based medicine, according to a Nature Medicine perspective by Vivek Subbiah. These technologies are empowering novel clinical trials that utilize real-world evidence (RWE) and patient-centric approaches in a wide range of fields and disease contexts.
Chiesi Acquires Amryt Pharma in $1.48B Deal to Broaden Rare Disease Efforts
Chiesi Farmaceutici S.p.A and Amyryr Pharma Plc have announced a new deal wherein the former will acquire the latter in an all-cash transaction topping $1.48 billion. The deal would provide Chiesi access to Amryt’s drug pipeline, with a focus on broadening its efforts in therapeutic development for treatments for rare diseases.
Fluctuating Income as a Social Determinant of Health
A freshly-published study has identified fluctuating income as a social determinant of health (SDoH) that negatively impacts health outcomes. The paper, published in the Journal of Applied Psychology, surveyed gig workers, servers, delivery drivers, and other workers whose daily income is not steady. These workers faced health disparities in several key areas of health.
NIH Looking to Fund ‘All of Us’ Data Precision Medicine Research Projects
The US National Institutes of Health (NIH) has announced new funding for precision medicine research projects that use data from the All of Us Project, a collaboration between 27 groups within the NIH. The details of these funding opportunities are presented in two Funding Opportunity Announcements (FOAs). The first FOA is an RO3 entitled, “Small Grants to Enhance the Use of the All of Us Research Program’s Data (R03)”.
Improving Recruiting of Black and Rural Patients
In the US, Black and rural residents are often significantly underrepresented in clinical trials. This negatively impacts these populations and decreases the clinical potential of new therapeutics. In a recent Clinical Leader article, Chief Editor Ed Miseta discusses the underlying causes of clinical trial under-representation and insights from two studies on how to address it.
AJMC Staffs 2022 Favorite American Journal of Accountable Care Papers
The AJMC staff have just published their top 5 favorite articles published in The Journal of Accountable Care (AJMC) this past year. These papers covered the cost-effectiveness of virtual healthcare, how employers can get the most from high-deductible health plans, value-based care at the end of life, and more.
GAO Report Finds Certain Demographics Underrepresented in Cancer Drug Trials
A newly released report from the US Government Accountability Office finds that, despite years of health equity efforts, several key demographics are routinely underrepresented in clinical trials for new cancer therapeutics. This includes people with lower-incomes, rural residents, teens, older adults, and women. In addition, people from certain ethnic and racial backgrounds are also underrepresented.
New Real-World Evidence Study on Cannabinoid-Based Transdermal Gel Begins
Avicanna and Santé Cannabis have announced the launch of a new real-world evidence study into the use of the RHO Phyto™ CBG Transdermal Gel Topical Product in treating musculoskeletal inflammation and pain. The study will enroll and follow 100 patients using the treatment over 3 months, with a 3 month follow-up. Participants will submit self-reported symptom data, which will be collected alongside demographic information and medical history.
