FDA Invites Public Comment On Adaptive Designs

October 25, 2018

FDA Invites Public Comment On Adaptive Designs

The Food and Drug Administration (FDA) released an updated draft guidance to industry in early October entitled, “Adaptive Designs for Clinical Trials of Drugs and Biologics: Guidance for Industry.”

The draft guidance reviews best practices for trial design, analysis, and reporting of clinical trials using different adaptive design strategies. The public is invited to submit comments by November 30th.

Adaptive clinical trials are designed to change in response to an interim analysis. Unlike non – adaptive clinical trials, an adaptive trial might close a treatment arm, might increase or decrease the sample size, or might change the randomization allocation. Adaptive clinical trials can increase statistical efficiency, can be more appealing to patients and families, and can increase generalizability of trial results. Adaptive trials also pose risks – increased Type 1 error and possible bias.

The draft guidance recommends that sponsors engage early with the FDA about possible adaptive clinical trials. Specifying the design and decision rules in advance are critical to avoid error and bias. Restricting access to interim trial results is important to avoid changing the behavior of the sponsor, contract research organization (CRO), clinical trial sites, or study participants. Analysis plans must account for the adaptive design and should consider the use of simulations in planning.

TRiNDS offers biostatistics and data management services. Are you thinking about starting an adaptive clinical trial? Submit a request for a quote today.

Newborn Screening Update for Duchenne Muscular Dystrophy

October 18, 2018

Newborn Screening Update for Duchenne Muscular Dystrophy

The Duchenne muscular dystrophy (DMD) community took two big steps toward newborn screening this month. Newborn screening programs are important because they allow early diagnosis even before symptoms are observed. Newborn screening facilitates early treatment and rapid development of new therapies for infants everywhere.

When infants are born in the United States, a heel prick blood sample is taken onto a paper card. The card is tested for a set of metabolic and genetic disorders at a public health laboratory. Once a disorder has been added to the national screening panel, each state determines whether to add it to their state testing card. You can learn more about newborn screening at http://www.babysfirsttest.org/.

Two major DMD patient advocacy groups shared news about their work to support newborn screening for DMD. Parent Project Muscular Dystrophy (PPMD) and CureDuchenne are working on different ways to make newborn screening a reality for everyone.

PPMD announced the launch of their newborn screening pilot in New York State (https://www.parentprojectmd.org/ppmd-launches-duchenne-newborn-screening-pilot-in-new-york-state/). Their pilot is a collaboration between industry, New York State Department of Health, and the Centers for Disease Control. The pilot study will collect data needed to add DMD to the national and state panels. Once DMD is added to the state testing cards, then every infant will be tested for free.

CureDuchenne announced a new partnership with Baebies to develop newborn screening tests for DMD (https://www.cureduchenne.org/press-release/cureduchenne-partners-with-baebies-to-accelerate-newborn-screening-for-duchenne-muscular-dystrophy/). These tests would be ordered by a doctor and paid for by health insurance. This would provide testing for infants born before the national panel is changed, or for infants born in places that do not have DMD on the state card.

The DMD community is supported by many strong and devoted advocacy groups. Everyone is committed to supporting families, advancing research, and thinking creatively about how to help people with DMD.

Success at WMS

October 10, 2018

World Muscle Society was a great success for TRiNDS, CINRG, and TRiNDS’ clients and collaborators. In addition to three days of presentations, researchers presented over 175 posters in a poster session. Poster presentations shared results from all different types of research and clinical trials about all different types of neuromuscular disorders. We had a great time sharing our results at the poster session and connecting with each other.

The Cooperative International Neuromuscular Research Group (CINRG) is a consortium of researchers who work together on industry sponsored and academic research projects about people with neuromuscular disorders. Everyone works hard to answer important research questions for patients and families everywhere. Congratulations CINRG researchers on your WMS presentations!

Conferences are a great opportunity for the scientific community to share results and build relationships for future projects. We always come home more energized and more committed to neuromuscular research than ever.

TRiNDS is the coordinating center for CINRG. TRiNDS serves industry and academic research clients working for neuromuscular disorders around the world.

Click here to request a quote for services.

Building Your Team: Becker Muscular Dystrophy

October 3, 2018

Building Your Team: Becker Muscular Dystrophy

Next week is Becker Muscular Dystrophy (BMD) Awareness Week, a time for everyone to recognize the families around the world living with BMD.

BMD affects 1 in 35,000 males worldwide. People with BMD show symptoms of muscle weakness in the teen years and can often keep walking into adulthood. Even though good outcomes are possible with BMD, it’s important to work with the medical team to maintain function and good health at diagnosis and beyond. Just like other kinds of muscular dystrophy, people with BMD can develop heart symptoms and other important changes that need management over the lifespan.

Gathering a strong medical team is the best way to get started with managing BMD. The Muscular Dystrophy Association has some great resources for providers and patients. Check out their medical management guide and get started today. (https://www.mda.org/disease/becker-muscular-dystrophy/medical-management)

TRiNDS works with academic and industry researchers on all types of neuromuscular disorders. If you’re getting ready to launch a NMD project, click here to request a quote for TRiNDS’ services.