Real-world evidence needed for new work in SMA, other disorders

TREAT-NMD, Aetion team up to promote treatments for rare diseases

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by Andrea Lobo |

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TREAT-NMD and Aetion have formed a partnership to deliver real-world evidence and promote the development of new treatments for people with rare neuromuscular diseases, including spinal muscular atrophy (SMA).

The projects will combine the expertise of the TREAT-NMD global registry network, which collects de-identified data from patients worldwide, with Aetion’s abilities in the design and analysis of real-world data studies. The goal, ultimately, is to generate rigorous real-world evidence to advance treatment development.

“TREAT-NMD is pleased to partner with Aetion on a range of projects to generate the scientific evidence necessary to develop and deliver novel treatment solutions to people with neuromuscular diseases around the world,” David Allison, CEO of TREAT-NMD, said in an Aetion press release.

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Partners cite need for ‘rigorous’ real-world evidence

In the field of neuromuscular diseases, researchers find it difficult to access high-quality registries of patients’ experience and medical information, hindering the subsequent generation of scientifically rigorous real-world evidence.

Such evidence results from the analysis of real-world data related to patients’ health status and healthcare, concerning its usage, and the benefits and risks of medical treatment.

These data are used by many of the players in rare-disease treatment development. For example, regulators, such as the U.S. Food and Drug Administration, use such data to monitor postmarket safety and adverse events. The information also is needed by the healthcare community, to develop guidelines and decision-support tools for use in clinical practice. Meanwhile, the developers of medical products use such data to support clinical trial designs and studies to generate novel treatments.

The new TREAT-NMD and Aetion collaboration aims to accelerate the delivery of robust real-world evidence using scientifically validated methodologies. This will enable easier access to essential rare disease data sources and scientific study designs to support biopharmaceutical researchers.

“We will work in concert to ensure that researchers have access to rare disease data while also leveraging Aetion’s trusted RWE [real-world evidence] capabilities,” Allison said.

Added Nicolas Deltour, vice-president of real world solutions at Aetion, “The richness of the TREAT-NMD registry network combined with Aetion’s RWE analytics technology and expertise will together play a pivotal role in supporting pharmaceutical companies in advancing treatments and better outcomes for neuromuscular patients in Europe and around the world.”

TREAT-NMD is pleased to partner with Aetion on a range of projects to generate the scientific evidence necessary to develop and deliver novel treatment solutions to people with neuromuscular diseases around the world.

Besides SMA, which is typically caused by mutations in the SMN1 gene, these neuromuscular disorders include Duchenne muscular dystrophy, Charcot-Marie-Tooth disease, and Becker muscular dystrophy.

TREAT-NMD is a network in the neuromuscular diseases field that supports every stage of the process of developing new medications, to ensure they reach patients as quickly as possible.

The network focuses on the development of tools that can be used by scientists, clinicians, and biopharmaceutical companies, from preclinical development to clinical practice, and on establishing the best care for patients with neuromuscular disorders.

Aetion is a healthcare analytics company that, through its Aetion Evidence Platform, provides scientifically validated software that analyzes real-world data to transform it into reliable evidence from real-life patients. The platform is used by biopharma companies and regulators to assess the safety, effectiveness, and value of medical treatments and technologies.

“Aetion is inspired by TREAT-NMD’s mission to operate a collaborative, inclusive global network and data infrastructure to advance the diagnosis, treatment, and care for neuromuscular patients globally,” Deltour said.