RARE Daily

CRO MMS Launches Pro-Bono Regulatory Assistance Program for Ultra-Rare Disease Patient Advocacy Groups

August 12, 2021

MMS Holdings has launched a pro-bono support program aimed at advancing research for ultra-rare diseases.

Photo: Uma Sharma, chief scientific officer and founder of MMS

The data-driven contract research organization said it will select from patient advocacy groups with demonstrable need and will provide complimentary regulatory assistance.

“We are proud to have built a program that affords patients and families with ultra-rare diseases the opportunity to move closer to effective therapies,” said Uma Sharma, chief scientific officer and founder of MMS.

Ultra-rare conditions make up the majority of rare diseases and require differing approaches within the framework of identical statutory requirements, MMS said. It will offer insight and solutions when navigating the laws, guidelines, and general regulatory standards that govern the required demonstration of a drug’s safety and efficacy.

To qualify for the MMS support, there must be a verified need for effective or alternate treatments, scientific rationale for efficacy in the ultra-rare population, a pre-established advocacy group, and a focus on a product with limited commercial viability in need of help to continue development or pursue licensing.

Among the services MMS will provide include regulatory strategy, medical writing support, grant writing, and other services dependent on assessed needs.

As part of the effort, MMS will create an advisory board with experts in regulatory strategy and writing, other industry thought leaders, and pharma or biotech sponsor-contributors who will be engaged with support of project selection, participation in reviews, and sharing of strategic input.

Over the past decade MMS has supported rare disease programs, including early phase research through approval for use in more than 12 therapeutic areas.

“Many of our colleagues come into this field with an altruistic focus – to help others,” said Sharma. “This program will provide a new perspective on radically new technologies and therapies that may never have made it to the licensing stage and is set to help improve the lives of many.”

Author: Rare Daily Staff

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