MMS support program to help ultra-rare disease research

By Emily Jarecki

- Last updated on GMT

(Marko Geber/iStock via Getty Images Plus)
(Marko Geber/iStock via Getty Images Plus)

Related tags Rare disease Patient centricity Patient recruitment orphan disease

The CRO has launched an initiative that offers help navigating regulatory issues for ultra-rare disease patient advocacy groups that demonstrate a need.

The contract research organization (CRO) MMS Holdings Inc. (MMS) has recently announced its new pro-bono support program. The initiative aims to advance research for ultra-rare diseases by providing regulatory assistance for patient advocacy groups that show the greatest need.

Certain patient advocacy groups that intend to help ultra-rare disease research and treatment often face certain challenges that make it difficult for them to move towards drug development and treatment. Every ultra-rare disease requires a different approach while remaining within the framework of statutory requirements and a CRO like MMS can provide insights and solutions when navigating laws, guidelines, and general regulatory standards for a drug’s safety and efficacy.

“Our target is the underserved patient population with ultra-rare serious diseases that have prevalence or other complicating factors effectively cutting them off from the traditional drug development model,” ​said Amanda Beaster, senior global regulatory affairs manager at MMS.

MMS will oversee and develop the necessary framework that will allow groups to have access to regulatory professionals experienced in drug development. The company will also create an advisory board for the support program equipped with experts, leaders, and contributors that will be engaged in project selection, participation in reviews, and sharing strategic input.

For the selected advocacy groups, support could include:

  • Regulatory strategy, including training on IND requirements and consulting on nonclinical and formulation development
  • Medical writing support, including the drafting of Investigator INDs, nonclinical study reports, Office of Orphan Products (OOPD) grant writing, or others suited to the program
  • Other services dependent upon assessed need

“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. “Many of our colleagues come into this field with an altruistic focus - to help others; 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.”

“Ultra-rare” selection

Within this program, pre-established patient advocacy groups that apply will be evaluated and selected based on demonstrable need and overall feasibility. Considering the term “ultra-rare” does not yet have a US regulatory definition, MMS is not assigning a strict numerical prevalence cut-off.

“We will most likely consider applications for diseases with fewer than 30 affected patients worldwide, as diseases with conditions with broader patient populations are more likely to find support with other entities,” ​Beaster said. “However, if a patient advocacy group feels they can supply adequate justification for ultra-rare status, we encourage them to submit an application.”

“Over time, as the way the medical community addresses these diseases changes, likely so will regulations and/or incentives,” ​Beaster added. “However, until those changes occur, MMS wants to help stand in the gap by donating our time and expertise to provide additional resources to this underserved patient population​.” 

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