This assistance program is well-suited to the MMS team and the organization’s expertise. Ultra-rare conditions make up the majority of rare diseases and require differing approaches within the framework of identical statutory requirements. An experienced partner can provide valuable insight and solutions when navigating the laws, guidelines, and general regulatory standards that govern the required demonstration of a drug’s safety and efficacy.
MMS understands the importance of a customized regulatory strategy and the challenges these programs face. Over the past decade MMS has supported rare disease programs, including early phase research through approval for use in more than 12 therapeutic areas and welcomes the opportunity to advance additional rare disease programs.
For those advocacy groups that MMS selects, team members’ support could include:
- Regulatory Strategy, including training on Investigational New Drug Applications (IND) requirements and consulting on nonclinical and formulation development
- Medical Writing support, including the drafting of Investigator INDs, nonclinical study reports, Office or 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 Dr. Uma Sharma, Chief Scientific Officer and Founder, 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.”
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 share strategic input.
“This program was brought to fruition by a colleague who recommended it as a better way to use pre-allocated volunteer hours,” said Alan Nicolle, Senior Director, European & South African Operations. “People like this who lead change make MMS a best place to work for those with an innovation-first, Sense of Urgency and Leadership (SOUL) mindset.”
Qualifications to apply for MMS support include the following:
- Verified need for effective/alternate treatments for the ultra-rare disease
- Scientific justification/rationale for efficacy in the ultra-rare disease population
- Must be a pre-established patient advocacy or parent group
- Focus on products with a limited commercial viability to continue development or pursue licensing
To apply for support, complete and send an application to the attention of the ‘Ultra-rare disease Assistance Program’ by emailing firstname.lastname@example.org. For details on MMS rare disease expertise, visit https://www.mmsholdings.com/rare-disease/.
MMS Holdings (MMS) is an innovative, data-focused CRO that supports the pharmaceutical and biotech industries with a proven, scientific approach to complex trial data and regulatory submission challenges. Strong industry experience, technology-enabled services, and a data-driven approach to drug development make MMS a valuable CRO partner, creating compelling submissions that meet rigorous regulatory standards. With a global footprint across four continents, MMS maintains a 97 percent customer satisfaction rating, and the company has been recognized as a leading CRO in Global Health & Pharma’s international awards programs for the last three consecutive years.