Having a combined 100 years of Industry, CRO and Consulting experience, allows us to confidently navigate the regulatory agencies.

Our experts have proven track records of navigating the agencies in both the United States and Europe.
4

Breakthrough Designations


46

US Orphan Drug Designations


18

EU Orphan Drug Designations


43

INDs


26+

Agency Meetings


Whether you are needing an application from start to finish or a detailed development plan, we support you and your team. Contact us >
FDA & EMA ORPHAN DRUG DESIGNATION
Having received over 60 Orphan Drug Designations in the United States and Europe combined, our team has the experience needed to confidently execute on our clients’ behalf.

Getting an orphan designation can be challenging. One of the major challenges is presenting adequate scientific rationale and translating that science into regulatory language, a common mistake seen on most orphan applications.

RareMoon’s team of US and EU Regulatory Scientists along with our Epidemiologist will offer strategic advice and further execute on Orphan Applications for our clients. A final draft will be submitted to you or the to the FDA’s Office of Orphan Products Development (OOPD) or the European Medicines Agency (EMA) at your request.

Key Benefits


    United States

  • Seven (7) years of marketing exclusivity
  • Protocol assistance
  • Research grants to help fund clinical investigations for orphan products
  • Waiver of the PDUFA application fee (>$2,000,000)
  • Tax credits covering up to 50 percent of clinical trial costs

    Europe

  • Ten (10) years of marketing exclusivity
  • Protocol assistance
  • Additional scientific advice specific for designated orphan medicines
  • Reduced fees throughout program development
FAST TRACK
The benefit of receiving a Fast Track designation is seen in the increase in communication between you and the FDA, identifying efficiencies in both your development plans and the agency review process. These are designated for drugs that address both an unmet medical need and used treat serious or life-threatening condition.

Over 30% of novel drugs approved in the US in the last two years were designated Fast Track.

Contact us to find out if you are ready to be “Fast Tracked.”
BREAKTHROUGH THERAPY DESIGNATION
Not only do you receive all the benefits you see with other expedited review pathways, but when you receive a Breakthrough Therapy designation you also receive enhanced communication and input from Senior Management. With this direct involvement in your drug’s development and with the assignment of a “cross-discipline project lead”, you have a personal driver moving things forward and ensuring reviews are efficient across all divisions.

We call that FDA love.
PRIME
The PRIME scheme, launched by the EMA in February 2016 is much like the FDA’s Breakthrough Therapy program. In a three-month period, the EMA received about 35 applications, and granted a mere 20% of those applicants. It’s a more difficult achievement however the potential benefits make this challenge acceptable.

Key Benefits


  • Appointment of a rapporteur from the Committee for Medicinal Products for Human Use (CHMP) or from the Committee on Advanced Therapies (CAT) in the case of an advanced therapy to provide continuous support and help to build knowledge ahead of a marketing-authorization application
  • Organization a kick-off meeting with the CHMP/CAT rapporteur and a multidisciplinary group of experts, so that they provide guidance on the overall development plan and regulatory strategy
  • Assignment of a dedicated contact point
  • Provides scientific advice at key development milestones, involving additional stakeholders such as health-technology-assessment bodies, to facilitate quicker access for patients to the new medicine
  • Confirmation of potential for accelerated assessment at the time of an application for marketing authorization
PRIORITY REVIEW VOUCHER
The Priority Review Voucher Program passed by Congress in 2007 has incentivized more and more pharmaceutical companies to develop drugs for neglected and rare pediatric diseases. Like all expedited approval programs, a product application must meet the criteria of either the Neglected Tropical Disease or Rare Pediatric Disease PRV Programs. Find out more.

The benefit of this program is a 6-month review over the typical 10-month review which can mean enormous market and financial benefits for competing companies to gain time, market access, brand recognition and more. Recently we’ve seen these vouchers sell for over $350M. If you meet the criteria, this is a no brainer.
AGENCY MEETINGS
FDA Types A, B, and C
EMA Meetings

Communicating with the Agencies is an important step to reaching your development goals and pivotal milestones. At RareMoon we believe in “inviting the fox into the hen house.” Gaining Agency support is imperative to accelerating your programs.

Depending on your development goals you will want to consider meetings with both the FDA and EMA.

We understand the proper use of these meetings and the specific topics of which you should be discussing which is imperative. We know the potential hurdles, challenges and questions the Agencies may pose. It is ultimately more economical, to proactively explore and mitigate potential issues than react when the Agency brings them to your attention.

Whether you need complete assistance or just a piece of the puzzle, we can help with:

  • Meeting Requests
  • Advice & Preparation
  • Meeting Package Content & Execution
  • Submission
  • Representation
  • Pre & Post Meeting Briefing
INVESTIGATOR'S BROCHURE
An IB serves many purposes and is useful to all parties involved in your program’s development including investigators, stakeholders and regulatory agencies. It is multidisciplinary, will summarize the main elements of your entire development program and should be updated regularly.

The main sections of the IB will include:

  • Summary
  • Introduction
  • Physical, chemical, and pharmaceutical properties and formulation
  • Nonclinical studies
  • Clinical studies & human effects
  • Summary of data and guidance for use

A concise and focused presentation style is a challenge in preparing your IB. While it needs to be comprehensive, it also needs to be readable. Our experts have the experience required to comprehend data and information, and author IBs in a language that is both informative and concise.
ANNUAL REPORT
Both the EMA and FDA require Sponsors to submit annual development reports. This might include ODD/OMP annual reports, reports for expedited program designations, or IND and IND amendments. While these should be concise, they can still be daunting.

Given our work and experience in authoring Agency documents, we can take these off your To-Do list and get them submitted on time.
INVESTIGATIONAL NEW DRUG (IND) GAP ANALYSIS
Our Nonclinical experts can thoroughly review and assess the current state of your development with an IND gap analysis. This analysis will outline a plan to advance your program to IND as swiftly and efficiently as possible.
REGULATORY ROADMAP
A Regulatory “driven” roadmap for your program is a key ingredient in your early development planning. A well-prepared map will align your proposed nonclinical and clinical trials with the objectives and ultimate goal of your program. With a thorough understanding of your ultimate goals, and working backwards from there, RareMoon can preemptively identify potential challenges, propose new approaches and offer a timeline to key milestones. Our experience with the regulatory authorities and our knowledge of their expectations, is the differentiating factor in our Regulatory Roadmaps.
STRATEGIC REVIEW & FEASIBILITY ASSESSMENTS
We realize that in today’s pharmaceutical world, and with virtual and “baby biotech’s” on the rise, our clients have varying resources and needs. We have found some to have the knowledge and regulatory expertise within their organization, yet do not have the time or recourses to execute on applications. Others have considerable drug development experience yet are seeking regulatory experts to help strategize and execute on program applications.

For this reason, we understand the need for a strategic or peer reviews, and feasibility assessments, allowing the Sponsor to confidently move forward.

This assessment may determine:

  • Prevalence estimate
  • Standard of care, describing current management of the disease
  • Unmet medical need
  • Risk assessment for market segment
  • Timelines or financial commitments
  • Peer review or QC of Regulatory applications