New Application
Please complete the form to request an external research application pack.
The objective of the BioMarin External Research Program is to provide support for investigator-sponsored clinical research that illustrates the potential to foster knowledge and understanding of BioMarin products, product candidates or disease-states of interest. BioMarin support is typically provided as funding and/or drug.
BioMarin will consider providing funding or drug product for legitimate clinical research activities related to a BioMarin medicine or disease area of interest.
The program supports the following types of investigator-sponsored studies:
Please note that applications for non-clinical research should not be submitted through this system. This program does not provide support for animal model studies, continuing medical education (CME) or non-CME educational activities, event sponsorships, or charitable contributions. Applications for educational activities or sponsorship support should be submitted via the BioMarin grants system.
Please click below to request your external research application pack. Prior to submitting your proposal, please make sure to contact your regional BioMarin contact to discuss your concept and review the information on this page carefully.
Once your request has been received, you will receive an external research application pack comprising of a proposal form and a funding and product form to complete. Please complete the required forms and submit them, together with a copy of your current CV, to complete the application.
Once a completed application is received, our normal review process is four to eight weeks, but can take longer depending on the proposal.
For any questions related to the BioMarin External Research Program process, please contact your regional BioMarin contact or send an email to externalresearch@bmrn.com
Please complete the form to request an external research application pack.
Please complete the form if you require changes to your ongoing external research study.
A complete protocol is not required for submission. The proposal form will request you fill in details regarding your concept and includes the following required sections:
Please provide the contracting institution’s information with your application on the proposal form. Contracting is generally the most time-consuming part of this process, and submitters are expected to understand their institution’s contracting process and policies, and be capable of ushering a contract through to execution in a reasonable amount of time.
Please contact your legal department in advance of submitting your contract to ensure support for this process.
An estimated budget is required for your application submission. Please complete the budget template thoughtfully and ensure that all study-related expenses have been appropriately included and itemized, and that all line items are commensurate with fair market value for the study’s geographic region. Please ensure your institution has approved your budget prior to submission.
Please note that BioMarin will not reimburse for the following items:
BioMarin accepts applications for support for investigator-initiated research all year.
Each application is reviewed by the External Research Review Committee and decisions are made based upon medical and scientific merit, as well as available resources and strategic research priorities. A formal notification on the outcome of your application will be sent after the committee meeting.
The External Research Review Committee makes every effort to meet approximately once per month, but this is subject to change based on the number of applications received and committee member availability. Your completed application will be placed sequentially on the next available open timeslot on the committee’s agenda. Typically, our normal review process is four to eight weeks, but can take longer.
If your application is approved, please note there will be many items required to be in place and approved in order for support to commence. Typical items that must be in place are discussed below and there may be additional items required depending on each individual case.
Draft protocol and consent forms must be received within the timeframe requested, generally three months. Protocols and consent forms must be reviewed by BioMarin prior to submission to your Ethics Committee (EC)/IRB and incorporation of standard safety language into the protocol is required in many cases. Please note that if the research described in your final protocol is materially different from that in your approved application, BioMarin reserves the right to reconsider its support.
Final budget proposals must be received within the timeframe requested, generally three months. Once received, each budget undergoes a formal fair market value (FMV) analysis, and BioMarin will not compensate for costs that exceed FMV or are not directly related to protocol-required research.
Approved budgets will be divided into milestone payments and listed in the contract. In order to receive payment for each milestone, an interim study progress report and invoice must be submitted as outlined in the contract.
BioMarin requires all recipients of monetary support to submit a completed copy of the payee IRS W-9 Form (U.S.) or IRS Form W8 (ex-U.S.). Please note that BioMarin discloses financial information in compliance with local regulations.
As the sponsor of the study, the investigator and the institution must ensure that the study is conducted in accordance with the provisions of the ICH GCP Guidelines and all applicable local and regulatory requirements. The principal investigator must assume all regulatory responsibilities, including, but not limited to, IRB/EC approvals, regulatory approvals, posting to clinicaltirals.gov or other registry websites, and any and all reporting obligations to local regulatory authorities. BioMarin cannot advise on any sponsor-related obligations. Required documents for our process include:
Once the study has begun, BioMarin requires periodic study status updates, which are expected to include pertinent information on enrollment, projected milestone completion dates, projected publication dates and updated study documents. BioMarin also requires notification of any amendment to the original protocol after the research has begun. Any material change in the protocol, budget or contract is required to be reviewed by the committee and may or may not be approved for continued support.
An investigator conducting an external research study is required to provide BioMarin with a written report of the final study results as described in the contract. Upon study closure, the investigator will be required to certify that the study was conducted as described in the protocol, that the BioMarin funds and/or drug product were used solely to conduct or report the study, and that all safety reporting obligations were met. Any unused funds are required to be returned upon completion of the study.
Any planned publications related to the study must be submitted for BioMarin to review in advance of submission in accordance with the terms agreed to in the contract. BioMarin expects Investigators to publish the results of the study, whether or not the results are favorable to the product being studied. The investigator is required to comply with recognized ethical standards concerning publications and authorship, including the Uniform Requirements for Manuscripts Submitted to Biomedical Journals, www.icmje.org, established by the International Committee of Medical Journal Editors. It is required that BioMarin financial support is disclosed publicly for any publication related to the study.
All details regarding requirements for study setup, conduct, closure and publication will be provided in the external research application pack.
No. BioMarin does not fund studies that are already in progress or that have already been completed. Applications must be submitted and approved before study activities begin.
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