Tips on Writing to the FDA

The voice of the patient has a role in the drug development and approval process. In fact, recent legislation mandates an increased emphasis on incorporating the patient perspective into the drug approval process.

There are no treatments currently approved by the FDA specifically for NTM lung disease. On August 8th, 2018 the FDA convened a meeting of the Antimicrobial Drugs Advisory Committee to review the potential new treatment, amikacin liposome inhalation suspension (ALIS), and make a recommendation to the FDA on whether to approve it for the treatment of non-cystic fibrosis bronchiectasis (NCFBE) patients with refractory Mycobacterium avium complex infections.

The committee voted to recommend approving the drug for this indication. The discussion at the meeting focused in part on the endpoint used during the Phase 3 clinical trial, sputum conversion to culture negative. Both the committee and the FDA questioned whether this outcome benefits the patient clinically, despite the fact that this is the outcome sought in the treatment guidelines specifically because it may lead to a patient no longer needing antimicrobial therapy.

The Advisory Committee provides recommendations for the FDA to consider. The FDA then makes the final decision. As part of this process, the FDA conducts a thorough review of the clinical trials already done, the evidence presented, and possible future studies the company may undertake once it is approved.

We are asking you to use the bullet point template provided (click here) as a guide to share your perspective with the FDA as they approach their September 28th deadline for a decision on approval of inhaled liposomal amikacin.

The Committee and approvals process highlights just how much we must show the FDA that there is urgent unmet need and that the patient community is just as, if not more, concerned about the side effects of the antibiotics used currently. As experts in your illness, you are in the best position to tell the FDA what your needs and wants are: the approval of a therapy for your disease.

By writing to the FDA to express your concerns and relay your own experiences, we show the FDA that there is an empowered and engaged patient community that stands ready to partner with them and with the companies willing to invest and study treatments to meet the needs of rare disease communities.

Click here for information on sending your letter to the FDA

Click here to read NTMir’s letter to the FDA