FDA: Rare disease treatments remain a top priority for the FDA during the COVID-19 pandemic.

During the COVID-19 pandemic, patients with rare diseases were the most vulnerable to direct or indirect infections.
the FDA's work during this period is becoming increasingly important in order to truly protect the interests of patients with rare diseases.
guarantee a stable supply of high-quality life-saving drugs is essential for many patients with rare diseases.
COVID-19 is a major public health priority, but advancing treatment for rare diseases to help ensure ongoing medical care for patients with rare diseases remains a top priority in the FDA's work.
a recent study conducted across the United States by the National Organization for Rare Diseases, which found that 74 percent of patients with amyotrophic lateral sclerosis (ALS), Dudsiyoyo (DMD), sickle cell disease, multiple cancers and other diseases said they had to cancel medical appointments because of the COVID-19 pandemic.
other disturbing findings, 29 percent of respondents said they lost their jobs temporarily or permanently because of the COVID-19 pandemic;
FDA staff, every day, do their best to promote the expanded use of research drugs by supporting sponsors and research institutions in clinical trials, and to help sponsors and research institutions work to prevent the COVID-19 pandemic from hindering the development of rare disease sedothing drugs and ensuring the continued availability of approved products critical to rare disease patients.
as the FDA continues to make it a priority to promote the development of new treatments for rare diseases, we will do everything we can to minimize the impact of COVID-19 on the progress being made.
mitigating the impact of pandemics on clinical trials of rare disease therapeutic drugs is critical, clinical trial design and implementation of rare disease therapeutic drugs are often more challenging.
in fact, the lives of patients with serious conditions depend on the timely completion of clinical trials and the continuous development of safe and effective therapies.
this is especially true for patients with rare diseases because there is often no available treatment.
Recognizing the threat posed by COVID-19 conducting clinical trials, the FDA has issued and revised updated the guidelines, which are "guidelines for industry, research institutions, and ethical review committees (IRB) to conduct clinical trials of pharmaceutical products during the COVID-19 pandemic", which is designed to help pharmaceutical companies, clinical research institutions, and others ensure the safety of clinical trial participants and minimize the risk of trial integrity.
quarantine isolation, clinical trial site closure, travel restrictions, interruption of the drug supply chain for clinical trials, and other factors may interfere with the normal completion of clinical trials, or patient participation in clinical trials, thus affecting the timeliness of the urgent availability of the drug.
based on regular contacts with stakeholders, the FDA has revised and updated the guidelines several times as needed.
will be a key tool for effectiveuse when discussing with the sponsors how to conduct clinical trials in response to the inevitable deviations caused by current public health emergencies.
, the FDA has released a source on its website for patients on the COVID-19 pandemic, and FDA staff participated in webinars and virtual meetings with patient advocacy groups to answer questions and help ensure that patients' voices are heard.
On June 8 this year, the FDA participated in a seminar organized by the Organization of Patient systorsis (MDA).
access to drugs in research, extended use, and trial rights For patients with rare diseases, certain malignancies, COVID-19 infections, and other serious or life-threatening diseases or conditions, the FDA's Extended Access Program is an important tool.
, without the absence of other comparable or satisfactory alternative therapies, extended use programs enable sending relevant patients access to therapeutic drugs in clinical trials outside of clinical trials.
FDA recognizes that, in many cases, extended use is the only way for patients with rare diseases to obtain drugs in clinical trials outside of clinical trials.
, patients will also have access to these drugs in clinical trials if clinical trials are completed.
the FDA has placed more emphasis than ever on helping patients and healthcare professionals evaluate access to drug-in-the-uthdrug, for example by involving relevant patients in clinical trials, or by expanding the use of drugs (sympathetic drugs), to give patients in dire need of access to drugs that are not approved outside of clinical trials. In addition,
for patients with amyotrophic lateral sclerosis (ALS) and certain other diseases, the FDA encourages pharmaceutical companies to provide access to drugs in research after clinical trials are completed and to continue to have promising drugs under the Extended Use Program.
However, it is important to note that the FDA does not allow pharmaceutical companies to offer products with extended usage coverage.
, therefore, in order for patients to be treated under this mechanism, the drug manufacturer or the applicant must be willing to provide the relevant drug.
Although the FDA reviews clinical trials and authorizes extended use, the FDA is not responsible for reviewing or approving requests for use of drugs under the Right to Trial Act.
the drug manufacturers have a greater say in the information on whether the drug under study meets the conditions for use under the Probation Act.
the potential impact of the continued availability of therapeutic drugs to patients with rare diseases FDA actively works with pharmaceutical manufacturers, including those focused on rare disease treatments, evaluating the entire supply chain of related drugs, including API, end-of-product formulations, and other components that may have problems in any part of the supply chain as a result of the COVID-19 pandemic.
, for example, FDA staff have been in close contact with pharmaceutical manufacturers of products such as intravenous immunoglobulins to help ensure continued supply of this critical drug to patients with primary immunodeficiency.
FDA accelerates reviews and approvals through rapid priority reviews to help effectively alleviate the shortage of FDA-approved treatments for lupus, a rare disease.
integrate patient perspectives and prepare for clinical trials Every rare disease may face specific challenges associated with the COVID-19 pandemic.
, for example, patients with amyotrophic lateral sclerosis (ALS) often experience breathing and lung function problems and therefore have a higher risk of serious complications.
is an important example of alS in the search for effective treatments for rare diseases.
despite some advances in treatment that could help patients prolong their lives, there is still no cure for the eventually deadly neurodegenerative disease.
However, there are a number of clinical trials under way, and the FDA hopes they will lead to more effective treatment options for ALS.
the FDA recently announced that it will publicly seek advice on setting up a network of clinical trials for rare diseases.
this is a valuable opportunity for the FDA to receive public advice and input, to hear public advice and input on practical steps to launch, implement and maintain the Global Clinical Trial network and ultimately to achieve its goals, including specific considerations for establishing a network of clinical trials for a range of rare diseases.
this is part of the FDA's efforts to continue the Rare Disease Cures Accelerator program. The
Rare Disease Syllabus Initiative is a collaborative scientific program to develop a common standardized platform to improve the characterization of rare diseases, incorporate patient stakes into clinical outcome assessments, and prepare for clinical trials before the drug is available.
With the development and change of THE PUBLIC health emergency of COVID-19, the FDA has been committed to promoting the development of relevant pharmaceutical products, helping to control the COVID-19 pandemic, and unswervingly fulfilling its mission to protect public health by ensuring the safety, effectiveness and quality of medicines.
FDA focuses on promoting pharmaceutical product development for the treatment of serious diseases, such as amyotrophic lateral sclerosis (ALS), Duxony muscular dystrophy (DMD), sickle cell disease and other rare diseases, and will continue to vigorously pursue development programs.
Original Title: FDA CDER Acting Director, CBER Director: During the COVID-19 pandemic, rare disease treatment drugs remain a top priority for the FDA.