The FDA has witnessed unprecedented and advanced medical breakthroughs over the past decade. However, progress for complex neurological diseases, such as amyotrophic lateral sclerosis (ALS), has been challenging due to complexity and the fact that the underlying causes of severe neurological diseases are not well understood. The lack of an ALS treatment has deeply frustrated patients and caregivers. After many meetings with the ALS community (including patients), the FDA issued a final guidance for industry: “Amyotrophic Lateral Sclerosis: Developing Drugs for Treatment”—offering industries with the FDA’s current scientific thinking to support ALS drug development and commercialization efforts. The FDA declares that ALS patients can tap into the expanded access (compassionate use) and Right to Try options if available. 

A Collaborative Work Product

The FDA reports that the final guidance results from intensive collaboration among experts across the agency and incorporation of inputs from all key stakeholders: from patients and their care givers to researchers and advocates.

Up-to-date Non-Binding Recommendations

The FDA declares that the final guidance reflects “up-to-date information on our recommendations to help advance the development of products for ALS patients” and these range from the design of ALS-focused clinical research to ways and means to measure effectiveness. A key underlying point to consider is their request for lots of communication—starting early in the drug development process-as the FDA seeks to support the expeditious, efficient quality-driven and safe development of treatments for ALS. The recommendations are non-binding, meaning they are open to alternative approaches.

Clinical Trial Design Points

All patients participating in ALS clinical trials should receive the best standard of care while no patient should be denied effective therapy in exchange for randomization to a placebo-only arm.  With a recognition and acknowledgement as to the importance of the placebo in clinical research, FDA offers alternative models such as master protocols, adaptive designs, and enrichment strategies.

Use of Expedited Development and Approval Program for ALS Drug Candidates

The FDA has positioned that it is ready to use the expedited development and approval programs now available to offer additional support to industry developing ALS treatments. The goal: produce treatments for ALS patients as fast as possible.

Compassionate Use

Additionally, the FDA stands ready to support ALS patients access investigational products where and when it can do so factoring in safety, risks, etc. through expanded access (compassionate use) or utilizing the new Right to Try Act. The FDA does note that the drug company must be willing and able to provide investigational product to ALS patients. Importantly, the FDA encourages the FDA to continue to make ALS investigational product available to ALS patients even after the clinical trial. This latter point is important as many very sick patients are denied access to certain investigational medicines once a clinical trial ends.

Many company may not share product in expanded access programs due to the fear that any serious adverse events may delay the drug development timeline—hence costing a company up to hundreds of millions of dollars. The FDA reminds the reader that FDA reviewers in the context of the expanded access must consider the context in which the investigational drug or biologic is provided. They emphasize it is a rare occurrence that drugs involved with expanded access actually lead to delays in the development timeline.

Confidentiality & Transparency

The FDA declares they are prohibited by law from releasing confidential information about investigational products and hence make a public request to ALS drug developer to offer more transparency about the status of their research as well as information on availability of products under expanded access or Right to Try to support patients, caregivers, and physicians discussions about what options are actually available.

Call to Action: TrialSite News seeks to make the business of clinical trials easier to understand for a wide audience.  With a focus on the clinical research site and investigator (and their staff) we cover what in all actuality is a massive, dynamic marketplace involved with the heavily controlled testing of pre-approved investigational drug products worldwide. Daily news aggregated, curated and summarized so that a wide audience can appreciate the current affairs of the world of clinical trials.

We are committed to tracking all trials and investigational products not to mention actual drug companies that help make the investigational product available for those in need. We have patients contacting us about situations where very sick people are denied access to expanded access due to a rule or a policy. We do not think that is right and surely are searching for situations where patients are denied access.

Source: U.S. Food & Drug Administration

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