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https://blogs.fda.gov/fdavoice/index.php/20...xpanded-access/

 

By: Scott Gottlieb, M.D.

 

FDA is committed to expanding access to safe and effective treatment options for patients with rare, debilitating, and sometimes fatal diseases. These patients face unique medical challenges. Sometimes there isnÃÆâ€â„¢ÃƒÆ’ƒâہ¡ÃƒÆ’‚¢ÃƒÆ’¢Ã¢Ã¢Ã¢Ã¢â€š¬Ã…¡Ãƒâ€šÃ‚¬ÃƒÆ’…¡Ãƒâہ¡ÃƒÆ’‚¬ÃƒÆ’¢Ã¢Ã¢Ã¢Ã¢â€š¬Ã…¡Ãƒâ€šÃ‚¬ÃƒÆ’…¾Ãƒâہ¡ÃƒÆ’‚¢t an FDA-approved drug to adequately address the needs of a patient with a rare disease. Therefore, the agency needs to take new steps to enable more patients with unmet needs to get access to promising treatments prior to full FDA approval.Two examples of the recent steps FDA has taken in pursuit of these goals are improvements we made to our Expanded Access Program and our Orphan Drug Program. These programs are high priorities of mine. They address the needs of patients with some of the most challenging conditions. Making sure thereÃÆâ€â„¢ÃƒÆ’ƒâہ¡ÃƒÆ’‚¢ÃƒÆ’¢Ã¢Ã¢Ã¢Ã¢â€š¬Ã…¡Ãƒâ€šÃ‚¬ÃƒÆ’…¡Ãƒâہ¡ÃƒÆ’‚¬ÃƒÆ’¢Ã¢Ã¢Ã¢Ã¢â€š¬Ã…¡Ãƒâ€šÃ‚¬ÃƒÆ’…¾Ãƒâہ¡ÃƒÆ’‚¢s a close relationship with the efforts we take to expand pre-approval access to promising treatments, and the work of our orphan drugs program, is a key step toward maximizing opportunities for patients. To further achieve these goals, weÃÆâ€â„¢ÃƒÆ’ƒâہ¡ÃƒÆ’‚¢ÃƒÆ’¢Ã¢Ã¢Ã¢Ã¢â€š¬Ã…¡Ãƒâ€šÃ‚¬ÃƒÆ’…¡Ãƒâہ¡ÃƒÆ’‚¬ÃƒÆ’¢Ã¢Ã¢Ã¢Ã¢â€š¬Ã…¡Ãƒâ€šÃ‚¬ÃƒÆ’…¾Ãƒâہ¡ÃƒÆ’‚¢re announcing that FDA is widening the scope of the new Expanded Access Navigator tool, a comprehensive online information resource maintained by the nonprofit Reagan-Udall Foundation to facilitate pre-approval access to drugs. Previously this tool was rolled out for drugs that treat cancer. It will now apply to drugs that treat orphan diseases.

 

As I discussed in my testimony at a recent hearing of the Energy and Commerce Committee, expanded access programs play an important role for terminal patients who donÃÆâ€â„¢ÃƒÆ’ƒâہ¡ÃƒÆ’‚¢ÃƒÆ’¢Ã¢Ã¢Ã¢Ã¢â€š¬Ã…¡Ãƒâ€šÃ‚¬ÃƒÆ’…¡Ãƒâہ¡ÃƒÆ’‚¬ÃƒÆ’¢Ã¢Ã¢Ã¢Ã¢â€š¬Ã…¡Ãƒâ€šÃ‚¬ÃƒÆ’…¾Ãƒâہ¡ÃƒÆ’‚¢t have access to conventional, FDA-approved treatments. Because many orphan diseases donÃÆâ€â„¢ÃƒÆ’ƒâہ¡ÃƒÆ’‚¢ÃƒÆ’¢Ã¢Ã¢Ã¢Ã¢â€š¬Ã…¡Ãƒâ€šÃ‚¬ÃƒÆ’…¡Ãƒâہ¡ÃƒÆ’‚¬ÃƒÆ’¢Ã¢Ã¢Ã¢Ã¢â€š¬Ã…¡Ãƒâ€šÃ‚¬ÃƒÆ’…¾Ãƒâہ¡ÃƒÆ’‚¢t have FDA-approved treatment options, patients with a rare disease can benefit from early access programs.

 

There are numerous challenges to developing treatments for patients with rare diseases. For example, it can be hard to recruit clinical trial participants because of the small patient populations. In addition, thereÃÆâ€â„¢ÃƒÆ’ƒâہ¡ÃƒÆ’‚¢ÃƒÆ’¢Ã¢Ã¢Ã¢Ã¢â€š¬Ã…¡Ãƒâ€šÃ‚¬ÃƒÆ’…¡Ãƒâہ¡ÃƒÆ’‚¬ÃƒÆ’¢Ã¢Ã¢Ã¢Ã¢â€š¬Ã…¡Ãƒâ€šÃ‚¬ÃƒÆ’…¾Ãƒâہ¡ÃƒÆ’‚¢s no single, consolidated place for patients with rare diseases to find companies that offer drugs on an expanded access basis, and then submit the information to enroll in these opportunities. This leaves a large, unmet need for access to investigational therapies among critically ill patients with rare diseases. Many of the innovative developers of orphan drug products are small companies that may face their own obstacles making this information available. We believe that patients shouldnÃÆâ€â„¢ÃƒÆ’ƒâہ¡ÃƒÆ’‚¢ÃƒÆ’¢Ã¢Ã¢Ã¢Ã¢â€š¬Ã…¡Ãƒâ€šÃ‚¬ÃƒÆ’…¡Ãƒâہ¡ÃƒÆ’‚¬ÃƒÆ’¢Ã¢Ã¢Ã¢Ã¢â€š¬Ã…¡Ãƒâ€šÃ‚¬ÃƒÆ’…¾Ãƒâہ¡ÃƒÆ’‚¢t face obstacles finding out which companies offer drugs on an expanded access basis, and then face additional unnecessary hurdles in accessing these programs.

 

Widening the scope of the Navigator program will help maximize the opportunity for patients to get access to promising treatments through clinical trials when these treatments are otherwise unavailable, and will advance the development of new drugs in a more transparent, accessible, and straightforward manner.

 

Scott Gottlieb, M.D., is Commissioner of the U.S. Food and Drug Administration

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What does this mean for Clinuvel in relation to expanded access? That's seems to be basically what Italy/Switzerland had been doing. To my knowledge, Clinuvel hadn't applied for this in the FDA right? It's probably too late for this to be of any use for EPP, but HHD has been granted orphan status by the FDA so maybe once EPP is approved then expanded access could be used for HHD.
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An interesting theory about the recent jump in sp from walltrader over at wallstreet-online:

 

deepl translate:

 

Homm is bloody intelligent!Probably smarter than all of us together! I believe that his "Buddies" buy shares to push the price to make the stock look more attractive. He plans such actions years in advance.

 

The steep rise in the share price, which we are currently seeing, is noticed by many stock screeners worldwide, which means that people are getting information about Clinuvel. And that's exactly what Homm wants: to put it mildly, he is doing IR (Investors Relations) indirectly.

 

When people look at CUV's balance sheet, they will notice that the company is extremely undervalued.

 

Source: https://www.wallstreet-online.de/diskussion...maceuticals-ltd

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Nice find CUV88, thanks for sharing.

 

Clinuvel - Orphan Drug Designations

 

SCENESSEÃÆâ€â„¢ÃƒÆ’ƒÂ¢Ãƒ¢Ã¢Ã¢â€š¬Ã…¡Ãƒâ€šÃ‚¬ÃƒÆ’…¡ÃƒÆ’â€Å¡Ãƒƒâہ¡ÃƒÆ’‚® (Afamelanotide) has been granted two Orphan Drug Designations (ODDs) by the FDAÃÆâ€â„¢ÃƒÆ’ƒâہ¡ÃƒÆ’‚¢ÃƒÆ’¢Ã¢Ã¢Ã¢Ã¢â€š¬Ã…¡Ãƒâ€šÃ‚¬ÃƒÆ’…¡Ãƒâہ¡ÃƒÆ’‚¬ÃƒÆ’¢Ã¢Ã¢Ã¢Ã¢â€š¬Ã…¡Ãƒâ€šÃ‚¬ÃƒÆ’…¾Ãƒâہ¡ÃƒÆ’‚¢s Office of Orphan Products Development, for erythropoietic protoporphyria (EPP) in July 2008 and for solar urticaria (SU) in December 2009.

http://www.clinuvel.com/clinuvel/item/10-s...han-drug-status

 

SCENESSEÃÆâ€â„¢ÃƒÆ’ƒÂ¢Ãƒ¢Ã¢Ã¢â€š¬Ã…¡Ãƒâ€šÃ‚¬ÃƒÆ’…¡ÃƒÆ’â€Å¡Ãƒƒâہ¡ÃƒÆ’‚®, has received orphan-drug designation from the US Food and Drug Administration (FDA) for the rare Hailey-Hailey Disease (HHD, also known as familial benign chronic pemphigus).

http://clinuvel.com/2014-announcements/ite...-hailey-disease

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I too say thanks for sharing this interesting piece of information.

 

I have read the article a couple of times and get that the idea behind this initiative is to, "help maximise the opportunity for patients to get access to promising treatments through clinical trials when these treatments are otherwise unavailable...

However, there are a couple of references in the article that suggest to me that ability to access treatments, "...by widening the scope of the new Expanded Access Navigation tool", [EANT] is intended for application to critically ill / terminal patients. I say this based on the following extracts from the article:

 

1. "Previously this tool [being the EANT] was rolled out for drugs that treat cancer[/i]." (end of para 1) Comment: I do note here that it then goes on to say that, "It will now apply to drugs that treat orphan diseases.

 

2. "..expanded access programs play an important role for terminal patients..... (second para )Comment: I also note that this does not necessarily exclude non terminal patients!

 

3. In the third paragraph, it refers to, "....a large unmet need for access to investigational therapies among critically ill patients with rare diseases."

 

My area of doubt is that although widening of the scope of the Navigation Program is said to "..now apply to drugs that treat orphan diseases", will any such orphan disease need to be of a critical/terminal nature in order to be approved?

 

not saying this is necessarily the case, but thought it worth sharing.

 

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