[Beacon Staff]

Easier access to unapproved myeloma drugs

by Beacon Staff on Wed Feb 04, 2015 3:54 pm

The U.S. Food and Drug Ad­min­is­tra­tion (FDA) today announced a proposal to sig­nifi­cantly simplify the pro­cess for getting per­mission for patients to be treated with investiga­tion­al drugs outside of standard clinical trials.

Investigational drugs are ones that have not yet been approved by the FDA for any disease whatsoever, but are undergoing clinical trials in the hopes that they may – at some point – gain FDA approval.

Until their approval by the FDA during the past few years, for example, Pomalyst and Kyprolis were investigational drugs. Investigational therapies for myeloma includes daratumumab, elotuzumab, ixazomib, filanesib (ARRY-520), SAR650984, PVX-410 (for smoldering myeloma), and a number of others.

Access to investigational drugs outside of a clinical trial is often described as "compassionate use." Many of you may recall that Beacon columnist Arnie Goodman was able to be treated with elotuzumab through a compassionate use approval.

In the past, it was very time consuming to get approval for a patient to be treated with an in­ves­tigational drug on a compassionate use basis. The FDA estimates that, just to complete the application form for a patient to be considered for compassionate use access, the patient's physician would have had to spend 100 hours (!) filling out the required forms.

Now, the FDA is opening for comment a revised process for applying for compassionate use access. At the core of the process is a revised application form, to be filled out by the patient's physician, which is just two pages long. The FDA estimates it will take just 45 minutes to complete the form.

The proposed new process is still just that: A proposal. But, if approved, it could make it much easier for myeloma patients, as well as others with difficult-to-treat diseases, to get access to investigational therapies.

We are including below the full text of the FDA's announcement, which was posted on its blog (and is not copyrighted)

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A big step to help the patients most in need
Posted on February 4, 2015 by FDA Voice
By: Peter Lurie, M.D., M.P.H.

Today, I had the pleasure of announcing an important measure intended to help streamline expanded access to investigational drugs. We heard concerns from patients and physicians that the process for gaining access to investigational drugs was too difficult, and pulled together a team to find a way to make that process simpler. Today, we’re introducing a much simpler draft form for comment that, when finalized, should accelerate patient access to investigational drugs. We know what an important tool this will be for physicians who treat those patients with serious or immediately life-threatening diseases or conditions for which there are no comparable alternative treatments.

The new draft document, entitled “Individual Patient Expanded Access Applications: Form FDA 3926,” includes a simplified application form that, when finalized, will be used for requesting the medications, and is designed to greatly simplify and accelerate the process by which a physician can request that FDA permit the use of an experimental — so-called “investigational” — drug or biological product while it’s still being tested to establish its safety and effectiveness.

The draft guidance and draft form continue a policy that started in the early years of the AIDS epidemic when FDA authorized, in certain cases, “compassionate use” of unapproved investigational drugs. In 2009, FDA made these rules broader and clearer. However, concerns persisted that the existing application form was too complex: it called for 26 separate types of information and seven attachments. In fact, it was originally designed for manufacturers seeking to begin human testing, not for physicians seeking use by single patients.

FDA authorizes the vast majority of expanded access requests, typically within days or even hours. However, FDA is committed to streamlining its processes wherever possible. The agency therefore tasked a special working group with designing a form more suitable for use by a physician not necessarily familiar with the IND process. The revised process, when finalized, will not change the agency’s rigorous requirement that all medical products on the market be studied in clinical trials in order to be FDA-approved as safe and effective. As before, expanded access to an investigational medication may be available when there is no other product that can diagnose, monitor, or treat the patient’s disease or condition, and the patient is not and cannot be enrolled in a clinical study testing it.

But we know why patients want access to these drugs and we know how busy their treating physicians can be. So we streamlined the new draft form to be shorter and simpler for physicians to fill out. The new draft form, when finalized, will require only eight elements of information and a single attachment. We estimate that physicians will be able to complete the finalized version of the form in just 45 minutes, as compared to the 100 hours listed on the previous form.

Additionally, to further assist the physician seeking access to an experimental therapy, we have redesigned our website to make it easier to navigate and to explain the new proposed process in detail.

For years, FDA has maintained a staff dedicated to assisting physicians and patients to navigate our system. These efforts will continue. The new draft guidance and draft form are the latest examples of FDA’s determined effort to minimize unnecessary red tape, increase efficiency and better serve patients in need.

Peter Lurie M.D., M.P.H. is associate FDA commissioner for public health strategy and analysis.

[LarryD]

Re: Easier access to unapproved myeloma drugs

by LarryD on Thu Feb 05, 2015 11:00 am

I see that the proposed form requires a Letter of Authorization from the drug manufacturer, and that the normal process is to obtain that before seeking FDA approval.

How easy is it to get the manufacturer to agree? In many cases, it seems to me that it is not in the manufacturer's interest to do so. Even if the FDA procedure is streamlined, the manu­fac­turer's ability to veto the requested access could be a big obstacle.

Who pays for the investigational drug, and how is the price determined?

--Larry

[Beacon Staff]

Re: Easier access to unapproved myeloma drugs

by Beacon Staff on Thu Feb 05, 2015 12:37 pm

Hi Larry,

It is our understanding that the manufacturer of an investigational drug still can decide whether or not it wishes to supply the drug for the requested compassionate use. Even if the FDA approves a request for compassionate use, this does not guarantee that the manufacturer will make the drug available to the patient.

It is also our understanding that, if the manufacturer decides to make the drug available for compassionate use, it sets the price for the drug, but that price is subject to oversight by the FDA. It's possible that, because compassionate use is not yet very common, that manufacturers do not charge much for drugs provided this way, or they may charge an amount comparable to other existing myeloma therapies. We don't know, but we will see if we can find out a bit more on the subject.

The FDA has a webpage with a lot of additional information about compassionate use / expanded access that addresses some of the questions you asked (and others you, and other readers, may have). You may want to check it out:

http://www.fda.gov/NewsEvents/PublicHealthFocus/ExpandedAccessCompassionateUse/

If you find out any additional information that may be of interest to others here in the forum, please let us know.

[Boris Simkovich]

Re: Easier access to unapproved myeloma drugs

by Boris Simkovich on Thu Feb 05, 2015 3:27 pm

I checked with someone who was involved in arranging for a myeloma patient to get access to an investigational therapy through the FDA's compassionate use / expanded access program.

There were no (or very limited) out-of-pocket expenses for the investigational drug. However, it is not clear whether this was because the patient's insurance was very generous, and covered the cost of the drug (whatever it was), or because the manufacturer decided not to charge much for the drug.

The patient's insurance is, in fact, usually quite generous. However, as I said, it's not clear in this case whether the low (or no) cost to the patient was due to the insurance company's policies or the manufacturer's decision about what to charge for the drug.

My colleagues and I will post more here on the topic if we learn anything further.

[Eric Hofacket]

Re: Easier access to unapproved myeloma drugs

by Eric Hofacket on Thu Feb 05, 2015 4:14 pm

I would be very interested in learning if this patient’s insurance company paid for the com­passionate use drug. If so, I think this would not be the norm. I do not see how it would be in the insurance company’s benefit to do this from a business perspective. If they did, I think a lot of Beacon readers would like to know which insurance companies have such polices.

My best guess is the drug company made the drug available at low cost out of compassionate use, knowing that most insurance companies would not cover the cost of the drug, so if they did not make the price reasonable, then it was not going to happen at all.

I hope these new FDA rules clear the bottlenecks for making new drugs available for com­pas­sionate use for those with advanced myeloma and are out of other options. We had a couple of Beacon writers last year who could have benefited from this and others who are still in this sit­u­a­tion.

It seems it may have been the FDA application process that was the bottleneck and not the drug companies. With a new streamlined FDA process, maybe we will see far more people writing about their compassionate use experience, or maybe we will discover that bottlenecks still remain with the drug or insurance companies.