Opinon: Off-Label Options and Compassionate Access to Phase II Drugs

There is much discussion—and frustration—within the ALS community over the lack of available therapies, and the FDA regulatory hurdles that impede bringing new drugs quickly into practice. Obviously these protocols are in place for the “greater good” of patients.

Yet it is excruciating to watch disease advance and take loved ones at the same rate it takes to complete a single drug trial.

The number of patient cohorts deemed acceptable in clinical trials often seems extreme, especially in small patient populations such as ALS. Placebo is another cruel, yet well-intentioned research “control.” It just doesn’t seem fair or humane when the vast majority of ALS patients are facing mortality rates of 2 – 5 years.

Finally, the current regulatory paradigm fails to take into account one of the most fundamental understandings in medicine: what works for one patient may not work for another. Therefore it becomes exceedingly difficult when dealing with rare or “orphan” status diseases to develop drugs that perform with mass uniform efficacy.

We know that from simple variances in dosing to “cocktails” of multiple drugs, different patients can respond drastically different. The severity of disease is also a variable that cannot be “controlled.”

Question: whatever happened to practicing medicine?

As the preceding article on Gilenya points out, physicians in many different disease categories have long prescribed off-label. Perhaps the physician community will take another look at therapies currently indicated for other neurological disorders.

No one should have the power to tell you what you can or cannot do in the efforts to save your own life. There can be no greater personal decision — or risk. And it should be your decision and your risk to take.

Any responsible physician will advise you of those risks, and manage your expectations. You in turn must also be willing to weigh those risks yourself, taking full responsibility in the process.

There has also been an increasing call in the ALS community for access to promising Phase II trial drugs. Again, with mortality rates being what they are, it seems only fair that patients should have the right to try everything they can in an attempt to slow disease progression.

Who knows, maybe even halt it or reverse it. Everyone’s different.

And that’s the point here. Despite the fact that ALS was identified nearly 150 years ago, we are now only beginning to understand how it works. With regard to the development of an efficacious treatment for ALS, we are still in its infancy. There is much more to be done—and much more quickly than we’ve seen.

If you want to get involved in an effort to gain access to Phase II drugs, check out ALS – Treat Us Now on facebook or the group’s change.org petition.

Their goals:

  1. To assure compassionate access for ALS patients to treatments that have demonstrated in Phase II trials the ability to slow progression or cure ALS.
  2. To cooperate with all willing ALS stakeholders.
  3. To assure that funds are made available to allow patients compassionate access to treatments.


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