THE "NEW" PROSTATE CANCER INFOLINK

As many of our readers may be aware, the “right to try” treatments using investigational new drugs outside of the clinical trials processes required by drug developers and overseen by the US Food and Drug Administration is a current “hot topic” on Capitol Hill. However, the degree to which this process may be relevant to the management of prostate cancer in general (and advanced forms of prostate cancer in particular) would seem to be rather small.

“Right to try” legislation has already been passed in 33 of the 50 states of the Union. However, passage of such laws has actually made almost no difference at all to how most patients get treatment, for a whole bunch of reasons.

In the case of prostate cancer, one of the most compelling reasons why “right to try” legislation might be important is if one is BRCA1/2-positive and one has an aggressive form of prostate cancer. In theory, it is possible that such men might respond well to one of the PARP inhibitors like olaparib (Lynparza), rucaparib (Rubraca), or niraparib (Zejula) — and there are more of these drugs to come. None of them are have been approved for the treatment of prostate cancer, and clinical trials of these drugs in the treatment of prostate cancer is in relatively early stages. Might they work? Yes they might. How well will they work if they do? We haven’t a clue.

Now the ability to actually get treated “off-label” with one of these drugs for an advanced or progressive form of prostate cancer (and particularly if the drug is truly investigational and unapproved for any indication) actually depends on a whole bunch of different things:

• The willingness of an appropriate physician to actually write a prescription for one of these drugs to treat your particular form of prostate cancer and work with you to get that prescription filled
• The quality of whatever data are available to suggest that the drug might work in your particular sub-type of prostate cancer
• Whether there is an ongoing clinical trial of the particular drug in the treatment of your particular sub-type of prostate cancer
• Whether the developer has an expanded access protocol in place that might allow you to get access to the relevant drug for the treatment of prostate cancer
• Whether the developer will be willing to provide the drug for off-label use outside a clinical trial or outside an expanded access program
• Whether (assuming that they are willing to provide it at all) the developer will require payment for the drug if it is being provided for off-label use outside a clinical trial or outside an expanded access program
• Whether you can afford what the drug company wants to be paid (if they do) — because your insurance company certainly won’t cover such costs

The easiest and best organized ways to get access to investigational drugs (whether they are approved are not) is through clinical trials and through expanded access programs. These processes are highly defined. The drug developer has worked with the FDA to ensure an appropriate process that protects all parties (to whatever extent is possible). And most eligible patients will actually get the investigational drug through one of these two processes (unless the clinical trial is a randomized trial, in which patients get either the investigational therapy or a known “standard of care” that does not include the investigational drug).

If you were to invoke a “right to try” law today, in an attempt to get treatment with a PARP inhibitor for prostate cancer, the chances that you would actually be able to get treatment with such a drug are extremely small … and it might cost a small fortune in legal fees to even try.

On the other hand, for almost every new investigational drug that has been developed for the treatment of prostate cancer, companies and the FDA have put “expanded access” initiatives in place as soon as there are data from clinical trials that strongly suggest effectiveness, so it has been possible for appropriate patients to get early and free treatment with these drugs prior to actual FDA approval. Recent examples have included abiraterone acetate (Zytiga), enzalutamide (Xtandi), and radium-223 diacetate (Xofigo).

The American Society for Clinical Oncology (ASCO) has come out strongly against the proposed federal “right to try” legislation. The full ASCO statement is available here, and an abbreviated version of the statement was issued in a media release a couple of weeks ago.

While The “New” Prostate Cancer InfoLink is sympathetic to the idea behind “right to try” legislation, we are also highly conscious that the practical application of such legislation is very limited indeed, most commonly because most drug companies will be unwilling to make investigational drugs available off-label without the FDA’s approval. Once a drug is approved (as the above three PARP inhibitors are, in the treatment of ovarian cancer), then a prescribing physician can use the drug to treat whatever he or she likes — on or off label. However, the controlling factor then is whether the insurance provider (Medicare or Medicaid or a commercial insurer) is willing to cover the cost of the drug when used off label. In the case of the PARP inhibitors, very few insurers would even consider covering this cost for an off-label use because the available data is not compelling yet.

The bottom line here is that the potential beneficiaries of “right to try” legislation are probably very limited — and are primarily going to be members of very affluent families who have the financial clout to be highly persuasive. For the rest of us, there are good reasons to believe, in any case, that the risks we may be taking (both financially and medically) in seeking and taking such drugs are unlikely to pay off.

Like ASCO, The “New” Prostate Cancer InfoLink thinks that the recently approved 21st Century Cures Act will improve the opportunities for appropriate patients to be able to get access to investigational drugs. However, if President Trump gets his way, he is about to cut the budget of the FDA in half, try to make drug companies pay twice as much (or more) to get their new drugs approved, and this will lead to yet another massive increase in drug prices that we can’t afford. No one will be able to get access to investigational drugs through “right to try” laws under that scenario — even if the proposed legislation makes it through Congress. Why? Because the FDA won’t have the budget to implement any of the activities required by the 21st Century Cures Act.

Filed under: Drugs in development, Living with Prostate Cancer, Management, Treatment | Tagged: "right to try", investigational, Legislation, off-label, Treatment, unapproved |