Miracle Drugs

[xoxoxoBruce]

Miracle Drugs, a much overused expression. But if you need them to fix you up, I guess they are a miracle. But it seems if you need cancer drugs, it may take a miracle to get them.

“Our results indicate that the vast majority of oncologists in the country are facing wrenching decisions about how to allocate lifesaving drugs when there aren’t enough to go around,” Gogineni says. “The potential impact of these drug shortages is vast: they’re putting patients at risk and driving up costs of cancer care.”

In addition to quantifying the prevalence of cancer drug shortages, the authors also identified a variety of ways in which oncologists say they’ve adapted when preferred or recommended drugs are scarce:
•78 percent of oncologists reported treating patients with a different drug or drug regimen
•77 percent substituted different drugs partway through therapy
•43 percent had to delay their patients’ treatment
•37 percent had to choose among patients who needed a particular drug
•29 percent omitted doses and 20 percent reduced doses
•17 percent referred patients to another practice

So far, little is known about how these adaptations may impact clinical outcomes, but the authors say the widespread treatment delays reported underscores the urgency of the problem. The most risky types of modifications, Gogineni says, occur when physicians substitute drugs partway through patients’ treatments, since there is often no established dose equivalence or known safety profile when the substitute is combined with other therapies. Clinical trials are also hampered when drugs necessary for properly testing new regimens are scarce. The team found that nearly 12 percent of the time, drug shortages prevented enrollment in studies, delayed administration of a study drug, or suspended involvement of patients on clinical trials.

So where the rubber meets the road, or where the board of directors meets the wall street analysts, your health is secondary to quarterly reports.
But hey, it's not personal, it's business.

[orthodoc]

It would appear obvious, here, to champion a system like that in Canada over the 'business first' American one. Unfortunately, getting new drugs past multiple bureaucratic committees that are trying to manage capped global health budgets means that Canadians often wait longer for new drugs. This happened with tamoxifen long after it was standard treatment in the US.

I don't know that there's a good answer. I'd think that pharm companies conducting clinical trials would at least want to have enough of their new drug available to give the study statistical significance, but stranger things have happened. And not all new 'miracle drugs' end up fulfilling their promise by any means.

I am still very relieved to have been here for my cancer treatment rather than in my native country. Which is not to say that everything is better here or worse there. But for imminently life-threatening issues I'm glad to be here.

[xoxoxoBruce]

I can see the problem and delays with getting new drugs approved.
I got the impression they were talking about drugs that had been approved and we supposed to be available, especially generic drugs.

[Happy Monkey]

We already have those committees, whether they're in the FDA or in a multitude of insurance companies.

[Lamplighter]

Here is a medical news topic that may well fit in this thread...

The headlines are current popular media are startling,
and may even generate visions of ménage à trois,

But the actual technique is more like supplying a miracle drug
to prevent a very rare (orphan) disease being inherited
by a baby from its mother.

There are lots of ethical issues too, but it is an issue that
will probably be decided in the UK via the popular press.

University of Oxford
Practical Ethics
Paula Boddington
June 29, 2013

Three person IVF

It was announced yesterday that the government is moving
towards allowing so-called three person IVF [In vivo fertilization]
for the creation of embryos free of mitochondrial disease.
<snip>

From Wikipedia: MELAS_syndrome

MELAS syndrome
Mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes
This condition is inherited in a mitochondrial pattern,
which is also known as maternal inheritance and heteroplasmy.
This pattern of inheritance applies to genes contained in mitochondrial DNA.

Because egg cells, but not sperm cells, contribute mitochondria to the developing embryo,
only females pass mitochondrial conditions to their children.
Mitochondrial disorders can appear in every generation of a family
and can affect both males and females,
but fathers do not pass mitochondrial traits to their children.
Less commonly, the disorder results from a new mutation
in a mitochondrial gene and occurs in people with no family history of MELAS.

There are a couple of ways of doing the new procedures,
involving 3 persons (#1, #2, and #3 below)

Basically the new proposed techniques take the nuclear DNA
of a egg from the affected mother[#1] and from the father [#2].

A donated egg is also taken from a non-affected woman [#3],
Its nuclear DNA is removed, leaving behind healthy mitochondrial DNA.

The nuclear DNA of the two parents is then transplanted into the body of the healthy egg,
resulting in an egg which has the DNA of the father and the affected mother,
and the mitochondrial DNA from the donor woman.

Pic from here
*167