New ALS drug
(`Motor Neuron Disease` or `MND` also known as `Lou Gehrig's disease`)
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A previously unknown problem protein has been discovered
A specifically designed new ALS drug, RCH4, successfully suppresses it
 ​RCH4 is available free of charge on compassionate basis depending on availability and location

Cost of developing a drug

The cost of developing a drug is primarily dictacted by the costs involved in obtaining Regulatory Authority approval for it.​​​​
The average cost for a drug to proceed from the laboratory bench to the bedside is often considered to be far more than one Billion Dolllars for a mainstream drug for a common disease.
This is easy to say, but to focus ones mind, a Billion Dollars is; $1,000,000,000.00.

Here are the costs per drug incurred by some of the major Pharmaceutical companies:

The cost to develop a drug for ALS:

The cost of developing a drug for a rare disease may be far lower due to the fact that the Regulatory Authorities may demand far fewer patients in trials of the drug.

Here are the development stages for any new drug:

Pre-clinical stages

Research, screening of candidate molecules, or accidental discovery to have a prospective drug can result in a cost of many millions. The cost all depends on how the potential drug is arrived at or discovered.

Once a candidate drug has been discovered, then:
Laboratory or in vitro testing to ascertain if the candidate drug appears to work in accordance with its hypothesised mode of action.
Typically the cost may be a few hundred thousand Dollars upwards.

Testing in an animal model  and in (usually) three species, costs a few hundred thousand Dollars.

Clinical stages

Phase l clinical trial in volunteers typically costs $200,000 but this depends on the number of volunteers used.

Phase ll clinical trial: between 30 and 100 patients are tested in a randomised, placebo controlled, double blinded trial to demonstrate that the drug works ("efficacy"). Cost $5 to $16 million.
Frequently a number of Phase ll trials of a drug are done, usually designated as Phase lla, Phase llb and so on.

Phase lll clinical trial usually comprises of hundreds to thousands of patients. When one assumes the cost per patient in a trial at about $25,000 to $60,000 each, it can be seen that it requires major funding.

So where did the figure of over $1 Billion come from?
Some 98% of all drugs fail to make it from the laboratory to the bedside. The very few successful drugs must pay for all the failures. Pharmaceutical companies must get a return for their investors who gamble their money in a very risky field.
No new money, no new drugs. We do not live in a perfect world.

Example: The $5 medicine for which our grandfathers had to pay $25 for, results in today having cardiac drugs which keeps millions of people alive.