When I was younger, Dupont Chemicals used the tagline - "Better living through chemistry." I'm a case study that indeed, at least sometimes it is true.
I have an hereditary condition called heterozygous familial hyperlipidemia. Basically there are two genes that regulate the way the body deals with cholesterol. In my disorder, one of those genes does not work correctly. This results in extremely high cholesterol numbers - my total count when it was first diagnosed was 613 (normal is under 200). There are some folk in whom neither gene works correctly - homozygous familial hyperlipidemia - whose total cholesterol counts are often above 1000.
You are probably aware that elevated cholesterol is associated with coronary artery disease. These crazy high numbers result in severe disease at earlier ages. My biological farther died at age 31 from the artery disease associated with his hyperlipidemia. An autopsy after his death revealed major blockages in every coronary artery.
Due to dysfunctional family issues, I was not aware of my problem until I had chest pains in my mid 30's. By the time my disorder was discovered, there were a number of drugs available to treat my cholesterol problem - niacin in large quantities, bile acid sequestrants, and statins - the first of which was released to the market about 1 month before my diagnosis. I began a regimen of all three classes of drugs. My blockages were too numerous and too severe to do stents or bypass so even with the lower cholesterol, my prognosis was unclear. Animal studies had shown that if the cholesterol could be brought to normal levels, the blockages may recede. The cardiologist told me that he didn't know whether the drugs would drop my cholesterol enough and that even if they did, the blockages may remain unchanged. He advised me to get my affairs in order just in case.
The drugs worked well and my cholesterol dropped more than anyone ever expected. The side effects were/are manageable. That hoped for result that hadn't really been observed in humans took place - my blockages began to recede. Still, some of my numbers never really got to "normal." My HDL's were still lower than they should be and my LDL's were still higher. I had a friend who was diagnosed with the same disorder as me about the same time. The drugs did not work for him and he died less than a year later.
Recently another new class of drugs have made their way to the market - PCSK9 inhibitors. They are aimed at folk like me with genetic causes for their high cholesterol. They are crazy expensive (about $14K per year). My cardiologist started me on one (Repatha) about 3 months ago in addition to my current regimen. The company - Amgen - is subsidizing some folk in using the drug and I fall into that category. For the first time in my life, my HDL's are crazy low, my HDL's are normal, and my overall cholesterol is insanely low - 97!!! If my blockages receded some before, they could recede even more now.
Assuming there are no terrible side effects, this bodes really well for me and for other folk like me... except... the price. What about the folk who will be excluded because they cannot afford the drug? I think of my friend for whom the earlier drugs did not work. Perhaps these new drugs would have been the miracle that saved his life... if his insurance would cover them with a copay he could afford or if he fell into the proper category to be subsidized. If not...
Again we see the reality in our culture with regards to medical care. Those with resources will live and those without... will die.