nce every two years, the World Health Organization releases a list of medications it thinks should be available, if needed, to all the people of the Earth. The latest iteration of the essential medicines list is slated to be released this week.
It’s a formulary, a compendium like the ones health insurers such as Kaiser Permanente or Harvard Pilgrim maintain to help them determine which medicines should be covered by their policies.
That may sound dull or at least rather wonky. But there are real-world implications when a drug makes — or is not approved for — this list.
The move to include HIV drugs in 2002 arguably helped to make lifesaving antiretrovirals available to AIDS patients in developing countries. More recently, the addition of game-changing hepatitis C drugs to the list appears to have put them on a similar trajectory.
The list is meant to help countries figure out how to prioritize spending on medications. It’s a model that many use to craft their own drug formularies — while individual countries may make tweaks here and there, they don’t each need to set about inventing this wheel.
While the process isn’t easy, the thinking behind it is.
“Some drugs are more important than others. … And this is independent of cost,” explained Nicola Magrini, a WHO scientist and secretary to the expert committee on the selection and use of essential medicines, which draws up the list.
Here are some facts about the essential medicines list — its history and some things to look for when the 2017 iteration is published.
Its birth was mired in controversy.
When the WHO set out to draft the first essential medicines list in the late 1970s, the United States and other pharmaceutical exporting countries weren’t pleased. Picking drugs for the list implied something about the drugs that didn’t make the cut.
“It was the concept that some medicines were more essential than others, which meant that some medicines were less essential than others,” said Dr. Richard Laing of Boston University’s Center for Global Health and Development. Laing formerly worked at the WHO and was involved in earlier iterations of the list. “No manufacturer wanted to have the suggestion that their medicine was less essential, less necessary than others.”
The list-making process has evolved.
For the first couple of decades, the list was drawn up based on expert advice. But that and other things changed shortly after the turn of the century.
Since that time, the selection process has used an evidence-based approach. The committee drawn together to update the list reviews the studies that have been conducted that either support a recommendation to include a drug or to delete it from the list.
There have been other changes, too. Before 2001, a drug had to be needed by a majority of the population to be included on the list, which meant medicines for uncommon diseases didn’t make the cut. But that criterion was dropped, said Magrini, and the list now includes medications like factor VIII, a clotting protein for hemophiliacs, and surfactant, which opens up the lungs of premature babies.
A drug’s cost was a deciding factor prior to 2001, Magrini said, with the idea being that a medication’s price should be low and affordable. But that’s a difficult bar to set when you’re looking at the whole world, he noted. “Even a dollar a month can be too much in sub-Saharan Africa.”
Now decisions are based on a drug’s usefulness, its safety and effectiveness, and the quality of the evidence supporting it.
The process is a two-way street.
Anyone — an individual, an organization, a pharmaceutical company — can submit a drug for inclusion. The WHO gets about 100 such applications every time it updates the essential medicines list, Magrini said.
Likewise, anyone can apply to have a drug stricken from the list. A malaria drug, amodiaquine, was added, dropped, then reinstated on the list in the 1990s.
One of the things people will be watching in this year’s list is whether oseltamivir — the flu medication sold as Tamiflu — will be cut. A group of academics who have long suggested the drug is more hype than help have asked that it be dropped.
Flu experts at the WHO have countered, saying the medication is the best of a very scant number of options for treatment of severe influenza.
Making the list can put expensive drugs within reach of more people.
In 2002, antiretroviral drugs, which can add decades to the lives of people infected with HIV, were listed for the first time. They were costly — well beyond the reach of the African countries hardest hit by the AIDS epidemic.
But their addition to the list occurred around the time of the creation of the Global Fund to Fight AIDS, Tuberculosis, and Malaria. The next year, PEPFAR — the President’s Emergency Plan for AIDS Relief — was born.
The confluence of those events spurred generic production of the listed HIV drugs, and that competition sent prices tumbling. The result: Millions more people had access to these critical drugs. Laing said this is one of the best examples of what adding a product to the list can do.
Some commonly used drugs have never made it to the list.
A number of drugs are widely prescribed to people with mild to moderate Alzheimer’s and other forms of dementia. The evidence for their efficacy is slender, yet they are still used.
None of those drugs is on the essential medicines list. “They have never been rejected, however,” Magrini said. “No one has ever proposed them — since they knew, probably, we would have rejected [them].”
The list can still kick up a controversy.
Medications are assessed based on how well they work and how much they are needed. Whether they will conform to a country’s religious or ethical beliefs is not part of the discussion.
So emergency contraception — the so-called morning after pill — is on the essential medicines list. This year the committee reviewed a proposal to include the “week-after pill,” Magrini said. He offered no clues on how the committee ruled on any of the applications before it.
A couple of things to watch for.
The essential medicines list is long. The full report, Magrini said, is about 500 pages. There were about 400 medicines listed in the 2015 iteration.
But here are a couple of issues Laing is watching for: Will long-acting analogue insulin be added to the list? And will the so-called polypill, the combination of four heart medicines, finally make the cut?
Laing opposes the insulin proposal, arguing — as do others — that the product is much more costly than human insulin but there’s minimal evidence to suggest it is any more effective.
He noted the case of Kyrgyzstan, which spends about half of its diabetes budget on analogue insulin — but by doing so only gets enough to meet about one-tenth of the country’s needs.
“If you’re in that situation, clearly you’re better off purchasing all your insulin as human insulin, because it’s pretty much the same as the analogues, and yet human insulin goes a lot further. You can buy a lot more,” he said.
As for the polypill, it’s a combination of a statin to lower cholesterol, a beta blocker, blood pressure medication, and aspirin. Proponents argue it is a cheap and effective way to improve the treatment of people who have had a heart attack or a stroke (each requires a different formulation), but some experts prefer individualizing treatment to the patient.
The polypill has been submitted and rejected twice before. Is 2017 its year? More to come.