Why are prescription drug prices so high in the US?
Let us start with insulin as an example. Insulin is the Achilles heel. If we understand insulin, we understand why it's so hard to fix our broken system.
1/ Existence of a vulnerable population needing a lifesaving medicine
2/ Monopoly
3 companies control the market for insulin. In a monopoly with significant regulatory and legal barriers to entry of competing products, the seller can set the price however high they want.
Here, the monopoly is not over a luxury item, but a lifesaving medicine.
3/ Patent Evergreening:
Making patent life extremely long & preventing competition.
Covert: By making newer version of a drug and patenting it (see insulin below)
Overt: Filing multiple new patents on same drug to stretch patent life, pay for delay schemes, lawsuits.
4/ Planned Obsolescence
When Pharma introduces new drugs, the new one is marketed as so much better, that using an older drug is not good clinical practice.
With insulin it's ok. But in other fields, new "me-too" drugs often provide minimal incremental value to justify cost.
5/ Biosimilar and Generic drug approval process is slow, expensive, and complicated. Numerous regulatory and legal barriers.
Studies show you need 4 or more competing biosimilars/generics to have an effect on price. One is not enough.
6/ Price "Collusion"
Absent true completion, if just 2 companies make similar products they can choose to increase prices in lock step. Both benefit. With insulin for years prices of competing drugs increased almost on same day to same level. It is not overt collusion. But...
7/ The Middlemen
Everyone in the supply chain from Pharma to Wholesaler to Pharmacy Benefit Manager to Pharmacy benefits from a higher price, except the patient. Profit is proportional to list price, which means it's in everyone's interest to have a high price.
8/ Influence of the Pharma Lobby
Pharmaceutical companies spend a lot on lobbying. Which is why nothing ever gets done.
Plus the factors for high price (#1-7) are many, and it's easy to point fingers.
9/ There are also other factors that make prices insanely high that are unique to the US:
Medicare must buy, but cannot negotiate.
Prescribers of meds administered in doctors offices stand to gain more by prescribing a more expensive option.
10/ So what are the solutions?
Every western nation has value based pricing. A maximum price that is negotiated for new drugs proportional to the value they provide. This ensures a reasonable launch price, & prevents the type of crazy price increases that are possible in the US
11/ Medicare must be able to negotiate for prices.
@ASlavitt once said almost 90% of Americans agree that Medicare should negotiate for drug prices, and 90% of Americans agree on very few things!
12/ Reform regulatory and patent process to make it easier for generics and biosimilars to end the market.
We cannot allow patent evergreening by repeated new patents filed on the same drugs as is the case with analog insulins and many new drugs.
13/ Eliminate reimbursement for drugs administered in doctors offices from a % of sales price to a fixed reimbursement. The current system encourages the use of a more expensive alternative when an equivalent cheaper one is available.
14/ Reforms to ensure that price increases are not related to rebates and lack of transparency in deals between Pharma and PBMs.
Many ways to do this. Including transparency, ending practice of rebates or passing rebates to patients. There will be many trade offs. It's complex.
15/ What can individual doctors do?
Help patients find the lowest cost options. Always prefer generics and biosimilars if possible.
For patients paying cash, prices of common drugs can vary dramatically. @GoodRx helps find the lowest price.
16/ Talk to patients about cost and affordability. Take cost and cost effectiveness into account when prescribing.
17/ I have discussed this in detail in a talk I gave @MayoClinic and you can access it here if interested. The problem of prescription drug prices is complex and unless we recognize the myriad factors and how they interact it is hard to fix.
cc: @hasanminhaj -- You have spoken about many of these issues on Patriot Act
In the year 2000, a few of us attended an angiogenesis meeting in Boston. We were there to discuss thalidomide
But a side meeting that evening led to trial that went on to get Velcade FDA approved for myeloma. @NEJM
Story in thread.
Velcade (bortezomib) was first introduced to cancer research by the name PS-341.
It was a novel proteasome inhibitor developed by Julian Adams and colleagues a a potential anti cancer agent. @CR_AACR @AACR aacrjournals.org/cancerres/arti…
The ubiquitin-proteasome garbage disposal pathway in cells is a Nobel prize winning discovery.
Proteins that need to be degraded are tagged with ubiquitin tails. Tagged proteins are degraded by the proteasome complex. (This review has details )
The fascinating story of Thalidomide: how this most notorious drug on the planet, banned in the 1960s, made an incredible comeback and revolutionized the treatment of myeloma.
I will also highlight one person whose role is not recognized: Without Dr. Leif Bergsagel there will be no thalidomide for myeloma.
Read on #MedTwitter
The thalidomide story has many takeaways and lessons.
It shows drug development from bedside to bench and back to bedside.
It shows the power and impact of astute clinicians
It shows the power of investigator courage
The role of serendipity
But let’s start at the very beginning.
Thalidomide was synthesized in 1954, and then developed as a sleeping pill by the German company Chemie Grünenthal in the 1950s.
At the time the only sedatives available were barbiturates which had risks of intentional or accidental overdose.
Because thalidomide was felt to be a drug that cannot cause death due to overdose it was marketed as one of the safest sedatives.
By 1961, it was sold in over 40 countries as a sleeping. It was also tragically used to control morning sickness of early pregnancy.
AQUILA trial for high risk smoldering myeloma published in @NEJM today.
@thanosdimop
Personally for me, it is a huge milestone along 25 years of work that started in 1998. #ASH24 #ASH24VR
This story below may help those interested in a clinical trialist career. 1/
In 1998, as a fellow @MayoClinic I was keen to determine if early intervention delayed progression and improved survival in SMM. #ASH24
In 1999, with the help of Tom Witzig, I led a small phase II trial of thalidomide for SMM. @LeukemiaJnl 2/
I was then so fortunate to examine the natural history of SMM, with the legendary Bob Kyle. Honored to be last author on @NEJM paper that also provided data that most progressions occur in the first 5 years of diagnosis.
The start of the concept of high risk vs low risk SMM. 3/
FDA approval doesn’t necessarily mean standard of care.
Thread.
1/
For example FDA approved Dara VMP for frontline therapy in myeloma in 2018.
Literally no one used the regimen in the US.
Literally no one felt the regimen was standard of care in the US.
Before or after approval!
Why?
FDA adjudicates a sponsors submission on whether a given drug/regimen has met the burden of proving safety and efficacy.
Standard of care in clinical practice is a different standard: judgment of risk/benefit of available alternatives, and assessment of trial design/end points.
Cure is a simple word. But there is confusion when it comes to cancer. What cure is in cancer, and what we should aspire for?
When can we say that a given type of cancer is curable?
Thread
1/
There is a difference between when we can say a particular cancer is a curable type versus whether individual patients with a given cancer can be considered potentially cured.
They are not the same.
2/
To call a cancer curable we must be able to treat the cancer for a finite duration, stop all therapy, and know that a certain % of patients will never relapse
Early stage solid tumors, Hodgkin lymphoma, DLBCL, ALL, AML are curable. Real cure. The definition of curable cancer
3/