Vincent Rajkumar Profile picture
May 18, 2022 35 tweets 13 min read Read on X
The high cost of prescription drugs and what we need to do about it. Why nothing ever gets done.

A full 360 of the issue using cancer drugs and insulin as examples. Check it out.
1/
2/ I'm not an enemy of Pharma. I understand the value of innovation first hand. I've led numerous clinical trials, and continue to work with Pharma on new drugs.

My interest started with the recognition that every drug in my field is priced sky high. Unsustainable.👇
3/ Prescription drug prices are generally higher in the US than in every other country. Simple drugs. Complex drugs. New drugs. Old drugs. Everything.
4/ We spend almost the same on prescription drugs for drugs to treat serious illnesses like cancer as all other countries combined.
5/ Another unique craziness in the US is that not only is the launch price high, but prices of new drugs increase each year for no reason. Way more than inflation. So while prices go down in other countries, we pay more.
6/ Why are prescription drug prices so high and so out of control? Many reasons which I list below.

But #1 is that prescription drugs are not luxury items that we can live without.
👇👇
7/ Second it's a MONOPOLY. The seller can dictate the price. When the monopoly is on a life saving drug, they really have you.

Even when there are many drugs to treat the same disease, because each drug may work only for a while, especially in cancer, each drug is a monopoly.
8/ Third, they have perfected the art of making patents last on and on and on. From filing newer patents on same drug to frivolous lawsuits to making slightly better versions of their drug and then filing new patents on them.
9/ Fourth, just like in tech, even when the current drug works well, it will be made to look obsolete by marketing. So the promise of low prices as drugs go generic may not be realized coz by then the drug is made to look old fashioned and obsolete.
10/ No wonder 2 of the top 3 drugs Medicare spends the most money on are new oral blood thinners. 4 of top 10 are diabetes meds.

These are better than older ones but don't fall for the prices will come down with time trick. They may, but not for the drug you are prescribed.
11/ Fifth, we have many regulatory and legal barriers for generics & biosimilars to enter. Even if a generic enters, getting market share is not easy in our system where contracts may go to who can offer the best profit margin than to who has the lowest price product.
12/ Sixth is the wink wink understanding among drug manufacturers to promptly increase price when a competitor increases their price. Collusion without collusion. See below with insulin. An astonishing lockstep price increase over decades.
13/ Seventh, the middlemen. Everyone in the chain benefits from a high list price (profit as a % of list price) except the patient.

Leads to ever rising drug prices.
14/ Eighth, Pharma lobbying power and money is hard to overcome. So despite overwhelming public support to change things, NOTHING gets done.
15/ There's more. Factors unique to the US that contribute to insanely high prices.
16/ Finally there are few allies in the fight to lower drug prices. Very few speak up for measures that truly reduce list price. They often support Pharma friendly policy measures or stay silent.
17/ By providing copay assistance through Pharma funded copay relief organizations, the patient voice is silenced. Pharma channels a few thousand to get tens of thousands back, and can now keep increasing prices without patients protesting. @NorthTxMSG
18/ So what can we do? What should we do.

First, we must have an organization that sets a value based price for all new drugs that derives legitimacy and is endorsed by Medicare and the federal government. Similar to other developed countries.
19/ Second, Medicare must be authorized to negotiate for prescription drugs. It's foolish to mandate Medicare to cover prescription drug costs and simultaneously order them to pay up whatever Pharma wants without negotiation.
20/ More than 80-90% of the public supports Medicare negotiation. Other developed countries negotiate prices. The fact that we don't negotiate affects the ability of other countries to negotiate prices because Pharma can walk away and make up loss by increasing prices in the US.
21/ See below how the price of the same drug can be increased year after year in the US. From insulin to Revlimid.
22/ Third, we need to make it far easier and competitive for generic and biosimilar manufacturers to enter the US market. At least 4 generics for each brand name drug is needed to have an effect on prices.
23/ We could consider reciprocal approval of generics approved in Europe to ease burden on FDA.
24/ We need more non profit minded generic drug manufacturers. @CivicaRx @mcuban @costplusdrugs
25/ Fourth we need to reduce the cost of clinical trials. This puristic drive for 100% monitoring and perfection makes our trials extraordinarily expensive. Revise FDA guidelines.
26/ Fifth, we need serious patent reform. 7-10 years and done. Or similar. @pritikrishtel @IMAKglobal
27/ From individual physician, professional institution/ organization standpoint what can we do?
28/ First, we must have strong National treatment guidelines and pathways that take cost into account. Guidelines that recommend two dramatically differently priced drugs as equivalent do not help.
29/ So the 2 drugs below are not similar just because the trial found them similar. They are not similar in price. Since our system rewards the higher priced drug with more reimbursement it is an invitation to prescribe the higher price drug if we say both are reasonable options.
30/ We must conduct strategic trials that reduce cost. Testing fixed duration therapy. Clever dosing schedules. Etc.
31/ Always support the lower lost price option even if your profit margin is higher with the brand name drug due to rebates or other deals. Always support generics and biosimilars.
32/ Always learn the cost of drugs. Learn how to help your patient get lowest price drugs. @costplusdrugs @GoodRx
33/ Advocacy is important. There are few organizations seriously trying to lower drug costs. Find a way to engage and support. @AARP @policydeb @DavidP4AD @P4AD_ @IMAKglobal
34/ Talk to your patients about access and affordability.
35/ For a talk in which I discuss most of this, check out.

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More from @VincentRK

Jun 27, 2025
What’s up with Beta 2 microglobulin (B2M) and myeloma?

Why is it the sole non-cytogenetic marker used in the new IMWG high risk definition?

Why does it matter whether the creatinine is high or not?

Thread
1/
Beta 2 microglobulin goes up in myeloma with either increasing tumor burden OR decreasing renal function.

Thus a high beta 2 microglobulin in myeloma is a reflection of high tumor burden or renal failure or both.

We need to distinguish why the B2M is high!
2/
When B2M is high, it confers adverse prognosis, but why it is high determines what you can do about it.

If the B2M is high because of tumor burden our strategy will be different than if it’s high due to renal failure.

3/
Read 7 tweets
May 24, 2025
The remarkable story of Velcade.

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. Image
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…Image
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 )

PS-341 was a proteasome inhibitorascopubs.org/doi/10.1200/JC…Image
Read 13 tweets
May 18, 2025
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 serendipityImage
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.Image
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. Image
Read 20 tweets
Dec 9, 2024
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/ Image
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/ Image
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/ Image
Read 12 tweets
Dec 9, 2024
Just out: Paradigm changing AQUILA randomized trial in high risk smoldering myeloma #ASH24
@thanosdimop @NEJM

Daratumumab significantly prolongs time to active myeloma and overall survival. Proud to be a lead investigator of this trial

Slides in thread nejm.org/doi/full/10.10…Image
Main Findings

Daratumumab prolongs:
-Time to active myeloma
-Time to CRAB
-PFS2
-Overall Survival

Plus: Low toxicity, no detriment to QOL, & limited duration therapy. #ASH24 #ASH24VR Image
390 patients with high risk SMM randomized. Largest trial in SMM ever conducted.
Limited duration therapy.

Significant improvement in time to progression to active myeloma or death with Dara.

Benefit seen in almost all subgroups. #ASH24 Image
Read 13 tweets
Aug 24, 2024
Why are prescription drug prices are far higher in the US that other developed countries.

I’ll break it down. A full 360.

1/ We don’t negotiate prices at launch of a new drug. Others do. Image
As a result, we spend billions on common drugs that other countries spend a fraction of the price on.

Some drugs we pay 10 or 100 times more!! Image
2) Generic and biosimilar entry, adoption, and utilization is slower in the US, and there are many barriers.

Timely and adequate free market competition is critically important for lowering price. Image
Read 21 tweets

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