For those of you interested in multiple myeloma, here is a brief state of the science for treatment.

1/ For newly diagnosed patients, triplets, VRd or DRd, are the standard of care. Current RCTs are examining if we can improve outcome using quadruplets. Eg. PERSEUS trial.
2/ In newly diagnosed myeloma, another question given the cost & potential toxicity of quadruplets is to start with a triplet, and based on MRD status identify patients who need 4 drugs versus this who do equally well with 3 drugs. Eg: EQUATE trial. @myelomaMD @mtmdphd @eaonc
3/ Auto stem cell transplant is still standard of care for eligible patients. Current data indicate overall survival is similar whether the transplant is done early or delayed. But for many reasons we prefer early transplant for most patients.
4/ Lenalidomide maintenance is standard of care after initial therapy. For high risk patients, lenalidomide + bortezomib. Current research questions include whether we can stop maintenance after 2 years, or adjust maintenance based on MRD status. Eg: SWOG trial; OPTIMUM trial
5/ For Relapsed myeloma numerous treatments are being investigated alone and in combination across the world. The more active drug classes we have, the longer the survival. Great progress.
6/ Immunotherapy is the major advance in myeloma. Bispecific antibodies and CAR-T cell therapy hold great promise. Multiple options available for myeloma refractory to lenalidomide, pomalidomide, bortezomib, carfilzomib, daratumumab, isatuximab, alkylators, and transplant.
7/ For smoldering myeloma, we now feel that early therapy is indicated for high risk patients. A comprehensive trial strategy is underway, specifically to see if patients with high risk SMM do better with myeloma like treatment like DRd, or if mild treatment like Rd is enough.

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More from @VincentRK

11 Jun
The delta variant is the triple threat I have talked about. I am not saying this lightly. I take no pleasure in alarming people. It's just the facts after 2 months of being closely involved with the India COVID crisis. This is not last years COVID. Get vaccinated.
The message is not alarm. The message is that there is an easy solution that works. #GetVaccinated
Read 5 tweets
10 Jun
When the FDA approves a drug, it is not only approving the drug. Its actions provide prolonged monopoly protection to a drug in a country where the seller can set whatever price they want, the buyer cannot negotiate price, and competitors will face more severe barriers to entry
If like other Western countries we had a 2-step process for prescription drugs: regulatory approval followed by value based pricing determination, I would have less problem with an FDA that is more lenient.
If like other countries barriers to market entry to competition are not so hard, then I would have less problem with an FDA that is more lenient.
Read 5 tweets
9 Jun
Vaccine hesitancy in alive and well in the US. And it is going to cost lives. The level of vaccination we have in the US is simply not sufficient against the delta variant.

Friends: tell unvaccinated friends to get vaccinated.

Leaders: lead. Talk to your constituents. Beg them
30 million people who got one dose of vaccine need second dose. One dose is not enough.

Another 60-100 million people need to be fully vaccinated.

Get vaccinated even if you had COVID before.

Im trying not to be alarmist even though I'm extremely worried.
The delta variant is a triple threat. It is here.

1) More transmissible: In India, If often if one family member had it, everyone got it.

2) Vaccine evasive: In UK study a single dose of Pfizer or AZ had only 33% protection against symptomatic COVID.

3) Likely more virulent
Read 8 tweets
8 Jun
Incidental COVID positive test after full vaccination without COVID symptoms is not COVID.

Better still I have been calling for Reinfection COVID and Post vaccination COVID to have their own names, because the natural history and prognosis will be different.
COVID is the name of a disease: SARS CoV-2 infection in people without prior immunity to SARS CoV-2 infection. It carried a certain hospitalization / mortality rate.

SARS CoV-2 infection in people who previously had COVID or were fully vaccinated are different diseases.
You have to define the natural history of each separately because both for the individual patient and for public policy the implications are different.

It doesn't mean Post Vaccination COVID cant be serious: it means the prognosis from such diagnosis will be different from COVID
Read 4 tweets
7 Jun
The reason Biogen's Alzheimer's drug is priced at $56,000 per year and not $112,000 is because they are being kind.

Nothing in the law or process that prevents them from setting whatever price they want. The system is not broken. There is no system.
Hope people at recent @icer_review meeting remember the comment I made at the myeloma meeting: Pharma is wary about lawmakers going after them for price increases. So the name of the game is going to be front loading. Get all you can get early.

@SarahKarlin @RESachs @DusetzinaS
Please for heaven's sake pass a law enabling Medicare to directly negotiate prescription drug prices. 90% of the public wants it.
Read 5 tweets
7 Jun
The FDA basically has the stance that as long as efficacy and safety are shown, the magnitude of the clinical benefit, no matter how tiny, doesn't matter. The cost however huge doesn't matter. It's not in their purview.

We need to reform the system.
nytimes.com/2021/06/07/hea…
In all other developed countries, regulatory approval is only the first of two steps.

I believe with existing law, CMS and insurance companies can play hardball and exercise the second step: Value. It would be better still if we had an automatic second step to assess value.
We are going to spend money we don't have. We are going to spend money that can go for other life saving treatments. No country has infinite resources.
Read 4 tweets

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