Why drug discovery needs you

Question 1

What was the cost of a life-changing drug for cystic fibrosis?

Answer 1

• cost $327,000 per year
• Cost $5,700 to make

most countries have refused to pay that (only 12% of patients are receiving it)

Question 2

How does Pharma decide the price of drugs?

Answer 2

they charge what the market will bear, not what is “right”

Question 3

What was the average price of a new drug in 2007 vs now?

Answer 3

$2,115 now it’s $180,000

Question 4

What big company should we be mad at, and why?

Answer 4

Pfizer
- played a major role in allowing us to get back to our normal lives, is making a profit of over $20 billion

Question 5

What profession’s code were medical men bound to back in the day?

Answer 5

to make all advances in healthcare freely available to humanity

it would violate a physician’s Hippocratic oath to engage in profiting from a discovery

when the details of the method of preparation are published, anyone will be free to prepare the extract

Question 6

At the current prices, how much would it cost to extend the lives of each cancer patient in the USA by 1 year?

Answer 6

$440 billion and no one would be cured

Question 7

How much do cancer drug rise in the USA per year?

Answer 7

10%

Question 8

How many new cancer drugs were approved in 2012? How much did they cost?

Answer 8

• 12
• 11 cost over $100,000 per year
  - only 3 found to improve survival
  - 2 increased survival by less than 2 months

Question 9

What is Cetuximab?

Answer 9

lung cancer drug
- 1.2 months of extra life ($80,000)
- skin toxicity on 85% of patients

Question 10

What is Bevacizumab?

Answer 10

lung cancer drug
- added 12-18 days of extra life

Question 11

When is the cost “too much”?

Answer 11

in the UK, over 30k pounds but doesn’t give more than one year of life, it won’t be approved.
- Over 15k pounds is harmful as it takes away from other essential healthcare services

Question 12

How much does CAR-T cost?

Answer 12

$300,000

Question 13

What is the most expensive drug in the world?

Answer 13

gene therapy for adults with hemophilia B
$3.5 million

Question 14

What is Elevidys?

Answer 14

gene therapy used to treat children with Duchenne (muscular disease)

costs $3.2 million

Question 15

What were some benefits of children using Elevidys?

Answer 15

• walking 10 meters improved by 0.42 seconds over the placebo
• Rising from the floor improved by 0.64 seconds over the placebo
• improvement on a scale of 0-17 (drugs: 2.7; placebo: 1.9)

Question 16

If we spend money on cancer drugs then we have to do without other things. What will we sacrifice?

Answer 16

• an extra year for your parent or child
• kidney transplant for a child
• fertility treatment for couples
• surgery for obesity
• free dental care
• treatment for drug addicts
• supported jobs for adults with autism
• Drugs to prevent HIV from mother to baby
• drugs to prevent children from catching malaria
• free education in Canada

Question 17

Why are such high prices “needed”?

Answer 17

• the highest risk of failure industry
• 5-10% of drugs that enter trials make it to market
• 1 in 3 drugs that reach the market make a profit
• it costs over $2 billion to develop a new drug (with failures)

Question 18

What do some models say about Pharma?

Answer 18

• some say, they are no longer sufficiently profitable (returns are below the cost of money)
• others say they are extremely profitable

Question 19

What happens if a company is not sufficiently profitable?

Answer 19

• share price slumps
• management fired
• company taken over
• employees fired and assets stripped
• advertising

Question 20

What is hepatitis C?

Answer 20

viral infection that affects the liver
over 170 million cases worldwide

Question 21

What was a Hepatitis C treatment before 2011?

Answer 21

• dual therapy
• cost $18k-36K
• 40-50% cure rate

Question 22

What was a Hepatitis C treatment in 2011?

Answer 22

Incivek
- cost $49k + 18K-36K
- 60-80% cure rate

Question 23

What was a Hepatitis C treatment in 2014?

Answer 23

Sovaldi/ Harvoni
- no need for the drug from before 2011
- cure rate over 95%
- only took 12-24 (less than 24-48, which was before 2011)

Question 24

What is the problem with the way people are treating pharmaceutical companies about making a profit?

Answer 24

If people are saying you can’t make a profit off of something you achieve (like apple), then we’re deciding pharmaceutical/ biotech industries can’t be run in the same fashion, but their still held to the same requirement to make money for investors

Question 25

How did the price of Sovaldi come down?

Answer 25

a market force
- Mavyret was a new Hep C treatment, but only $24k per year making Sovaldi prices to come down to match

Question 26

When did management change in pharma?

Answer 26

in the early 1980s, drugs became commodities (people only saw profit)

Question 27

What happened to Lance Armstrong?

Answer 27

had testicular cancer that spread to his brain, he was a cyclist and was cured

Question 28

Who was Greg Louganis?

Answer 28

a driver who cracked his head open, had HIV, and was scared he was going to spread it

Question 29

What would’ve happened if AIDs wasn’t a global disease (eventually giving rapid progress)?

Answer 29

It would’ve been ignored

Question 30

What was the timeline of AIDS?

Answer 30

1981: Aids first reported (1 year life)
1983: HIV cause of AIDS
1887: AZT (raised to 21 months)
1995: Saquinivir (huge improvement in survival)
2007: Raltegravir (normal life)

Question 31

How much difference did it make to have drugs against two different targets?

Answer 31

we can avoid resistance to developing

Question 32

What are HIV Integrase Inhibitors?

Answer 32

Raltegravir (2007)
- responsible for integrating viral DNA into human DNA, to allow for replication
- no human equivalent, good for selectivity

Question 33

What is Atripla?

Answer 33

a combination pill for HIV
a three-drug fixed-dose combination (3 drugs in one pill)

Question 34

How can HIV be a disease of the past?

Answer 34

we have the tools
- if the viral load is kept low by ART (combination pills for HIV), then the risk of transmission will be eliminated

Question 35

How does ART eliminate transmission?

Answer 35

Mother-to-child transmission is 15-25% (35-40% with breastfeeding). There is near elimination when anti-retroviral therapy is used (down to 1.5%)

Question 36

What is the problem with eliminating HIV?

Answer 36

fewer than half of HIV infected people are receiving treatment, most have no been diagnosed, an estimated 21 million deaths could be avoided with better testing and better ART coverage

Question 37

Why is HIV/ AIDS not a disease of the past?

Answer 37

Hype drugs are a big threat to healthcare budgets. Always a new Alzheimer’s disease cure taking the budget away from other projects, and always seem to fail

Question 38

What is Aducanumab?

Answer 38

a failed Alzheimer’s drug

Question 39

What were the results of Biogen’s Alzheimer’s drug?

Answer 39

two trials: one failed, the other showed an improvement of 0.39 on a scale of 0-18
($56,000)

Question 40

How much could Aduhelm cost Medicare?

Answer 40

$66.9 billion

Question 41

How can the prices change for new drugs?

Answer 41

informed people shouting louder than uninformed people
(several private insurers won’t cover Biogen’s Alzheimer’s drug)

Question 42

What happened when people spoke out about Biogen’s Alzheimer’s drug?

Answer 42

cut prices in half, $500 million in cost-cutting

Question 43

What drug was approved that is slightly better than Biogen’s?

Answer 43

Lecanemab was shown to moderately slow cognitive decline in the early stages of the disease (a score ranging from 0-18 did 0.45 points better)

Question 44

When did public pressure and outrage work in the medical field?

Answer 44

the cost of insulin, a hormone the body needs to survive, were too high. Prices were cut by 70% by Eli Lilly

Question 45

What was a positive side effect for women who are HPV vaccinated?

Answer 45

no cervical cancer cases

it’s the most common cancer in women 25-35

Question 46

What is Dracunculiasis?

Answer 46

infection by a parasite
1980s: 3.5 million cases per year
2023: 13 cases

Question 47

What did Jonas Salk and Albert Sabin do?

Answer 47

created polio vaccines and didn’t put them under patents. They eradicated polio leaving fewer children from becoming paralysed

Question 48

What are some ways to improve pharmacoeconomics?

Answer 48

• refuse to approve drugs that only show meaningless gains or impose price restrictions and link price to efficacy (companies won’t pursue borderline drugs, fewer phase 3 failures, lower developmental costs)
  -more emphasis on disease prevention, detection and diagnosis (cheaper and better for everyone to avoid cancer than to treat it)
• Act globally (must be led by the USA, the largest market by far)
• link price to gross domestic product for each country
• remove advertising from pharmaceuticals