EP352: Some Big Actionable Surprises About the Efficacy and Effectiveness of Specialty Pharmaceuticals, With Pramod John, PhD

Efficacy vs. Effectiveness in Specialty Pharmaceuticals, With Pramod John, PhD. Why Only 2% of Patients Get the Expected Benefit From Some Expensive Specialty Drugs. Episode 352.

Stacey Richter talks with Pramod John, PhD, founder and CEO of VIVIO Health, about the gap between a specialty drug's clinical trial efficacy and its real-world effectiveness—and why limiting a formulary to just one or two drugs per category can mean paying huge sums for treatments that don't work for most of the patients taking them.

WHAT YOU'LL LEARN

✅ Why specialty drugs are less than 2% of prescriptions but account for roughly half of the $500 billion the US spends annually on prescription drugs

✅ The difference between a drug's efficacy (how it performs in trials) and its effectiveness (how it performs in the real world)

✅ Why limiting formulary access to one or two drugs per therapeutic category reduces the odds that any given patient lands on the drug that actually works for them

✅ How the concepts of NNT (number needed to treat) and NNH (number needed to harm) should factor into patient decision-making

✅ Why expanding access to more drugs in a therapeutic category, rather than narrowing formularies, can improve outcomes and lower total cost

✅ How VIVIO's model has generated 35% to 40% savings on drug acquisition costs while improving outcomes

WHY THIS MATTERS

When formularies are built around rebate economics rather than which drug actually works for which patient, purchasers pay enormous sums for treatments that a majority of patients get no benefit from—and sometimes serious side effects instead. Understanding efficacy versus effectiveness, and NNT versus NNH, gives employers and plan sponsors the vocabulary to demand a formulary strategy built around what actually helps patients.

MENTIONED IN THIS EPISODE

EP334 with Sunita Desai, PhD: Apple Podcasts | Spotify | Other Apps

EP303 with Anna Kaltenboeck: Apple Podcasts | Spotify | Other Apps

=== LINKS ===

🔗 Show Notes with all mentioned links: Episode Page

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00:00 Introduction.

05:34 What does a good response mean in pharmaceutical products?

06:06 "Different people get different utility out of something."

06:31 Why doesn't efficacy mean what you think it means in terms of pharmaceutical products?

08:40 What is the difference between efficacy and effectiveness in Pharma?

09:10 Why aren't drugs' major side effects factored into a drug's efficacy and effectiveness?

10:14 "What's the benefit of this versus what's the harm in this?"

13:35 "Clearly as consumers, we all feel that we're special. But what about physicians?"

14:14 "The benefit itself—what does it have to be?"

17:11 "We tend to think of things as a binary distribution—it works or it doesn't."

18:22 "The default choice that we start with is often the wrong one."

20:54 "It doesn't matter why if we can't fix the reason."

22:02 "At some point, the question becomes, 'Do we have any information?'"

22:36 Why do other developed countries pay less for their drugs?

24:21 How do we end up with crappy drugs on the market that don't really move the dial?

27:22 "We can build a better system. And that's what we do every day."