Regulatory Affairs & Industrial Marketing

Question 1

FDA, EMA, HC, NMPA/CDE are what?

Answer 1

Regulatory governance, but also sparring partners for companies.

Question 2

What is ICH?

Answer 2

International Conference for Harmonization – tries to align data, applications, vocabulary etc. for drugs.
- Focus on Quality, Safety, Efficacy

Question 3

What is a common technical document?

Answer 3

Sufficient and complete documentation for clinical trials and all requirements defined in the legislation complied with

• Quality, Q – manufacturing and control
• Safety, S – preclinical safety
• Efficacy, E – clinical efficacy

Question 4

What is the trend in documentation in regards to drug approval?

Answer 4

Increasing demands since the 1960s initially due to the thalidomide disaster. From 7 pages in 1952 to millions of pages today.

Question 5

What is Marketing Authorizations?

Answer 5

Approval from health authorities = Marketing Authorisation

• Needed to sell a drug

Question 6

Why is the Target Product Profile/Label important?

Answer 6

• The label is the outcome and conclusion of the entire product development
• It is the most important part of the marketing authorisation as it provides:
  1) Information to physician for use of the product – important efficacy and safety information
  2) Information to patients (package leaflet): Use and side effects
  3) Framework for appropriate promotion and differentiation vs. competitors

Question 7

How many species do you have to test therapies in?

Answer 7

At least two animal models
How many years does it take to develop a drug?
6-10 years (closer to 6 if it gets special permissions)

Question 8

What is posology?

Answer 8

The part of medicine concerned with dosage.

Question 9

Off-label promotion – is it allowed, can they do active advertising?

Answer 9

NOT Legal!
- Communication about an unapproved drug or an off-label use of an approved drug that promotes or is designed to promote the unapproved drug/off label-use

Question 10

What is off-label use and is it legal?

Answer 10

• A use that fails to comply with the currently approved label
• Legal if physician decides to do it (not the company)

Question 11

What is off-label information and is it legal to supply from the company?

Answer 11

• Legal, if not promotional!

Communication about an unapproved drug or an off-label use of an approved drug that does not constitute promotion

Question 12

What is a patent?

Answer 12

A negative right – prohibits somebody in using your invention.
- Term of 20 years + up to 5 years of extension in some countries

Question 13

What is RDP?

Answer 13

Regulatory Data Protection – Meaning others can’t refer to your data for their trials
* Exclusive right to benefit from data
* Automatically in force from date of marketing authorisation
* Enforcement of RDP is the responsibility of governments
* Term of 5-12 years varying from country to country

(RDP = Data Exclusivity + Market Exclusivity)

Question 14

Oprhan Drug Excusivity is how long in EU and US?

Answer 14

EU: 10 years from approval
US: 7 years from approval

Question 15

What are generics?

Answer 15

A drug that contains the same active moiety as a previously approved drug and is intended for the same use as the previously approved drug

Question 16

What are biosimilars?

Answer 16

A drug that contains the same principal molecular structural features (but not necessarily all of the same structural features) and is intended for the same use as a previously approved drug

Question 17

Industrial enzymes business case looks like?

Answer 17

1) Major economic scale advantage (economics of scale = high entry barrier) + Significant IP in main applications and ability of players to enforce.
2) High level of rivalry (small player enter niche applications)
3) Buyer power varies between industries and markets. High switching cost as enzymes often are strategic resources.
4) Possibility to substitute petrochemicals which are increasingly unattractive.
5) Low supplier power: As Novozymes is buying commodities (electricity etc.)

Question 18

Exclusivity deals are only made with?

Answer 18

Big players that can buy big volumes, they are often time limited before being released to the rest of the industry.

Question 19

When market sizing what can you do?

Answer 19

Make it a range instead of a solid number (ie. Market size = 10-20 mio DKK)

Question 20

Is market sizing always spot on?

Answer 20

No. Your estimates will tend to overshoot the potential account for the complexity of the market place!