Chapter 13: The medical department
In the development, we see a third wave of innovation with a potential to develop
“Transformational” therapies, this will require us to rethink and challenge existing paradigms. In the
third wave, we see an upcoming of the genetic medicines which will treat the underlying cause. This
includes RNAi, gene therapy and gene editing. The Business Development and deal licenses are
critical in building the innovation substrate for research and development. More than 60% of the
launched products results in collaboration. There are different sources of innovation for the
thoughtful integration of internal capabilities and external substrate. The more mature your
molecule (compound), the type of collaboration will be different. When you are in the beginning of
the research and development phase, you will have a lot of research collaborations. However, when
you are ending phase III, the collaborations will change into licensing and marketing authorization
collaborations for access existing opportunities.
Medicine development
The development of new medicines is a
highly regulated and long process. Are
there opportunities to shorten the
development timelines during all stages
of drug development? Yes, there are,
think about AI and COVID learnings. The
drug development phase and the pre-
clinical phase together takes 4 – 6
years! To reduce the development
time, we can maybe combine phase I
and phase II.
The medical department
The Medical Department works in the four domains of registering a product:
1. Research and development: In humans, so from phase I studies. This is typically a global
effort. You are developing a medicine, and, if it works, it is produced on a big scale. This is the
global effort
2. Regulatory
3. Access
4. Commercialization
1. Research and development
Overview of clinical trials per region
30 – 40% of the clinical studies are sponsored by the industry. There is an emerging role of China,
they grew a lot in clinical trials. For 10 years ago, this was significantly less!
Clinical trial program
The Clinical Trial Program was developed in close early consultation with regulatory authorities (e.g.
the FDA, EMA and PMDA). The European Medicines Agency’s Committee for Medicinal Products for
Human Use prepares scientific guidelines in consultation with regulatory authorities in the European
Union (EU) Member States, to help applicants prepare marketing authorization applications for
human medicines. Guidelines reflect a harmonized approach of the EU Member States and the
Agency on how to interpret and apply the requirements for the demonstration of quality, safety and
efficacy set out in the Community directives. The Agency strongly encourages applicants and
marketing authorization holders to follow these guidelines. Applicants need to justify deviations from
guidelines fully in their applications at the time of submission. Before that, they should seek scientific
advice, to discuss any proposed deviations during medicine development. So, the guidelines are
Pagina 1 van 5
In the development, we see a third wave of innovation with a potential to develop
“Transformational” therapies, this will require us to rethink and challenge existing paradigms. In the
third wave, we see an upcoming of the genetic medicines which will treat the underlying cause. This
includes RNAi, gene therapy and gene editing. The Business Development and deal licenses are
critical in building the innovation substrate for research and development. More than 60% of the
launched products results in collaboration. There are different sources of innovation for the
thoughtful integration of internal capabilities and external substrate. The more mature your
molecule (compound), the type of collaboration will be different. When you are in the beginning of
the research and development phase, you will have a lot of research collaborations. However, when
you are ending phase III, the collaborations will change into licensing and marketing authorization
collaborations for access existing opportunities.
Medicine development
The development of new medicines is a
highly regulated and long process. Are
there opportunities to shorten the
development timelines during all stages
of drug development? Yes, there are,
think about AI and COVID learnings. The
drug development phase and the pre-
clinical phase together takes 4 – 6
years! To reduce the development
time, we can maybe combine phase I
and phase II.
The medical department
The Medical Department works in the four domains of registering a product:
1. Research and development: In humans, so from phase I studies. This is typically a global
effort. You are developing a medicine, and, if it works, it is produced on a big scale. This is the
global effort
2. Regulatory
3. Access
4. Commercialization
1. Research and development
Overview of clinical trials per region
30 – 40% of the clinical studies are sponsored by the industry. There is an emerging role of China,
they grew a lot in clinical trials. For 10 years ago, this was significantly less!
Clinical trial program
The Clinical Trial Program was developed in close early consultation with regulatory authorities (e.g.
the FDA, EMA and PMDA). The European Medicines Agency’s Committee for Medicinal Products for
Human Use prepares scientific guidelines in consultation with regulatory authorities in the European
Union (EU) Member States, to help applicants prepare marketing authorization applications for
human medicines. Guidelines reflect a harmonized approach of the EU Member States and the
Agency on how to interpret and apply the requirements for the demonstration of quality, safety and
efficacy set out in the Community directives. The Agency strongly encourages applicants and
marketing authorization holders to follow these guidelines. Applicants need to justify deviations from
guidelines fully in their applications at the time of submission. Before that, they should seek scientific
advice, to discuss any proposed deviations during medicine development. So, the guidelines are
Pagina 1 van 5
