The current system for developing and pricing orphan drugs is unsustainable and will lead governments to impose price limits. A new approach called Sustainable Manufacturing Practice (SMP) is proposed that focuses on personalized medicine, companion diagnostics, small scale production, and conditional approval of drugs based on real-world efficacy data from public registries. SMP requires drug development and pricing to shift from a blockbuster model to one tailored for small patient groups through regulatory and legal changes.

1. From GMP
to
SMP?

2. The current system, although provides successes,
will not provide accessibility and sustainability
2
OD development is, under
current system, too slow
time
Price limit
Current pricing level for OD x New drugs in pipeline = Unsustainable
Governments or payers
will impose a price limit in
coming years
With current pricing levels OD
become unsustainable
With 90 out of 6000 OD covered
and pipeline very focussed on
cancer, development is too slow
With current pricing levels (still
rising) and pipeline the
inconvenient truth is that OD will
be unsustainable

3. The inconvenient truth: the cost challenge will
lead to a price or budget limit
3
time
Price limit
Current pricing level for OD x New drugs in pipeline = Unsustainable
Governments or payers
will impose a price limit in
coming years

4. A partnership for the future:
Sustainable Manufacturing Practice (SMP)
4
GMP
Sustainable
Manufacturing
Practice
Within
pricing limits
Personalised
Based on
companion
diagnostic
effective
New technological options
- Personalised medicine
- Companion diagnostic
(example organoids)
- Big data
- Small Scale Production
Legal – societal innovations
- Fast track
- Pressure to change the model

5. “SMP” requires a paradigm shift
5
Research Development Treatment
university
Public funded:
only sell/licence
under SMP….
Regulatory affairs
Pharma
Framework to
develop and test
for small groups.
Conditional
Approval
Pending
efficacy
In practice
Companion
diagnostic
registry
Public
registry
Small
Scale
Production
Admission
GMP
Sustainable
Manufacturing
Practice
time
Price limit
Governments or payers
will impose a price limit in
coming years

6. If the research is funded with ‘public money’
there should be an obligation to enforce SMP
6
university Pharma
Public money to fund
research
- Grants
- Taxes
- Funds from patient
organisations
GMP
Sustainable
Manufacturing
Practice
time
Price limit
Governments or payers
will impose a price limit in
coming years
A legal obligation to research under
‘SMP’ when using public funds
The innovation is sold / licensed under
SMP:
- The next party has the obligation to
ensure that end pricing level doesn’t
exceed Sustainable Pricing Limit

7. Switch from the large volume framework to small
group framework
7
Regulatory affairs
Pharma
The regulatory framework is
not suited for small groups
Our frameworks were designed
in the blockbuster era
Large scale trials, strict focus
on production
Pharma
Create a framework for small
groups: faster and cheaper
Create a new framework that
still focusses on safety but
acknowledges the difficulty of
small groups

8. Under ‘SMP’: conditional admission pending
efficacy evidence from registry
8
Conditional admission Pending efficacy in practice
Admission based on Companion Diagnostic
‘If it works it’s reimbursed
registry
Under ‘SMP” price is not
the issue… admission
should focus on efficacy
Accessibility is greatly improved if drugs are
conditionally approved for a short term and
based on CD
During this approval period the efficacy has to
be established, in practice, using a public
registry
Example
Drugs for CF not based on
gen mutation but on CD:
Organoids

9. Companion Diagnostics facilitate Personalised
Medicine and reduces waste
9
Companion
diagnostic
60% no effect
Within patients with
designated gen
mutation
40% of patients with
designated gen
mutation show effect
Actual
case
Under ”SMP” Companion
diagnostic is compulsory for OD

10. New technologies for small scale production
could facilitate personalised medicine at low cost
10
From large scale production To small scale production

11. We will hit the wall…. The ‘SMP’ can help us to
ensure accessibility and sustainability
11
Unsustainable
Research Development Treatment
university
Public funded:
only sell/licence
under SMP….
Regulatory affairs
Pharma
Framework to
develop and test
for small groups.
Conditional
Approval
Pending
efficacy
In practice
Companion
diagnostic
registry
Public
registry
Small
Scale
Production
Admission
GMP
Sustainable
Manufacturing
Practice
time
Price limit
Governments or payers
will impose a price limit in
coming years