189
Global Pharmaceutical and Biologics Regulatory Strategy, Second Edition
1
2
3
4
5
6
7
8
9
10
11
12
13
14
15
16
17
18
planned product presentation. The sponsor TPP
will establish the global development plan frame-
work, incorporating data and regulatory require-
ments and strategies to gain marketing approval
in the shortest possible timeframe. Ultimately,
the company may ask a regulatory professional
the likelihood of achieving the product’s targeted
labeling claims and when it will be achieved.
Increasingly, the overall goal is a global
development program with simultaneous, mul-
timarket submissions and approvals. However,
many factors can influence the need for prioritiz-
ing an individual country or region, identifying
opportunities to enable early or more cost-ef-
fective market entry. For example, orphan drug
programs vary significantly. Some are limited to a
reduction in regulatory agency fees, while others,
like the US, have more generous programs that
include additional agency support and devel-
opment grants. A second important example is
FDA’s accelerated approval program that pro-
vides an opportunity to gain early approval based
on surrogate or intermediate endpoints, thus
reducing premarket development costs and time,
and leading to early market entry.
Similar programs to support innovative
new medicines are available in Europe, includ-
ing accelerated assessment of medicines of
major public health interest via the Centralised
Procedure, especially ones that are therapeutic
innovations. Accelerated assessment usually takes
150 evaluation days, rather than the standard 210
days. In addition, in 2016, a program to enable
accelerated assessment of PRIority MEdicines
(referred to as PRIME) was introduced. PRIME
eligibility criteria are similar to those for acceler-
ated assessment but result in additional dialogue,
support and connectivity within the EU regu-
latory network. It is intended for medicines in
development to address an unmet medical need.
There also are mechanisms in some jurisdictions
to allow approval based on more limited data
(e.g., Phase 2 studies or surrogate endpoints)
due to the new medicine’s promising nature. The
conditional approval mechanism in Europe is
intended for this purpose, where benefit-risk is
positive, but more data are required to confirm
benefit post-license.
Conversely, local regulations requiring addi-
tional studies (aiming to ensure clinical data are
relevant to the local population) or having sig-
nificantly longer approval times may negatively
influence a country’s prioritization. Increased
development costs or delayed market entry to
generate additional data may reduce financial
returns compared to marketing a product in a
country without these challenges. Ideally, a com-
pany will consider an integrated evidence gener-
ation strategy that considers evidence required by
the healthcare system’s multiple stakeholders and
will gather such evidence in the smallest number
of separate studies minimizing cost while meet-
ing stakeholder objectives for evidence.
Clearly articulating claims and global market
entry timing enables potential sales in key mar-
kets, development costs and return on investment
to be modeled.
In early development stages, many
unknowns and assumptions need to be built
into the model. Scenario planning and sensi-
tivity analysis will take these uncertainties into
account, which will reduce as development
progresses and challenges are overcome, creating
an increasingly robust financial model to support
business planning.
During development, decisions made by
the multidisciplinary team will impact poten-
tial business outcomes, the financial return and
subsequent patient access. Decisions should
be based on data available, moving the prod-
uct forward toward the overall TPP objectives.
If available data indicate deviations from the
planned pathway are required, a detailed TPP
review and business outcome impact evaluation
should follow.
The sponsor TPP and development plan are
living documents and require regular monitoring
and review to evaluate progress against objectives
and updating as appropriate.
Global Pharmaceutical and Biologics Regulatory Strategy, Second Edition
1
2
3
4
5
6
7
8
9
10
11
12
13
14
15
16
17
18
planned product presentation. The sponsor TPP
will establish the global development plan frame-
work, incorporating data and regulatory require-
ments and strategies to gain marketing approval
in the shortest possible timeframe. Ultimately,
the company may ask a regulatory professional
the likelihood of achieving the product’s targeted
labeling claims and when it will be achieved.
Increasingly, the overall goal is a global
development program with simultaneous, mul-
timarket submissions and approvals. However,
many factors can influence the need for prioritiz-
ing an individual country or region, identifying
opportunities to enable early or more cost-ef-
fective market entry. For example, orphan drug
programs vary significantly. Some are limited to a
reduction in regulatory agency fees, while others,
like the US, have more generous programs that
include additional agency support and devel-
opment grants. A second important example is
FDA’s accelerated approval program that pro-
vides an opportunity to gain early approval based
on surrogate or intermediate endpoints, thus
reducing premarket development costs and time,
and leading to early market entry.
Similar programs to support innovative
new medicines are available in Europe, includ-
ing accelerated assessment of medicines of
major public health interest via the Centralised
Procedure, especially ones that are therapeutic
innovations. Accelerated assessment usually takes
150 evaluation days, rather than the standard 210
days. In addition, in 2016, a program to enable
accelerated assessment of PRIority MEdicines
(referred to as PRIME) was introduced. PRIME
eligibility criteria are similar to those for acceler-
ated assessment but result in additional dialogue,
support and connectivity within the EU regu-
latory network. It is intended for medicines in
development to address an unmet medical need.
There also are mechanisms in some jurisdictions
to allow approval based on more limited data
(e.g., Phase 2 studies or surrogate endpoints)
due to the new medicine’s promising nature. The
conditional approval mechanism in Europe is
intended for this purpose, where benefit-risk is
positive, but more data are required to confirm
benefit post-license.
Conversely, local regulations requiring addi-
tional studies (aiming to ensure clinical data are
relevant to the local population) or having sig-
nificantly longer approval times may negatively
influence a country’s prioritization. Increased
development costs or delayed market entry to
generate additional data may reduce financial
returns compared to marketing a product in a
country without these challenges. Ideally, a com-
pany will consider an integrated evidence gener-
ation strategy that considers evidence required by
the healthcare system’s multiple stakeholders and
will gather such evidence in the smallest number
of separate studies minimizing cost while meet-
ing stakeholder objectives for evidence.
Clearly articulating claims and global market
entry timing enables potential sales in key mar-
kets, development costs and return on investment
to be modeled.
In early development stages, many
unknowns and assumptions need to be built
into the model. Scenario planning and sensi-
tivity analysis will take these uncertainties into
account, which will reduce as development
progresses and challenges are overcome, creating
an increasingly robust financial model to support
business planning.
During development, decisions made by
the multidisciplinary team will impact poten-
tial business outcomes, the financial return and
subsequent patient access. Decisions should
be based on data available, moving the prod-
uct forward toward the overall TPP objectives.
If available data indicate deviations from the
planned pathway are required, a detailed TPP
review and business outcome impact evaluation
should follow.
The sponsor TPP and development plan are
living documents and require regular monitoring
and review to evaluate progress against objectives
and updating as appropriate.