CHAPTER 1:
The Drug Development Continuum, Preclinical to Market Access
Regulatory Affairs Professionals Society 15
Scottish Medicines Consortium (SMC), and the All Wales Medi-
cines Strategy Group (AWMSG). In France, the Haute Autorité
de Santé (HAS) uses an HTA to determine the percentage of the
price to be reimbursed by the government and paid by the patient.
In China, a drug needs to be included in the National Reimburse-
ment Drug List (NRDL) to be eligible for reimbursement.50 Eli-
gibility for inclusion in the NRDL is decided by the central
government in Beijing and the provincial governments. The pro-
cess for negotiation and inclusion into the NRDL was simplified
in 2016, and a significantly larger number of drugs has since be-
come eligible for reimbursement.44
Reimbursement is also country-specific. In many European
countries, an HTA that measures the relative effectiveness assess-
ment (REA) of a new drug compared with the standard of care is
a requirement for reimbursement. The health-related QoL is a
recognized REA end point. However, there is a lack of consensus
on which QoL data to use, for example, quality-adjusted life years
or equal value of life years gained.51
While it is important to target the broadest market access possi-
ble with the most favorable pricing, it is also important to define the
value of the medicine to each stakeholder. Medicinal product devel-
opment companies are now including real-world evidence (RWE)
tools in the product lifecycle to help achieve launch success and opti-
mal market access, positioning the right drug to the right patient.
In simplest terms, RWE is informing and supporting decision
making across the product lifecycle using analytics that provide real-
world knowledge on patients, different clinical phenotypes, and the
burden of disease, which was not possible in the past.52 RWE can
facilitate comparative effectiveness, help determine new product in-
sights, and differentiate products concerning broad-based outcomes
such as medication adherence, patient satisfaction, resource utiliza-
tion, and associated costs.52 These data are then used to inform the
target product profile, define product specific-attributes, inform
clinical trial design (right disease and patient population), and target
specific markets. Clinicians and patients are also using RWE to un-
derstand advancement in care, efficiency, and health outcomes.
Moving forward, RWE is a tool that is increasing in use across the
drug development continuum to maximize value and increase the
probability of market success.
Market access, including pricing and reimbursement, adver-
tising, and other factors that influence market success, are dis-
cussed in more detail in Chapters 35 and 36.
In parallel with market access, the MAA holder is responsible
for postmarketing regulations and lifecycle management of the ap-
proved medicine. The MAA holder must ensure product quality,
safety, and efficacy and mitigate any potential identified postmarket
References1
All references verified 12 February 2025.
1. Food and Drug Administration. The drug development process. Last updated
4 January 2018. https://www.fda.gov/patients/learn-about-drug-and-device-
approvals/drug-development-process
impact on patient health and safety through ongoing compliance
with cGMP regulations and guidance related to quality systems, in-
cluding product complaints. In addition, the MAA holder must
keep the application up to date regarding labeling, CMC changes
(e.g., manufacturing or formulation), and pharmacovigilance
(safety), including adverse event reporting. These activities are typi-
cally categorized as postauthorization activities, discussed in more
detail in Section 6: Postmarketing Authorization.
Conclusion
Medicinal product development is a continuum with specific steps
that are globally recognized. The process is long, complex, costly,
and made more challenging by the different country-specific re-
quirements. ICH continues to work on harmonization of regula-
tory requirements and guidelines beyond the founding ICH
regions, and emerging markets are adopting and implementing
standards such as the CTD and eCTD submission formats, ICH
E8(R1) on General Considerations for Clinical Studies and ICH
E6(R3) Good Clinical Practice, ICH S6 (R1) Preclinical Safety
Evaluation of Biotechnology-Derived Pharmaceuticals – Scientific
Guideline. CMC development is an integral part of the develop-
ment continuum that ensures the quality, consistency, safety, and
availability of the medicinal product.
Regulatory agencies recognize the need to expedite the au-
thorization and availability of medicinal products – especially for
serious, life-threatening, rare, and orphan indications – while
maintaining safety and effectiveness. Agencies such as the FDA,
EMA, and PMDA have worked to create and publish guidelines
and approaches to expedite development programs and nonstand-
ard review and authorization pathways to facilitate the availability
of new medicines. Agencies are encouraging early and frequent in-
teractions for novel medicines and new indications.
Marketing authorization is key to allowing medicines to be
sold in the regions authorized. In many countries, however, na-
tional pricing and reimbursement need to be determined and
agreed upon before the medicine is prescribed and patients can ac-
cess the medicine. Often, decisions also are made at the local, re-
gional, or hospital levels. Market success needs to take into
account pricing, reimbursement, and stakeholder (payer, physi-
cian, and patient) needs. Health technology assessments, health-
related quality of life metrics, and real-world evidence are playing
important roles in the product development continuum and pro-
vide input to clinical trial design, target product profile, and more,
helping to ensure the right drug is developed for the right patient
at the right price.
2. European Medicines Agency. From lab to patient: Journey of a medicine. Last
updated 10 February 2020. https://www.ema.europa.eu/en/about-us/what-we-
do/authorisation-medicines#from-lab-to-patient
The Drug Development Continuum, Preclinical to Market Access
Regulatory Affairs Professionals Society 15
Scottish Medicines Consortium (SMC), and the All Wales Medi-
cines Strategy Group (AWMSG). In France, the Haute Autorité
de Santé (HAS) uses an HTA to determine the percentage of the
price to be reimbursed by the government and paid by the patient.
In China, a drug needs to be included in the National Reimburse-
ment Drug List (NRDL) to be eligible for reimbursement.50 Eli-
gibility for inclusion in the NRDL is decided by the central
government in Beijing and the provincial governments. The pro-
cess for negotiation and inclusion into the NRDL was simplified
in 2016, and a significantly larger number of drugs has since be-
come eligible for reimbursement.44
Reimbursement is also country-specific. In many European
countries, an HTA that measures the relative effectiveness assess-
ment (REA) of a new drug compared with the standard of care is
a requirement for reimbursement. The health-related QoL is a
recognized REA end point. However, there is a lack of consensus
on which QoL data to use, for example, quality-adjusted life years
or equal value of life years gained.51
While it is important to target the broadest market access possi-
ble with the most favorable pricing, it is also important to define the
value of the medicine to each stakeholder. Medicinal product devel-
opment companies are now including real-world evidence (RWE)
tools in the product lifecycle to help achieve launch success and opti-
mal market access, positioning the right drug to the right patient.
In simplest terms, RWE is informing and supporting decision
making across the product lifecycle using analytics that provide real-
world knowledge on patients, different clinical phenotypes, and the
burden of disease, which was not possible in the past.52 RWE can
facilitate comparative effectiveness, help determine new product in-
sights, and differentiate products concerning broad-based outcomes
such as medication adherence, patient satisfaction, resource utiliza-
tion, and associated costs.52 These data are then used to inform the
target product profile, define product specific-attributes, inform
clinical trial design (right disease and patient population), and target
specific markets. Clinicians and patients are also using RWE to un-
derstand advancement in care, efficiency, and health outcomes.
Moving forward, RWE is a tool that is increasing in use across the
drug development continuum to maximize value and increase the
probability of market success.
Market access, including pricing and reimbursement, adver-
tising, and other factors that influence market success, are dis-
cussed in more detail in Chapters 35 and 36.
In parallel with market access, the MAA holder is responsible
for postmarketing regulations and lifecycle management of the ap-
proved medicine. The MAA holder must ensure product quality,
safety, and efficacy and mitigate any potential identified postmarket
References1
All references verified 12 February 2025.
1. Food and Drug Administration. The drug development process. Last updated
4 January 2018. https://www.fda.gov/patients/learn-about-drug-and-device-
approvals/drug-development-process
impact on patient health and safety through ongoing compliance
with cGMP regulations and guidance related to quality systems, in-
cluding product complaints. In addition, the MAA holder must
keep the application up to date regarding labeling, CMC changes
(e.g., manufacturing or formulation), and pharmacovigilance
(safety), including adverse event reporting. These activities are typi-
cally categorized as postauthorization activities, discussed in more
detail in Section 6: Postmarketing Authorization.
Conclusion
Medicinal product development is a continuum with specific steps
that are globally recognized. The process is long, complex, costly,
and made more challenging by the different country-specific re-
quirements. ICH continues to work on harmonization of regula-
tory requirements and guidelines beyond the founding ICH
regions, and emerging markets are adopting and implementing
standards such as the CTD and eCTD submission formats, ICH
E8(R1) on General Considerations for Clinical Studies and ICH
E6(R3) Good Clinical Practice, ICH S6 (R1) Preclinical Safety
Evaluation of Biotechnology-Derived Pharmaceuticals – Scientific
Guideline. CMC development is an integral part of the develop-
ment continuum that ensures the quality, consistency, safety, and
availability of the medicinal product.
Regulatory agencies recognize the need to expedite the au-
thorization and availability of medicinal products – especially for
serious, life-threatening, rare, and orphan indications – while
maintaining safety and effectiveness. Agencies such as the FDA,
EMA, and PMDA have worked to create and publish guidelines
and approaches to expedite development programs and nonstand-
ard review and authorization pathways to facilitate the availability
of new medicines. Agencies are encouraging early and frequent in-
teractions for novel medicines and new indications.
Marketing authorization is key to allowing medicines to be
sold in the regions authorized. In many countries, however, na-
tional pricing and reimbursement need to be determined and
agreed upon before the medicine is prescribed and patients can ac-
cess the medicine. Often, decisions also are made at the local, re-
gional, or hospital levels. Market success needs to take into
account pricing, reimbursement, and stakeholder (payer, physi-
cian, and patient) needs. Health technology assessments, health-
related quality of life metrics, and real-world evidence are playing
important roles in the product development continuum and pro-
vide input to clinical trial design, target product profile, and more,
helping to ensure the right drug is developed for the right patient
at the right price.
2. European Medicines Agency. From lab to patient: Journey of a medicine. Last
updated 10 February 2020. https://www.ema.europa.eu/en/about-us/what-we-
do/authorisation-medicines#from-lab-to-patient