Minireview

Nephron Clin Pract 2009;113:c125–c131 Published online: August 12, 2009

DOI: 10.1159/000232592

Drug Development: From Concept to

Marketing!
Nihad A.M. Tamimi Peter Ellis
Pfizer Inc., Sandwich, UK

Key Words ty and efficacy in the intended patient population and its
Drug discovery ⴢ Clinical trials ⴢ Drug approval ⴢ Drug safety benefits must outweigh its risks before it will be approved
by the regulatory agencies. Strict regulatory standards gov-
ern the conduct of pre-clinical and clinical trials as well as the
Abstract manufacturing of pharmaceutical products. The assessment
Drug development is an expensive, long and high-risk busi- of the new medicinal product’s safety continues beyond the
ness taking 10–15 years and is associated with a high attri- initial drug approval through post-marketing monitoring of
tion rate. It is driven by medical need, disease prevalence adverse events. Copyright © 2009 S. Karger AG, Basel
and the likelihood of success. Drug candidate selection is an
iterative process between chemistry and biology, refining
the molecular properties until a compound suitable for ad-
vancing to man is found. Typically, about one in a thousand Introduction
synthesised compounds is ever selected for progression to
the clinic. Prior to administration to humans, the pharmacol- Getting drugs to the market is an expensive and high-
ogy and biochemistry of the drug is established using an risk business which takes on average 10–15 years to com-
extensive range of in vitro and in vivo test procedures. It is plete. The Tufts Center for the Study of Drug Develop-
also a regulatory requirement that the drug is administered ment announced in November 2001 that the average cost
to animals to assess its safety. Later-stage animal testing is to develop a new prescription drug was USD 802 million
also required to assess carcinogenicity and effects on the [1]. When the costs of failed prospective drugs are factored
reproductive system. Clinical phases of drug development in, the actual cost for discovering, developing and launch-
include phase I in healthy volunteers to assess primarily ing a single new drug would have exceeded 1.5 billion.
pharmacokinetics, safety and toleration, phase II in a cohort This compares with USD 4 million in 1962 and USD 231
of patients with the target disease to establish efficacy and million in 1987 [2, 3]. The problem is compounded by the
dose-response relationship and large-scale phase III studies high attrition rate, as it is estimated that approximately
to confirm safety and efficacy. Experience tells us that ap- only 1 in 10 drugs that enter clinical trials will make it to
proximately only 1 in 10 drugs that start the clinical phase the market. In a recent study, it was shown that the aver-
will make it to the market. Each drug must demonstrate safe- age success rate for drugs to be approved for all therapeu-
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© 2009 S. Karger AG, Basel Pfizer Laboratories

1660–2110/09/1133–0125$26.00/0 Ramsgate Road
Fax +41 61 306 12 34 Sandwich CT13 9NJ (UK)
Selçuk Universitesi

E-Mail [email protected] Accessible online at: Tel. +44 1304 641 627, Fax +44 1304 652 629
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 Pre- Approval
Discovery clinical Phase I Phase II Phase III and post-
approval

Fig. 1. Phases of drug development.

Hypertension Asthma Chronic kidney disease

High
Hyperlipidaemia Psychotic disorders Obesity
Arthritis Type II diabetes mellitus Malignancy
Stroke
Disease prevalence
Heart failure
Medium Gastro-oesophageal reflux Epilepsy Liver cirrhosis
disease Type I diabetes mellitus Chronic obstructive
pulmonary disease
AIDS

Cystic fibrosis
Inflammatory bowel Multiple sclerosis
Low

Genetic storage diseases disease Septic shock

Irritable bowel syndrome Transplant rejection

Low Medium High

Fig. 2. Drivers for discovering new drugs Medical need

with examples.

tic areas is approximately 11%. The success rate varies be- covers only 27% of new drugs whilst the reality is that
tween therapeutic areas ranging from 20% for cardiovas- more than 90% of all new drugs are discovered by the in-
cular drugs to only 5–8% for oncology and central nervous dustry [5].
system disorder drugs [4]. Improvements in predicting This minireview will address the process of drug de-
the potential success or failure of a product in clinical tri- velopment from discovery through the stages of develop-
als is essential to aid in reducing the spiralling devel- ment up to approval and marketing (fig. 1).
opment costs. Unfortunately, increasing costs combined
with the high attrition rate are forcing pharmaceutical
companies to reduce investment in research and develop- Discovery
ment, focussing on a more limited product portfolio.
Drug development is a significant challenge. Every Selecting therapeutic areas or indications to invest in
product must not only be safe and efficacious, but its ef- is driven by ‘medical need’ and the prevalence of the dis-
ficacy has also to be proven across racial and ethnic ease (fig. 2). Additional factors also include technical fea-
groups as well as across different age groups. Every drug sibility, research and development costs and commercial
has to pass a global regulatory review in what is current- considerations such as competition in the market place
ly the most regulated industry in the world. Once this is and potential market share. Even if these criteria are met,
done, approved products must appeal to global markets there is only a limited research and development budget
across different cultures, healthcare systems and distri- and each new project must be prioritized against the
bution systems. company research and development portfolio, with only
It is interesting to note that in a recent survey, the pub- high priority projects within the budget being selected for
lic perception was that the pharmaceutical industry dis- progression. For many companies, this is typically an an-
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nual review process for products at all stages of develop- ent expiry date. The patent life is typically 25 years but as
ment. This may lead to stopping a programme even at an it takes 10–15 years to develop a drug, there could only be
advanced stage of development. 10 years remaining to sell the product and recoup the
Early chemical starting points have been identified high development costs.
from naturally occurring substances in plants, humans or
animals but lead compounds are more often sourced from
targeted chemical synthesis directed to bind to the known Phases of Clinical Drug Development
structures of receptors and enzymes or from random or
receptor-targeted high-throughput screening [6]. This has Phase I. Phase I starts with the first administration of
become more popular in the last few years as it is helpful the new medicinal product to humans. Usually this phase
in accelerating drug discovery. Initial problems encoun- involves healthy volunteers with the exception of cyto-
tered in the last decade have eased with improving tech- toxic drugs (e.g. oncology drugs) which get tested in pa-
nology. With the advent of modern computer technology, tients without the requirement to test in healthy volun-
robotics and multi-well assay plates (384 growing to 1,536 teers first. The purpose of this stage is to evaluate the safe-
wells per plate), high-throughput screening can test vast ty, tolerability, pharmacodynamic (effect of the drug on
‘libraries’ of chemical compounds in multiple screens the body e.g. effect on heart rate, blood pressure, electro-
(which can deliver up to 120,000 assays every 24 h). An- cardiogram (ECG), etc.) and pharmacokinetic (effect of
other method of lead identification is ‘virtual screening’ the body on the drug i.e. absorption, distribution, metab-
(also named in silico screening) which is defined as the olism and excretion) effects of the tested drug. Phase I
‘selection of compounds by evaluating their desirability in studies are usually conducted in dedicated phase I units
a computational model’ [7]. Compounds testing positive which are research units attached to a general or teaching
in screening have their potency and selectivity confirmed hospital and manned by research physicians who are fa-
by in vitro biochemical or cellular assays. This is typi- miliar with conducting such studies. Full resuscitation fa-
cally followed by functional biochemical and pharmaco- cilities are available at these units. Phase I studies require
logical testing in vitro, followed by pharmacodynamic approval from an ethics committee and the relevant regu-
and pharmacokinetic testing in vitro and in vivo [8]. The latory agency. In the United States, an Investigational
next step is to complete pilot toxicology testing to inform New Drug (IND) application, which summarises the es-
us of the likely safety profile. Once all preclinical testing tablished preclinical and manufacturing information
has satisfied the minimum selection criteria, the com- along with investigator guidance, must be in place prior
pound transitions from a ‘lead’ to a ‘candidate’ and is to starting clinical trials. A pre-IND consultation pro-
nominated for progression to the clinic. gramme is offered by the US Food and Drug Administra-
At this stage, drug production is scaled up to meet the tion (FDA) to provide guidance on the data necessary for
increased compound demand, work commences on de- the IND submission. Subjects are usually compensated for
veloping a suitable formulation for clinical use (often a participating in these studies. Development of the drug
tablet is the preferred dosage form) and the candidate is could be stopped if it is found that the half-life of the drug
progressed through the required toxicology testing (in- is too short or too long or if it has poor bioavailability.
cluding genotoxicity, safety pharmacology in all biologi- Similarly, if the drug is not well tolerated at effective con-
cal systems, single and multiple dose toxicity and toxico- centrations it is dropped from development. Phase I stud-
kinetic studies) to enable the first in human and subse- ies usually start with single sub-pharmacological doses
quent clinical studies. Reproductive toxicology in male which are escalated gradually and followed by multiple
and female animals (required prior to testing in women doses. Stopping rules to dose escalation include severe ad-
of child-bearing potential) and long-term carcinogenici- verse events, clinically significant ECG abnormalities and
ty testing are also prerequisites for filing a drug approval clinically significant laboratory abnormalities.
request [9]. Other phase I studies to support drug development are
In parallel with lead development/candidate nomina- conducted throughout phase II and II of development.
tion, a key decision on when to patent the compound or These include drug-drug interaction studies, effect of
chemical series is taken. Early patenting mitigates against food on absorption, age and genetic influences. A typical
competitors beating a company to a claim, but delaying phase I study can cost up to USD 500,000, with speciality
the patent application allows for introduction of addi- studies (such as detailed QTc ECG assessments) costing
tional data to strengthen the patent and extends the pat- up to USD 1.5 million.
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 Role of Translational Medicine/Biomarkers called the ‘proof of concept’. Phase IIb follows on from
The American Physiological Society has defined trans- the proof of concept in which several dose levels are test-
lational research as ‘the transfer of knowledge gained ed in the target population (dose-ranging studies) to de-
from basic research to new and improved methods of pre- fine the minimally effective or non-effective dose and to
venting, diagnosing, or treating disease, as well as the decide the optimal dose, based on clinical efficacy and
transfer of clinical insights into hypotheses that can be safety, to take to the next stage. Occasionally phases IIa
tested and validated in the basic research laboratory’ [10]. and IIb are combined in one large study. A complete
Biomarkers are quantitative measures of biological ef- phase II programme could involve several hundred pa-
fects that provide informative links between mechanisms tients and can cost several million dollars.
of action and clinical effectiveness [11]. Effectively apply- With ever increasing development costs and expiry of
ing translational research measures to a development valuable patents on major products, the pharmaceutical
programme in phase I and phase II results in earlier iden- industry is compelled to develop more efficient and cost-
tification of efficacy (or just as important, lack of effica- effective ways of doing drug development. These include
cy) resulting in increased overall productivity and poten- the use of biomarkers, as discussed, but also application
tially a quicker route to drug approval. There are 3 fun- of enhanced quantitative drug design ‘EQDD’ to under-
damental classifications of biomarkers: (1) markers of stand exposure-response relationships and optimise dose
disease e.g. proteinuria as a biomarker of chronic kidney selection, thus facilitating regulatory review and maxi-
disease (CKD); (2) markers of pharmacological activity of mising the commercial value of the drug.
a drug e.g. inhibition of angiotensin-converting enzyme However, positive phase II data is no guarantee of pro-
increases plasma levels of angiotensin-1 and decreases gression to phase III. At this key stage of development,
plasma levels of angiotensin-2; (3) surrogate biomarkers costs will increase significantly and detailed analyses of
of efficacy e.g. using a measure of penile rigidity mea- the drug candidate and the market (patient, payer and
sured by plethysmography (Rigiscan) as a surrogate for physician perspectives) are conducted. This will include
sexual intercourse. An example of a biomarker with di- drug efficacy relative to the competitors, safety profile,
agnostic rather than efficacy potential is neutrophil gela- probability of technical and regulatory success, remain-
tinase-associated lipocalin or NGAL, which serves as ing patent life of the drug, cost of goods to produce the
biomarker of acute renal injury as increased levels are de- drug, potential market share and pricing and reimburse-
tected in urine and blood within hours of kidney injury. ment. Once again, the drug will be prioritised against all
Taking the example of proteinuria in CKD, interven- other candidates in the portfolio and only if the outlook
tions that reduce proteinuria can be potentially beneficial is favourable and the priority is within the research and
in the treatment of CKD. Therefore measuring changes development budget will it go forward.
in the biomarker in both preclinical models (e.g. sub-total A successful phase II is followed by an ‘end of phase II’
nephrectomy model in the rat) and the clinic can be in- meeting with regulatory agencies such as the FDA to dis-
dicative of activity of a potentially new drug for treating cuss the results from phase II and discuss and agree the
that indication (i.e. slowing progression of non-diabetic clinical and statistical analysis plans for phase III. This
CKD). The challenge is to use or develop a biomarker in negotiation, which also includes the target labelling, is
which we have confidence that it will reflect changes in critical to ensure alignment between the regulatory agen-
the important registrable endpoints that we will assess in cy and sponsor.
phase III trials and which are essential to gain regulatory Phase III. This is the final stage of drug development
approval. prior to registration and will confirm the clinical doses,
Phase II. Once the drug’s safety, pharmacokinetics and frequency and timing of administration for approval. Be-
dose selection has been established in healthy volunteers, fore embarking on a costly phase III programme, the
the next step is to investigate the efficacy and safety of the sponsor should have a high level of confidence in the
drug in the target population. For example, if a drug is drug’s safety and efficacy in the target patient population
being developed for the treatment of hypertension, phase and the dose range to be tested. Phase III trials (usually a
II trials will involve investigating the drug in a hyperten- minimum of 2) can involve up to several thousands of
sive patient population. Phase II is usually divided into patients, depending on the indication, so that an ade-
phase IIa and phase IIb. Phase IIa is when the drug (usu- quate database (with 90% power to detect statistically sig-
ally limited to a single high/maximal tolerated dose level) nificant differences) is created to assess the efficacy and
is tested in a small cohort (12–100) of patients; this is safety profile, in addition to enabling accurate drug label-
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ling. Phase III trials are primarily designed and powered if the rate is too high, additional study centres will be re-
to test the hypothesis of efficacy but at the same time, ad- cruited. This ensures adequate patient numbers for ap-
verse events are collected to assess benefit-risk potential proval, but is costly in incurring delays to the programme.
of the drug. Use of novel endpoints in phase III is a high- The overall success rate of phase III is around 70% and
risk strategy, but can prove valuable in demonstrating depending on the size can cost up to USD 100 million. A
benefits relative to competitors or established therapies; successful phase III is usually recognised by the financial
however, these endpoints do require validation and markets with an impact on the sponsor’s share price.
should be included in phase II and discussed with the
regulatory authorities prior to the start of phase III.
Clinical studies that use mortality and morbidity end- Regulatory Submission/Approval
points are often very large and can take several years to
complete. Oncology is an exception, with phase III stud- Once the phase III studies have completed and deliv-
ies often limited to a few hundred patients. In diseases in ered a positive outcome, compilation of the data to sub-
which there is an established ‘gold standard’ treatment, mit to the regulatory agencies starts. This usually takes
European regulatory authorities will require phase III several months and can be done by one region at a time,
studies to include a comparator arm to demonstrate non- e.g. in the United States, or could be done globally, target-
inferiority or superiority compared to the standard ther- ing major regions simultaneously. Classically, the major
apy. Efficacy can be demonstrated either by demonstrat- markets include the United States, the European Union
ing superiority to placebo in placebo-controlled trials or and Japan. However, recently more attention is given to
by showing superiority to an active-control treatment. the ‘emerging markets’ such as Latin America, India and
Sometimes the new drug entity is compared to a reference China, amongst others. As for the United States, a routine
treatment without the objective of showing superiority. New Drug Application ‘NDA’ can take up to 15 months
This can be either an equivalence trial, which shows that for review. However, in cases of particularly high medical
the response to treatments differs by an amount which is need or in areas lacking treatments (e.g. oncology and hu-
clinically not significant (specify upper and lower equiv- man immunodeficiency virus), an expedited review can
alence margins), or a non-inferiority trial which has the be granted. If the new drug is a biologic, then a biologic
objective of showing that the new drug is not clinically license application ‘BLA’ rather than a ‘NDA’, is submit-
inferior to the comparator (only lower equivalence mar- ted.
gin is specified). The choice of specified margins should In Europe, the sponsor submits a marketing authori-
be clinically justified. sation application (MAA), which could be granted either
Depending on the nature of the study and the end- under the centralised procedure (valid for the entire com-
points used for the indication, a ‘Data Safety Monitoring munity market) or through the mutual recognition pro-
Board’ (DSMB) may be required throughout the conduct cess.
of the trial. This is especially so in studies that incorpo- During the review by the regulatory agencies, ques-
rate mortality and morbidity as primary or secondary tions are referred back to the sponsor. To facilitate the
endpoints. DSMB members must include a clinician with review process, the sponsor will typically establish a rap-
expertise in the disease area under investigation as well id response team to coordinate the responses to the au-
as a biostatistician as a minimum. Each DSMB must have thority. Drug label negotiations take place during the re-
a charter and written operating procedures detailing view process. Regulatory agencies could request post-ap-
members’ responsibilities and the plan of communica- proval studies from the drug companies to address any
tion. DSMB members must disclose potential conflict of safety concerns that the regulatory agencies may have. At
interest to the sponsor. the same time, the drug company will have presented its
For the sponsor, phase III trials involve a large cross- plans to detect, assess and report adverse events.
functional team which involves, amongst others, clini- Pharmacovigilance is the term used in Europe de-
cians, project management, data management, drug safe- scribing the ongoing evaluation of the safety of the drug
ty monitoring, document management, regulatory sup- in the post-marketing period; it is a requirement that all
port and clinical quality assurance. A key consideration pharmaceutical companies with a post marketed product
for phase III is selection of study centres to ensure appro- must comply. The drug company will also provide peri-
priate patient recruitment and timely completion of the odic safety update reports on the new drug after its ap-
study. Estimations of patient drop-out rates are made, but proval. Post-marketing or safety surveillance trials are
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sometimes referred to as phase IV clinical trials. Harmful sus immediate release, combinations with other drugs for
effects discovered during phase IV trials can lead to the improved efficacy, as well as seeking new indications.
withdrawal of the drug from the market as seen in the Once a new indication is confirmed, the drug company
example of rofecoxib (Vioxx) and cerivastatin (Lipobay, can apply for a supplementary new drug application (s-
also known as Baycol in the United States). NDA). Publication strategies are also another important
Orphan Drug Status. Pharmaceutical products devel- part of lifecycle management, as additional benefits of the
oped to treat rare diseases have been referred to as orphan drug that cannot be added to the label, such as patient-
drugs. The FDA Orphan Drug Act specifies the require- reported outcome measures, are published in peer-re-
ments for granting a drug orphan status. The disease that viewed journals.
the drug is intended for should affect less than 200,000
people in the United States. This designation grants the
company fast-track review process as well as market ex- Interaction between Pharmaceutical Industry and
clusivity for a period of 7 years. In addition, it will be eli- Healthcare Professionals
gible for direct guidance from the FDA for the design of
a clinical plan to further develop the drug. In Europe, The Pharmaceutical Research and Manufacturers of
some drugs used to treat tropical diseases that are pri- America (PhRMA) represent research-based pharma-
marily found in developing countries can also be desig- ceutical and biotechnology companies. PhRMA have de-
nated as orphan drugs. For the drug companies, the cost veloped guidelines on the basis of interactions between
of developing such drugs and marketing them will not be US healthcare professionals and the pharmaceutical in-
covered by the expected sales. Hence, the economic and dustry. The PhRMA code was last updated in January
regulatory incentives to encourage pharmaceutical com- 2009 and regulates amongst other things: informational
panies to develop such drugs are needed. presentations by pharmaceutical company representa-
tives and accompanying meals, prohibition on entertain-
ment and recreation, pharmaceutical company support
Regulatory Standards for continuing medical education, pharmaceutical com-
pany support for third-party educational or professional
Preclinical studies are conducted according to good meetings, the employment of healthcare professionals as
laboratory practice (GLP) guidelines, which regulate how consultants, speaker programmes and speaker training
laboratory studies are performed. Clinical trials are con- meetings, prohibition of non-educational and practice-
ducted according to good clinical practice (GCP) guide- related items as well as scholarships and educational
lines, which are internationally required quality and safe- funds. In the United Kingdom, the Association of the
ty standards for designing, conducting and reporting British Pharmaceutical Industry (ABPI) code was estab-
clinical trials. GCP-compliant clinical trials are essential lished in 1958 and covers advertising, activities of repre-
to ensure the rights and safety of clinical trial subjects. sentatives, supply of samples, provision of hospitality,
These standards are subject to inspection by regulatory promotional meetings and the sponsorship of scientific
agencies at any time; regulatory agencies have the right to and other meetings, including payment of travelling and
halt ongoing clinical studies if they have concerns that accommodation expenses. The ABPI code does not apply
the studies are not GCP-compliant. Finally drug manu- to the promotion of over-the-counter medicines to the
facturing is done according to good manufacturing prac- general public [12].
tice (GMP) guidelines, which dictates the standards for
manufacturing and quality control of pharmaceutical
products. This is also subject to regulatory inspection. Conclusion

Drug development is a long, expensive and highly reg-

Lifecycle Management ulated process. The risks are high, but continued invest-
ment in pharmaceuticals is vital if we are to enjoy the
The drug company will plan the lifecycle of the drug benefits of long-term improvements in patient health-
throughout the patent life and beyond into the future ge- care.
neric marketplace. This may include different drug deliv-
ery systems such as prolonged release formulations ver-
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 References

1 Tufts Center for the Study of Drug Develop- 5 Protección de la propiedad intelectual para 8 Rester U: From virtuality to reality – virtual
ment pegs cost of a new prescription medi- productos farmacéuticos: qué es y por qué es screening in lead discovery and lead optimi-
cine at $802 million. 2001. http://csdd.tufts. esencial para la innovación en salud? Con- zation: a medicinal chemistry perspective.
edu/NewsEvents/RecentNews.asp?newsid=6 sideraciones a la luz del DR-CAFTA. http:// Curr Opin Drug Discov Devel 2008;11:559–
(accessed January 2, 2009). www.amchamsal.com/uploaded/content/ 568.
2 Drug development: the short story 7. Cost category/2000065261.pdf (accessed January 9 Tweats DJ, Scales MDC: Toxicity testing; in
of drug development. Network Science Cor- 2, 2009). Griffin JP and O’Grady J (eds): The Textbook
poration. http://www.netsci.org/scgi-bin/ 6 Dutta A: Discovery of new medicines; in of Pharmaceutical Medicine. London, BMJ
Courseware/projector.pl?Course_num=cour- Griffin JP and O’Grady J (eds): The Textbook Books, 2002, p 134.
se1&Filename=slide07.html (accessed Janu- of Pharmaceutical Medicine. London, BMJ 10 Hall JE: The promise of translational physi-
ary 2, 2009). Books, 2002, p 25. ology. Am J Physiol 2002;283:1235–1236.
3 Drugresearch.com: drug development costs 7 International Union of Pure and Applied 11 Biomarkers Definitions Working Group:
hit $1.7 billion. 2003. http://www.drugre- Chemistry: Glossary of terms used in com- Biomarkers and surrogate endpoints: pre-
searcher.com/Research-management/Drug- binatorial chemistry, U-Z3. Research Trian- ferred definitions and conceptual frame-
development-costs-hit-1.7-billion (accessed gle Park, International Union of Pure and work. Clin Pharmacol Ther 2001;69:89–95.
January 2, 2009). Applied Chemistry. 1999. http://www.iupac. 12 What is the code of practice. http://www.
4 Kola I, Landis J: Can the pharmaceutical in- org/reports/1999/7112maclean/u-z.html pmcpa.org.uk/?q=whatisthecodeofpractice
dustry reduce attrition rates? Nat Rev Drug (accessed January 2, 2009). (accessed March 29, 2009).
Discov 2004;3:711–715.

Editorial Comment
M. El Nahas, Sheffield

This minireview by Tamimi and Ellis, 2 senior execu- ly, investments in lengthy clinical trials addressing chron-
tives at Pfizer UK with considerable experience in drug ic diseases such as CKD may also fall victim to the credit
development and with a genuine interest in nephrology crunch. This editor knows of more than one clinical trial
and chronic kidney disease (CKD), is timely. It reminds that has been cancelled or stopped as the sponsors’ finan-
the reader of the huge and often prohibitive cost of new cial situation worsened with the global credit crunch. Fi-
drug developments. It highlights the fact that thousands nally, drug development may itself be shelved for a more
of new potentially promising products never make it to cost-effective approach consisting of acquiring generic
the bedside. Giving the cost associated with drug develop- drug makers. Over the last few weeks alone, Novartis pur-
ment and the current global financial situation, this mini- chased Ebewe Pharma an Austrian maker of generic can-
review sheds considerable light on the direction major cer drugs for USD 1.3 billion, Pfizer agreed a licencing
pharmaceutical companies may be taking. First, the drug deal with 2 Indian generic makers and GlaxoSmithKline
industry is re-evaluating its research priorities moving to- acquired a stake in South Africa’s Aspen. Such an ap-
wards safer clinical areas with projected quicker financial proach may be the way forward for the drug industry to
return. Pfizer has moved away from its prior top research reach a sustainable business model. It may also offer the
priorities and successful drug development areas, namely industry an opportunity to unlock emerging markets that
atherosclerosis and heart failure research. Instead, re- may account for 70% of new pharmaceutical sales by 2020.
search and development of drugs to tackle the growing Drugs development and clinical trials have not been
market of Alzheimer’s disease are gathering pace. Second- spared the ravages of the credit crunch.
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