Applied Health Economics and Health Policy (2023) 21:831–840

https://doi.org/10.1007/s40258-023-00816-6

CURRENT OPINION

Drug Repurposing of Generic Drugs: Challenges and the Potential Role

for Government
Karel H. van der Pol1 · Mohamad Aljofan2,3 · Olivier Blin4 · Jan H. Cornel5,6 · Gerard A. Rongen1 ·
Aurélie‑Gaëlle Woestelandt7 · Michael Spedding7

Accepted: 17 May 2023 / Published online: 3 July 2023

© The Author(s) 2023

Abstract
Drug repurposing is the process of identifying a new use for an existing drug or active substance in an indication outside the
scope of the original indication. Drug repurposing has important advantages including reduced development time and costs,
and potentially large societal healthcare cost savings. However, current generic drug repurposing research faces a number
of challenges in obtaining research funds. Furthermore, regardless of the success of a repurposing trial, commercial parties
often lack interest in pursuing marketing authorisation for financial reasons, and academic researchers lack the knowledge,
time and funding. Therefore, the new indication of a repurposed drug often does not make it ‘on label’. We propose a large
increase in public funding for generic drug repurposing research, including funds for the marketing authorisation process
when a trial is successful, and a reduction in the regulatory burden of the marketing authorisation process for repurposed
generic drugs.

Key Points for Decision Makers

Advances in generic drug repurposing are hampered by

lack of finances and legislative barriers.
Public funding and public–private partnerships for
generic drug repurposing should be increased.
Regulatory requirements for label extension of generic
drugs should be reduced.
* Gerard A. Rongen
Gerard.Rongen@radboudumc.nl
1
Department of Internal Medicine, Radboud Institute
for Health Sciences, Radboud University Medical Center,
Nijmegen, The Netherlands
2 1 Introduction
Department of Biomedical Science, Nazarbayev University
School of Medicine, Nur‑Sultan, Kazakhstan
3
The field of drug repurposing has been receiving increasing
National Laboratory Astana, Nazarbayev University,
Astana 010000, Kazakhstan attention over the past 5 years, especially since the corona-
4
virus disease 2019 (COVID-19) pandemic. A search on the
Institut de Neurosciences des Systèmes, Aix Marseille
Université, Inserm UMR 1106, Marseille, France term ‘drug repurposing’ on PubMed yields over 8000 results
5
in the past 5 years, of which 6000 have been published since
Department of Cardiology, Radboud Institute for Health
Sciences, Radboud University Medical Center, Nijmegen, 2020 [1]. The goal of drug repurposing is to use an existing
The Netherlands active substance, or medicine, for a new indication for which
6
Department of Cardiology, Northwest Clinics, Alkmaar, it does not have marketing authorisation. The European
The Netherlands Medicine Agency (EMA) and the Safe and Timely Access
7
Spedding Research Solutions SAS, 78110 Le Vesinet, France to Medicines for Patients (STAMP) working group define

Vol.:(0123456789)
832 K. H. van der Pol et al.

drug repurposing as: “The process of identifying a new use thus reducing societal healthcare costs. These lower costs
for an existing drug/active substance in an indication outside are of increasing importance in the light of ever-increasing
the scope of the original indication”. prices for new drugs, an issue discussed at length elsewhere
Drug repurposing can be subdivided into three main [35, 36]. Despite clear advantages, there are several barriers
categories: (i) repurposing of an active substance which faced by researchers, especially when the repurposed drug
has never previously received marketing authorisation; (ii) is a generic. These barriers need to be addressed to allow
repurposing of a medicine with marketing authorisation, and this growing field to fulfil its full potential. In this article
with remaining marketing protection in the form of a patent we will be focusing on the challenges faced when repurpos-
and/or data exclusivity; and (iii) repurposing of a medicine ing generic drugs, and their potential solutions, from the
with marketing authorisation for which all marketing pro- perspective of clinical pharmacologists and drug discovery
tection has expired (i.e. generic drug). New drug targets, or scientists working in academia.
targets shown to be critical in rare diseases, are routinely
screened with a chemical library of generic drugs, whether
the experiments are performed in academia or industry. 2 Financial Barriers to Drug Repurposing
An example is the ReFrame collection at Calibr at Scripps and Possible Solutions
[2]. Another approach is based on deep learning, and using
artificial intelligence to perform in silico screening. While One of the major challenges is the funding of repurposing
generic drugs may be used to merely validate further screen- trials, and the subsequent marketing authorisation process
ing for new chemical entities, they themselves may also be if a trial is successful. This is, on the one hand, due to lack
proposed for new therapeutic uses based on the screening. of interest from commercial parties and, on the other, lack
Examples of the therapeutic breadth of some of these pro- of public funding.
posals is shown in Table 1 [3–30]. However, nearly all of the
proposals remain untested in the clinic, many for the reasons 2.1 The Role of Commercial Parties, Marketing
outlined in this article. Protection and Pricing of Repurposed Generic
There are several advantages to drug repurposing of Drugs
generics compared with the development of new drugs.
The preclinical toxicology, pharmacokinetics, pharmaco- Generic drugs lack marketing protection, which leads to
dynamics and safety profile of the drug are usually well higher market competition. Thus, commercial parties are
known provided that dose, treatment duration and target often hesitant to invest in repurposing of generic drugs, as
population are comparable. Therefore, preclinical, phase I the chances of recuperating their investment are deemed low
and phase II studies are not required for (phase III) clinical [37]. Marketing protection comes in the form of patents (for
trials, thus reducing the chances of late attrition. However, generics usually ‘second medical use’ patents) issued by a
phase II studies are required when the dose–response curve patent office and data/marketing exclusivity granted by regu-
might be different in the new targeted indication/population latory authorities. While the EU offers 1 year, and the US 3
(e.g. paediatric population). Despite these advantages, as years, of market exclusivity for a repurposed generic drug,
we discuss below, the existing drug dossier may not satisfy provided certain conditions are met, these periods are too
registration criteria. short to recuperate investments costs [38, 39]. Additionally,
The importance of generic drugs has increased as the obtaining ‘second medical use’ patents for generic drugs
body of prescription drugs has aged, and patents have can be difficult, as the repurposed indication may not be
expired. The US Food and Drug Administration (FDA) novel due to already published literature [38, 40]. Further-
has set up an office of generic drugs, as 9 out of 10 drug more, enforcing a patent and/or marketing exclusivity of a
prescriptions are for generic drugs, saving an approximate repurposed generic may be difficult [38]. Another issue for
2.2 trillion USD between 2009 and 2019 [31]. The FDA commercial parties is that in many countries drug prices are
has issued 93 first-time generic drug approvals in 2021, as fixed at the national level, and they may not be allowed to
opposed to 50 new drug (i.e. new molecular entity), or use increase the price of a drug when they add a new indication,
approvals [32, 33]. Generally, while pharmaceutical com- especially when the drug is old and sold at a low price [37].
panies are willing to invest in the aforementioned first two This makes it harder to recoup their investment. For exam-
drug repurposing categories, they lack interest in the repur- ple, the repositioning and price negotiation of hydroxyurea
posing of generic drugs [34]. Research into the repurposing for the treatment of chronic myeloid leukaemia was chal-
of generic drugs is, therefore, often performed by academic lenging, and even involved the highest national legal court
researchers and non-profit organisations. Naturally, a repur- in France. All these factors make it difficult for academic
posed generic drug has lower developmental times and costs researchers to attract commercial partners. An example
that translate into a more inexpensive product for patients, of a successfully repurposed generic drug is viloxazine, a
Drug Repurposing of Generic Drugs 833

Table 1  Indications where generic drugs have been screened on potential targets and proposed for therapeutic use, without or with insufficient
clinical advancement
Potential use Drug name or class Original indication or use References

Acute coronary syndrome Calcium channel blocker Hypertension Shimada et al. [25]
Amyotrophic lateral sclerosis Ambroxol Mucolytic agent Bouscary et al. [5]
Alcohol use disorders Topiramate Epilepsy Burnette et al. [6]
Gabapentin Epilepsy
Varenicline Smoking cessation
Aripiprazole Schizophrenia
Ondansetron Nausea
Mifepristone Medical abortion
Ibudilast Asthma
Prazosin Hypertension
N-acetylcysteine Mucolytic agent
Suvorexant Insomnia
Alzheimer’s disease Bupivacaine Local anaesthetic Lee et al. [15]
Topiramate Epilepsy
Selegiline Depression
Iproniazid Depression
Skin infections Bumetanide Edema Palaniappan et al. [17]
Breast cancer Ferumoxytol Iron-deficiency anemia Sillerud et al. [26]
Chagas disease Levothyroxine Hypothyroidism Bellera et al. [4]
Clozapine-induced constipation Lubiprostone Constipation Torrico et al. [29]
Cocaine use disorders Zolmitriptan Migraine Garcia et al. [9]
Dermatological therapy Levothyroxine Hypothyroidism Paus et al. [18]
Epilepsy Losartan Hypertension Klein et al. [12]
Isoflurane Anaesthetic
Anakinra Immunosuppressive agent
N-acetylcysteine Mucolytic agent
Atorvastatin Hypercholesterolemia
Ceftriaxon Antibiotic
Sirolimus Immunosuppressive agent
Fingolimod Multiple sclerosis
Familial amyloid polyneuropathy Benzbromarone Gout Cotrina et al. [8]
Gaucher’s disease Ambroxol Mucolytic agent Kumar et al. [28]
Hematopoietic progenitor cell Transplan- Treprostinil Pulmonary hypertension Kazemi et al. [11]
tation
Invasive mycosis Erythromycin Antibiotic Rossi et al. [21]
Chenodiol Gallstones
Riluzole Amyotrophic lateral sclerosis
Nortriptyline Depression
Promazine Psychosis
Nisoldipine Hypertension
Suloctidil Arterial vasospasm
Ciclopirox Dermatomycosis
Auranofin Rheumatoid arthritis
Tamoxifen Infertility
Triclabendazole Fascioliasis
Leprosy Thalidomide Morning sickness Thangaraju et al. [27]
Minocycline Antibiotic
Apremilast Psoriasis
Tenidap Rheumatoid arthritis
834 K. H. van der Pol et al.

Table 1  (continued)
Potential use Drug name or class Original indication or use References

Lichen planopilaris Pioglitazone Type 2 diabetes mellitus Sanz et al. [24]

Tofacitinib Rheumatoid arthritis
Barcitinib Rheumatoid arthritis
Apremilast Psoriasis
Myasthenia gravis Ambroxol Mucolytic agent Cao et al. [7]
Myeloma Tofacitinib Rheumatoid arthritis Lam et al. [13]
Neurodegenerative disease Omarigliptin Type 2 diabetes mellitus Ayoub et al. [3]
Neuorinflammation Thalidomide Morning sickness Jung et al. [10]
Non-alcoholic fatty liverdisease Rosuvastatin Hypercholesterolemia Rotman et al., 2016 [22]
Pentoxifylline Peripheral arterial disease
Solithromycin Antibiotic
Primary biliary cholangitis Rituximab Lymphoma Ronca et al. [20]
Abatacept Rheumatoid arthritis
Ustekinumab Psoriasis
Fenofibrate Hypertriglyceridemia
Simtuzumab Fibrosis
Rattlesnake bites Batimastat Metastatic cancer Layfield et al. [14]
Marimastat Metastatic cancer
Refractory chronic cough Amitriptyline Depression Ryan et al. [23]
Gabapentin Epilepsy
Pregabalin Epilepsy
Morphine Analgesic
Tramadol Analgesic
Renal cell carcinoma Penfluridol Psychosis Tung et al. [30]
Metformin Type 2 diabetes mellitus Rausch et al. [19]
Zika virus Sunitinib Renal cell carcinoma Lin et al. [16]

selective noradrenaline uptake inhibitor, which was origi- marketing protection may be difficult if the generic drug is
nally approved and marketed for the treatment of depres- already on the market, especially if the drug is available
sion in Europe but was never approved or marketed in the at the same dose as would be used in the rare, or orphan,
USA [41]. When viloxazine was subsequently repurposed disease and in a cheap dose form, as it may still be bought
for the treatment of attention-deficit hyperactivity disorder, at the cheaper price. On the other hand, older drugs are not
it could therefore be classified as a new molecular entity in necessarily marketed around the world, so in some cases this
the USA (i.e. in the USA it was deemed a new drug instead problem can be avoided, as was the case with viloxazine.
of an existing drug with a new indication). This allowed for
pricing that may cover development costs. 2.3 The Role of Public Research Funding

2.2 Orphan Drug Status There also is a lack of research funding agency and/or gov-
ernmental research funding for larger drug repurposing tri-
Many drugs are repurposed for rare, or orphan, diseases, als and especially the subsequent marketing authorisation
where the patient population and potential market may be process, which represent an integral part of the drug repur-
small. The Innovative Medicines Initiative recently reviewed posing process. While there are successful publicly funded
this aspect [42]. Obtaining ‘orphan drug status’ from the projects, for example in cancer or COVID-19 research
EMA or FDA or in Japan can lead to 10 years of market pro- [43–45], most funding initiatives are aimed at earlier stage
tection, free regulatory advice for academic investigators and development and/or have insufficient funds for larger-scale
small companies, and potentially faster development. Thus, clinical trials [46, 47]. Another issue, especially in disease
this can be a way of protecting future sales to reimburse areas where pharmaceutical companies are performing well-
development costs. However, as mentioned earlier, enforcing funded trials testing patented new molecular entities, is that
Drug Repurposing of Generic Drugs 835

these well-funded trials compete with marginally funded [64]. Two of these trials were completely publicly funded
drug repurposing trials for participating sites. Therefore, [61, 62]. The other trial was funded by a large contribution
hospitals may be more reluctant to participate in drug repur- from a consortium of pharmaceutical companies and contri-
posing trials due to budgetary constraints. butions from several public sources [60]. These are examples
of successful academia-driven repurposing trials as well as
2.4 Examples of Repurposed Drugs: Successes a model for the potential of collaborative funding for such
and Failures projects. However, as of yet colchicine does not have market-
ing authorisation for the treatment of cardiovascular disease
A famous success for repurposing was the use of sildenafil despite one of the trials being co-sponsored by a consortium
for erectile dysfunction, when the drug was being devel- of pharmaceutical companies [60]. This is partly due to an
oped for cardiovascular disease [48]. Propranolol is used for earlier lack of interest from these companies to pursue mar-
shrinking infantile haemangiomas, based on serendipitous keting authorisation, as they were not allowed to increase
clinical observation, and is now a first-line therapy [49]. drug prices. Consequently, colchicine can only be prescribed
Similarly, sirolimus showed high efficacy in congenital ther- off-label for this indication at this time, which hampers its
apy-resistant low-flow vascular malformations [50]. More implementation in clinical practice. The legal responsibility
recently, Hutchinson–Gilford progeria syndrome has been of prescribers will not automatically be covered, for exam-
a target for repurposing. This rare disease, causing acceler- ple. This ultimately results in an unnecessary public health
ated ageing, was modelled by analysing the steps involved loss, which, at its core, is a governmental concern.
in the production or degradation of mutant progerin, key to
the disease [51]. Multiple agents were identified for repur- 2.5 Potential Solutions to Increase Funding
posing, and some have progressed to clinical trials, with for Generic Drug Repurposing
beneficial effects being reported for pravastatin, zoledronate
and lonafarnib [51]. While this approach in a clearly defined In our opinion, the largest stakeholder (i.e. the government)
rare disease may be hopeful, the small number of patients should bear the greatest financial burden, as it stands to gain
limits the number of clinical trials which can be performed. the most from the public health benefit. Therefore, govern-
Progressing such an approach to ageing itself is fraught with mental organisations should increase their funding for drug
difficulties. Agents such as metformin, rapamycin, aspirin, repurposing research and the implementation process of
resveratrol, statins, melatonin and antioxidants have as of repurposed drugs, which have shown benefit in clinical trials
yet not revealed remarkable efficacy, as trials would require and are proven to be cost-effective. In the European Union,
vast numbers of patients to counter the variables involved for example, a large dedicated drug repurposing funding pro-
in testing the aged population [52]. However, the very pre- gramme similar to ‘Horizon Europe’ and the ‘Computer-
cise decline in human performance in healthy individuals aided Drug Repurposing for Cancer Therapy Project’ could
indicates that ageing may be related to entropy and may be be created [46, 65]. Second, insurance providers, whether
difficult to treat [53]. public in the form of a governmental institution [e.g. the
Ambroxol is a safe mucolytic drug sold in 77 countries. National Health Service (NHS) in the UK], or privatized in
Ambroxol has been found to modify glucosylceramidase the form of insurance companies (e.g. as in the USA), are
activity by inhibiting the non-lysosomal enzyme, GBA2, an important stakeholder in the healthcare system. These
while acting as a chaperone for the lysosomal enzyme GBA1 providers may benefit directly, in the form of cost-savings,
[5, 54–56]. Consequently, the drug is proposed for testing if an inexpensive and cost-effective drug can be used for
in phase II for amyotrophic lateral sclerosis (ALS), and in the treatment of a disease that places a significant financial
phase III for Parkinson’s disease and Lewy body disease; burden on healthcare expenditure. Therefore, insurance pro-
however, funding has limited the extent of clinical trials [5, viders should be incentivized to fund, or increase funding
57, 58]. Fortunately, it was recently announced that Cure for, research on promising repurposed drugs in diseases that
Parkinson’s will co-fund a phase III trial of ambroxol in are costly to the healthcare system. Third, pharmaceutical
Parkinson’s disease [59]. companies should be encouraged to put more effort in the
Another example is the implementation of colchicine in assessment of potential business models for repurposed
the treatment of cardiovascular disease. Two large and one drugs on a case-by-case basis. For instance, some repur-
smaller randomised controlled trials showed the potency posed drugs may have a large target population, and there-
of colchicine in the prevention of cardiovascular events fore a potentially large yearly production volume providing a
[60–62]. In addition, colchicine reduced healthcare costs in good business model for generic pharmaceutical companies.
cost-effectiveness analyses [63]. This has led to the inclu- Indeed, this was the case for colchicine, and a consortium of
sion of colchicine in the most recent European Society of pharmaceutical companies are now pursuing label extension.
Cardiology guideline on cardiovascular disease prevention Academic researchers performing drug repurposing could
836 K. H. van der Pol et al.

play a role in convincing potential commercial parties. Oth- significant efforts. Discussions with regulatory bodies, and
ers suggested extension of market exclusivity for repurposed ethical committees, are critical, particularly if the pharmaceuti-
(generic) drugs, which will attract commercial parties or cal company which originally developed the compound is not
other funding strategies such as ‘social finance’ as a possible cooperative in releasing data.
solution for the lack of funding issue [38, 66].
3.2 Current Initiatives to Ease the Marketing
Authorisation Process
3 Regulatory Barriers to Drug Repurposing
and Possible Solutions This issue has not gone unnoticed, and the EMA has recently
launched a pilot project aimed at supporting academia and
Despite confirmation of clinical effectiveness in clinical tri- non-profit organisations to generate sufficient evidence on the
als, a repurposed drug must obtain marketing authorisation use of a repurposed generic drug with the aim of having the
from the regulatory authorities before the new indication new indication formally authorised by a regulatory authority
can be added to the label and fully implemented in clinical [67, 68]. This is a good initiative, but it does not, however,
practice. address the lack of funding issue. While the EMA (or national
competent authority) encourages the generation of a data pack-
3.1 Barriers to Obtaining Marketing Authorisation age, academics still have to secure their own funding to per-
form the necessary research. Furthermore, they still have to
Obtaining marketing authorisation is an important hurdle for fulfil the same strict regulatory requirements. Meanwhile, in
academic researchers, as the regulatory process is complex. the UK the NHS is developing a multifaceted strategy to sup-
While academic researchers have knowledge on how to per- port generic drug repurposing [69].
form high-quality clinical trials, they often lack detailed knowl-
edge of the authorisation process, time and other resources to 3.3 Potential Solutions for the Marketing
obtain regulatory approval. Furthermore, academic researchers Authorisation Process of Repurposed Generic
are usually not marketing authorisation holders, and often have Drugs
no interest in becoming marketing authorisation holders them-
selves. Commercial parties (i.e. pharmaceutical companies), We propose that the regulatory authorities simplify the mar-
which are marketing authorisation holders, often lack interest keting authorisation process for repurposed generic drugs. A
in pursuing authorisation. Currently, regulatory requirements potential solution is to provide the regulatory authorities with
to extend a label are not risk proportionate for generic drugs tools to adapt rules, originally developed for the first market-
which have been on the market for a long time. A critical factor ing authorisation of a drug, when they have to assess a new
is dose definition for safety and efficacy in the new indication, indication for a generic drug, provided that the safety profile
taking into account the publicly available data for a generic of the generic drug has been proven in clinical practice (by
drug. The available toxicology data may be very old, and bal- exposure of many thousands of patients) in the same dose
ancing safety based on clinical experience means being very as proposed for the new indication, and with no (or trivial)
careful about dosage increments. An unfortunate issue has manufacturing change. This would reduce the burden of the
arisen with the EMA in 2022, as they can now demand the marketing authorisation process both financially and in time
original toxicology reports for registration. This means that investment. Consequently, more lucrative opportunities for
the original pharmaceutical developer can negotiate a go/no-go commercial parties (or other parties) to pursue the market-
situation after academic institutions, or small to medium enter- ing authorisation process will be created. Interestingly in the
prises, have taken on board all the risks and financed the clini- UK, a NHS-owned pharmaceutical contract manufacturer is a
cal trials, even for very rare diseases where additional regula- marketing authorization holder for some drugs [69]. This, at
tory studies are prohibitively expensive to justify for the patient least in some countries, could be an interesting model to get
population. The original marketing authorisation holder might repurposed drugs on label if researchers are unable, or unwill-
also be reluctant to disclose a dossier that was regulatory com- ing, to become marketing authorization holders themselves.
pliant 40 years ago but that will encounter numerous gaps if
assessed using the current regulations. Furthermore, pharma-
covigilance data in other populations will possibly raise new
questions for their own marketing authorisation. In addition, it
might be tempting to keep the dossier just in case new oppor-
tunities occur. Finally, after the marketing authorisation has
been obtained, and the project leader moves to other activi-
ties/companies, retrieving all relevant documentation requires
Drug Repurposing of Generic Drugs 837

Table 2  Lipophilic drugs liable

to cause phospholipidosis Amiodarone Chlorprothixene Fluoxetine Opipramol Tamoxifen
which have been reported to Amodiaquine Clemastine Flupenthixol Promethazine Thioridazine
be active in reducing severe Astemizole Clomipramine Fluphenazine Propafenone Tilorone
acute respiratory syndrome Bix01294 Clomiphene Haloperidol Quinidine Trifluoperazine
coronavirus 2 (SARS-CoV-2)
replication in vitro (adapted Cepharanthine Desloratadine Loperamide Raloxifene Trimipramine
from Tummino et al. [71]) Chloroquine Ebastine Maprotiline Sertraline Triparanol
Chlorpromazine Fendiline Nortriptyline Spiperone

4 Other Topics in Generic Drug Repurposing cheaper drugs may have a big health impact, especially
for complex tropical diseases which may not be actively
4.1 Drug Repurposing During the COVID‑19 researched in high-income countries. The drug development
Pandemic process is very complex, frequently requiring resources that
only pharmaceutical companies can provide, and it is there-
The COVID-19 pandemic highlighted both the potential, fore difficult for RLCs to meet requirements when they are
as well as pitfalls, of generic drug repurposing. During the the main beneficiaries of such trials. This is a highly relevant
COVID-19 pandemic many generic drugs were claimed to societal debate, and it is the same situation for rare diseases
be useful for the therapy of COVID-19, but were in many where financial returns do not relate to potential therapeutic
cases proven to be inefficacious. There are 3050 PubMed value.
entries associated with the terms ‘repurpose’ and ‘COVID-
19’. Hydroxychloroquine, azithromycin, lopinavir/ritonavir 4.3 Post‑Marketing‑Authorisation Issues
and umifenovir were the most prescribed drugs worldwide
up until the end of 2020, but with very little, or no, thera- A final issue is that academic scientists usually do not have
peutic benefit [70]. Nearly all the drugs that were proposed any experience in sourcing, manufacturing, quality control
were lipophilic amphiphiles (Table 2) which can accumu- (CMC) and supply chain, as well as regulatory affairs. Fur-
late massively in cell membranes, and this can give posi- thermore, maintaining a pharmacovigilance department in
tive results in vitro which do not translate in the clinic [71]. compliance with regulations and assuring drug supply to
While ultimately not providing clinical success, the WHO- patients is a full-time job. This further limits the ability of
funded Solidarity trial is a good example of the value of academic scientists to successfully bring a repurposed drug
large, well-designed, drug repurposing trials [45]. This trial to market.
was a collaboration between many countries and research-
ers, and set out to provide high-quality randomized clinical
evidence on several drugs at a time when such evidence was 5 Conclusion
lacking [72]. Dexamethasone was proven to have clinical
benefit in the largely publicly funded RECOVERY trial [44]. In summary, the current generic drug repurposing research
These trials clearly demonstrate the value of drug repurpos- faces a number of challenges in obtaining research funds.
ing and should encourage similar large-scale collaborations Furthermore, regardless of the success of a repurposing trial,
and funding for drug repurposing trials in the future. Follow- commercial parties often lack interest in pursuing marketing
ing the RECOVERY trial, the EMA initiated a review by the authorisation for financial reasons, and academic researchers
Committee for Medicinal Products for Human Use (CHMP) lack the knowledge, time and funding. Therefore, the new
which defined its safe and effective use in COVID-19. This indication of a repurposed drug often does not make it ‘on
allowed generic companies to quickly add this indication to label’. We propose a large increase in public funding for
their label using the recommendation issued by the CHMP. generic drug repurposing research, including funds for the
Unfortunately, if such a mechanism were to be generalised, marketing authorisation process when a trial is successful,
there would be little incentive for private companies to per- and a reduction in the regulatory burden of the marketing
form clinical trials without full patent protection. authorisation process for repurposed generic drugs.

4.2 Tropical Diseases and Resource‑Limited Declarations

Countries Funding sources No specific funding was obtained for the writing of
this article.
One of the great therapeutic needs is for the development of
generic drugs in resource-limited countries (RLCs) where
838 K. H. van der Pol et al.

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