Regulation Of Clinical Trials Research Articles

Call for More Effective Regulation of Clinical Trials with Advanced Therapy Medicinal Products Consisting of or Containing Genetically Modified Organisms in the European Union.

Advanced therapy medicinal products (ATMPs), such as gene therapies that consist of or contain genetically modified organisms (GMOs) need to comply with the European Union (EU) GMO legislation, as implemented in each EU Member State, before a clinical trial can commence. Complying with GMO requirements is complex, varies significantly across EU Member States and is leading to delays to clinical trials with ATMPs. Such delays and varying implementation of the GMO legislation makes the EU less attractive as a region to conduct clinical trials with investigational gene therapies. This is detrimental to EU patients, since their timely access to these transformative potentially curative medicines is delayed. Despite recent initiatives coordinated by the European Commission (EC) to facilitate and reduce discrepancies across the EU regarding the application of the GMO requirements, it remains particularly difficult to conduct multicenter clinical trials with ATMPs containing or consisting of GMOs involving several EU Member States. The recent decision for the EC to temporarily derogate potential coronavirus disease 2019 treatments and vaccines from some provisions of the GMO requirements was made on the basis of a clear recognition of such complexities and resulting delays to clinical development. The Alliance for Regenerative Medicine, the European Federation of Pharmaceutical Industries and Associations, and the European Association for Bioindustries call upon the EC, together with national competent authorities, to exempt ATMPs containing or consisting of GMOs from the GMO legislation. Such a simplification will eliminate the delays currently reported to occur when submitting environmental risk assessments and GMO applications to the national competent authorities. An exemption from GMO requirements will make the EU a more attractive region for clinical development of gene therapies and could accelerate European patients' access to these potentially life-saving medicines. Maintaining a system for GMO assessment that is different across countries may also prevent ATMPs from realizing the full benefits of a harmonized clinical trial approval process under the Clinical Trials Regulation. The undersigned organizations to this publication urge the EC to use its right of initiative to put forward a legislative proposal to exempt ATMPs in clinical development from the EU GMO legislation, within the timeframe proposed in the 2020 EU Pharmaceutical Strategy (by 2022). Implementation of a GMO exemption scheme before the end of the transition period for the Clinical Trial Regulation (the end of 2023) is important to avoid new Clinical Trial Application submissions for ATMPs under the Clinical Trial Regulation having to conduct the whole GMO assessment process in parallel. It is considered that ATMPs pose negligible risk to the environment. Such ATMPs include the following: human somatic cells modified ex vivo; recombinant virus-based vectors, including those containing genome editing nucleic acid sequences (which may also be delivered nonvirally); and bacterial vectors. Outside of controlled storage conditions, gene therapies cannot survive for any appreciable length of time. Upon clinical administration, any recombinant gene therapy viral vector particles that do not enter host cells are diluted within the body and if excreted are in such low multiplicity to no longer be viable or considered infectious to persons, animals, or living organisms within the environment. Any nucleic acids released into the environment are rapidly degraded.

From sandbox to pandemic: Agile reform of Canadian drug regulation

Public health urgency for emerging COVID-19 treatments and vaccines challenges regulators worldwide to ensure safety and efficacy while expediting approval. In Canada, legislative amendments by 2019 Omnibus Bill C-97 created a new "agile" licensing framework known as the "Advanced Therapeutic Pathway" (ATPathway) and modernized the regulation of clinical trials of drugs, vaccines, and medical devices. Bill C-97′s amendments are worthy of attention in Canada and globally, as health product regulation bends to COVID-19. The amendments follow reforms elsewhere to accommodate health product innovation, however, the Canadian ATPathway is broader and more flexible than its counterparts in other jurisdictions. In addition, Bill C-97 informed Canada's COVID-19 response in important ways, particularly in relation to clinical trials. The measures adopted by the drug regulatory authority, Health Canada (HC) during COVID-19 may become the new norm in Canadian regulatory practice insofar as they help achieve the amendments introduced by Bill C-97. Finally, despite government rhetoric of transparency, the agenda-setting, formulation, and implementation of the amendments have occurred with little opportunity for scrutiny or public engagement.

A deep learning-based approach for simultaneously segmenting T2 lesions and T1 hypointensities in multiple sclerosis regulatory clinical trials (4379)

A deep learning-based approach for simultaneously segmenting T2 lesions and T1 hypointensities in multiple sclerosis regulatory clinical trials (4379)

A Novel Fluorescent c-Met Targeted Imaging Agent for Intra-Operative Colonic Tumour Mapping: Translation from the Laboratory into a Clinical Trial

Background: Curative resection of colon cancer requires accurate intraoperative identification of tumour margins and lymph node (LN) burden. The c-Met protein is important for cell differentiation and migration and is overexpressed in many gastrointestinal cancers. We explored EMI-137, a novel c-Met targeting fluorescent probe, for application in fluorescence-guided colon surgery, in HT-29 colorectal cancer (CRC) cell line and an in vivo murine model. This informed a first-in-man clinical trial of EMI-137. Methods: HT-29 cells were used as a representative CRC model. SiRNA was used to demonstrate the specificity of EMI-137 for c-Met. A HT-29 CRC xenograft model was developed in female BALB/c mice. Tumours were cultured for 14 days to 10mm diameter. EMI-137 (0.18mg/kg) was injected into tail veins and biodistribution analysed through in vivo fluorescent imaging. Nine patients, aged 67-77 years, received a single intravenous dose of EMI-137 (0.13mg/kg), 1-3 hours before laparoscopic-assisted colon cancer resection surgery (NCT03360461). Tumour and LN fluorescence was assessed with a modified Karl Storz® laparoscope. Immunohistochemistry with a monoclonal antibody analysed c-Met expression in eight resected samples. Findings: c-Met expression in HT-29 cells was effectively silenced and imaged with EMI-137. Strong EMI-137 uptake in tumour xenografts was observed for up to 6 hours post-administration. At clinical trial, no serious adverse events related to EMI-137 were reported. Marked background fluorescence was observed in all trial participants, 4/9 showed an increase in tumour fluorescence over background; 5/9 had histological LN metastases; no fluorescent LN were detected intraoperatively. All primary tumours (8/8) and malignant LN (15/15) exhibited moderate to high c-Met protein expression. Interpretation: EMI-137, binds specifically to the human c-Met transmembrane protein, is safe in patients and with further refinement, shows potential for long-term application in fluorescence-guided colon surgery. Trial Registration: The trial was registered with ClinicalTrials.gov identifier NCT03360461. Funding: Research was supported by internal University of Leeds funding. Declaration of Interests: The authors declare no conflict of interest. Ethics Approval Statement: Ethical approval was granted by the Yorkshire and Humber Leeds West Research and Ethics Committee (Ref 17/YH/0263), on 19th October 2017 and Regulatory approval was granted by the Medicines and Healthcare products Regulatory Agency (MHRA), under The Medicines for Human Use (Clinical Trials) regulations on 29th August 2017.

Assessing the impact on pharmacists’ time by introducing a technician screening process for clinical trial prescriptions

IntroductionVarious national guidance from the Lord Carter 2016 report to the NHS Long term plan have emphasised the need to transform traditional hospital pharmacy and make work streams more efficient.[1] A clinical trials pharmacist has historically validated clinical trial medicines. Whilst this is good practice for non-chemotherapy prescriptions, it is not a requirement of the Clinical Trial Regulations.[2] Interruption to validate trial prescriptions can have a negative impact on pharmacists’ duty and consequently patient outcomes. With limited data available, this issue has been highlighted by anecdotal evidence. Due to the often complex requirements associated with trials, the research team are responsible for assessing the suitability of treatment. This includes checking interactions with concomitant medication, reviewing blood results and patient counselling. The clinical aspect of the pharmacist validation is therefore removed, allowing technicians to be involved in the screening of suitable prescriptions. Much is written on technicians extending their roles in the clinical setting, but this service improvement focuses on enhancing their role within the pharmacy clinical trials department.AimTo evaluate the amount of pharmacists’ time saved by the introduction of technician screening of clinical trial prescriptions.MethodA risk-based proforma was created and used by a pharmacist to assess clinical trial prescriptions for the suitability of screening by a Band 7 technician. Only prescriptions with pre-printed doses, no aseptic preparation or additional medicines, were approved for technician screening. The process of screening therefore only involves the checking of patient and prescriber details, allergy status and possibly a medication randomisation. The technicians under-went an in-house training including the screening of prescriptions under pharmacist supervision. A quantitative data collection tool was used to review the screening / validation of all nonchemotherapy clinical trial prescriptions received at two sites over a two-week period in September 2020. The data collection tool was piloted and all data was analysed using Microsoft Excel.ResultsA total of 89 prescriptions were received. 56 (63%) were eligible for technician screening, of which a suitable technician validated 50%.Across both sites a total time of 360 minutes were spent validating/screening prescriptions including solving prescription related issues. Combining the time taken by a pharmacist to return from a clinical area and screening time consequently saved a total of 227 minutes of pharmacists’ time.ConclusionDistributing the workload amongst trained staff saves pharmacist’s time, which can be utilised on clinical and complex tasks. This does not eliminate the requirement of a pharmacist to validate prescriptions however; it reduces the frequency and streamlines the service. Further data collection is required to analyse the direct impact on patients’ and any changes in the number of reported errors. A limitation to the study is the lack of data prior to implementation as a comparator. Additionally, during data collection there were no suitable technicians available at one site due to the Covid-19 pandemic, resulting in only 50% of eligible prescriptions being screened by a technician. Ultimately, this does not change the outcome; enhancing technician’s roles allows pharmacists’ time to be used more efficiently.

Analysis of clinical trial agreement and insurance policy submitted to the ethics committee of a tertiary care teaching institute in central India.

Purpose:Very few studies conducted in India have analyzed insurance policies and clinical trial agreement (CTA) submitted to ethics committee (EC). This study was conducted to review and find out deficiencies in it.Materials and Methods:This was a retrospective observational study. All the protocols for regulatory clinical trials and academic research sponsored by the Indian Council of Medical Research or other funding agency were included. Insurance documents and CTA submitted with the study protocols were analyzed.Results:A total of seventy CTA and insurance policies were analyzed. CTA mentioned that parties involved in 60 (86%) forms, scope of the agreement in 15 (21%) forms, responsibilities of the party in 68 (97%) forms, and payment details in 58 (83%) forms. Nearly 88.5% of the insurance policies mentioned whether the policy covers the participants for injury due to all clauses and 91% of the policies mentioned the validity period of insurance.Conclusion:It was found that both the documents contained almost all the required elements. This was probably because this institutional EC insisted on and thoroughly reviewed the documents to ensure that adequate compensation of research-related injuries has been provided for and this fact is informed to the trial subject. As very few studies are available in the literature, we could not compare majority of the findings of this study with others.

Comparative study of updated Clinical Trial Regulations in India with respect to Australia, Europe, Japan and US

The national regulatory authorities, such as the US Food and Drug Administration (FDA), the European Medicines Agency (EMA), Central Drugs Standard Control Organization and the Australian Therapeutic Goods Administration (TGA), approve every drug that is prescribed for patients around the world. The process of approval undertaken by pharmaceutical companies for drug candidates consists of a series of phases to ensure the product is safe and effective at treating the disease.
 In this paper, we will compare the clinical trial regulations of India with respect to Australia, Europe, Japan & USA in order to determine the safety and efficacy of pharmaceutical products like drugs, biologics and medical devices in different geographical regions and to confirm the clinical trials studies follow strict scientific standards.

Heterogeneity of national legislation and practice on clinical trials with vulnerable patients based on the EU Clinical Trials Directive by the example of adults permanently incapable of giving informed consent.

In principle, persons wishing to participate in a clinical trial must give informed consent in advance after comprehensive information has been provided. Under certain conditions, it is possible to deviate from this requirement in the European Union (EU) in order to enable the participation of so-called vulnerable persons who are incapable of giving their informed consent. Kuthning et al. [1] have already dealt with general and specific aspects of vulnerable patients and the principle of informed consent in clinical trials. One group of vulnerable persons, for example, are adults temporarily or permanently incapable of giving consent due to their state of health. For a long period of time, no systematic and uniform legal basis for clinical trials existed in the EU as a whole. The Clinical Trials Directive (CTD) [2], adopted in 2001, aimed to change this by harmonizing all legal regulations on clinical trials applicable in the EU, but nevertheless allowing national deviations in implementation into national laws through opening clauses and aspects that were left unregulated. In view of the Clinical Trials Regulation (CTR) [3] which, according to the current status, will with high probability be applied from 2022 on, and which in future will be the legal basis for clinical trials with medicinal products in humans, applied directly in all EU member states, the necessity to take stock of the effects of the CTD was evident.The national deviations with regard to the participation of patients incapable of giving informed consent were investigated qualitatively and quantitatively by means of a systematic analysis of legislation in 16 EU countries and a retrospective database analysis of a European clinical trial registry over a ten-year observation period. Although the analysis initially showed a predominantly homogeneous picture, the differences between the EU member states became apparent in a detailed examination. The database analysis yielded a clear result, since in some countries the majority of clinical trials are carried out. The clearest difference was found between the legal analysis and the results of the evaluated clinical trials concerning adults who are permanently incapable of giving informed consent. A presumed association between the “degree of liberality” of the national law and the frequency of clinical trials conducted in the respective country could not be confirmed. In the past, the selection of countries for conducting a clinical trial was based less on legal requirements and more on experience and financial considerations.

Development of sustainable research excellence with a global perspective on infectious diseases: Centre de Recherches Médicales de Lambaréné (CERMEL), Gabon.

SummaryMedical research in sub-Saharan Africa is of high priority for societies to respond adequately to local health needs. Often enough it remains a challenge to build up capacity in infrastructure and human resources to highest international standards and to sustain this over mid-term to long-term periods due to difficulties in obtaining long-term institutional core funding, attracting highly qualified scientists for medical research and coping with ever changing structural and political environments. The Centre de Recherches Médicales de Lambaréné (CERMEL) serves as model for how to overcome such challenges and to continuously increase its impact on medical care in Central Africa and beyond. Starting off as a research annex to the Albert Schweitzer Hospital in Lambaréné, Gabon, it has since then expanded its activities to academic and regulatory clinical trials for drugs, vaccines and diagnostics in the field of malaria, tuberculosis, and a wide range of poverty related and neglected tropical infectious diseases. Advancing bioethics in medical research in Africa and steadily improving its global networks and infrastructures, CERMEL serves as a reference centre for several international consortia. In close collaboration with national authorities, CERMEL has become one of the main training hubs for medical research in Central Africa. It is hoped that CERMEL and its leitmotiv “to improve medical care for local populations” will serve as an inspiration to other institutions in sub-Saharan Africa to further increase African capacity to advance medicine.

Clinical Trial Regulations of Personalized Medicine

Clinical Trial Regulations of Personalized Medicine

Novel standards and emerging therapies for systemically treatment-naïve clear cell renal cell carcinoma - a rapidly changing landscape

The last decade has brought about major advances in systemic therapy for patients with advanced clear cell renal cell carcinoma. The introduction of angiogenesis targeting agents, immune checkpoint inhibitors, and combinations thereof has resulted in a multitude of therapeutic standards for patients with newly diagnosed advanced disease. With the rapid adoption and increasing number of available options for patients, selection amongst treatment regimens has become complex. Further, a new balance is also being sought to optimize treatment outcomes and limit treatment-related toxicities. With a rising bar against which novel therapeutics are being measured, the field looks toward an evolved understanding of tumor biology to help pave new ways forward for individualized therapy. Here, we review pivotal studies that led to regulatory approvals and ongoing clinical trials conducted in patients with systemically untreated clear cell renal cell carcinoma and provide perspective on how newly emerging data can be integrated into this rapidly changing landscape.

アカデミア臨床研究環境のこれまで

アカデミア臨床研究環境のこれまで

Implementation of the EU clinical trial regulation transforms the ethics committee systems and endangers ethical standards

The upcoming Regulation (EU) No 536/2014 on clinical trials on medicinal products for human use (Regulation), which will replace the current Clinical Trial Directive at the end of 2021, has...

Comparison of clinical outcomes with two Transcranial Magnetic Stimulation treatment protocols for major depressive disorder

BackgroundTranscranial magnetic stimulation (TMS) is an effective treatment for major depressive disorder (MDD). The rest time between pulse trains is the inter-train interval (ITI). Since 2016, some TMS clinicians have adopted a stimulation protocol with shorter ITIs than were used in regulatory clinical trials. ObjectiveTo contrast treatment outcomes with the Standard TMS protocol (38.5 min per session) and the “Dash” protocol, which, at the shortest ITI, has a session duration of 18.75 min. MethodsRegistry data were collected at 103 practice sites. Of 7759 participants, 5010 were included in an intent-to-treat (ITT) sample, defined as a primary MDD diagnosis, age ≥ 18, and completion of the PHQ-9 before TMS and with at least one PHQ-9 assessment after baseline. Completers (N = 3814) were responders or had received ≥ 20 sessions and had an end of acute treatment PHQ-9 assessment. Within the ITT sample, 613 patients were treated with the Standard NeuroStar 38-min protocol and 1493 patients with the new Dash protocol. CGI-S ratings were obtained in smaller samples. Treatment outcomes were also examined in subgroups considered Completers, as well as the subgroups who met criteria for Full Adherence to the Standard or Dash protocol parameters. ResultsIn the ITT, Completer, and Fully Adherent samples, response (58–72%) and remission (28–53%) rates were notably high across PHQ-9 and CGI-S ratings. The Standard and Dash protocols did not differ in number of treatment sessions, and both manifested strong antidepressant effects. ConclusionsThe Standard and Dash protocols did not meaningfully differ in efficacy.

The analysis of the regulatory and legal support for clinical trials of medicines and veterinary preparations in Ukraine and the EU

Aim. To analyze the regulatory and legal support for clinical trials of medicines and veterinary preparations in Ukraine and the EU.Results. The analysis of normative documentation for clinical trials of medicines and veterinary preparations has determined significantly different approaches in Ukraine regarding the approved procedures, specified laboratories and clinical sites regulating communications between sponsors and researchers, information support of the procedure, availability of public information, reporting, etc. It has been proven that in veterinary medicine the regulatory framework for clinical trials in Ukraine needs to be updated, a number of critical procedures and documents for planning, monitoring, reporting and various forms of analysis have not been regulated. The absence of interconnection and harmonization of procedures for the use of animals in research has been determined. The main problems in the regulation of clinical trials in veterinary medicine remain the lack of transparent procedures, the absence of regulated guidelines for applicants and sponsors, the low quality of expertise and the lack of coordination between the various government agencies involved.Conclusions. The regulatory and legal bases existing at the moment in Ukraine for conducting clinical trials of medicines complies with international standards and EU Directives. The lack of the regulatory and legal support has been proven; it requires and determines the conduct of clinical trials in veterinary medicine not only in the form of guidelines, but has legal force. Significantly different approaches have been identified regarding the procedures for conducting clinical trials, namely, human clinical trials focus on protecting the ethical standards of the study subject, while the first area of the current animal clinical trial standards is the development and testing of veterinary preparations at all levels, species of target animals; issues of the animal welfare and protection do not cover the legislative and regulatory framework for clinical trials.

Redundant trials can be prevented, if the EU clinical trial regulation is applied duly

The problem of wasteful clinical trials has been debated relentlessly in the medical community. To a significant extent, it is attributed to redundant trials – studies that are carried out to address questions, which can be answered satisfactorily on the basis of existing knowledge and accessible evidence from prior research. This article presents the first evaluation of the potential of the EU Clinical Trials Regulation 536/2014, which entered into force in 2014 but is expected to become applicable at the end of 2021, to prevent such trials. Having reviewed provisions related to the trial authorisation, we propose how certain regulatory requirements for the assessment of trial applications can and should be interpreted and applied by national research ethics committees and other relevant authorities in order to avoid redundant trials and, most importantly, preclude the unnecessary recruitment of trial participants and their unjustified exposure to health risks.

Plain Language Summaries of Clinical Trial Results: A Preliminary Study to Assess Availability of Easy-to-Understand Summaries and Approaches to Improving Public Engagement.

BackgroundEasy-to-understand, stand-alone factual summaries of clinical trial results have the potential to improve public understanding of and engagement with pharmaceutical research. The European Clinical Trial Regulation (EU) No. 536/2014 is a major regulatory initiative that will result in a large number of such plain language summaries (PLSs) posted in the public domain. Today, however, little is known about the extent to which PLSs are written and are available to the general public.ObjectivesThis preliminary study assessed (i) 20 top pharmaceutical companies’ positions on improving transparency and commitment to disclosing trial result summaries in an easy-to-understand format and (ii) the availability of such summaries in the public domain and the ease of locating them via general web searches.MethodsThe availability of PLSs in the public domain was estimated based on the number of EudraCT technical result summaries in four disease areas: chronic obstructive pulmonary disease, asthma, meningitis, and influenza. The likelihood of PLSs being easy to find through internet search engine queries by members of the public was assessed using Google.ResultsAll 20 sponsors had committed to improve clinical trial transparency, 17 committed to sharing PLSs with trial participants, and 14 had at least one PLS available in the public domain. A total of 99 clinical studies in these four disease areas had technical summaries posted on EudraCT between 1 January 2017 and 30 June 2020. Of these 99, 14 studies had PLSs in the public domain. A total of 12 of 14 PLSs were directly captured by search engine. However, the sponsor trial identifier or EudraCT number had to be included in the search term to locate them. Generic search terms resulted in large volumes of non-relevant results.ConclusionDespite the progressive movement towards clinical trial transparency, easily accessible PLSs on clinical trials are currently scarce. The provision of a European mandate and framework for non-technical result summaries by Regulation (EU) 536/2014 will be a major step to bring about positive change.

Standard Operating Procedure to Derive Homoeopathic Prophylaxis in COVID-19 through Prophylactic Clinical Trial—A Perspective

AbstractThe pandemic coronavirus disease 2019 (COVID-19) has become a public health emergency of international concern affecting human health and economy across the world. The outbreak in India is accelerating even after enforcement of lockdown as well as promotion of hand hygiene, respiratory etiquettes and social distancing mainly due to nonavailability of specific antiviral medicine and vaccine. Historically, for the purpose of prevention and treatment, homoeopathy has shown its usefulness in recent and past epidemics using carefully selected medicines as genus epidemicus (GE) for each epidemic. In the present era of evidence-based medicine, usefulness of therapeutic agent as prophylaxis is recognised only after obtaining significant result in prophylactic clinical trial. Claims of usefulness of certain homoeopathic medicine as prophylaxis or GE of COVID-19 can be used to generate research hypothesis and can be tested through prophylactic clinical trial. In this article, outbreak investigation procedures are highlighted, necessity of epidemic preparedness with engagement of community is emphasised, several recent developments in clinical trial regulations for an outbreak are pointed out, among which online submission of research protocol, expedition of ethical review procedure, virtual meeting of the ethics committee, Good Participatory Practice Guidelines for Trials of Emerging (and re-emerging) Pathogens are mentioned. Standard operating procedures are discussed to conduct a comparative effectiveness research using cluster randomisation in a multidisciplinary approach to investigate the effectiveness of the homoeopathic medicine as a prophylaxis for COVID-19 which can be used by the policy makers as an effective public health intervention to stop transmission and prevent community spread of this highly infectious disease.

Informed consent approaches for clinical trial participation of infants with minor parents in sub-Saharan Africa: A systematic review.

BackgroundRegulations are vague regarding the appropriate decision-maker and authority to consent for children of minor parents participating in clinical trials. In countries with high rates of underage mothers, such as in sub-Saharan Africa, this lack of guidance may affect the rights of potential paediatric participants already bearing increased vulnerability. It can also influence the recruitment and generalizability of the research. We provide evidence and discuss informed consent management in such cases to inform best practice.Materials and methodsWe searched PubMed/MEDLINE, Embase, CINAHL, and Google Scholar for articles published up to March 2019. In total, 4382 articles were screened, of which 16 met our inclusion criteria. Studies addressing informed consent in clinical trials involving children with minor parents in sub-Saharan Africa were included. We performed descriptive and qualitative framework analyses. The review was registered in PROSPERO: CRD42018074220.ResultsVarious informed consent approaches were reported. Articles supporting individual consent by minor parents based on emancipation or “mature minor” status lacked evidence in the context of research. National laws on medical care guided consent instead. When no laws or guidance existed an interpretation of the local decision-making culture, including community engagement and collaboration with local ethics committees, defined the informed consent approach.ConclusionsThe review emphasises that the implementation of informed consent for children with minor parents may be variable and hampered by absent or ambiguous clinical trial regulations, as well as divergent local realities. It may further be influenced by the research area and study-specific risks. Clear guidance is required to help address these challenges proactively in clinical trial planning. We provided a set of questions to be considered in the development of an ethically acceptable informed consent approach and proposed information that should be integrated into international clinical trial guidelines.

Tackling the unique challenges of capacity planning for autologous cell therapies

<h3>Background & Aim</h3> Capacity planning has been a long-standing issue in the pharmaceutical industry; setting up Good Manufacturing Practices (GMP) compliant facilities is time-consuming and capital-intensive, but for products to be able to generate maximal economic returns during the market exclusivity period, timely availability of manufacturing capacity is vital. Capacity crunches faced by the monoclonal antibody (mAb) industry in the 1990s were estimated to have caused around $3-$4 billion losses in total revenue due to insufficient product supply. In addition, the uncertainties in clinical trial outcomes, market pressures, and regulations make the multi-objective optimisation of capacity planning and product development decisions critical for the industry. In this work, we present a detailed analysis of the above challenges, and propose to develop a mixed-integer linear programming optimisation model to address the unique challenges of autologous cell therapies. <h3>Methods, Results & Conclusion</h3> Previous studies have considered the optimisation of investment strategies for product development, manufacturing facility investment decisions and global multi-site supply chain optimisation for drugs and multi-site continuous bioprocess optimisation for biologics. As more advanced therapeutic medicinal products (ATMPs) move from bench to clinic, the unique challenges for autologous cell therapies present a new problem in the capacity planning domain. Firstly, the unique requirement for autologous cell therapies to be produced on-demand with cell material originating from patients has proven to be commercially challenging. Secondly, with geographical and supply chain constraints, companies are considering building multiple facilities in different regions or finding alternative arrangements such as licensing and outsourcing. Thirdly, fast-track regulatory frameworks introduced by various regulatory authorities are improving the speed-to-market and have allowed faster commercialisation of ATMPs for life-threatening diseases. The clinical development duration can be reduced from 6-7 years (phase 1, phase 2, phase 3 clinical trial) to 2-3 years (small scale clinical trials with surrogate end-points). Therefore, the commercialisation timeline for process, CMC development, and capacity planning decisions should also be considered earlier. The approach can potentially allow the industry to make better-informed investment decisions and accelerate the commercialisation of these life-saving therapies.