Small Pharmaceutical Companies Research Articles

The Impact of Lean Inventory Management on Small Pharmaceutical Companies

"Inventory lean management" is a key strategy for cost savings and quick market response in fast-moving consumer goods industries such as food, retail, and beauty. Recently, the pharmaceutical industry, with its stringent requirements on product shelf life, supply efficiency, and storage methods, has also begun to focus more on effective inventory management. For SMEs, poor inventory management can lead to lost sales and customers, as shoppers will turn to other pharmacies when they cannot find the desired products.

The Effect of the RMAT Designation on Biotechnology Stock Prices, Drug Development Timelines and Outcomes: An Empirical Analysis

This research examines the impact of the U.S. FDA's Regenerative Medicine Advanced Therapy (RMAT) designation on biotechnology and small pharmaceutical companies, focusing on both stock price reactions and drug development timelines. The research employs an event-study methodology to analyze share price movements following RMAT announcements and evaluates the long-term product development outcomes for these companies. Findings reveal that while RMAT designation leads to an initial short-term stock price increase of approximately 8.11% within five days, this optimism diminishes over time, with long-term trends showing a decline in stock value. Furthermore, products under RMAT designation exhibited longer development timelines compared to non-RMAT therapies, raising questions about the designation's effectiveness in accelerating product availability. The study underscores the complex implications of RMAT for stakeholders, emphasizing the need for strategic planning and further regulatory refinement to better align short-term investor expectations with long-term product development realities.

Review of the impact of the FDA’s Fast Track Designation on biotechnology companies’ share prices

This study examines the effect of the FDA's Fast Track Designation (FTD) on biotech company share prices. Using an event-study approach on 25 FTD announcements between June 2019 and June 2020, notable short- and long-term share price hikes were observed, with a 5-day cumulative average abnormal returns of 21.59%, 30-day at 38.34%, 1-year at 76.64% and 3-year at 111.37% against the XBI benchmark. These surges surpass prior research findings, indicating stronger investor reactions. The role of the COVID-19 pandemic as a confounder is discussed. Although the sample size is limited, the results offer valuable insights for investors and small pharma firms. Further research is recommended to delve into the driving market factors.

Anzalone Prime: An Interview with Prime Editing Developer Andrew Anzalone

Anzalone Prime: An Interview with Prime Editing Developer Andrew Anzalone

Small pharma vendor management practices in clinical trials: A case study within Faron Pharmaceuticals

Many small pharmaceutical companies find that they lack the resources, knowledge and expertise of the regulatory landscape for adequate vendor management in clinical trials, making the organization vulnerable. Recent research suggests that some pharmaceutical companies have found themselves out of compliance with ICH, FDA or EMA guidelines. This paper aims to perform a comprehensive review of the regulatory landscape for vendor selection, oversight and ongoing evaluation in clinical trials. In addition, the case study performed studies the practices recently implemented at small pharmaceutical company Faron Pharmaceuticals to assess regulatory compliance and identify any potential best practices. Faron Pharmaceuticals conducted a process improvement activity at the beginning of 2022 to improve the vendor selection, oversight and evaluation of their clinical trial partners. The results of this case study indicate that Faron Pharmaceuticals' processes are regulatory compliant, suggesting that QTLs, KPIs, SOPs and communication plans are effective vendor oversight mechanisms for small pharmaceutical companies to utilize.

Psychedelic Psychiatry

The last decade has seen a remarkable resurgence of interest in psychedelic drugs such as psilocybin (from magic mushrooms) LSD and DMT (dimethyl tryptamine – the active ingredient of ayahuasca). This has been driven by the discovery that these psychedelics all act agonists of 5-HT2A receptors. Human imaging studies have revealed this action leads to profound alterations in brain measures of activity particularly in terms of increased entropy of EEG MEG and fMRI signals and reduced within-network, but increased between-network, connectivity. In addition they all can increase synaptic growth and brain plasticity. These findings not only explain the subjective nature of the psychedelic experience but also have implications for the treatment of internalising disorders such as depression addiction anorexia and OCD that are characterised by increased within network connectivity especially of the default mode network. Subsequent trials, particularly of psilocybin, in these disorders has revealed significant clinical benefits from even just a single administration. A number of companies have now been set up to extend these discoveries with regulatory-level trials that could result in market authorisations within a few years. My talk will explore these brain mechanisms and clinical data and discuss the potential place of psychedelic medicine in the future of psychiatry.DisclosureI am an advisor to Compass pathways and Beckley Psytec two companies that are developing psychedelics for depression and other psychiatric indications. Several members of my research group receive support from these companies and also from Small Pharma.

The role of artificial intelligence in business transformation: A case of pharmaceutical companies

Artificial intelligence (AI) is of great interest to researchers and practitioners as a means of achieving the necessary progress in the pharmaceutical industry. However, the role of AI and ways of transforming companies are not well studied. The purpose of the paper is to identify exactly how AI affects the key and support business processes of pharmaceutical companies. We offer a qualitative interview study of five large, five medium, and five small pharmaceutical companies. Based on scarce literature on the role of AI in the pharmaceutical industry, we considered which business processes are subject to transform within it and how they do so. We determine that small pharma companies significantly change research and development, master data management, analysis and reporting, and human resource business processes under the influence of AI. Large pharma companies use AI to transform production, sales, marketing, and analysis business processes. In turn, medium-sized companies are in the middle and individually transform their business processes depending on their specialization.

PRS25 Transparency Helped a Small Pharmaceutical Company to Have the Price of Their Drug with Few Million Euro Annual Revenue in Poland Practically Increased by 168% by the Minister of Health - a Case Study of Publicly Available Documents from Administrative Proceedings on Novo-Helisen Depot

A small (6.5 million EUR reimbursement cost in Poland in 2018) pharmaceutical company was under pressure by German HQ to increase the official price of Novo-Helisen Depot (NHD) by est. 2 times to catch up with other European countries prices. The drug is indicated for dust mites allergen immunotherapy with no reimbursed equivalents in Poland. This study aims to present the case of how unconventional transparency-based approach helped to achieve a seemingly impossibly large price increase which could potentially result in 8 million EUR budget impact. We analysed documents made public either directly by the company or by the Foundation for Transparency and Predictability of Administrative Decisions. Having submitted on 2019-05-15 the applications (1) to increase NHD initial set price by 128%, (2) to increase NHD one-vial maintenance set price by 228%, (3) to reimburse NHD two-vials maintenance set with a price per vial 228% higher than the reimbursed product price, the company published unredacted argumentation to MoH at https://allergopharmanexter.pl/application/files/5715/5860/5403/Pismo_do_MZ.pdf. They also sponsored the press conference “Does the Minister of Health in Poland Have the Absolute Power in the Reimbursement Proceedings?” on 2019-09-12 during which the Foundation’s president and first author of this study highlighted MoH failures to proceed lawfully based on administrative documents shared by the company (available at https://www.dlaprzejrzystosci.pl/pytanie-z-konferencji-czy-minister-zdrowia-jest-wladca-absolutnym-refundacji-w-polsce-ciagle-aktualne/). Just a month later on 2019-10-14 the Minister of Health took a positive decision to reimburse the two-vials maintenance set with ex-factory price per vial 168% higher than the most recent official reimbursed price (490.00PLN vs 183.10PLN), without a content-related justification although the company provided it. In countries like Poland where positive reimbursement decisions on drugs and their prices lack content-related justification smaller companies may use openness and transparency to communicate the rational justification of their applications and to obtain fair decisions which otherwise apparently would not be achieved.

Evolving landscape and academic attitudes toward the controversies of global immuno-oncology trials.

This cross‐sectional and longitudinal descriptive analysis aimed to track the evolving landscape of global immuno‐oncology (IO) trials and provide insight into the resolution of IO‐related controversies. Clinical trials (n = 4510) registered on ClinicalTrials.gov in 2007 to 2019 studying immune checkpoint inhibitors (ICIs), adoptive cell transfer (ACT), cancer vaccines and immune modulators were included. Most of IO trials are Phase 2 and focus on ICIs and multiple IO therapies. The United States leads global IO research, with stable growth and the best methodological quality. Mainland China ranks first in the number of ACT trials but has the lowest article publication rate (6.2%). A multiple‐arm comparative design is often adopted in multiple IO therapies trials (44.0%). Trials studying ICIs and multiple IO therapies are likely to use early registration (80.0% and 86.6%) and stringent corticosteroid‐/infection‐related criteria. Hospitals have provided the most extensive and strongest support for all IO categories. Big pharma prefers to fund Phase 3‐4 ICI trials (6.98%), while small pharma has a wider sponsorship favoring Phase 1‐2 trials. The “partial‐use‐of‐corticosteroids” strategy is generally well accepted in ICI trials with a definitive trend (32.5%; P < .001) but is associated with the poor dissemination of results (P ≤ .020), while the complete disclosure and standardization of dose/timing limits are still lacking. Disparities in design features and dissemination of results are widespread in IO trials and are modulated by IO category, cancer type and sponsor. We propose policy reforms to redefine the timely publication of IO trials and standardize the resolution of corticosteroid‐/infection‐related issues.

Companies performance management: the role of operational flexibility

The article summarizes the arguments on minimizing the uncertainty level caused by numerous unforeseen circumstances due to using operational flexibility to increase companies' efficiency (example of small and medium enterprises in the pharmaceutical sector of Iran). The research aims at investigating the relationship between operational flexibility and a company's performance to examine the impact of environmental uncertainty on these relationships. This study was conducted as quantitative. The deductive method using the synergy of systematization of scientific background on the problem and the empirical proof of the formulated hypotheses became a methodological study tool. The article provides evidence of the economic-mathematical model based on data from small and medium-sized pharmaceutical Iranian companies. The study hypotheses are as follows: 1) operational flexibility has a positive effect on the productivity of the pharmaceutical sector of Iran, represented by small and medium-sized companies; 2) uncertainty determines the relationship between the operational flexibility and efficiency of small and medium-sized pharmaceutical companies in Iran. The model constructed by the authors allowed measuring the relationship between variables using regression analysis and moderation analysis (Hayes model). The total number of companies included in the sample is 113. In turn, 228 managers of these pharmaceutical companies took part in the surveys (Iran example). The empirical analysis results showed that the mix flexibility indicator has practically no effect on companies' efficiency, and the volume flexibility and product development flexibility indicators generally have a positive effect on the performance of companies in the pharmaceutical sector. On the other hand, the environmental uncertainty indicator does not help reduce the relationship between the operational flexibility indicator and companies' performance in the pharmaceutical sector of Iran's economy. The study results could be useful for planning small and medium enterprises' activities in the context of improving their performance.

국내 중소 제약회사의 선진국 제네릭 시장 진출 성공요인 분석 : 점안제 사례를 중심으로

국내 중소 제약회사의 선진국 제네릭 시장 진출 성공요인 분석 : 점안제 사례를 중심으로

Study of deviant use of international non-proprietary name of medicines in India

Introduction: International Nonproprietary Names (INNs) are internationally documented names for medicines recommended by the World Health Organization (WHO) and provide a standardized system of nomenclature. Objective: To analyse the current pattern of use of INNs by different pharmaceuticals companies in India. Methodology: The relevant material was systemically collated and analysed after collecting material from websites of WHO, pharmaceutical companies, MIMS and CIMS. We then analysed what is the severity of misuse of INNs in India and categorize the gaps and loopholes at the different level. Results: It has been found that for implementation of INN, most important problem lies in the policy regime along with and deficient coordination in between drug authorities in India. Both big and small pharmaceutical companies were involved in the misuse (10-40%) of INNs. Number of INN stems have been exploited by different companies. Most commonly used INN were by “-grel”, followed by “-pril “-pride” and “gli-”. A great number of cases of misuse of INNs in our country involve drugs for cardiac, renal diseases, and diabetes. Although drug authority has issued INN protection letter by WHO to few pharmaceutical companies but in spite of that number of brand names are still derived from INN. Conclusion: Many lacunae exist in the INN implementation in India. It is essential to develop improved coordination, specific mandate and suitable policy to safeguard INNs in India. Keywords: Brand name, Generic, Propriety name.

Small pharma and biotech firms dominated 2020 drug approvals

Small pharma and biotech firms dominated 2020 drug approvals

Expert Roundtable: getting a gene therapy product to market: pitfalls and how to prevent them

<div class="authors authors-lg"><div class="author"><div class="authors authors-lg"><div class="author"><div class="author-img"><img src="https://bioinsights.blob.core.windows.net/uploads/5665fd4b0e0d43fd9457b130647e8679.png" data-image="1"></div><div class="author-det"><h3 class="author-name" style="color:#5D538A;">Hetal Brahmbhatt</h3><h5 class="author-title" style="color:#5D538A;"><strong><strong>Principal Investigator - Science and Technology Team, Pharma Services, Viral Vector Services, Thermo Fisher Scientific</strong></strong></h5> Dr Hetal Brahmbhatt is a Principal Investigator in the Science and Technology Team, Pharma Services, Viral Vector Services at Thermo Fisher Scientific. She has a PhD in Biochemistry and Biomedical Sciences from McMaster University where she characterized small molecule modulators of apoptotic proteins. In her present role, she contributes to the development of platform processes and technologies for the manufacturing of viral gene transfer vectors. </div></div></div></div></div><div class="authors authors-lg"><div class="author"><div class="author-img"><strong><strong><img src="https://bioinsights.blob.core.windows.net/uploads/119afb6b1b5b4b119cd918769348eab8.jpg" data-image="1"></strong></strong></div><div class="author-det"><h3 class="author-name" style="color:#00A795;"><strong><strong>Elizabeth Simmons</strong></strong></h3><h5 class="author-title" style="color:#00A795;"><strong><strong>Associate Director QC Analytics, Homology Medicines Inc</strong></strong></h5> Beth has a Master’s degree from Lehigh University in Analytical Chemistry and Regulatory Affairs with 13 years of industry experience. She has managed small and large Quality Control laboratories including method transfer, qualification and validation teams in both small pharma and CMO settings. During that time Beth has supported 2 facility start ups (Commercial and Clinical) and managed technical teams focusing on small molecule, oligonucleotides, and now gene therapy. </div></div></div><div class="authors authors-lg"><div class="author"><div class="author-img"><strong><strong><img src="https://bioinsights.blob.core.windows.net/uploads/thomas.png" data-image="1"></strong></strong></div><div class="author-det"><h3 class="author-name" style="color:#007589;"><strong><strong>Thomas Guarinoni</strong></strong></h3><h5 class="author-title" style="color:#007589;"><strong><strong>Manager, Downstream Process team, Viralgen</strong></strong></h5> Thomas Guarinoni, MSc was recruited in January 2017 as manager of the Downstream Process team at Viralgen. He was successful in transferring the entire purification process from Askbio to Viiralgen in record time. Thomas Guarinoni obtained his MSc in 2011 at ENSTBB (National Superior College of Biotechnology) in Bordeaux, France. </div></div></div>

An Analysis of Antibacterial Drug Development Trends in the United States, 1980-2019.

We present a longitudinal analysis of investigational new drug applications (INDs) for new, systemic antibacterial drugs under active development between 1980 and 2019, evaluating the characteristics of these investigational drugs and the outcomes of these drug development programs. The number of INDs in active development declined by two-thirds, from 39 active INDs at its peak in 1987 to a low 13 in 2001, with decreased development of new cephalosporin, quinolone, and macrolide drugs and reduced participation from large pharmaceutical firms. Antibacterial drug development activity rebounded substantially from 2002 to 2009, primarily led by involvement of small pharmaceutical companies. As of 31 December 2019, the number of active INDs has declined to an 11-year low, and the number of antibacterial INDs initiated with the US Food and Drug Administration during 2010-2019 was lower than any of the previous 3 decades. Antibacterial drug development programs initiated in the 1980s and 1990s had high success rates, with >40% of INDs obtaining marketing approval, in a median time of about 6 years from IND receipt to approval. For drug development programs initiated between 2000 and 2009, we found that IND-to-approval rates reduced to 23%, with median development times for approved antibacterial drugs increasing to 8.2 years. The majority of INDs in development as of 31 December 2019 come from already established drug classes, most in early stages of development, and few are sponsored by large pharmaceutical companies.

A Critical Study of Access to Medicines in Light of Indian Patent (Amendment) Act, 2005

Indian Patent Act,1970 was the law governing patents before the formulation of the TRIPS agreement. The subject matter of patentability according to this legislation was the process of making chemicals, drugs and medicines which lead to the emergence of many domestic drug manufacturers who produced new drugs by way of reverse engineering. Indian drug market was considered as the cheapest drug manufacturers and the cost of medicines was affordable by people of every income group. The TRIPS agreement, 1995 was signed by India and major changes were incorporated in the Indian Patents Act, 1970 through various amendments. Before the TRIPS agreement was signed Indian patent regime was focussed on process patents because of which generic market flourished, but by way of the 2005 Amendment to the Patents Act, patents on food, chemical and pharmaceuticals were includes as product patents. This change proposed by the TRIPS was a major blow to the generic market of pharmaceutical manufacturers as they could no longer manufacture drugs by reverse engineering and created monopoly over the patent owners of the drugs. Another major change suggested by the TRIPS agreement was a result of the Doha Declaration, 2002 which was with the objective to strike a balance between the interests of the Patent owner and to ensure access to medicines at affordable prices. The Patents (Amendment)Act, 2002 was an attempt to establish this balance by implementing the principle of compulsory licensing. This principle was laid down in Article 30 of the Agreement that allowed the Member states to legislate compulsory licensing provisions. In the Indian Patent Act,1970 the compulsory licenses can be granted only after 3 years of patent except in case of national emergency or when reasonable requirements of the public are not satisfied etc as per the provisions of Section 84 of the Act. Even though the TRIPS agreement is exhaustive about the balance between the economic interest and the public health, the Indian legislation has failed to specify what circumstances would be considered to be “reasonable requirements of the public”, the procedural requirements are onerous and result in delay, patents result in monopolistic control of the pharmaceuticals and lead to anti-competitive practices. Another lacuna in effective implementation of compulsory licensing is the payment of remuneration to the original patent owner and there is no rate of payment of remuneration and it is left to the discretion of the parties at that time which may be unreasonable if the demand is too high and the government in need is not able to make the payment. The scope of this topic is to critically analyse the limitations and exceptions in the TRIPS agreement with respect to pharmaceutical industry and the effective application of these exceptions to fulfil the objective of striking a balance between the public health and protecting the interest of the patent owner. This dissertation will also discuss about the compulsory licensing procedure adopted in the Indian Patent Act, the impact on the generic manufacturer after TRIPS agreement , the changes in the pharmaceutical industry and the impact of these changes on small pharmaceutical companies at par with the large pharmaceutical companies and give suggestions for better implementation to achieve the objective of Doha Declaration.

Collaboration between academics, small pharmaceutical company and patient organizations in the development of a new formulation of cysteamine in nephropathic cystinosis: A successful story

Rare diseases usually concern small and disseminated population. Implementing clinical research with the right design, outcomes measures and the recruitment of patients are challenges. Collaborations, training and multidisciplinary approach are often required. In this article, we provide an overview of a successful collaboration in nephropathic cystinosis (NC), focusing on what was the key of success, the interactions between academics, the pharmaceutical company and patients organizations. NC is considered as a very rare disease. In 2010, a new formulation of cysteamine, the only available treatment to improve renal outcome of the disease, was proposed by a small American company. Studies were implemented in France under the coordination of an expert of the disease and the clinical investigation center of Lyon. The collaboration resulted in a good recruitment and retention of the patients in the study and most of all in the availability of the new formulation in France. Patients could have facilitated the research by being involved in the early stages of the studies. Involving patients and public early in the process is particularly important in rare diseases as the patient is a great source of knowledge and has his own expectations. Priorities of research, design, conduct and reporting of clinical trials can be defined in collaboration with adults but also with young patients or public, the first concerned in rare diseases. This concept is still to be developed and improved especially with paediatric patients.

The Yew Tree and the Crab: A Case Study of Big Pharma, Small Pharma, and an Anti-Cancer Drug

The Yew Tree and the Crab: A Case Study of Big Pharma, Small Pharma, and an Anti-Cancer Drug

Segmentation Strategies:Empirical Evidences from Pharmaceutical Companies

The Indian Pharmaceutical sector is highly fragmented with more than 20, 000 registered units. The organized sector of India's pharmaceutical industry consists of 250 to 300 companies, which account for 70 percent of products on the market, with the top 10 firms representing 30 percent. Multinationals represent only 35% of the market, down from 70% thirty years ago. Because of differences in the industry structure and combination of market opportunities for domestic companies and multinationals, they are likely to differ on strategic issues. Hence domestic companies and multinational firms need to pursue different strategies to achieve desired goals. The study covers pharmaceutical companies operating in Madhya Pradesh state of India. The data or information required is related to segmentation strategies adopted by these pharmaceutical companies. The participants are selected from the directory of pharmaceutical manufacturing units in India, published by national pharmaceutical pricing authority. ANOVA is used for testing of hypothesis. The results show that there are visible differences in the segmentation strategies followed by different pharmaceutical companies of Madhya Pradesh. Small & medium domestic pharmaceutical companies have very little inclination towards segmentation strategies. MNCs and large domestic pharmaceutical organizations in Madhya Pradesh are following the segmentation strategies but this is also limited to demographic and geographic segmentation mostly. They also follow integrated segmentation strategies to some extent.

Transdiagnostic and Illness-Specific Functional Dysconnectivity Across Schizophrenia, Bipolar Disorder and Major Depression and Relationships with Working Memory

Background: Mental disorders are typically defined as distinct diagnostic entities but similar patterns of clinical impairments are frequently found in practice across the diagnostic groups. We investigate whether the transdiagnostic deficits result from a common neurocognitive mechanism across disorders, or various illness-specific mechanisms, or a combination of both. Methods: Functional MRI data were collected from the clinically stable patients with major depressive disorder (MDD; n=53), bipolar disorder (BIP; n=78), or schizophrenia (SCZ; n=100), and the matched healthy control subjects (n=109) using a single scanner. Group comparisons were conducted to identify both the transdiagnostic and the illness-specific features. A multivariate approach with cross-validation was used to assess the association between the brain functional dysconnectivity and the cognitive deficits. The confounding effect of medication on the findings was also tested. Findings: Compared with the healthy controls, the patients displayed shared working memory deficits [Cohen's d (d) in MDD/BIP/SCZ=0·73/0·65/0·89], and functional dysconnectivity within brain networks [somatomotor (d:0·50- 0·58) and salience (d:0·52-0·58)] and between brain networks [subcortical-limbic (d:0·55-0·69) and subcortical-dorsal attention (d:0·56-0·61)]. The illness-specific dysconnectivity were found between the executive control and default-mode networks in SCZ (d:0·46-0·56), between the salience and subcortical networks in BIP (d:0·47-0·48), and between the salience and default-mode networks in MDD (d:0·53-0·55). Working memory deficits were associated with a linear combination of 10 transdiagnostic and 4 illness-specific dysconnectivity patterns (r = 0·368, p = 2·45e-12, n = 340). The associations between the dysconnectivity patterns and the medication dosage did not reach statistical significance. Interpretation This study emphasizes that transdiagnostic deficits in cognition may be mediated by multiple interactions between many brain networks, and including diagnostic-specific patterns of functional dysconnectivity in different psychiatric disorders. Funding Statement: Ministry of Science and Technology, National Health Research Institutes (Taiwan). National Natural Science Foundation, National Key Research and Development Program, Shanghai Municipal Science and Technology Major Project, Shanghai Science and Technology Innovation Plan, Shanghai AI Platform for Diagnosis and Treatment of Brain Diseases, the Project of Zhangjiang Hi-Tech District Management, and Zhangjiang Lab (China). Academic Medical Organsiation of Southwest Ontario and the Bucke Family Funds. Declaration of Interests: Dr Palaniyappan reports educational grants and personal fees from Otsuka Canada and Janssen Canada, educational grants from Sunovion, personal fees from SPMM Course (UK) and Canadian Psychiatric Association, and royalties from Oxford University Press outside the submitted work. Dr. Robbins reports personal fees from Cambridge Cognition, Unilever, Mundipharma, Greenfiled Inc, grants from Shionogi, Lundbeck, Small Pharma, personal fees from Elsevier, Springer Verlag, outside the submitted work. No other disclosures were reported Ethics Approval Statement: The study was approved by the Institutional Review Board of Taipei Veterans General Hospital. Written informed consent was obtained from all participants before commencement of the study.