Monthly Archives: February 2018

Rare Disease Day: Raising Awareness towards Rare Diseases and Orphan Drugs

Awareness Day/Month

Rare Disease Day

On the 28th of February, rare disease day is celebrated globally every year. This day was established for the first time by EURORDIS (The European Rare Disease Organization) in 2008 to spread awareness amongst the public about rare diseases and their impact on the lives of patients suffering with them. Further, it sought to raise awareness about the challenges faced by these patients, their unmet needs and the importance of research and development in the field. In 2009, NORD [The National Organization for Rare Disorders, United States (US)] supported the initiative by EURORDIS and Rare Disease Day was sponsored in the US by both the organizations in partnership. The concept was then propagated beyond Europe and US to spread awareness around the world.

Rare/ Orphan Diseases

Rare diseases also called as orphan diseases affect small number of patients. These diseases might cause serious and life threatening or fatal consequences. The designation of disease as rare/ orphan differs from country to country. As per World Health Organization (WHO), a disease affecting 6.5-10 individuals for every 10,000 people is referred to as rare/orphan disease. However, as per European Union (EU), the diseases with prevalence of 5 per 10,000 people are considered as orphan. While, USA designates a disease as rare if it has an incidence lesser than 7.5 individuals per 10,000 of population. As a clear definition of rare diseases in Indian context was not defined, a non-profit organization namely Organization for Rare Diseases in India (ORDI) took the initiative to define them. According to ORDI, a disease with a prevalence of ≤1 per 5000 people may be referred to as a rare/orphan disease. The estimated prevalence of rare diseases in South Asian countries according to Foundation for research on Rare Diseases and Disorders (FRRDD) is given in Table-1.

Table:1 Prevalence of rare diseases in South Asian countries

 

Challenges Related to Rare Diseases

There are numerous challenges that pertaining to rare diseases. These are as follows:

  • Lack of general understanding about the underlying mechanism responsible for these diseases.
  • Difficulty in recruitment of clinical trials and experimental studies owing to the rarity of diseases and their widely dispersed prevalence.
  • Centers for their diagnosis and treatment are scarce.
  • Research and development is this field is also highly neglected.

Orphan Drugs

Orphan drugs are those that are used for the treatment of an orphan disease. Apart from disease rarity, other concepts also underlie the designation of a drug as orphan. Certain drugs are assigned as orphan based upon their feasibility and financial profits. Illustrating this, few vaccines and drugs which are employed to manage tropical diseases are categorized as orphan drugs. This is attributed to the fact that financial benefits to the drug developers is minimal. The population suffering from these diseases is often incapable to bear their cost and thus they must be provided at a subsidized rate thereby, giving minimal financial benefits to drug developers.

In addition, the drug is also categorized as orphan if it might have earlier been withdrawn from the market and later revives back afresh with recognition of its role in treating some other affliction. For instance, thalidomide, which was once withdrawn from market due to the landmark tragedy in mid-1960s has revived back in recent years is an orphan drug to treat lupus erythematosus.

The recent paradigm shift in market trends and profitability in research and development of orphan drugs and related regulation shall be discussed in our upcoming blog.

Turacoz Healthcare Solutions understands its responsibility towards society and this blog is an initiative to draw attention of healthcare providers and pharmaceutical companies towards the unaddressed needs of patients with rare diseases.

Practicalities of Working From Home

HR

Work-From-Home (WFH) is one of the main argument topics lately. WFH as well as flexible working hours are the two major retention tools for working mothers and even other employees. Time is changing very fast, and it has become necessary to build a transparent culture within, which includes trusting employees to work whenever they want. The benefits of working from home are shown in Figure-1.

 

Figure-1: Practicalities of Working from Home

 

We @Turacoz Healthcare Solutions understand the significance of a better work-life balance and realize that employees often value the flexibility of working from home. We firmly believe that best work does not always happen within the restricted office hours of 9-5 and hence have integrated remote working arrangements and flexi time in our culture. So, if you are interested in what we do and looking for a gig that will let you work from home completely/occasionally, reach out to us at [email protected] . To know more about our company visit https://turacoz.com/ and http://www.turacozskilldevelopment.org/.

Regulations of Medical Devices in India: An Update

Medical Devices

Medical devices comprise of a vast range of equipment, ranging from a single pair of tongs to a heart-lung machine. Along with medicines and allied healthcare technologies, medical devices are important for modern patient‑centric care in all sorts of clinical settings like bedside, rural clinics, or metro hospitals. Medical devices have improved the quality of healthcare by making diagnosis, prevention, treatment, and monitoring of diseases simpler and efficient.

Indian medical device industry

Medical device industry is a huge industry, manufacturing wide range of products. In India, the medical device industry has a relatively small share of the global market when it is compared to the size of its economy. India ranks twentieth in the world in medical device market and it is fourth largest market in Asia. Most of the medical devices are imported in India which constitutes over 65% of the total market. The Indian medical device industry is driven by foreign manufacturers. The largest medical device manufacturing segment (53.7%) in India comprises of medical electronics, hospital equipments, and surgical apparatus. Around 31.3% of the medical device industry deals with medical disposables and consumables while diagnostic reagents and implants contributes to 7.9% and 7.1% of the market share respectively (Figure 1).

 

Figure 1*: Components of Indian Medical Devices Industry

*Source: Sethi R, Popli H, Sethi S. Medical Devices Regulation in United States of America, European Union and India: A Comparative Study. Pharm Regul Aff. 2017;6(1):1-9.

Medical devices regulations in India (CLAA scheme)

The Central Drug Standard Control Organization (CDSCO) has assigned a particular division i.e. Medical Device and Diagnostics Division which is dedicated to regulate the medical devices. Under the Central Licensing Approval Authority (CLAA) scheme, the CDSCO notified some of the medical devices and diagnostic kits which are being regulated for the manufacture, import and sale of the medical devices. These medical devices are also called as notified devices. The medical devices are regulated by Drugs and Cosmetics Act (1940) and Rules (1945) for the purpose of import, manufacture, sales and distribution. Currently, fourteen medical devices are notified under the CLAA scheme which are mentioned below:

  1. Disposable Hypodermic Syringes
  2. Disposable Hypodermic Needles
  3. Disposable Perfusion Sets
  4. In-vitro Diagnostic Devices for HIV, HBsAg and HCV
  5. Cardiac Stents
  6. Drug Eluting Stents
  7. Catheters
  8. Intra Ocular Lenses
  9. V. Cannulae
  10. Bone Cement
  11. Heart Valves
  12. Scalp Vein Set
  13. Orthopaedic Implants
  14. Internal Prosthetic Replacements

Current medical device rules (2017) in India

The Government of India has realized the need for framing separate rules and regulations for medical devices in order to improve the Indian regulatory system for medical devices. Hence, Government of India has issued a draft of medical devices to help the medical device industry and will separate the regulatory norms for manufacturing of medical devices from drugs. The draft of medical device was issued on January 31, 2017 and came into force on January 31, 2018.

The new rules and regulations for medical devices will create a novel and different regulatory apparatus by creating a separate registration process for medical devices. The key features of medical device rules, 2017, are summarized below:

  • The new rules have classified the medical devices on the basis of risk. These classes include:
  • Class A – Low risk

(e.g. catheters, surgical dressings)

  • Class B – Low moderate risk

(e.g. disposable hypodermic needles, orthopedic implants)

  • Class C – Moderate high risk

(e.g. cardiac stents, internal prosthetics replacements)

  • Class D – High risk

(e.g. drug eluting stents, heart valves)

  • The Indian regulators will have unique identification for their devices.
  • The medical devices should follow the standards established by the Bureau of Indian Standards (BIS). Where no relevant standard of any medical device has been laid down, then those devices should follow the standards laid down by International Organization for Standardization (ISO) and International Electrotechnical Commission (IEC) standards.
  • Licenses will remain valid indefinitely which are issued to the device registrants, along with the payment of the license retention fees, unless cancelled or surrendered.
  • Licenses and registration certificates received before the implementation of the new rules will be valid till expiry or after an 18-month period following implementation, whichever is later.
  • Test license will be valid only for one year.
  • The manufacturing sites of the devices will be audited by the notified bodies.
  • Medical devices for which trials are not required will need only a pilot study on a smaller population in order to test the safety and performance of the device followed by a pivotal trial on a large population.
  • These rules would be imposed by Central Licensing Authority and State Licensing Authority
  • The Central Licensing Authority is responsible for importing medical devices of all classes, and manufacturing of class C and D medical devices. Also, it is responsible for clinical investigation and approval of investigation of devices, and co-ordination with the State Licensing Authorities.
  • The State Licensing Authorities are responsible for the manufacturing, sale, and distribution of the devices that fall under class A or B, and also, control of sale, stock, exhibit, or offer for sale or distribution of in-vitro medical devices (IVDs) of all classes.

The new rules for medical devices will improve the accessibility and affordability of medical devices globally and will create a favourable and encouraging environment for development and innovation of new medical devices in India. The main objective of Government of India, through these rules, is to develop the confidence of the investors in medical device industry in India and to maintain the quality of all devices as per the international standards.

Turacoz Healthcare Solutions (THS) always aims to keep professionals updated on the current rules and regulations pertinent to regulatory affairs. Turacoz is dedicated towards providing scientific/medical writing support to the industry and academia. With expertise in various clinical trial related documents like Investigator Brochures (IBs), Protocols, Clinical Study Reports (CSRs), Common Technical Document (CTD), and Prescribing Information (PI), we aim to deliver the best quality and guideline compliant documents to the pharma and different sectors of healthcare industry.

Clinical Evaluation Report for Medical Devices-Role of Medical Writer

Medical Devices

Clinical Evaluation Report for Medical Devices

Clinical evaluation report (CER) is a document containing information regarding clinical evaluation of a medical device. CER is intended to be reviewed by the Notified Body (NB). Clinical evaluation refers to an organized procedure (Figure 1) used to collate, compile, and analyze clinical data related to a medical device. The clinical data includes updated published information on a medical condition and the medical device which is obtained from various databases. It also includes pre-clinical and clinical data, quality control data, and risk management and post-market surveillance related inputs which are obtained from manufacturers, quality and safety scientists, respectively. The clinical information also includes the instructions to be followed while using the medical device. The CER is prepared as per the guidelines mentioned in MEDDEV 2.7/1 Rev. 4 (June 2016). It specifies the structure as well as content to be added in the CER.

 

Figure 1: Organized procedure followed for Clinical Evaluation of a Medical Device

(Reference: MEDDEV 2.7/1 Rev. 4, June 2016, Section 6.3)

 

MEDDEV 2.7/1 Rev. 4 (June 2016)

MEDDEV 2.7/1 Rev. 4 (June 2016) is the guideline followed to prepare the CER. It includes summary of CER, updated published information about medical condition for which the medical device is used, as well as complete information about device under evaluation (Figure 2).

Figure 2: Table of content for development of Clinical Evaluation Report

(Reference: MEDDEV 2.7/1 Rev. 4, June 2016, Section 6.3)

Role of Medical Writer in the Development of Clinical Evaluation Reports

Medical writer plays an essential role in development of CERs. According to the requirements of MEDDEV 2.7/1 Rev. 4 (June 2016), the medical writer performs various activities (Figure 3).

Figure 3: Role of a medical Writer in development of Clinical Evaluation Report

Turacoz Healthcare Solutions, a medical communication company, provides latest updates on medical devices. Turacoz is dedicated in providing scientific/medical writing support to the industry and academia. With expertise in several clinical trial related documents such as Investigator Brochures (IBs), Protocols, Clinical Study Reports (CSRs) and Common Technical Documents (CTDs), Prescribing Information (PI), we aim to deliver the best quality and guideline compliant documents to the Pharma and different sectors of Healthcare Industry.

 

Recent Advancements in Cancer Diagnostics

Cancer

Global Cancer Diagnostics Market

The global cancer diagnostics market was estimated to be USD 16.55 billion in year 2016. It is expected to reach USD 232.7 billion by 2025 as per a new report released by Grand View Research, Inc. This boost in the cancer diagnostics market is majorly attributed to the advances in diagnostic technology. Although the newly developed diagnostic modalities remain costly, but they provide an incremental benefit.

Recently Developed Cancer Diagnostic Methods

  1. Magnetite Nanoparticles (MNPs)

Iron oxide, owing to their higher biocompatibility and least toxicity, is the only metal oxide nanoparticle that is approved for clinical use. These nanoparticles are used for diagnostic imaging to enhance image contrast as they accumulate selectively at tumour sites and thereby, provide precise details about tumour. Besides diagnostics, MNPs, when loaded with therapeutic agents such as daunorubicin, are used for the treatment of various cancers such as blood cancer, prostate cancer, etc. Their properties have also been harnessed to induce localized magnetic hyperthermia for cancer treatment.

With the recognition of MNP’s potential for theranostics (diagnostics + therapeutics), further researches are ongoing to develop new techniques for localization of non-palpable breast lesions and its applications in therapy for breast cancer and other tumours.

  1. Biosensors

Biosensor is a diagnostic device used to detect a biological analyte, i.e. tumour biomarkers (certain molecules/ proteins expressed and/or secreted by tumour cells). Biosensor rapidly analyses and measures the cancer biomarkers present and thereby helps in detection of cancer and its nature benign/malignant). In addition to diagnosis, biosensors also help in monitoring the efficacy of anticancer agents. They provide instant information to the treating physician at office (point of care) which greatly influence treatment planning and patient management. Their use is extended to diagnose wide range of cancers including lung cancer, oral cancer, breast cancer and many more.

A biosensor device is formed by three components, namely recognition component (detects signal), signal transducer (responsible for conversion of biological signal to electrical output), and processor (displays result). These are simple tool having huge potential for sensitive and specific detection of cancer biomarkers. Most of the currently available biosensors utilize single biomarker for detection of cancers. However, cancers usually express multiple biomarkers. Therefore, there is a need to develop biosensors with improved diagnostic accuracy and repeatability that can perform multiple analysis.

  1. Next-Generation Sequencing (NGS)

The advent of NGS technology have revolutionized the cancer diagnostics. In contrast to traditional DNA sequencing methods, simultaneous sequencing of numerous DNA fragments can be performed using NGS technique even without any prior knowledge about DNA sequences. This technique allows sequencing of complete genome within few days at a cost less than $1000/genome.

Presently, NGS technique is available in two types, namely short read sequencing and long read sequencing. The short-read technique is relatively cheaper and can read sequences that are less than 300 base pairs. The contrast long-read technique is costly and allows reading of sequences larger than 2.5 Kilo bases.

Cancer diagnostics is an evolving field and numerous researches are always underway to develop more advanced modalities. Turacoz Healthcare Solutions aims to keep everyone updated on the newer technologies and help spread awareness on the current and upcoming trends.

Chimeric Antigen Receptor (CAR) T-cell Therapy: An Emerging Therapy for Cancer

Cancer,

Cancer is the second most common cause of death after heart disease. The treatment of cancer depends upon the type of cancer and at what stage it is being diagnosed. Due to low socioeconomic status and low literacy rate, patients often opt traditional methods and alternative medicines instead of seeking proper treatment. There are numerous methods such as surgery, radiation therapy, chemotherapy, stem cell transplant, etc., which are being used for the treatment of cancer. However, the major concerns associated with these therapies are their adverse effects and the recurrence of the disease. Nowadays, immunotherapy is budding as a promising method for the treatment of cancer as it is associated with minimal adverse effects and low risk of disease recurrence.

The emergence of immunotherapy, i.e., using body’s own immune system, to fight with cancer has been seen from the past two decades. William B. Coley, father of immunotherapy, was the first person to use immune system to treat cancer in the late 19th century. Recently, researchers have developed and approved a new Chimeric Antigen receptor (CAR) T- cell therapy for the treatment of cancer.

CAR T-cell Therapy: A Promising Therapy to Fight Cancer

CAR T-cell is a new therapy that has shown favorable result in the treatment of the cancer. This therapy uses T-cells from the patient’s cell which are further modified to target cancer cells. The therapy is a type of cellular therapy which functions by using body’s own immune system to destroy cancer cells.

Since the treatment involves extraction of T-cells from the blood, the patients may face the risk of infection and may temporarily have low blood cell counts and need transfusion support.

CAR T-cell Therapy: Updated Status

CAR T-cell therapy has been found to be effective in the treatment of cancer and also received approval from Food and Drug Administration (FDA) for cancer treatment. Novartis was the first ever company to receive the United States (US) FDA approval for CAR T-cell (Kymriah (TM) (CTL019)) therapy in August 2017 after years of research and clinical trials. Kymriah is the first gene therapy that is approved for the pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL).

Kite’s Yescarta (Axicabtagene Ciloleucel) is the second most CAR T-cell therapy which got FDA approval in October 2017 to treat adults with certain types of large B-cell lymphoma. Yescarta is the first approved therapy for certain types of non-Hodgkin lymphoma (NHL).

An ongoing phase I clinical trial (NCT00968760) is focusing on the use of gene transfer in patients with advanced B-cell lymphoma. Another interventional clinical trial, in phase I (NCT02186860), aims to determine safety and effectiveness of third generation CART-cells in patients with refractory acute lymphoblastic leukemia. The results of both the clinical trials are awaited.

CAR T-cell therapy: Pharmaceutical development

Several companies such as Novartis, Bluebird bio, Kite Pharma and Juno therapeutics are in lead to develop CAR T-cell therapy. Recently, Gilead and Kite Pharma associated with Pfizer to work on a combination of Yescarta-Utomilumab, which could be beneficial in treating certain blood cancers.

CAR T-cell therapy is one of the recent advancements in the field of cancer immunotherapy and Turacoz Healthcare Solutions (THS) aims to keep professionals updated on the recent advancement in cancer therapeutics and patient care. Turacoz is dedicated in providing scientific/medical writing support to the industry and academia. With expertise in several clinical trial related documents such as Investigator Brochures (IBs), Protocols, Clinical Study Reports (CSRs) and Common Technical Documents (CTDs), Prescribing Information (PI), we aim to deliver the best quality and guideline compliant documents to the Pharma and different sectors of healthcare Industry.