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What is food for special medical purposes (FSMP)?

Food for Special Medical Purposes

With a greater recognition of the importance of nutritional support in medical therapy and improved technology, the “food for special medical purposes” (FSMP) market is currently surging. However, many questions arise, what is food for special medical purposes? Where can patients access them? Is it classified as therapeutic goods? Who is the regulator of such products?

As per Standard 2.9.5 – Food for special medical purposes under Food Standards Australia New Zealand Act 1991 (Cth), FSMP means food that is:

  1. Specially formulated for the dietary management of individuals:
    1. By way of exclusive or partial feeding, who have special medically determined nutrient requirements or whose capacity is limited or impaired to take, digest, absorb, metabolise or excrete ordinary food or certain nutrients in ordinary food; and
    2. Whose dietary management cannot be completely achieved without the use of the food; and
  2. Intended to be used under medical supervision; and
  3. Represented as being:
    1. A food for special medical purposes; or
    2. For the dietary management of a disease, disorder or medical condition.

In Short, FSMP may be used to help people suffering certain diseases, medical conditions or disorders and cannot meet the nutritional requirements via normal diet.

Even though they are food, consumers, cannot purchase them from local supermarkets. FSMP can only be provided via health care professionals, such as a medical practitioner or dietitian or pharmacists.

However, they are not considered therapeutic goods. The line between a medicine and therapeutic food has become even greyer over the past few years. TGA has developed an online tool – Food-Medicine Interface Guidance Tool – and encourages customers to familiarise themselves with the basics of food and medicine and regulation. Manufacturers and sponsors can also use the Tool to work out which regulatory regime would apply to their products.

Unlike therapeutic goods regulated by the TGA, FSMP are regulated by Food Standards Australia New Zealand (FSANZ), who developed the Food Standards Code. Standard 2.9.5 of the Food Standards Code regulates the composition, labelling and sale of food for special medical purposes. It limits FSMP to make any therapeutic claims, for example, FSMP cannot make a claim in relation to the prevention of the disease.

Because FSMP is so similar to a medicine, it may cause undesired effects as well. However, which regulatory authority should we report to? Under the mandatory reporting obligations section of the Australian Competition and Consumer Commission (ACCC) guidelines: “Individual suppliers are responsible for reporting incidents where consumer goods have been associated with a death or serious injury or illness of any person.” The manufacturers of the FSMP may wish to be conservative and report such cases to ACCC.

 

Our Medical Services team has extensive experience in Food for Special Medical Purposes. Call us on +613 9251 0777 to discuss how we can assist.

This article was written by Wenna Zhang, from our Medical Services team.




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Stressed? Well your products should be!

Forced Degradation Studies

Treating your drug substance or drug product with an acid, a base, an oxidising substance, heat or exposing it to direct light might sound extreme, but that is exactly what the TGA want to see in your registration dossier.

Assay results of around 100% are certainly ideal, but if your test method cannot distinguish the active ingredient from any impurities formed through degradation, then how can you be sure your product will remain safe and efficacious throughout its shelf life?

Forced degradation studies, also known as ‘stress testing’ studies, are used to demonstrate that the assay and impurity test methods are sensitive to the full range of potential degradation products that could form over the shelf-life of a drug product or drug substance. Regulatory authorities will expect to see this data in Module 3 of a registration dossier; however, it is important that the studies are performed correctly.

So how much stress?

Detailed instructions on how to perform stress testing are not readily available in published regulatory guidance documents. This is because there is no “one size fits all” when it comes to stress testing. What may be adequate for one product may be too extreme for another. Instead, stress testing studies should be designed considering the nature of the drug substance and drug product, along with some common sense.

Companies often apply conditions that are either too drastic, resulting in irrelevant degradation, or sometimes the conditions applied are not harsh enough and result in little or no significant degradation being observed. The ideal result under each of the five stress conditions (acid, base, oxidising, heat, light) is that the main compound is degraded by around 5-15% (ideally 10%). The conditions applied should also allow for some predictability of degradation pathways. Chromatographic conditions should ensure that degradants can be separated (as much as possible) and that they don’t interfere with measurement of the main compound or previously known impurities and degradants.

What about mass balance?

Another important measure of an acceptable stability-indicating test method is its ability to account for all the components produced under stressed conditions and over the ordinary shelf-life of the product. Ideally, the amount of decrease in the main active component has a directly corresponding increase in the amount of all the measured impurities and degradants. Where this is not the case, a few factors will need to be considered to ensure that mass balance can be achieved (or differences understood) such as improvements to the test method(s), changes to the stress conditions or possible degradation pathways which may hinder the detection or measurement of specific compounds.

Stress-testing is an important validation consideration and should be conducted very early in the development or implementation of new test methods rather than leaving it until regulatory authorities raise questions on the results.

 

Want to know more?

For assistance in reviewing or designing your forced degradation studies, please contact our expert Regulatory Services team on +61 3 9251 0777

This article was written by Tim Dow and Colleen Turnbull, from our Regulatory Services team




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Proposed updates to GVP Module VI and what this might mean for our clients

european-medicines-agency

Good pharmacovigilance practices (GVP) are a set of measures drawn up to facilitate the performance of pharmacovigilance in the European Union (EU). GVP apply to marketing-authorisation holders, the European Medicines Agency and medicines regulatory authorities in EU Member States. The TGA and Medsafe also occasionally refer to the GVPs in their regulations and guidance documents. It is important to keep abreast of any major changes to the GVPs, so that we are best able to help clients prepare for necessary changes.

The new draft of GVP Module VI (Management and reporting of adverse reactions to medicinal products) was released for public consultation in August 2016, with a deadline for comments mid October 2016.  Of interest are the following proposed changes: 

  1. Definition/terminology changes and additions – The definition of Occupational Exposure has been amended (clarification that it means only the medicinal product and not active ingredients/excipients during manufacture), the definitions of Medication Error and Falsified Medicinal Product have been added.  These updates or new definitions may be important for clients in terms of capturing good quality information. 
  2. Electrowinnic reporting modalities of ICSRs under the new ICH-E2B (R3) format (becomes compulsory in June 2017) – there have been many changes made to Module VI with regards to ICH-E2B (R3).  MAHs will need to understand these changes and be able to adopt them before the deadline.  Two important changes include:
    1. The addition of ‘Primary Source for Regulatory Purposes’ which covers multiple sources by identifying the source of the worldwide case number (enabling global pharma companies/clients with SDEAs to identify duplicate cases)
    2. The ability to capture information on the seriousness of multiple adverse reactions in a single ICSR for each individual reported adverse reaction
  3. ICSR reporting, and data quality management – The addition of a sub-section called Nullflavours (a collection of codes specifying why a valid value is not present in an ICSR, e.g. a value is applicable but is unknown such as age of patient).  The client will have to be familiar with these codes to produce valid reports.  A new section on amendment and nullification of reports (might be applicable following an internal audit, where a report is found to be erroneous or in need of correction, but no follow-up information is available. With regards to data quality management, the section which discusses correct data entry has been moved to the Quality Management section.  
  4. Validation of ICSRs based on patients and reporters’ identifiability – At least one patient identifier must be available to consider the case validated for reporting (e.g. initials, gender, age, identification number).  Also the HCP must now be characterised by their qualification AND at least one of the following parameters; name, address or phone number.  If these identifiers are not present, it will not be considered a valid report. This will be important for clients to ensure that all of their reports are valid. 
  5. Management of ICSRs described in the scientific literature – There are changes to literature monitoring requirements for MAHs.  Exclusion criteria for the reporting of ICSRs published in the scientific literature has been listed.  The EMA will be performing monitoring of medical literature and will make available a list of active substances and medical literature subject to this monitoring.  This will affect clients’ processes with regards to literature monitoring. 
  6. Collection of information on patients’ age – More emphasis has now been placed on the importance of obtaining the patient’s age/age group.  This is especially important for identifying issues in elderly or paediatric populations. Clients should be aware of this, so that collection of age can be attempted in initial case or follow-up.  
  7. Guidance on spontaneous reports (management of suspected adverse reactions (not related to any organized data collection systems and which are notified through medical enquiry/product information services or which are consequent of the distribution of information materials), management of reports from post-authorisation efficacy studies, cases notified by different reporters, referring to the same patient and same suspected adverse reaction, and at least one notification is done in an unsolicited manner) – this will be important for MAHs to enable them to correctly identify spontaneous reports.
  8. Transfer of the guidance on Emerging Safety Issues to another GVP Module (Module IX) 

The proposed draft can be found on the European Medicines Agency website.

 

Call our experienced Pharmacovigilance Team on +61 3 9251 0777 to discuss how these changes might affect you

This article was written by Beverley Worrall, from our Pharmacovigilance Team




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Medicine labels: TGO 91 and TGO 92. What’s the impact?

Medicine Labels

Following years of consultation and review the TGA has issued two new labelling orders to replace the current Therapeutic Good Order No. 69 for specific medicine labelling requirements:

  • Therapeutic Goods Order No. 91 – Standard for labels of prescription and related medicines (TGO 91)
  • Therapeutic Goods Order No. 92 – Standard for labels of non-prescription medicines (TGO 92)
  • The creation of separate orders for prescription and non-prescription medicines was implemented to reflect how the products are used and ensure adequate information is available for the safe use of products.

    Some key changes introduced in the new TGOs will require that the labels for the majority of products supplied in Australia are revised. These include:

  • Increased prominence of the active ingredient with specified placement and size
  • Medicine name must be a cohesive unit without graphics of text
  • Additional warning statements and allergen declarations
  • For prescription medicines, addition of a dispensing label space
  • For non-prescription medicines, addition of a Critical Health Information table
  • Although the TGA has implemented a 4-year transition period before the new TGOs become mandatory, it is recommended that where new labels are being created, the new TGOs are adhered to now. This will avoid a rush to update labelling at the end of the period.

    For currently supplied products, the timing of labelling updates will need to be carefully managed over the transition period to ensure there is minimal write-off of packaging components and the appropriate variation applications are submitted (and approved) in a reasonable time period to ensure continued regulatory compliance.

     

    For assistance in updating or developing medicine labels that comply with the new orders, please contact our expert Regulatory Services team on +61 3 9251 0777.

    This article was written by Lynda Notting from our Regulatory Services team




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    Patient support program – the key driver of better health outcomes

    patient support program

    Pharmaceutical companies play a key role in supporting the quality use of medicines through patient focused programs. These programs are designed to aid compliance and lifestyle choices. They provide significant value to patients through the accessibility of information and support.

    Each program run by a pharmaceutical company is managed under strict ethical guidelines and for a specific medicine. Participation is only available to patients who have already been prescribed a medicine and supported by their healthcare professional.

    Programs can provide information to patients about their condition, how to better manage their health, or encourage adherence to the medicines they have been prescribed. They are aimed to complement the important roles their doctors and pharmacists play in the management of their condition.

    Companies must ensure that all aspects of a program including written materials or verbal interactions are patient focused and are not in any way promotional nor have the intention of promoting a prescription medicine to members of the general public.

    A rationale must be developed for each program which describes the clinical significance of the program, the anticipated number of patients to be enrolled, the type of educational/informational material to be provided to a patient, the number of interactions (if any) that may be had with a patient and the duration of the program. Any communication with a patient considering enrolling in a patient support program should clearly identify the company, what materials or calls the patient may receive and their rights under the program. Companies have a duty of care to ensure that patients are fully aware how their privacy and confidentially is maintained and that they can elect to opt out of a program at any time. Patients should also be advised how and who will be holding their data and assurances given that data will not be used for purposes other than for the designed program. At specified intervals companies should evaluate whether a program is delivering on its initial objectives including improvements in compliance and patient wellbeing.

    The importance of reporting adverse events identified through interactions with patients enrolled in patient support programs cannot be over emphasised. Careful monitoring of programs ensures accurate and timely reporting of de-identified product safety related information. It also aids our understanding of medicines in the post market environment.

    Medicines Australia Code of Conduct has detailed provisions on how such programs should be administered and recognises that they offer important support to patients and have a key role to play in delivering positive health outcomes.

     

    Our Medical Services team has extensive experience in patient focused programs. Call us on +613 9251 0777 to discuss how we can assist.

    This article was written by Terence Khoo, from our Medical Services team.




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    Commercial Eyes Recruiting: Regulatory Services Manager

    Regulatory Services Manager

    Commercial Eyes has a great opportunity for a Regulatory Services Manager

    Reporting to the Director, Regulatory Services, the Manager is responsible for the day to day operational management of the regulatory team and the assigned project portfolio within the Regulatory Services business unit.

     

    To be successful in this role, you will have…

    • A degree in science, pharmacy or a related discipline.
    • A minimum of 10 years of experience in Australian and New Zealand regulatory affairs, preferably in a commercial environment.
    • Ability to develop sound strategies to resolve complex regulatory problems.
    • Good working relationships with relevant TGA staff and experience with TGA pre-submission meetings.
    • Experience in the review, critical evaluation and presentation of clinical data.
    • Good knowledge of TGA legislation, regulations, guidelines and working procedures.
    • High level written and oral communication skills.
    • Proven ability to manage projects.
    • Experience or aptitude for mentoring and leadership
    • Experience in staff management in a regulatory environment.
    • Ability to deal with high level of confidentiality.

     

    Key responsibilities include:

    • Manage regulatory projects and staff to ensure optimum project outcomes.
    • Ensure agreed financial and organisational targets are met.
    • Undertake key account management and day-to-day liaison with clients as agreed with the Director, Regulatory Services.
    • Undertake business development and assist the Director, Regulatory Services to develop business development and other growth strategies.
    • External Representation of the business unit where possible
    • Attract new talented regulatory professionals to join the business.
    • To provide support to clients on the registration and listing of pharmaceuticals and medical devices in Australia and New Zealand.
    • Provide strategic advice to clients regarding the Australian and New Zealand regulatory environment.

     

    In return….

    This role is a rewarding one that will offer a competitive salary to the right candidate and a rare opportunity to lead a dynamic and successful team. You will also have access to a flexible work environment, life-work balance and the opportunity to expand your professional network working with local and multinational pharmaceutical companies, as well as a range of other companies developing novel and cutting edge health technologies. 

    Becoming part of the CEPL team will also provide you with a strong sense of teamwork, job satisfaction and achievement whilst working in a well-known professional services organisation that recognises talent and expertise as one of its biggest assets. 

     

    For more information about this role, please visit our Seek ad.

    Please contact Jeanette Lodge on 03 9251 0777 if you have queries about the role that have not been addressed in the job ad. 




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    Commercial Eyes Recruiting: Senior Market Access Consultant

    Market Insight Manager

    Commercial Eyes has a great opportunity for a Senior Market Access consultant

    to join our dynamic Market Access team. Reporting to the Health Economics and Market Access Manager, this senior consulting role offers the successful candidate the opportunity to gain valuable market access experience across a wide variety of projects for the pharmaceutical, medical device and wider health care industry.

     

    To be successful in this role, you will have…

    • Minimum undergraduate qualifications in economics or a life sciences discipline.
    • Preferably post graduate qualifications in health economics, statistics, or health sciences.
    • Minimum 6 years commercial life sciences experience.
    • Excellent numerical, verbal reasoning and written communication skills.
    • Excellent problem solving and project management skills.
    • Proficiency in building productive relationships and networks.
    • A keen interest in the practice of consulting.

     

    Key responsibilities include:

    • Providing high quality expert consulting services to clients, in the key areas of reimbursement strategy, health economics, statistics, submission development and private/public market pricing.
    • Working with existing and potential clients in the life sciences sector to understand their business needs and identify areas or activities with which CEPL may be able to assist.
    • Translating these conceptual discussions into concrete project proposals which provide a realistic description of the scope of work to be undertaken and a competitive cost estimate for this.
    • Delivery of strategically and technically sound submissions, reports and presentations according to agreed deadlines and client requirements.
    • Building strong interpersonal networks across the life sciences sector and drawing on these to identify potential short and long term business opportunities for CEPL.
    • Working with the Manager and other stakeholders to identify important skills and knowledge gaps in the current CEPL reimbursement and pricing service offering; and to implement solutions to these service gaps; e.g. mentoring and developing junior consulting staff, negotiating access to key information sources, and identifying/working with new/existing partner organisations.

     

    In return….

    This role is a rewarding one that will offer a competitive salary to the right candidate. You will also have access to a flexible work environment, life-work balance and the opportunity to expand your professional network working with local and multinational pharmaceutical companies, as well as a range of other companies developing novel and cutting edge health technologies. 

    Becoming part of the CEPL team will also provide you with a strong sense of teamwork, job satisfaction and achievement whilst working in a well-known professional services organisation that recognises talent and expertise as one of its biggest assets.

     

    For more information about this role, please visit our Seek ad.




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    Warning: This medical device literature review may contain traces of LBS

    Medical Device Literature Review

    With the release of new clinical evidence guidelines for medical devices, sponsors and manufacturers will be expecting some further guidance on the structure, content and quality of their clinical evaluation reports (CERs). Once final, this guidance will be used in conjunction with the existing Australian Regulatory Guidelines for Medical Devices (ARGMD).

    Clinical evidence for devices can vary depending on the class of the medical device. It is common for manufacturers to provide a CER derived from a variety of sources, such as clinical trials, literature reviews and/or post-market data for the proposed device. It is also common to collate data from equivalent or similar devices as long as there is an explanation of how evidence for the equivalent or similar device is applicable to the proposed device. Most of the time, literature reviews form a part of the CER.

    The features of a literature review provided in this draft clinical evidence guideline closely resemble those for a literature-based submission (LBS) for medicines, such as:

    • A documented methodology, which captures published and unpublished scientific literature, both favourable and unfavourable,
    • A search protocol, which would include the database searched, search terms and limits used, inclusions/ exclusion criteria, to ensure the search can be replicated,
    • A selection strategy, which involves examining the quality of the literature, including the design of the study/ investigation, quality and completeness of the data reported, its scientific impartiality, and validity of any conclusions drawn in the paper,
    • Critical analysis of the data by an expert in the therapeutic area, rather than a summary of each literature article and a general conclusion at the end.

    Some of the common errors made in CERs have also been highlighted by the TGA in this draft guideline.  Errors tend to relate to the proposed literature search strategy, such as the lack of a comprehensive literature review with documented methodology, an inadequate demonstration of substantial equivalence between reference device and the device under review, absence of critique or discussion of the evidence or its validity.

    There is no need to obtain prior TGA approval for the proposed search strategy, however based on the above criteria, the TGA will be expecting a CER that delivers strongly on the quality of data.

    The TGA consultation for the draft clinical evidence guideline closes COB on 10 June 2016, so make sure you take a look and provide input where necessary.

     

    For assistance developing a suitable CER for your product, please contact our expert Regulatory Services team on +61 3 9251 0777.

    This article was written by Wai Zin Wong, from our Regulatory Service team.




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    Ready? Get Set…Go! Planning the most appropriate strategy for your product to enter the Australian market

    exploding-idea

    As we weave our way through life, we plan…plan our career, plan our finances, plan a holiday and most of us would have at least once in our lifetime planned a party. The key to a successful plan is finding the right resources to help us make informed decisions that suit our needs.  Building a successful commercialisation strategy for a new medicine is no different and requires a plan that must include the right resources and knowledge.

    Introducing a novel treatment into Australia requires the sponsor to align with governance factors that shape the local pharmaceutical industry. An in-depth understanding of the current regulatory and reimbursement environment is critical for success. As such, planning for product launch should begin with the development of a cohesive strategy that occurs long before the submission of a regulatory registration dossier or government reimbursement application.

    Consequently, resourcing and investing in accurate market insight advice specific to the Australian market can be invaluable. Government authorities are demanding more data than ever before, so coordinating relevant activities in the lead-up to launch cannot be underestimated. Market insight advice can formulate valuable assessments that will ensure any key gaps in various inputs are identified and filled which is critical to strategic decision-making. Market Insight professionals are similar to a knowledgeable party planner who meticulously researches and plans to bring the most useful and relevant resources together to set the scene and create a successful event.

    Of course, it does not stop there, optimising a planned strategy also requires timely and appropriate execution. There is no point in the DJ arriving three days before the party, just as it can be fruitless to attempt a cost-effectiveness analysis with incomplete data or an unconfirmed indication. At the same time, it is also wise to show up a little early to scope the scene and identify where information is missing. As the demand for detailed clinical and economic evidence increases, a timely market access strategy allows a sponsor to paint a clear picture of the new treatment’s value offering to decision-makers at the earliest feasible point, having addressed any previously identified shortcomings in the acquired data.

    Capturing clinical data that encapsulates the needs of both a regulatory and PBAC application is a worthwhile consideration prior to the drafting of a TGA/PBAC parallel process submission. Each individual TGA and PBAC application should be well structured and complement the other to form a compliant, comprehensive and cohesive submission for the proposed product. The timing of these types of submissions requires effective pre-planning and communication, a process that is significantly strengthened by synergy between the Market Access and Regulatory Services experts involved.

    Just as a party host should be checking in to make sure that hot food is continuously available for guests, for commercialisation, inputs should be checked for accuracy and completeness. As a medicine gets closer to launch, marketing strategy development, pricing considerations, and cost effectiveness modelling help support reimbursement goals. When accessed through a single source such as Commercial Eyes, coaction is assured between the experts working within these distinct workflows, helping to harmonise and strengthen each of the applications required to achieve the optimal launch of a medicine on the Australian market.

    Early implementation of a well-conceived strategy, specific to the needs of the product, will benefit both registration and reimbursement applications. As with any successful event, a commercialisation strategy must be well planned and properly executed to achieve optimal results. Engaging Commercial Eyes opens a specialised avenue to multiple experts in all areas of Market Insights, Market Access, and Regulatory Services. As a unified team, we can offer an integrated approach to any product launch and help you effectively manage the entry of your new drug into the Australian marketplace.

     

    Commercial Eyes offers a full and integrated suite of consulting services across Market Insights, Market Access, and Regulatory Services. Call us on +61 3 9251 0777 for our insights into commercialisation strategies for healthcare products.

    This article is co-written by our Market Access team and Matthew Douglas from our Market Insights team




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    Data and Safety Monitoring of Clinical Trials

    Safety Monitoring

    Randomised clinical trials for medicines and medical devices are progressively being monitored for safety and other interim results by both Sponsors and Contract Research Organisations (CROs). There are a variety of methods to aid in proactive monitoring and gauging of patient safety and risk, including the utilisation of independent panels of expert physicians and biostatisticians, or what are known as Data Safety Monitoring Boards (DSMBs) and Data Monitoring Committees (DMCs).

    DSMBs/DMCs have as their top priority the ongoing assessment of subject safety during the course of a trial where subjects are exposed to heightened risks of trial participation due to the use of experimental medicines and/or medical devices.

    The importance and growing popularity of monitoring panels has been recognised, evidenced by the FDA’s guidance and recommendations to Sponsors regarding when such committees should be convened as well as a general operational framework for each member’s role and responsibilities.  In order to maintain an independent unbiased role in their oversight of a trial, the physicians and biostatisticians of a DSMB/DMC may not have any fiduciary association with the Sponsor and may not be operationally involved in the trial as steering committee members, investigators or as the active study biostatistician.

    A common example of when a DSMB/DMC panel may be convened surrounds central adverse event adjudication. Another area where DSMB/DMC panels play a key role is concerning trial protocols. Before a trial protocol is finalised for regulatory approvals, the DSMB/DMC may participate in a review of the trial protocol to provide feedback to the Sponsor as to operational characteristics that may be problematic and lead to subject safety and/or trial scientific integrity concerns. If there are formalised interim stopping rules defined in the trial protocol, these will be reviewed so that all parties understand the timing and mechanisms by which these decisions will be made.

    Following are some of the ongoing safety and scientific integrity questions that a DSMB/DMC may need to consider:

    • Are subjects being exposed to reasonable risks given the scientific research that is being conducted?
      • Are there unexpected events not initially envisioned at the outset of the trial?
      • Are there evolving safety signals that might suggest that subjects in the trial are being exposed to an elevated level of risk and, with an eye toward the future, is it reasonable to continue the trial given the projected risks to subjects yet to be enrolled?
      • If continuing the trial is reasonable, a DSMB/DMC may make recommendations to the Sponsor to communicate safety related issues to the study steering committee, the investigators and even the FDA to raise awareness
      • If the safety issues are material and the risks to subjects are no longer acceptable, the DSMB/DMC can make a recommendation to stop the study early due to these concerns
    • Are the a priori assumptions that led to the power and sample size calculations in the trial protocol still valid?
      • Is the observed data deviating from these assumptions, perhaps leading to the need for an interim sample size re-estimation which would most commonly be an increase in the enrolment target?
      • Can the new enrolment target be achieved?
    • Is there new data from an external source (e.g. a similar or clinically relevant trial) where the results that have become available suggest that it would be unethical to continue the trial being monitored?

    In trials where there are formalised interim stopping rules, these must be pre-defined in the trial protocol. These stopping rules would be derived through detailed statistical methods.

    • Stopping for Safety
      • Predefined criteria for the incidence of serious adverse events, especially if they are deemed to be treatment emergent/related or are indicative of treatment failure. These are typically expected events, which are occurring at rates that raise questions about the risk/benefit balance of the treatment(s) under study
    • Stopping for Efficacy
      • Predefined criteria, which would demonstrate that the benefit observed in the treatment of interest is sufficiently large, that to continue the study to its logical conclusion would not alter or reverse this finding
      • Stopping the study for efficacy on an interim basis would have stricter than typical p value thresholds for statistical testing (e.g. p ≤0.001 as opposed to p ≤0.05) to control for false positive (Type I) errors, and would account for multiple looks at the data
      • Ifthe criteria are met, stopping would reduce the required sample size for the study, reducing risks to subjects not yet enrolled and address the ethical questions, for example, of continuing to treat subjects with an alternative that is now demonstrably inferior. It would allow the Sponsor to proceed to regulatory submissions earlier and at a lower cost than originally budgeted
    • Stopping for Futility
      • Predefined criteria that would indicate that there is a lack of a clinically meaningful benefit attributed to the treatment of interest. If the study were to continue to its logical conclusion, the probability that this finding would be altered or reversed to demonstrate a benefit is sufficiently low (typically <10%) as to raise ethical concerns to continue.

     

    Commercial Eyes’ experienced Clinical Development team can help you navigate the data and safety monitoring requirements of your clinical trials. Call us on +61 3 9251 0777 to discuss your needs.

    This article was written by Ric DeGaris, a member of our Clinical Development team.




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