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EMA to have debate on the use of epidemiology in the assessment of drug effects

admin — Fri, 03 Sep 2010 09:29:17 +0000

EMA to have debate on the use of epidemiology in the assessment of drug effects.

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The European Medicines Agency is organising a public debate on the use of epidemiology in the assessment of drug effects at the International Conference on Pharmacoepidemiology & Therapeutic Risk Management congress in Brighton on 20 August 2010. The debate will be moderated by Hans-Georg Eichler, the Agency’s Senior Medical Officer. The Executive Director of the European Medicines Agency, Thomas Lönngren, will deliver the keynote address at ICPE on the same day. Participation is free to attendees of the ICPE congress.

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EMA and FDA Seek Candidate for Joint GMP Inspection Programme

admin — Thu, 02 Sep 2010 09:26:20 +0000

EMA and FDA Seek Candidate for Joint GMP Inspection Programme.

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The European Medicines Agency (EMA) and the Food and Drug Administration of the United States of America (US FDA) continue to seek potential candidate companies for a joint GMP inspection pilot programme for manufacturers of medicinal products. Companies that have submitted in parallel two equivalent marketing authorisation applications for the same medicinal product to both the EMA and the US FDA can request to participate in the pilot programme for joint pre-approval inspection should such an inspection be considered necessary by both agencies.

The overall objective is to see whether greater international collaboration can help to distribute inspection capacity allowing more manufacturing sites to be monitored and reducing unnecessary duplication.

Companies can also participate in the pilot exercise by hosting a single join re-inspection (routine surveillance) where both the EMA and the US FDA have separately planned routine surveillance inspections (re-inspections) to take place within a similar time period at a manufacturing site of a medicinal product authorised in the USA and centrally authorised in the European Union.

Companies that wish to participate should contact either gmp@ema.europa.eu and/or CDERInternationalGMP@fda.hhs.gov.

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The FDA, Issues Assessments of the 510(k) Programme and Use of Science in Decision-Making

admin — Wed, 01 Sep 2010 09:25:10 +0000

The FDA, Issues Assessments of the 510(k) Programme and Use of Science in Decision-Making.

The U.S. Food and Drug Administration today issued two comprehensive evaluations containing recommendations that address three key objectives of the agency’s public health mission as it relates to medical devices – foster device innovation, create a more predictable regulatory environment, and enhance device safety.

The FDA’s Center for Devices and Radiological Health assessment consists of two preliminary reports. One report focuses on ways to strengthen and clarify a premarket review process called the 510(k) program for medical devices that do not need to undergo a full premarket approval review. The other evaluates CDRH’s use of science in decision-making, with an eye toward adapting to new scientific information, while maintaining regulatory predictability necessary for innovation.

The two documents overlap in several places and cross-reference information. The documents can be found online.

CDRH established two staff committees on these issues in late 2009 as part of its 2010 strategic plan. The committees collected and reviewed input from public meetings, open dockets, data analyses, and input from CDRH staff over the course of several months to prepare the complementary evaluations.

In recent years, concerns have been raised both inside and outside of the FDA about whether the current 510(k) program achieves its goals of making safe and effective devices available to the public while fostering innovation. Concerns about the program have centered on whether it allows devices to enter the market without sufficient safety and effectiveness evidence and whether a lack of predictability, consistency, and transparency is hindering device development.

CDRH uses science to guide its regulation of medical devices across the total product lifecycle. At any stage of that lifecycle, new, unfamiliar or unexpected scientific information may arise that warrants a change in the FDA’s thinking, expectations, and actions. CDRH is seeking to strike the right balance between the ability to adapt its approach as new science emerges and to provide predictable regulatory pathways.

“Taken together, these preliminary reports show a smarter FDA – an agency that recognizes both sides of our mission to protect and promote public health,” said CDRH Director Jeffrey Shuren, M.D. “The agency is ready to make necessary improvements to support device innovation while assuring patients receive safe and effective devices.

“Even with our significant outreach, it’s important to remember that these recommendations are preliminary,” said Shuren. “CDRH opened another public docket to receive additional comments on both reports. We will make a decision on which recommendations to adopt only after a thorough review of additional comments.”

Selected recommendations and the key public health objectives they address include:.

Fostering Device Innovation

The 510(k) report recommends major improvements to the regulatory pathway for lower-risk novel devices that cannot be cleared through 510(k) but which do not warrant the more rigorous premarket approval review applied to higher-risk devices. The report calls for major reforms in the implementation of this process – called the de novo classification process. The recommendations include streamlining the process and clarification of CDRH’s expectations for submissions that undergo this type of review.
The science report recommends that CDRH make better use of scientific experts outside of the agency by developing a web-based network of external experts using social media technology. This network would help CDRH staff leverage outside knowledge without serving in an advisory capacity.

Enhancing Regulatory Predictability

The 510(k) report recommends that CDRH develop a guidance document defining a subset of moderate-risk (Class II) devices, called Class IIb, for which clinical or manufacturing data typically would be necessary to support a substantial equivalence determination. This guidance document would help clarify what information submitters should include in their 510(k) submissions so that they can plan accordingly. In addition, this would also help the center’s review staff obtain the type and level of evidence necessary to make well-supported decisions without as much need for time-consuming follow-up requests for information.
The science report recommends use of a standardized “Notice to Industry” letter that would generally be issued as a “Level 1 – Immediately in Effect” guidance document to quickly communicate when CDRH has changed its premarket regulatory expectations due to scientific information that has emerged about a certain device type. CDRH currently communicates this kind of information through individual interactions during the review process, which can lead to delays. These letters would provide greater clarity to affected manufacturers, in a timelier manner, about CDRH’s expectations with respect to a particular group of devices.

Improving Patient Safety

The 510(k) report recommends that CDRH consider revising regulations to explicitly require 510(k) submitters to provide a summary of all scientific information known or that the submitter should reasonably know regarding the safety and effectiveness of the device under review. This is not required now for 510(k) submissions and, as a result, relevant information may not be included in an initial submission. This summary would help CDRH review staff to more efficiently make decisions, and potentially avoid extensive follow-up inquiries and questions.
The 510(k) report recommends that CDRH develop a guidance document that clarifies when a device should not be used as a predicate, such as when the device has been removed from the market because of safety concerns. The report also recommends that the center consider issuing a regulation that would clarify the circumstances under which the center would exercise its authority to rescind a 510(k) clearance to remove an unsafe device from the market and preclude its use as a predicate and also consider whether additional authority is needed.
Both reports recommend that CDRH build upon public databases to include meaningful, up-to-date information that supports good decision making and promotes the safe use of devices. This could be accomplished by improving the current 510(k) database so that it includes summaries of FDA review decisions, current labeling and photos. In addition, the science report recommends that CDRH build upon the existing transparency website to provide more immediate information on how devices are regulated.

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EMA THe European Drug Regulator, Publishes CHMP Efficacy Working Party Therapeutic Subgroup on Pharmacokinetics (EWP-PK) Q&A on Positions on Specific Questions Addressed

admin — Tue, 31 Aug 2010 14:57:51 +0000

EMA The European Drug Regulator, Publishes CHMP Efficacy Working Party Therapeutic Subgroup on Pharmacokinetics (EWP-PK) Q&A on Positions on Specific Questions Addressed

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In the context of assessment procedures, the Therapeutic Subgroup on Pharmacokinetics of the Efficacy Working Party (EWP-PK subgroup) is occasionally consulted by the CHMP or, following CHMP’s agreement, by other Committees, Working parties or the CMD(h). The objective is to address specific questions in relation to pharmacokinetic evaluations and particularly the requirements and assessment of bioequivalence studies. The positions, which are being elaborated by the EWP-PK subgroup in response to such questions, are being forwarded to the enquiring party for consideration in their assessment.
It is understood that such position will be reflected in the procedure-related assessment reports if applicable. In some cases however, these position might also be of more general interest as they interpret a very specific aspect that would not necessarily be covered by guidelines. This paper summarises these positions which have been identified as being within this scope. It should be noted that these positions are based on the current scientific knowledge as well as regulatory precedents. They should be read in conjunction with the applicable guidelines on bioequivalence in their current version. As the questions have initially been raised in the context of specific assessment procedures, details of these procedures have been redacted for reasons of confidentiality. This compilation will be updated with new positions as soon as they become available. Likewise, if a position is being considered outdated, e.g. due to new evolutions in the scientific knowledge including revisions to the applicable guidelines, positions will be removed from this document. The positions in this document are addressing very specific aspects. They should not be quoted as product-specific advice on a particular matter as this may require reflection of specific data available for this product. By no means should these positions be understood as being legally enforceable.

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Testimonial SkinSecure – Joanne Reed

admin — Tue, 31 Aug 2010 09:46:14 +0000

Testimonial SkinSecure – Joanne Reed

Damien helped me pull together my business plan in a quick and effortless way, he got the information from my head and down onto the page in a logical way that told the story that my funders needed to see. I was stuck before working with Damien, but he helped me overcome the writers block to produce a fantastic business plan. – Joanne Reed CEO SkinSecure ltd

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EMA, The European Drug Regulator, Publish Overview of Comments Received on “Guideline on the clinical investigation of Medicinal Products for the Treatment of Attention Deficit Hyperactivity Disorder, (ADHD)

admin — Mon, 30 Aug 2010 14:53:22 +0000

EMA, The European Drug Regulator, Publish Overview of Comments Received on “Guideline on the clinical investigation of Medicinal Products for the Treatment of Attention Deficit Hyperactivity Disorder, (ADHD)

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Stakeholder no. Name of organisation or individual
1 European College of Neuropsychopharmacology (ECNP) I
2 European College of Neuropsychopharmacology (ECNP) II
3 Shire Pharmaceuticals
4 European Association for Clinical Pharmacology and Therapeutics (EACPT)
5 EFPIA
6 EUNETHYDIS Guidelines Group
7 International Federation of Associations of Pharmaceutical Physicians (IFAP)
8 J&J
9 H. LUNDBECK A/S
10 M. Rösler, W. Retz, R.-D. Stieglitz
11 F. Hoffmann-La Roche Ltd.

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NICE, the UK HMO, Publishes full Guidance on the Diagnosis and Management of Metastatic Malignant Disease of Unknown Primary Origin

admin — Mon, 30 Aug 2010 09:38:37 +0000

NICE, the UK HMO, Publishes full Guidance on the Diagnosis and Management of Metastatic Malignant Disease of Unknown Primary Origin.

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Every hospital with a cancer centre or unit should establish a carcinoma of unknown primary (CUP) team, and ensure that patients have access to the team when malignancy of undefined primary origin (MUO) is diagnosed. The team should:
− consist of an oncologist, a palliative care physician and a CUP specialist nurse or key worker as a minimum
− have administrative support and sufficient designated time in their job plans for this specialist role and
− have a named lead clinician.

Every hospital with a cancer centre or unit should assign a CUP specialist nurse or key worker to patients diagnosed with MUO or CUP. The CUP specialist nurse or key worker should:
− take a major role in coordinating the patient’s care in line with this guideline
− liaise with the patient’s GP and other community support services
− ensure that the patient and their carers can get information, advice and support about diagnosis, treatment, palliative care, spiritual and psychosocial concerns
− meet with the patient in the early stages of the pathway and keep in close contact with the patient regularly by mutual agreement and
− be an advocate for the patient at CUP team meetings.

Refer outpatients with MUO to the CUP team immediately using the rapid referral pathway for cancer, so that all patients are assessed within 2 weeks of referral. A member of the CUP team should assess inpatients with MUO by the end of the next working day after referral. The CUP team should take responsibility for ensuring that a management plan exists which includes:
− appropriate investigations
− symptom control
− access to psychological support and
− providing information.

A CUP network multidisciplinary team (MDT) should be set up to review the treatment and are of patients with confirmed CUP, or with MUO or provisional CUP and complex diagnostic or treatment issues. This team should carry out established specialist MDT responsibilities.

Every cancer network should establish a network site-specific group to define and oversee policies for managing CUP. The group should:
− ensure that every CUP team in the network is properly set up (see recommendation on page 15)
− ensure that the local care pathway for diagnosing and managing CUP is in line with this guideline
− be aware of the variety of routes by which newly diagnosed patients present
− advise the cancer network on all matters related to CUP, recognising that many healthcare professionals have limited experience of CUP

maintain a network-wide audit of the incidence of CUP, its timely management, and patient outcomes
− arrange and hold regular meetings for the group to report patient outcomes and review the local care pathway.

Offer the following investigations to patients with MUO, as clinically appropriate, guided by the patient’s symptoms:
− comprehensive history and physical examination including breast, nodal areas, skin, genital, rectal and pelvic examination
− full blood count; urea, electrolytes and creatinine; liver function tests; calcium; urinalysis; lactate dehydrogenase
− chest X-ray
− myeloma screen (when there are isolated or multiple lytic bone lesions)
− symptom-directed endoscopy
− computed tomography (CT) scan of the chest, abdomen and pelvis
− prostate-specific antigen (PSA) in men (see recommendation on page 24)
− cancer antigen 125 (CA125) in women with peritoneal malignancy or ascites (see recommendation on page 24)
− alpha-fetoprotein (AFP) and human chorionic gonadotrophin (hCG) (particularly in the presence of midline nodal disease) (see recommendation on page 24)
− testicular ultrasound in men with presentations compatible with germ-cell tumours
− biopsy and standard histological examination, with immunohistochemistry where necessary, to distinguish carcinoma from other malignant diagnoses.

Do not use gene-expression-based profiling to identify primary tumours in patients with provisional CUP

Perform investigations only if:
− the results are likely to affect a treatment decision
− the patient understands why the investigations are being carried out
− the patient understands the potential benefits and risks of investigation and treatment and
− the patient is prepared to accept treatment.

Include the patient’s prognostic factors in decision aids and other information for patients and their relatives or carers about treatment options.

If chemotherapy is being considered for patients with confirmed CUP, with no clinical features suggesting a specific treatable syndrome, inform patients about the potential
benefits and risks of treatment.

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EMA, Publishes Guidance on the Clinical Investigation of Medicinal Products for the Treatment of Attention Deficit Hyperactivity Disorder (ADHD)

admin — Sun, 29 Aug 2010 14:48:40 +0000

EMA, Publishes Guidance on the Clinical Investigation of Medicinal Products for the Treatment of Attention Deficit Hyperactivity Disorder (ADHD)

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Attention Deficit Hyperactivity Disorder (ADHD) is among the most common disorders in child- and adolescent psychiatry. Its impact on learning and development is considered substantial. The benefit of pharmacotherapy has empirically been proven, and several products are on the market. Although primarily a disorder diagnosed in childhood and adolescence, signs and symptoms may not be selflimiting but may persist into adulthood. These new insights in the ADHD syndrome are a challenge in the field of drug development. However, this Guideline is intended to provide guidance on the evaluation of new medicinal products in ADHD with focus on the childhood onset. It is the first guideline written in psychiatry to address a (child) psychiatric disorder from this perspective, and it should be read in conjunction with other EMA and ICH guidelines, which may apply to similar conditions and patient populations.

Attention Deficit Hyperactivity Disorder, ADHD, is a well defined disorder with core features of inattention, hyperactivity, and impulsivity, but also impairment in executive functions. It has its origin in childhood and is often diagnosed for the first time in school-aged children because of learning problems and problems with social behaviour. Treatment is therefore directed towards improvement of attention and reduction of hyperactivity/impulsivity in order to be able to focus on tasks and performance, and improve associated behavioural and relational problems. Methylphenidate is among the first effective drugs reported to treat the ‘hyperkinetic syndrome’ in the 1950s. Although often regarded as the standard of treatment, new products have come to the market, e.g. atomoxetine with a different mode of action. Psycho-education, and psycho-education in combination with pharmacotherapy are usually the standard of care in Europe, and behavioural treatment is often provided to sustain success of pharmacotherapy, and to modify conduct problems. In the context of non-pharmacological interventions, cognitive treatment, neurofeedback training and dietary measures can be regarded as potential, but not yet evidence based strategies. It has long been assumed that the core symptoms of ADHD ameliorate with age. It has recently been recognized that symptoms and impairments may persist into adulthood, thereby extending treatment to this age group. Usually, inattention and restlessness predominate at adult age, interfering with work and social functioning. As ADHD is a chronic disorder, long term treatment can be foreseen, thereby emphasizing the need for
long term safety data in a group of patients that does include many otherwise healthy individuals.

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NICE, the UK HMO, Publishes Guidance on Dietary Interventions and physical activity interventions for weight management before, during and after pregnancy

admin — Sun, 29 Aug 2010 09:32:02 +0000

NICE, the UK HMO, Publishes Guidance on Dietary Interventions and physical activity interventions for weight management before, during and after pregnancy

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The Department of Health (DH) asked the National Institute for Health and Clinical Excellence (NICE) to produce public health guidance on dietary and physical activity interventions for weight management before, during and after pregnancy.
The guidance does not cover:
• women who are underweight (that is, those who have a body mass index [BMI] less than 18.5 kg/m²)
• clinical management of women who are obese during pregnancy
• those who have been diagnosed with, or who are receiving treatment for, an existing condition such as type 1 or type 2 diabetes
• food safety advice.
The guidance is for NHS and other commissioners, managers and professionals who have a direct or indirect role in, and responsibility for, women who are pregnant or who are planning a pregnancy and mothers who have had a baby in the last 2 years. This includes those working in local authorities, education and the wider public, private, voluntary and community sectors.
It is particularly aimed at: GPs, obstetricians, midwives, health visitors, dietitians, community pharmacists and all those working in antenatal and postnatal services and children’s centres. It may also be of interest to women before, during and after pregnancy and their partners and families, and other members of the public.
The guidance complements but does not replace NICE guidance on: obesity, maternal and child nutrition, antenatal care, postnatal care, physical activity, behaviour change, antenatal and postnatal mental health and diabetes in pregnancy (for further details, see section 7).

NICE public health guidance 27: Weight management before, during and after
pregnancy
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The Public Health Interventions Advisory Committee (PHIAC) developed these recommendations on the basis of a review of the evidence, economic modelling, expert advice, stakeholder comments and fieldwork.
Members of PHIAC are listed in appendix A. The methods used to develop the guidance are summarised in appendix B.
Supporting documents used to prepare this document are listed in appendix E. Full details of the evidence collated, including fieldwork data and activities and stakeholder comments, are available on the NICE website, along with a list of the stakeholders involved and NICE’s supporting process and methods manuals. The website address is: www.nice.org.uk

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EMA, The European Drug Regulator Publishes Recommendation on Elements Required to Support the Medical Plausibility and the Assumption of Significant Benefit for an Orphan Designtion

admin — Sat, 28 Aug 2010 14:40:15 +0000

EMA, The European Drug Regulator Publishes Recommendation on Elements Required to Support the Medical Plausibility and the Assumption of Significant Benefit for an Orphan Designtion

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According to the European Parliament and Council Regulation (EC) 141/2000 (Art 5) and the Commission Regulation (EC) 847/2000 “a sponsor applying for designation of a medicinal product shall apply for designation at any stage of the development of the medicinal product before the application for marketing authorization is made”. Furthermore, in the criteria for designation (Article 3 of Regulation (EC) 141/2000) it is stated that a medicinal product shall be designated as an orphan medicinal product if its sponsor can establish that “there exists no satisfactory method of diagnosis, prevention or treatment of the condition in question that has been authorized in the European Union or, if such method exists, that the medicinal product will be of significant benefit to those affected by that condition”. This Discussion Paper has two aims. Firstly, to outline the level of evidence normally required to support the medical plausibility of using the product in the applied condition, and secondly, the level of evidence required to support the assumption of significant benefit. The paper is based on the experience accumulated over recent years with several hundred orphan drug designation applications, approximately 70% of which included a discussion on significant benefit since satisfactory methods for diagnosis, prevention or treatment existed in the European Union at the time of the submission of the application. General guidance is already available on what is considered necessary to support ‘medical plausibility’ at the time of the submission of an orphan designation application and on what is necessary for the justification of the assumption of ‘significant benefit’ if this criterion applies. This is included in the “Commission Guideline on the format and content of applications for designation as orphan medicinal products and on the transfer of designations from one sponsor to another” (ENTR/6283/00) and in the “Communication from the Commission on Regulation (EC) 141/2000 of the European parliament and of the Council on orphan medicinal products” (Commission Communication 2003/C 178/02 of 29 July 2003). This discussion paper should be read in conjunction with these documents. According to the Commission Guideline (ENTR/6283/00), the medical plausibility section should be completed for all applications. There are two aspects to “Medical Plausibility”: (1) the rationale for use of the medicinal product in the proposed orphan indication; and (2) where the orphan indication refers to a subset of a particular condition, a justification of the medical plausibility for restricting the medicinal product in the sub-set. The ‘rationale for development’ is closely and necessarily linked with both the nature of an orphan drug as a ‘medicinal product’ and with the designation criterion set out in Article 3.1(a) of Regulation (EC) No 141/2000.

A product which is the subject of such application must be a medicinal product as defined in Article 1, Directive 2001/83/EC and consideration of the ‘medical plausibility’ at an early stage of product development provides a means of verifying this. Article 3.1 which lays down the criteria for designation states that “a medicinal product shall be designated as an orphan medicinal product if its sponsor can establish: that it is intended for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition….”. Based on this wording, the Committee for Orphan Medicinal Products (COMP) will consider the notion of ‘medical plausibility’ when assessing an application for designation.The Commission Communication (2003/C 178/02) section B.1, furthermore, recognises that the COMP may take into account available data to modify the condition under application (for example, because the Committee considers that the designatable condition is broader than the one under application). To
define a suitable condition for designation, the COMP must look at the rationale for development of the medicinal product in the proposed orphan indication. This is imperative to prevent the slicing of common conditions into invalid sub-sets (e.g. different stages of a condition such as “metastatic cancer”; subgroups of frequent diseases where the product would have interest in the rest of the disease; conditions defined based on the therapeutic use of the product such as “treatment in patientsnot responding to X”). It is important that sponsors, when preparing designation applications, are aware that this is an important issue that will be reviewed by the Committee. It should be noted that for the purpose of designation and to support the rationale for the development of the product in the proposed condition some preliminary preclinical or clinical data are generally required. A pharmacological concept, not supported by any form of evidence, would generally not be considered by the COMP as sufficient justification for the designation of the medicinal product in the proposed condition. Article 3(1)b of Regulation EC 141/2000 states that in the case where a satisfactory method of
diagnosis, prevention or treatment of the condition exists, the sponsor has to establish ‘that the medicinal product will be of significant benefit to those affected by that condition’. In the Commission Communication it is stated, “a treatment for a particular disease or condition may be associated with certain risks. These risks are balanced against the expected benefits when considering whether to grant or refuse a marketing authorisation in accordance with the criteria of safety, quality and efficacy as laid down in Directive 2001/83/EC. A marketing authorisation is granted if the benefit risk assessment is positive”. As mentioned in the Commission Regulation (EC) 847/2000, authorised medicinal products are therefore considered satisfactory methods of diagnosis, prevention or treatment. Commonly used methods of diagnosis, prevention or treatment that are not subject to marketing authorization (e.g. surgery, medical devices) may be also considered satisfactory methods, if there is scientific evidence as to the value of those methods. Significant benefit is defined in Commission Regulation (EC) 847/2000 as ‘a clinically relevant advantage or a major contribution to patient care.’ The applicant is required to justify the assumption that the medicinal product will be of significant benefit compared to the existing authorized medicinal products or methods at the time of designation. As there may be little or no clinical experience with the orphan medicinal product in question, the justification for significant benefit is likely to be made on assumptions of benefit by the applicant. As stated in the Guideline (ENTR/6283/00), at the time of designation “significant benefit should be based on well justified assumptions. Assumptions of potential benefit(s) should be plausible and where possible based on sound pharmacological principles.” In the same Guideline it is also stated that “In general a demonstration of potentially greater efficacy, an improved safety profile, and/or more favourable pharmacokinetic properties than existing methods may be considered to support the notion of significant benefit.” In addition, the Commission Communication on Regulation (EC) No 141/2000 gives some clarification on the possibility to base the significant benefit on the availability of the medicinal product (e.g. European Union availability versus availability in one Member State; supply insufficient to meet patients’ needs with the exclusion of either transient or artificial problems in supply), documented safety problems in relation to the origin of the medicinal product; serious and documented difficulties with the formulation or route of administration; long term interruption in supply of an authorized product; favorable and clinically relevant pharmacokinetic properties. In all cases the COMP is required to assess whether or not these assumptions are plausible and are supported in the application by appropriate evidence.

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