Compassionate use is a treatment option….allows the use of an unauthorised medicine.

 EMA, EU  Comments Off on Compassionate use is a treatment option….allows the use of an unauthorised medicine.
Aug 202014




Compassionate use is a treatment option that allows the use of an unauthorised medicine. Compassionate-use programmes are for patients in the European Union (EU) who have a disease with no satisfactory authorised therapies or cannot enter aclinical trial. They are intended to facilitate the availability to patients of new treatment options under development.



Compassionate-use programmes are often governed by legislation in individual EU Member States, to make medicines available on a named-patient basis or to cohorts of patients.

In addition to this, EU legislation provides an option for Member States to ask the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) to provide an opinion to all EU Member States on how to administer, distribute and use certain medicines for compassionate use. The CHMP also identifies which patients may benefit from compassionate-use programmes. This is described in Article 83 of Regulation (EC) No 726/2004External link icon and is complementary to national legislation.



The objectives of Article 83 are to:

  • facilitate and improve access to compassionate-use programmes by patients in the EU;
  • favour a common approach regarding the conditions of use, the conditions for distribution and the patients targeted for the compassionate use of unauthorised new medicines;
  • increase transparency between Member States in terms of treatment availability.

More information is available in:



Compassionate-use opinions from the CHMP

Name of medicine Ledipasvir/Sofosbuvir
Active substance ledipasvir, sofosbuvir
Dosage 90mg / 400 mg
Pharmaceutical form Film coated tablet
Member State notifying the Agency Ireland
CHMP opinion documents Conditions of use, conditions for distribution and patients targeted and conditions for safety monitoringSummary on compassionate use
Date of opinion 20/02/2014
Company contact information Gilead Sciences Limited
Granta Park
CB21 6GT
United Kingdom
Tel. +44 (0)208 5872206
Fax +44 (0)1223 897233
Status Ongoing
Related documents  –


Name of medicine Daclatasvir
Active substance daclatasvir
Dosage 30 and 60 mg
Pharmaceutical form Film coated tablet
Member State notifying the Agency Sweden
CHMP opinion documents Conditions of use, conditions for distribution and patients targeted and conditions for safety monitoringSummary on compassionate use
Date of opinion 21/11/2013
Company contact information Bristol-Myers Squibb Pharma EEIG
Uxbridge Business Park
Sanderson Road
Uxbridge UB8 1DH
United Kingdom
Tel. +44 (0)1895 523 740
Fax +44 (0)1895 523 677
Status Ongoing
Related documents  –


Name of medicine Sofosbuvir Gilead
Active substance Sofosbuvir
Dosage 400 mg
Pharmaceutical form Film-coated tablet
Member State notifying the Agency Sweden
CHMP opinion documents Conditions of use, conditions for distribution and patients targeted and conditions for safety monitoring
Summary on compassionate use
Date of opinion 24/10/2013
Company contact information Gilead Sciences International Ltd
Granta Park, Abington
Cambridgeshire CB21 6GT
United Kingdom
Tel. +44 (0)1223 897496
Fax +44 (0)1223 897233
Status Ongoing
Related documents  –


Name of medicine IV Zanamivir
Active substance Zanamivir
Dosage 10 mg/ml
Pharmaceutical form Solution for infusion
Member State notifying the Agency Sweden
CHMP opinion documents Conditions of use, conditions for distribution and patients targeted and conditions for safety monitoring
Summary on compassionate use
Date of opinion 18/02/2010
Company contact information GlaxoSmithKline Research & Development Limited
980 Great West Road, Brentford
Middlesex TW8 9GS
United Kingdom
Tel. +44 (0)20 8047 5000 or +44 (0)20 8990 3885
Status Ongoing
Related documents  –


Name of product Tamiflu IV
Active substance Oseltamivir phosphate
Dosage 100 mg
Pharmaceutical form Powder for solution for infusion
Member State notifying the Agency Finland
CHMP opinion documents Conditions of use, conditions for distribution and patients targeted and conditions for safety monitoring
Summary on compassionate use
Date of opinion 20/01/2010
Company contact information F. Hoffmann-La Roche Ltd.
Pharmaceuticals Division
PBMV Bldg 74/3O 104
CH-4070, Basel
Tel. +41 61 688 5522
Fax +41 61 687 2239
Status Closed
Related documents Public statement on Tamiflu IV: Closure of compassionate-use programme in the EU
Tamiflu IV compassionate-use programme EMEA/H/K/002287 – Closure of programme



Expanded access (also known as compassionate use) refers to the use of an investigational drug outside of a clinical trial by patients with serious or life-threatening conditions who do not meet the enrollment criteria for the clinical trial in progress. Outside the US, such access is allowed through Named patient programs. In the US this type of access may be available, in accordance with United States Food and Drug Administration (FDA) regulations, when it is clear that patients may benefit from the treatment, the therapy can be given safely outside the clinical trial setting, no other alternative therapy is available, and the drug developer agrees to provide access to the drug. The FDA refers to such a program as an expanded access program (EAP).[1] EAPs can be leveraged in a wide range of therapeutic areas including HIV/AIDS and other infectious diseases, cancer, rare diseases, and cardiovascular diseases, to name a few.

There are several types of EAPs allowed in the United States. Treatment protocols and treatment INDs provide large numbers of patients access to investigational drugs. A single-patient IND is a request from a physician to the FDA that an individual patient be allowed access to an investigational drug on an emergency or compassionate use basis.[2] When the FDA receives a significant number of requests (~10 to 100) for individual patient expanded access to an investigational drug for the same use, they may ask the trial sponsor to consolidate these requests, creating an intermediate-size group.[3] “Compassionate use” is a more colloquial term that is not generally used by the FDA.

FDA regulations

Since 1987, the FDA has had rules in place that have enabled patients, under specific circumstances, to access drugs or biologics that are still in development for treatment purposes. These expanded access program rules were amended in 2009 by the FDA to ensure “broad and equitable access to investigational drugs for treatment.”[4]

The regulations include the following:[4]

  • Criteria that must be met in order to authorize the expanded access use
  • Requirements for expanded access submissions
  • Safeguards to protect patients and the clinical trial process

The regulations also include general criteria for granting expanded access:[3]

  • The patient must have a serious condition or disease for which there is no comparable alternative therapy available
  • The patient must be unable to participate in a clinical trial
  • The potential benefit must outweigh the potential risk of using the treatment
  • There should be no impact on the completion of the clinical trial or the drug’s approval

Despite the updated regulations, debate remains over key elements of expanded access:

  • Deciding at what point in the clinical trial process access should be given. Some stakeholders support expanded access programs after phase I testing in clinical trials. The FDA has stated that most drugs should not be eligible until some point during phase III when efficacy data have been obtained, unless compelling phase II data on safety and efficacy are available.[3][5]
  • Weighing risks to the patient against the potential benefits. The FDA requires that a physician and an institutional review board (IRB) determine that a treatment will not pose undue risk to the patient, relative to the condition he or she is suffering from.[6] However, the FDA maintains the right to overrule the physician and IRB.[3]
  • Determining who should get access. The FDA states that expanded access should only be considered for patients with a serious disease or condition, but the FDA’s rules do not provide a definition of “serious”; instead it provides examples of diseases and conditions that fall into this category.[3] In the case of a cancer drug, the sponsor of an expanded access program must define exactly which patients will get access. Most often, access is limited to those patients with the same type of cancer the drug is being tested for.[7]

A number of challenges can exist when patients seek access to investigational drugs:

  • Obtaining an IRB review. Finding time on an IRB’s schedule can be difficult, particularly for doctors who are not based at research centers where IRBs are readily available. The fee for the review may pose a problem as well. It may be unclear who is responsible for the cost of the IRB review, which can be as much as $2,000. Many IRBs conduct reviews pro bono but others that charge will often only waive their fees for research done in their hospital.[6][8]
  • Protecting physicians against liability risk. Currently, physicians may be concerned that they could face a liability risk for investigational drugs that they recommend to patients or help them gain access to, potentially discouraging them from doing so. The FDA does not have jurisdiction over this issue but there is a bill in Congress, the Compassionate Access Act of 2010 (H.R. 4732), that would address the situation.[6][8][9]
  • Paying for the drug. While the FDA allows drug companies to recover the costs of providing a treatment through an EAP, many companies may hesitate to do so because it requires disclosing the cost of their drug, which is often a closely guarded secret. In addition, many insurance companies won’t cover the costs of experimental treatment so access could be limited to patients with the means to pay for it.[6][8]
  • Assessing the potential impact of adverse events on drug development. Adverse events (AEs) that result from expanded access programs must be reported to the FDA in the same way AEs are reported in the case of a clinical trial. The FDA states that, to their knowledge, no drug candidate has been turned down for approval because of an adverse event that appeared in an expanded access program.[3][6]

Outside the United States

Outside the U.S., programs that enable access to drugs in the pre-approval and pre-launch phase are referred to by a variety of names including “named patient programs,” “named patient supply” and “temporary authorization for use.”[10] In the EU, named patient programs also allow patients to access drugs in the time period between centralized European Medicines Agency (EMEA) approval and launch in their home countries which can range from one year to eighteen months.[11]


  1. Jump up^ US National Cancer Institute – Access to Investigational Drugs accessed April 22, 2007
  2. Jump up^ FDA Final Rules for Expanded Access to Investigational Drugs for Treatment Use and Charging for Investigational Drugs
  3. Jump up to:a b c d e f Final FDA Rules on Expanded Access to Investigational Drugs for Treatment Use
  4. Jump up to:a b FDA website
  5. Jump up^ Expanded Access to Investigational Drugs Genetic Engineering & Biotechnology News, January 15, 2010.
  6. Jump up to:a b c d e Access to Investigational Drugs Remains Challenge Despite New FDA Rules ‘’The Pink Sheet’’
  7. Jump up^ Managing Access to Drugs Prior to Approval and Launch ‘’Life Science Leader’’[dead link]
  8. Jump up to:a b c FDA webinar accessed May 5, 2010
  9. Jump up^ FDA Law Blog accessed May 5, 2010
  10. Jump up^ Helene S (2010). “EU Compassionate Use Programmes (CUPs): Regulatory Framework and Points to Consider before CUP Implementation”Pharm Med 24 (4): 223–229.
  11. Jump up^ [Ericson, M., Harrison, K., Laure, N. & De Crémiers, F., Compassionate Use Requirements in the Enlarged European Union. RAJ Pharma, May 2005: 83.

External links



Road map to 2015, The European Medicines Agency’s contribution to science, medicines and health

 EU, regulatory, Uncategorized  Comments Off on Road map to 2015, The European Medicines Agency’s contribution to science, medicines and health
Aug 182014


One of the European Medicines Agency’s long-term strategic goals is to foster researchand the uptake of innovative methods in the development of medicines.

READ………….Road map to 2015

The European Medicines Agency’s
contribution to science, medicines and health……………..

This helps the Agency to meet its objective of making safe and effective medicines available to patients in a timely manner, following evaluation using state-of-the-art methods.

The Agency also supports the development of new therapies and technologies by working with interested parties in the European Union (EU).

Activities at the Agency

In 2004, the Agency set up the European Medicines Agency/Committee for Medicinal Products for Human Use (CHMP) Think-Tank Group on Innovative Drug Development.

This group included Agency staff and members of the CHMP and its working parties. Its work focused on identifying scientific bottlenecks and emerging science in the development of medicines, both in industry research and development and in academia, and on generating recommendations for future activities at the Agency:

In 2008 the EMA and its Scientific Committees integrated the recommendations made by the Think Tank in its strategy for supporting innovative medicines developments. Key areas of action included the strengthening of the EU scientific network model, emphasis on communication during the lifecycle of medicinal products development and international activities. Overview of measures implemented in the period 2008-2010.

The recently published Road Map to 2015 further expands on the role the Agency plays to promote innovation in pharmaceuticals.

The Agency also contributes to the Innovative Medicines InitiativeExternal link icon (IMI). This is a public-private initiative that aims to speed up the development of better and safer medicines for patients:

Support for business

The Agency provides support for business on issues related to innovative medicines:


European Medicines Agency …Clinical trials in human medicines

 EMA, EU  Comments Off on European Medicines Agency …Clinical trials in human medicines
Aug 132014


The European Medicines Agency relies on the results of clinical trials carried out by pharmaceutical companies to reach its opinions on the authorisation of medicines. Although the authorisation of clinical trials occurs at Member State level, the Agency plays a key role in ensuring that the standards of good clinical practice (GCP) are applied across the European Economic Area in cooperation with the Member States. It also manages a database of clinical trials carried out in the European Union.

Clinical trials are studies that are intended to discover or verify the effects of one or more investigational medicines. The regulation of clinical trials aims to ensure that the rights, safety and well-being of trial subjects are protected and the results of clinical trials are credible.

Regardless of where they are conducted, all clinical trials included in applications for marketing authorisation for human medicines in the European Economic Area (EEA) must have been carried out in accordance with the requirements set out in Annex 1 ofDirective 2001/83/ECExternal link icon. This means that:

In the EEA, approximately 4,000 clinical trials are authorised each year. This equals approximately 8,000 clinical-trial applications, with each trial involving two Member States on average. Approximately 61% of clinical trials are sponsored by the pharmaceutical industry and 39% by non-commercial sponsors, mainly academia.

Role of the Agency

Clinical-trial data is included in clinical-study reports that form a large part of the application dossiers submitted by pharmaceutical companies applying for a marketing authorisation via the Agency.

The Agency’s Committee for Medicinal Products for Human Use (CHMP) is responsible for conducting the assessment of a human medicine for which an EU-wide marketing authorisation is sought. As part of its scientific evaluation work, the CHMP reviews the clinical-trial data included in the application.

Assessments are based on purely scientific criteria and determine whether or not the medicines concerned meet the necessary quality, safety and efficacy requirements in accordance with EU legislation, particularly Directive 2001/83/ECExternal link icon.

Good clinical practice

The Agency plays a central role in ensuring application of good clinical practice (GCP). GCP is the international ethical and scientific quality standard for designing, recording and reporting clinical trials that involve the participation of human subjects.

The Agency works in cooperation with GCP inspectors from medicines regulatory authorities (‘national competent authorities’) in EEA Member States on the harmonisation and coordination of GCP-related activity at an EEA level.

The Agency does not have a role in the approval of clinical-trial applications in the EEA. The approval of clinical-trial applications is the responsibility of the national competent authorities.

EudraCT database and the EU Clinical Trials Register

The Agency is responsible for the development, maintenance and coordination of the EudraCT database. This is a database used by national competent authorities to enter clinical-trial data from clinical trial sponsors and paediatric-investigation-plan (PIP) addressees.

A subset of this data is made available through the European Union Clinical Trials Register, which the Agency manages on behalf of EU Member States and forms part ofEudraPharmExternal link icon, the EU database of medicines.

Users are able to view:

  • the description of phase-II to phase-IV adult clinical trials where the investigator sites are in the EEA;
  • the description of any clinical trials in children with investigator sites in the EU and any trials that form part of a PIP including those where the investigator sites are outside the EU.

As of 21 July 2014, it will be mandatory for sponsors to post clinical trial results in the EudraCT database. A subset of the data included in EudraCT is made available to the public in the European Union Clinical Trials Register. The content and level of detail of these summary results is set out in a European Commission guideline and in its technical guidance. A typical set of summary results provides information on the objectives of a given study, explains how it was designed and gives its main results and conclusions.

The Agency is also working towards the proactive publication of data from clinical trials carried out on the medicines that it authorises. For more information, see release of data from clinical trials.

Clinical trials conducted in countries outside the EU

Clinical trials conducted outside the EU but submitted in an application for marketing authorisation in the EU have to follow the principles which are equivalent to the provisions of the Directive 2001/20/ECExternal link icon.

In April 2012, the Agency published the final version of this paper:

This paper aims to strengthen existing processes to provide assurance that clinical trials meet the required ethical and GCP standards, no matter where in the world they have been conducted.

The number of clinical trials and clinical-trial subjects outside Western Europe and North America has been increasing for a number of years. More information is available in this document:

Revision of EU clinical trial legislation

In July 2012, the European Commission published a proposal on a regulation to revise the EU clinical trial legislation.

More information is available at: Revision of the clinical trials directiveExternal link icon.

Clinical Trials Facilitation Group

The Clinical Trials Facilitation GroupExternal link icon (CTFG) is a working group of the Heads of Medicines Agencies that:

  • acts as forum for discussion to agree on common principles and processes to be applied throughout the European medicines regulatory network;
  • promotes harmonisation of clinical-trial-assessment decisions and administrative processes by national competent authorities;
  • operates the voluntary harmonisation procedure for assessment of clinical-trial applications involving several Member States.

The Group is composed of representatives from the clinical-trial departments of the national competent authorities.


Europe green light for GSK COPD drug Incruse (umeclidinium)

 EU  Comments Off on Europe green light for GSK COPD drug Incruse (umeclidinium)
Apr 302014

Europe green light for GSK COPD drug

Regulators in Europe have given the green light to GlaxoSmithKline’s new chronic obstructive pulmonary disease drug Incruse.

Specifically, the European Commission has granted marketing authorisation for Incruse (umeclidinium) as a once-daily treatment to relieve symptoms in adults with COPD. The drug is a once-daily long-acting muscarinic antagonist (LAMA) delivered by GSK’s Ellipta inhaler.

Read more at:
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First mAb biosimilars backed by EMA advisors

 EU  Comments Off on First mAb biosimilars backed by EMA advisors
Jul 022013

Among the latest opinions issued by the European Medicines Agency’s Committee for Medicinal Products for Human Use, perhaps the most eye-catching are recommendations for two biosimilar versions of Johnson & Johnson’s monoclonal antibody blockbuster Remicade.

The CHMP has backed Remsima from South Korea’s Celltrion and Hospira’s Inflectra, both of which contain the same active substance as Remicade (infliximab). They have been recommended for authorisation in the same indications as the J&J drug, ie a range of autoimmune diseases such as rheumatoid arthritis, Crohn’s disease, ulcerative colitis, ankylosing spondylitis, psoriatic arthritis and and psoriasis.




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