Compassionate Use

from Wikipedia, the free encyclopedia

The English expression compassionate use means the use of drugs that have not (yet) been approved on patients in particularly serious cases of illness who cannot be treated satisfactorily with approved drugs (Art. 83, Paragraph 2, Regulation (EC ) No. 726/2004 ). In the literature, expressions or paraphrases such as release out of mercy or compassionate use or use of an unauthorized drug to patients or prematurely tolerated use of a drug that has not yet been approved for humanitarian reasons are used. Mostly, however, the English term compassionate use is used directly. Even if the term essentially refers to human medical patients, it is also used in veterinary medicine .

The European Medicines Agency defines as follows: “ Compassionate use is a treatment option that enables the use of an unapproved drug. Under certain conditions, drugs in development can be made available to groups of patients suffering from a disease for which there are no satisfactory approved therapies and who cannot enter clinical trials. "

Legal situation

The use of unapproved drugs on patients is regulated differently depending on the state. A number of member states of the European Union have set up specific national programs to make development products that are still in clinical trials available to patients in need in certain cases.

The national compassionate use programs in the respective states ensure in different ways that certain patients - either personally or because they belong to a group of people with the same indication - have access to new, promising drugs before approval, as is the case in the USA has been the case since 1987. In Denmark , Finland , France , Greece , Luxembourg , the Netherlands , Sweden and UK both programs are possible in other states only the individual patient-specific regulation is often possible. Only a few member states have no specific regulation.

Germany

In the 14th Amendment to the Medicines Act (AMG), “compassionate use” was also included in German pharmaceutical law. The placing on the market as part of compassionate use has been legally permissible since then.

Previously, patients in Germany could only be treated with unauthorized drugs on the basis of the legally uncertain Section 34 StGB ( justifying emergency ).

Section 21 (2) AMG provides under No. 6:

“(2) A license is not required for medicinal products that
1. […]
6. are made available free of charge under the conditions set out in Article 83 of Regulation (EC) No. 726/2004 for use in patients who suffer from a disease leading to a severe disability or whose disease is life-threatening and who have a authorized medicinal products cannot be treated satisfactorily; Procedural regulations are determined in a statutory ordinance according to § 80. [...] "

The ordinance according to § 80 AMG ( Medicines Hardship Ordinance , AMHV) came into force on July 22, 2010. It is the implementation of the "Guideline on Compassionate Use of Medicinal Products, Pursuant to Article 83 of Regulation (EC) No 726/2004". According to this, drugs can be made available to a specific group of patients without approval or approval if there is sufficient evidence of the drug's effectiveness and drug safety and a clinical study is carried out for it or if an application for approval has been submitted to the European Medicines Agency , the Federal Institute for Drugs and Medical Devices (BfArM) or an authority of a member state responsible for approval. A case of hardship within the meaning of this Ordinance exists if a group of patients who suffer from a disease that would lead to a severe disability or that is life-threatening cannot be treated satisfactorily with a drug that is intended to be placed on the market within the scope of the Medicines Act is approved or approved. The responsible person must report the compassionate use program to the competent authority (BfArM or PEI ). The free distribution of the drug was included as a basic requirement for a compassionate use program so that there is no financial incentive for drug manufacturers to initiate such programs. The treatment of individual cases in compassionate use is excluded from the drug hardship ordinance and the associated notification procedure.

Switzerland

In principle, only medicinal products may be placed on the market whose safety, efficacy and quality have been checked by the responsible authorities and, where this is not the case, similarly strict rules must be observed. Any use for a medicinal product that is subject to authorization and is not authorized by Swissmedic therefore requires a license. This option is specifically given for the treatment of life-threatening diseases in the Therapeutic Products Act (HMG) (Art. 9 Para. 4) under certain conditions. Swissmedic defines the framework conditions for the use of products as follows:

  • The patient suffers from a serious, potentially life-threatening or debilitating illness.
  • There is no satisfactory alternative therapy for the treatment of the disease in Switzerland (no approved preparation) or the risk-benefit ratio of an approved drug is poor or an alternative therapy has already been carried out without the hoped-for success.
  • It is primarily an emergency intervention or one in the sense of the last possible therapy.
  • The drug has already been approved by a recognized approval authority in a third country or is in the approval process. If both are not the case, solid scientific results from clinical trials and good product information from the manufacturer (documentation on effectiveness and safety as well as documentation on the quality of the preparation) must be available. The latter must be submitted or at least communicated as references.
  • It is used on the basis of a single patient known by name.
  • An assessment and approval of the use by the local ethics committee is not necessary.
  • The license is only granted to a single attending physician. He must have the information specific to the drug used. He is responsible for the careful documentation and data storage of each individual patient ( data protection ). After completing the therapy, a short summary report must be submitted.
  • The obligation to report undesired effects and incidents, as set out in Art. 59 TPA, also applies in cases of the use of preparations on the basis of a special license.
  • The patient must be informed about the application and his written declaration of consent must be obtained.
  • Insurance coverage must be ensured through the private liability insurance of the attending physician.
  • Swissmedic can impose additional requirements or request information.
  • Addition: If the preparation to be used is a radiopharmaceutical , the approval of the BAG must also be submitted ( Radiation Protection Ordinance ).

France

Compassionate use is widely used in France . The assumption of treatment costs by the statutory health insurance is also regulated.

classification

Compassionate drug therapy measures are a form of unlicensed use , i. H. treatment with a drug that is not approved for use in the country in which it is used. Compassionate use is particularly important for the application of drugs for the treatment of rare diseases that are life-threatening or serious and for which there are no satisfactory treatment options (orphan drugs) .

Demarcation

Demarcate the compassionate use of off-label use , a pivotal-border application for an approved drug. B. for an indication or a patient group that is not covered by the approval. This means that in off-label use a drug is used that has already been approved for certain other applications and has also been extensively clinically tested on humans.

The Federal Social Court had in a judgment in 2002 the Compassionate Use a controlled off-label use an equal footing, but this is not true according to many doctors and lawyers. The Federal Association of Company Health Insurance Funds therefore stated in a letter to the Bundestag Committee for Health and Social Security on the draft of the Fourteenth Act to Amend the Medicines Act : “ Compassionate use must also be clearly distinguished from the regulations on off-label use ; in no case are the terms to be understood as synonymous. Compassionate use only regulates drugs that are already in phase III clinical trials or cover the period between the end of the approval study and market launch. However, these requirements are not met with off-label use. "

In Switzerland, on the other hand , the term compassionate use says nothing about the status (clinical phase) of the drug itself.

Examples of compassionate use

The best known is the compassionate use of cannabis in the United States of America . The Compassionate Investigational New Drug Program began in 1978 with the distribution of marijuana cigarettes to selected patients suffering from diseases such as glaucoma and epilepsy . There has been a number of legal disputes in the United States in this Compassionate Use case .

literature

Individual evidence

  1. ↑ a b heise.de: Precious pills
  2. ↑ wdr.de Glossary ( Memento from September 29, 2007 in the Internet Archive )
  3. ↑ a b c Federal Association of the Pharmaceutical Industry e. V. (BPI): BPI position paper Orphan Drugs - Compassionate Use, Economic Incentives, Prescribability at the expense of the statutory health insurance ( Memento of March 4, 2016 in the Internet Archive ) from September 2008, accessed on November 15, 2015
  4. ↑ Compassionate use , accessed March 27, 2018.
  5. ↑ bfarm.de , accessed on April 1, 2020
  6. ↑ The revision of the EC pharmaceutical legislation - challenges and opportunities for patients, regulatory authorities and the pharmaceutical industry. , Association of Research-Based Pharmaceutical Companies , April 27, 2005.
  7. ↑ Federal Law Gazette 2010 I No. 37, 935
  8. ↑ EMEA Guideline on Compassionate Use of Medicinal Products (PDF; 56 kB)
  9. ↑ a b Authorization ( memento of April 6, 2009 in the Internet Archive ) for the use and import of a preparation that is not authorized in Switzerland ("special authorization")
  10. ↑ [1]  ( Page no longer available , search in web archives )  Info: The link was automatically marked as defective. Please check the link according to the instructions and then remove this notice.@1@ 2Template: Dead Link / www.lumrix.de  
  11. ↑ Dierks, Christian, off-label use points setting to the BSG judgment in 2002 , university print shop Wolf & Sohn, Munich, 2002
  12. ↑ Blickpunkt Apotheke, May 2007 ( Memento from July 14, 2014 in the Internet Archive ) (PDF; 276 kB)
  13. ↑ Introgen provides ADVEXIN p53 therapy for cancer patients with Li-Fraumeni syndrome .
  14. ↑ Questions and answers on the withdrawal of the application for a marketing authorization for Retaane ( Memento of July 30, 2007 in the Internet Archive )
  15. ↑ S. Hayek, M. Scherrer, D. Barthelmes, J. Fleischhauer, M. Kurz-Levin, M. Menghini, H. Helbig, F. Sutter: First Clinical Experience with Anecortave Acetate (Retaane). In: Clinical monthly sheets for ophthalmology. 224, 2007, pp. 279-281, doi: 10.1055 / s-2007-962848 .
  16. ↑ Gefitinib - Pre-therapeutic known parameters II  ( page no longer available , search in web archives )  Info: The link was automatically marked as defective. Please check the link according to the instructions and then remove this notice.@1@ 2Template: Toter Link / www.lungenkarzinom-info.de  
  17. ↑ AMN107 / Nilotinib / Tasigna® (monotherapy) ( Memento from August 15, 2007 in the Internet Archive ).
  18. ↑ International Working Group on Cannabis as Medicine ( Memento of October 8, 2007 in the Internet Archive ).