Emergence of Compassionate Use programmes

Transcription

1 Emergence of Compassionate Use programmes Rosanna Melchior, PharmD, MS IDRAC, Thomson Scientific

2 Overview What is compassionate use (CU) Overview of CU in Europe Authorities and Sponsors Roles and Responsibilities CU outside Europe Industry s challenges

3 What is Compassionate Use? It is a European term which refers to a programme facilitating the early access of promising experimental new drugs to desperately needing patients In Europe, national CU programmes are available on a named patient basis or to a cohort of patients.

4 What is not Compassionate Use? It is not off label use of authorised medicinal products It is not a substitute for properly conducted clinical trials It is not a pre-marketing launch tool

5 Why emergence of CU programmes? Products are under development Patients cannot enter clinical trial Products are not yet marketed Products are marketed elsewhere Unmet medical need

6 Compassionate Use: Legal basis (1) Directive 2001/83/EC provides the legal basis for Member States to implement national programmes Article 5 - A Member State may, in accordance with legislation in force and to fulfil special needs, exclude from the provisions of this Directive [requirement for a marketing authorisation] medicinal products supplied in response to a bona fide unsolicited order, formulated in accordance with the specifications of an authorised health-care professional and for use by an individual patient under his direct personal responsibility.

7 Compassionate Use: Legal basis (2) Regulation (EC) No 726/2004 provides the legal basis for the management of a compassionate use programme at the European level Art 83 : Medicinal products eligible for the Centralised procedure Medicinal products undergoing clinical trials or subject of an application for a marketing authorisation Group of patients (cohort programme) For chronically or seriously debilitating disease, life threatening disease, unmet medical need

8 Art 83 of Reg 726/2004 / EMEA guidance A Member State notifies the EMEA when it envisages the need to make a medicinal product available for CU EMEA/CHMP ensures the eligibility of the product to the centralised procedure CHMP may adopt an opinion including criteria relating to the conditions for use and distribution and the patients targeted CHMP opinions are regularly updated and published on the EMEA website. They are not binding on MSs Administrative fees rely on the Applicant

9 Overview of CU in Europe CU Programmes available throughout EU MSs vary considerably * : 15 MSs allow products to be provided only for named patients; others have put in place nominative and cohort programmes Different terminology (named prescription, named patient supply, nominative importation; collective prescription, general licence, temporary authorisation for use) Programme initiator/applicant (Company, Physician, Patient) Competent Authorities approval (and validity) vs notification Documentation required Costs Liability All MSs require ADR reporting *from RAJ Pharma Feb 2005

10 Authorities and Sponsors Roles and Responsibilities Applicant Sponsor or Physician Liable Guarantee supply continuity Notify ADRs to MSs Submit MA under centralised procedure * Competent Authorities/MSs Coordinate and implement CU programmes Evaluate Application received by Sponsor/Physician Notify EMEA of cohort national programme in place * Take into account CHMP opinion * Report serious ADRs to EMEA/MAH EMEA & CHMP * Adopts opinion on CU Keeps opinion updated Publishes opinion * when Reg. 726/2004 applies

11 Situation outside Europe USA: Treatment /Emergency IND Canada: Special Access Programme (SAP) Australia: Special Access Scheme (SAS)

12 USA: Treatment IND (t-ind) Legal basis: 21 CFR , Treatment IND allows the use of an investigational drug (and biologic and devices) in patients not in the clinical trials Conditions the drug is intended to treat a serious or immediately lifethreatening disease; there is no satisfactory alternative treatment available; the drug is already under investigation, or trials have been completed; the trial sponsor is actively pursuing marketing approval. Procedure initiated by IND sponsor for group of patients (treated according to existing protocol) medical practitioner for a single patient (treated under sponsor s IND or under new IND filed by the medical practitioner )

13 USA: Emergency IND (e-ind) Legal basis: 21 CFR In case of emergency situation FDA may authorize shipment of the drug in advance of submission of an IND and before obtaining IRB approval for such use Conditions: Sponsor making an appropriate IND submission as soon as practicable after receiving the authorization Physician to notify the IRB within 5 working days

14 Canada: Special Access Programme Sections C , C of the Food and Drug Regulations Sale of New Drug for Emergency Treatment The SAP provides access to nonmarketed drugs and medical devices for physicians treating patients with serious or life-threatening conditions when conventional therapies have failed, are unsuitable, or unavailable. Scope: Pharmaceutical, biologic, radiopharmaceuticals, natural health products, medical devices, veterinary products

15 Canada: Special Access Programme SAP requests: Are initiated by the physician Are generally for single patients May be renewed A max quantity equivalent to 6 months treatment is allowed for chronic treatments Are processed by TPD within few business days. HC keeps database (SAMS) listing all drugs eligible through the SAP

16 Australia: Special Access Scheme Therapeutic Goods Act 1989 (Sect. 18, 19 and 41 HA), Therapeutic Goods Regulations 1990 (Reg. 12A), Medical Devices Regulations (Reg. 7.2) The SAS refers to arrangements which provide for the import and/or supply of an unapproved therapeutic good for a single patient, on a case by case basis. Temporary measure for supply unapproved medicinal products and medical devices pending general marketing approval of the product Procedure is initiated by physician

17 A or B patient? If the health status of a patient meets the criteria defined for a Category A patient, "a person who is seriously ill with a condition from which death is reasonably likely to occur within a matter of months, or from which premature death is reasonably likely to occur in the absence of early treatment the medical practitioner can supply unapproved goods without TGA approval. For all other patients the medical practitioner needs to seek TGA approval

18 Overview European Union USA Canada Australia Program me Named patient Compassionate use Cohort Treatment IND / single patient IND Emergency use Product Medicinal products Drug/biologic/device under investigation (phase 3/ 2) or trials have been completed; The drug is to be approved Special Access Programme Drug/biologic/ Device/ Radiopharm./ NHP and veterinary Special Access Scheme Drug/device N patients individual group group / single single single single Applicant sponsor/practitioner (at MS level) sponsor / practitioner practitioner practitioner practitioner Type of applicati on Depending on national legislation Depending on national legislation; MS to notify EMEA CHMP to issue opinion Treatment protocol/ind Phone, rapid request Request (rapid means) Notification/ Request (rapid means) Evaluatio n (timelines) Depending on national legislation Depending on national legislation; EMEA informed Yes (Max 30 days) No (1-2 days) No (1-2 days) No (A) /Yes (B) (2 days)

19 Industry s challenges Get to know local applicable CU provisions Promote an harmonised CU approach across Europe Internally, be ready to handle: drug supply safety issues related costs admin burden increasing demands/pressure

20 Conclusion Competent Authorities have put in place CU programmes allowing the use /supply of unapproved drugs Sponsors are fully engaged and committed in making investigational/unapproved drugs available to needing patients Patients right to access promising medicinal product is ensured

21 Further reading EMEA Draft Guideline on compassionate use RAJ Articles Feb 2005 issue August 06 issue