Bill C-265: An Act to amend the Food and Drugs Act (List of Therapeutic Products Pre-approved for Special Access)

R.S., c. F- 27 Food and Drugs Act 1 The Food and Drugs Act is amended by adding the following after section 2.4: Clinical judgment 2. 5 For greater certainty, nothing in this Act or the regulations is to be interpreted as preventing, in an emergency situation, a person who is entitled to practise medicine under the laws of a province from temporarily administering to a person under their care any therapeutic product that was lawfully manufactured in or imported into Canada and that is available to them if doing so is the best available treatment based on their clinical judgment. 2 The Act is amended by adding the following after section 21.96: PART I. 1 List of Therapeutic Products Pre-approved for Special Access Interpretation Definitions 21. 9701 In this Part, establishment licence  means a licence issued under section C.01A.008 of the Food and Drug Regulations or under section 46 of the Medical Devices Regulations . ( licence d’établissement ) List of Therapeutic Products Pre-approved for Special Access  means the list established under subsection 21.9702(1). ( Liste des produits thérapeutiques préapprouvés aux fins d’accès spécial ) non-marketed , in respect of a therapeutic product, means that it is not the subject of a therapeutic product authorization or it is no longer marketed in Canada. ( non commercialisé ) pharmacist  means a person who (a) is registered or otherwise entitled under the laws of a province to practise pharmacy; and (b) is practising pharmacy in that province. ( pharmacien ) practitioner  means a person who (a) is entitled under the laws of a province to treat patients with a therapeutic product; and (b) is practising their profession in that province. ( praticien ) site licence  means a licence issued under section 29 of the Natural Health Products Regulations . ( licence d’exploitation ) List of Therapeutic Products Pre-approved for Special Access Establishment of list 21.9702 (1) The Minister shall establish and maintain a list of pre-approved non-marketed therapeutic products or classes of such products for use in the treatment or prevention of the progression of serious or life-threatening conditions, including rare diseases, and for use where a comparable therapeutic product is not available on the Canadian market. Clarification (2) For greater certainty, therapeutic products are not comparable if there is a significant difference in their (a) cost; (b) formulation; (c) efficacy; (d) contraindications; or (e) rates of adverse drug reactions or medical device incidents in specific populations. Publication (3) The Minister shall publish the List of Therapeutic Products Pre-approved for Special Access and any changes to it on the website of the Department of Health. Contents of list (4) The List of Therapeutic Products Pre-approved for Special Access must set out (a) the name of the therapeutic product; (b) the uses for which it is authorized; and (c) any limitations on the practitioners who may use it or on the settings in which it may be used. Additions to list (5) The Minister may add a therapeutic product to the List of Therapeutic Products Pre-approved for Special Access for the purposes of the uses listed in subsection (1) if the Minister considers that it meets appropriate quality and safety standards, provided that, (a) in the case of a new drug, it has been the subject of a letter of authorization under subsection C.08.010 (1) of the Food and Drug Regulations , for the purposes of a use that will be associated with it on the list; or (b) in any other case, the therapeutic product (i) was previously the subject of a therapeutic product authorization that was not cancelled for safety reasons, or (ii) is authorized by a foreign regulatory authority that the Minister considers comparable to Canada. Acceptable uses (6) In the case of a therapeutic product referred to in subparagraph (5)(b)(ii), the uses for it set out on the List of Therapeutic Products Pre-approved for Special Access need not be authorized by the foreign regulatory authority but must be supported by generally accepted guidelines or clinical evidence. Limitations on practitioners or uses (7) If the Minister considers that limiting the use of a therapeutic product on the List of Therapeutic Products Pre-approved for Special Access is necessary to ensure safe and appropriate use of the therapeutic product, the Minister may (a) limit the use of a therapeutic product on the list to practitioners with specified training or expertise or to specified situations or clinical care settings; or (b) impose conditions on the use of a therapeutic product on the list. Removal from list (8) The Minister may remove a therapeutic product or an associated use from the List of Therapeutic Products Pre-approved for Special Access for any of the following reasons: (a) new safety concerns arise about the use of the therapeutic product; (b) the therapeutic product becomes the subject of a notice of compliance or a medical device licence under the regulations; (c) the therapeutic product is marketed in Canada; (d) the manufacturer withdraws the therapeutic product from other international markets or discontinues production; or (e) the therapeutic product gives rise to misuse, diversion or public health risks. Notice of removal (9) If the Minister removes a therapeutic product or an associated use from the List of Therapeutic Products Pre-approved for Special Access, the Minister shall publish a notice of removal setting out the reasons for the removal on the website of the Department of Health. Advisory committee 21.9703 (1) Before establishing or amending the List of Therapeutic Products Pre-approved for Special Access, the Minister shall consult a non-partisan committee composed of experts in medical research and clinical practice, including those in specialized medicine most likely to access therapeutic products on the list, such as those who work with pediatric populations. No remuneration or reimbursement (2) No member of the committee shall receive remuneration for acting as a committee member or be reimbursed for any expenses incurred in the course of acting as a committee member. Submission by practitioner, pharmacist, hospital or organization 21.9704 (1) A practitioner, a pharmacist, a hospital or a medical non-profit organization may, in the form and manner specified by the Minister, submit to the Minister the name of a non-marketed therapeutic product and the uses associated with it for addition to the List of Therapeutic Products Pre-approved for Special Access. Decision within 120 days (2) The Minister shall decide whether to add a therapeutic product or a use submitted under subsection (1) to the List of Therapeutic Products Pre-approved for Special Access within 120 days after the day on which the submission is received. Report 21.9705 (1) Within the first three months of each calendar year, the Minister shall, in consultation with the advisory committee, practitioners, pharmacists, hospitals and medical non-profit organizations, prepare a report on the effectiveness of the List of Therapeutic Products Pre-approved for Special Access and on measures to improve its effectiveness. Publication (2) The Minister shall publish the report on the website of the Department of Health. Access to Therapeutic Products on the List of Therapeutic Products Pre-approved for Special Access Practitioner request 21.9706 (1) Subject to any limitations on practitioners set out in the List of Therapeutic Products Pre-approved for Special Access, a practitioner may make a request, in the form specified by the Minister, to a manufacturer of a therapeutic product on that list to purchase a specified quantity of the therapeutic product for use in the treatment of a person under the care of the practitioner, if that use corresponds to a use of the therapeutic product that is included in the list. Required information (2) The request must include any information specified by the Minister. Identity (3) For the purposes of this section, the practitioner is not required to know the identity of the person under their care at the time the request is made. Sale by manufacturer 21.9707 (1) A manufacturer may sell a therapeutic product on the List of Therapeutic Products Pre-approved for Special Access (a) in accordance with a request made under subsection 21.9706(1); or (b) in anticipation of such a request, subject to any limitations or conditions specified by the Minister. Exemption (2) The sale of a therapeutic product made in accordance with subsection (1) is exempt from the provisions of this Act and any regulations made under it other than the provisions of this Part and any regulations made for the purposes of this Part. Import 21.9708 (1) The holder of an establishment licence or a site licence that authorizes the importation of a therapeutic product in the same category as the one to be imported may import a therapeutic product on the List of Therapeutic Products Pre-approved for Special Access (a) in accordance with a request made under subsection 21.9706(1); or (b) in anticipation of such a request, subject to any limitations or conditions specified by the Minister. Exemption — import (2) Subject to subsection (3), the importation of a therapeutic product made in accordance with subsection (1) is exempt from the provisions of this Act and any regulations made under it other than the provisions of this Part and any regulations made for the purposes of this Part. Applicable regulatory provisions (3) The Minister may, by order, specify provisions of the regulations that apply to the importation of a therapeutic product under subsection (1) for the purposes of storage, transportation, quality control, tracking and record keeping. Distribution (4) The holder of an establishment licence or a site licence who imports a therapeutic product under this section may distribute that therapeutic product in accordance with a request made under subsection 21.9706(1). Exemption — distribution (5) The distribution of a therapeutic product in accordance with this section is exempt from the provisions of this Act and any regulations made under it other than the provisions of this Part and any regulations made for the purposes of this Part. Oversight Report to Minister by practitioner 21.9709 (1) A practitioner who makes a request under subsection 21.9706(1) shall submit a report to the Minister, in the manner and form specified by the Minister, within 90 days after the day on which they made the request. Required information (2) The report must include any information specified by the minister. Additional information (3) The Minister may at any time order the practitioner to provide the Minister with any information that the Minister believes is necessary in relation to the use of a therapeutic product. Ministerial order (4) The Minister may make an order prohibiting a practitioner from using a requested therapeutic product or establishing limitations on its use, if the Minister determines that the use poses a significant risk to the health or safety of any person. Notice to Minister by manufacturer — sale 21.971 (1) A manufacturer who sells a therapeutic product under subsection 21.9707(1) shall submit notice of the sale to the Minister, in the manner and form specified by the Minister, within 90 days after the day of the sale. Required information (2) The notice must include (a) the name of the practitioner who requested the therapeutic product; (b) the name and the civic address of the person to whom the therapeutic product was shipped; (c) the name of the therapeutic product and its intended use; and (d) the quantity of the therapeutic product provided. Notice to Minister by holder of establishment or site licence 21.9711 (1) A holder of an establishment licence or site licence who imports a therapeutic product under subsection 21.9708(1) shall submit notice of the importation to the Minister, in the manner and form specified by the Minister, within 90 days after the day of the importation. Required information (2) The notice must include any information specified by the Minister. Reasons for not seeking regulatory approval 21.9712 (1) If the Minister believes on reasonable grounds that a manufacturer is using the List of Therapeutic Products Pre-approved for Special Access to circumvent the regulatory process for approval of a therapeutic product, the Minister may require the manufacturer to give reasons for not submitting the therapeutic product to that process. Reasons not sufficient (2) If the Minister is of the opinion that the manufacturer’s reasons are not sufficient, the Minister may (a) maintain the therapeutic product on the List of Therapeutic Products Pre-approved for Special Access until the manufacturer obtains approval; or (b) remove the therapeutic product from the List of Therapeutic Products Pre-approved for Special Access. 3 (1) Subsection 30(1) of the Act is amended by adding the following after paragraph (j): (j.01) respecting the establishment, maintenance and publication of the List of Therapeutic Products Pre-approved for Special Access referred to in Part I.1 and any additional criteria for addition or removal of a therapeutic product to or from the list; (j.02) respecting the creation and maintenance of an electronic information exchange to facilitate requests and notifications required under Part I.1; (2) Subsection 30(1) of the Act is amended by adding the following after paragraph (o): (o.1) respecting a process to request the Minister to reconsider the refusal to issue a letter of authorization under subsection C.08.010(1) of the Food and Drug Regulations ; 4 The Act is amended by adding the following after section 30: Emergency Access to New Drugs Definition of letter of authorization 30. 001 In sections 30.002 to 30.008, letter of authorization means a letter of authorization issued under subsection C.08.010(1) of the Food and Drug Regulations . Criteria for issuing letter of authorization 30. 002 In determining whether to issue a letter of authorization, the Minister shall (a) weigh the benefits of the new drug against its risks to the person being treated, while considering the risk to the person should the use of the new drug not be authorized; and (b) consider all available medical evidence in respect of treatment with the new drug, including (i) evidence derived from routine clinical practice, (ii) evidence derived from controlled clinical trials, (iii) evidence derived from observational studies, and (iv) clinical data recognized by a foreign regulatory authority. Publication of guidelines for issuing letters of authorization 30. 003 The Minister shall publish and keep up to date on the website of the Department of Health the guidelines that the Minister uses to determine whether to issue a letter of authorization. Presumption — clinical judgment 30.004 (1) If the Minister considers that the information under section 30.002 is insufficient to issue a letter of authorization, the Minister shall, subject to subsection (2), issue a letter of authorization if two practitioners who are expert clinicians with specialized knowledge in the management of the condition for which a person with a serious or life-threatening condition is being treated provide to the Minister a treatment plan with a new drug in respect of the person and a statement that (a) the treatment plan is the most appropriate treatment for the person; (b) the benefits of the treatment plan likely outweigh the risks of authorizing the new drug; and (c) the person being treated has provided informed consent and is aware of the potentially significant risk of using a new drug. Exception (2) The Minister may refuse to issue a letter of authorization under subsection (1) if the Minister determines that the use of the new drug is likely to result in diversion or other significant public harm. Written reasons for denial 30.005 (1) If the Minister refuses to issue a letter of authorization, the Minister shall provide the practitioner with written reasons for the refusal, which must include (a) the regulatory or scientific reasons for the refusal; (b) any conditions or additional information that would result in the Minister deciding to reverse the Minister’s refusal; and (c) an outline of the process for the practitioner to request that the Minister reconsider the refusal. Limitations (2) The Minister shall not refuse to issue a letter of authorization solely on the basis of any of the following grounds: (a) a clinical trial for the new drug is currently active or recruiting; (b) the evidence supporting the request for a letter of authorization is derived primarily from foreign jurisdictions or off-label use; (c) the evidence supporting the request for a letter of authorization is not recent, provided that it has not been superseded by more recent evidence; (d) other forms of treatment for the condition are available on the Canadian market; or (e) other forms of treatment have not been received by the person under the practitioner’s care. Emergency line 30. 006 The Minister shall make every effort to ensure that an employee of the Department of Health is available at all times to assist practitioners in obtaining, as quickly as possible, a letter of authorization for emergency treatment of persons under the practitioners’ care. Emergency clinical judgment 30.007 (1) In an emergency situation, a practitioner may, without a letter of authorization, temporarily administer to a person under their care any new drug that was lawfully manufactured in or imported into Canada and that is available to them if doing so is the best available treatment based on their clinical judgment. Notice (2) A practitioner who administers a new drug under subsection (1) shall submit notice of that fact to the Minister, in the manner and form specified by the Minister. Clarification (3) For greater certainty, nothing in this section authorizes the continued use of a new drug without a letter of authorization. Annual review 30.008 (1) Within the first three months of each calendar year, the Minister shall, in consultation with practitioners, pharmacists, hospitals and medical non-profit organizations, prepare a report on the operation of sections C.08.010 to C.08.011.3 of the Food and Drug Regulations and setting out (a) the number of requests for letters of authorization, the number of requests refused and the average response times; (b) a list of new drugs that could be added to the List of Therapeutic Products Pre-approved for Special Access; and (c) proposed measures to improve emergency access to new drugs and, in doing so, to reduce the administrative burden on practitioners and hospitals. Publication (2) The Minister shall publish the report on the website of the Department of Health. Coming into Force One year after royal assent 5 This Act comes into force one year after the day on which it receives royal assent.