Proposal to list bedaquiline (Sirturo) and siltuximab (Sylvant) for rare disorders

Medicines

Consultation Closed

PHARMAC is seeking feedback on a proposal to list bedaquiline (Sirturo) and siltuximab (Sylvant) resulting from a provisional agreement between Janssen-Cilag Pty Ltd and PHARMAC.

The provisional agreement is the fourth that PHARMAC has reached with a bidder in a Request for Proposals we ran in 2014, related to the supply of medicines for rare disorders.

In summary, this proposal would result in:

  • bedaquiline (Sirturo) being funded in the community and in DHB hospitals for treatment of extensively drug resistant tuberculosis (XDR-TB). Listing in the Pharmaceutical Schedule would occur subject to Medsafe approval of the pharmaceutical; and
  • siltuximab (Sylvant) being funded in the community and in DHB hospitals for treatment of HIV-negative, HHV-8 negative multicentric Castleman’s disease.  This listing would occur from 1 June 2016.

Detail of the proposal and background information follow.

Feedback sought

PHARMAC welcomes feedback on this proposal. To provide feedback, please submit it in writing by 5pm on Wednesday, 27 April 2016 to:

Caroline De Luca
Senior Therapeutic Group Manager / Team Leader
PHARMAC
PO Box 10 254
Wellington 6143

Email: caroline.deluca@pharmac.govt.nz
Fax:      04 460 4995

All feedback received before the closing date will be considered by PHARMAC’s Board (or its delegate) prior to making a decision on this proposal.

Feedback we receive is subject to the Official Information Act 1982 (OIA) and we will consider any request to have information withheld in accordance with our obligations under the OIA. Anyone providing feedback, whether on their own account or on behalf of an organisation, and whether in a personal or professional capacity, should be aware that the content of their feedback and their identity may need to be disclosed in response to an OIA request.

We are not able to treat any part of your feedback as confidential unless you specifically request that we do, and then only to the extent permissible under the OIA and other relevant laws and requirements. If you would like us to withhold any commercially sensitive, confidential proprietary, or personal information included in your submission, please clearly state this in your submission and identify the relevant sections of your submission that you would like it withheld.  PHARMAC will give due consideration to any such request.

Details of the proposal

Following Medsafe approval, bedaquiline (Sirturo) would be listed in Section B (the Community) and Part II of Section H (the Hospital Medicines List, HML) of the Pharmaceutical Schedule as follows:

Chemical

Presentation

Brand

Pack size

Price and subsidy

Bedaquiline

Tab 100 mg

Sirturo

188

$24,160.00

  • Sirturo would be listed as soon as practicable following Janssen’s notification to PHARMAC that Medsafe has granted registration.
  • Sirturo would be listed subject to the following Special Authority criteria in the community and equivalent restrictions in the HML:

Special Authority for subsidy

Initial application from any relevant practitioner.  Approvals valid for 6 months where the patient has extensively drug-resistant tuberculosis (XDR-TB) for whom treatment with bedaquiline has been recommended by a multidisciplinary clinical team with expertise in the treatment of tuberculosis, with input from infectious disease and respiratory specialists.

Note: Bedaquiline is indicated for a course of treatment of 24 weeks.   

  • Sirturo would have subsidy and delisting protection until 30 June 2018.
  • Prior to a listing on the Pharmaceutical Schedule (ie until Medsafe approval is granted) the proposed list price would apply to any individual patient funding applications approved via the Named Patient Pharmaceutical Assessment (NPPA) Policy.
  • PHARMAC is considering the best way to manage the supply and distribution of bedaquiline to patients with XDR-TB and seeks particular feedback on this issue (see Background section below).

From 1 June 2016, siltuximab (Sylvant) would be listed in Section B (the Community) and Part II of Section H (the Hospital Medicines List) of the Pharmaceutical Schedule as follows:

Chemical

Presentation

Brand

Pack size

Price and subsidy

Siltuximab

Inj 100 mg vial

Sylvant

1

$770.57

Siltuximab

Inj 400 mg vial

Sylvant

1

$3,082.33

  • A confidential rebate would apply to Sylvant, which would reduce the net price of the treatment to the Funder.
  • Sylvant would be listed subject to the following Special Authority criteria in the community and equivalent restrictions in the Hospital Medicines List:

Special Authority for subsidy

Initial application only from a haematologist or rheumatologist.  Approvals valid for 6 months for applications meeting the following criteria:

All of the following:

  1. Patient has severe HIV/HHV-8 negative multicentric Castleman’s Disease; and
  2. reatment with at least 6 months of corticosteroids has proven ineffective; and
  3. Treatment with chemotherapy is clinically inappropriate; and
  4. Siltuximab is to be administered at doses no greater than 11 mg/kg every 3 weeks.

Renewal only from a haematologist or rheumatologist. Approvals valid for 12 months where the treatment remains appropriate and the patient has sustained improvement in inflammatory markers and functional status.

  • Sylvant would have Sole Supply Status in the community, Hospital Supply Status in DHB hospitals (DV limit 1%) and subsidy and delisting protection until 30 June 2018.
  • Prior to 1 July, the contracted net pricing would apply to any individual patient funding applications approved via the Named Patient Pharmaceutical Assessment (NPPA) Policy.

Background

Bedaquiline

Bedaquiline is an oral treatment for extensively drug resistant tuberculosis (XDR-TB). Tuberculosis is a bacterial communicable disease that generally affects the lungs primarily but also many body organ systems. Extensively drug resistant tuberculosis is a rare form caused by bacteria resistant to a number of the usual treatments for tuberculosis. The FDA defines XDR-TB as resistance to isoniazid, rifampicin, a fluoroquinolone, and a second line treatment such as amikacin.

People with active tuberculosis infection may experience symptoms such as cough with sputum and blood at times, chest pains, weakness, weight loss, fever and night sweats. Tuberculosis infection may lead to death. People who have an active tuberculosis infection can transmit the disease to other people. It is estimated that a person with active disease can infect 10-15 other people they have been in close contact with over the course of a year.

PHARMAC estimates that less than four patients per year in New Zealand may require bedaquiline as part of multi-drug regimen.

One pack of bedaquiline 100 mg tablets (188 tablets) would provide a 6-month treatment course. Due to the small patient population, cost and large pack size of this treatment, PHARMAC is considering the best way to manage the supply and distribution of bedaquiline to patients with XDR-TB.

PHARMAC is seeking particular feedback on the following information:

  • how would a patient with XDR-TB be treated? (e.g. Directly Observed Treatment, via hospital or community managed services)
  • how frequently would these medications be dispensed to a patient during a course of treatment?

To date, PHARMAC has not received any NPPA applications for the funding of bedaquiline for individuals. 

Bedaquiline is approved in Europe and the United States. Janssen has submitted an application for registration to Medsafe, and it is currently under assessment [link no longer available].

Siltuximab

Siltuximab is an intravenous infusion treatment for HIV-negative, HHV-8 negative multicentric Castleman’s disease (also known as idiopathic MCD), a rare condition that affects the multiple lymph nodes and other tissues. It involves an increased production of interleukin 6, which has an important role in the immune system. People with HIV/HHV-8 negative MCD often have problems such as serious infections, fevers, weight loss, fatigue, night sweats, and nerve damage that can cause weakness and numbness. Clinical findings consistent with interleukin-6 excess include a high C-reactive protein (CRP), low albumin, polyclonal increase in immunoglobulins, lymphadenopathy, microcytic anaemia. Symptoms can be very serious, including organ system involvement (kidney, liver, bone marrow) and may lead to death. 

Siltuximab is registered with Medsafe for the treatment of HIV/HHV-8 negative MCD [link no longer available] and is the only pharmaceutical that has been subjected to a randomised controlled trial for the management of this condition.

PHARMAC estimates there would initially be 20 to 40 patients in New Zealand with HIV/HHV-8 negative MCD that could be treated with siltuximab, with 5 to 6 new patients each year. MCD has a higher incidence in Pacific peoples and this contributes to the higher rates seen in New Zealand compared to other countries.

Siltuximab is a hospital out-patient infusion treatment; however the proposal would include a listing in Section B of the Pharmaceutical Schedule to enable hospitals to claim prescriptions from the CPB for out-patients.

PHARMAC has not received any Named Patient Pharmaceutical Assessment (NPPA) applications for the funding of siltuximab for individuals.  PHARMAC has received a number of NPPA applications of other unregistered treatments for HIV/HHV-8 negative MCD, including tocilizumab and rituximab. Some of these applications were approved and some were declined.

PHARMAC’s evaluation of bedaquiline (Sirturo) and siltuximab (Sylvant) followed the process described in Schedule 2 of the Request for Proposals (RFP) for the supply of medicines for rare disorders. The RFP document can be found on the PHARMAC website.