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Research – Alternative Funding Sources for Rituximab

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FOI 24/25- 0013 DOCUMENT 16

Research – Alternative Funding Sources for Rituximab

Brief Please investigate alternative options a participant could possibly access funding to assist them to purchase medications (in this case Rituximab) not approved by the PBS.
Date 19/03/2021
Requester(s) Lee redacted: s22(1)(a)(ii) - irre – (Director – Compensation Recoveries Branch)
Researcher Jane redacted: s22(1)(a)(ii) - irrelev (Research Team Leader - TAB)
Cleared N/A

Please note:

The research and literature reviews collated by our TAB Research Team are not to be shared external to the Branch. These are for internal TAB use only and are intended to assist our advisors with their reasonable and necessary decision-making.

Delegates have access to a wide variety of comprehensive guidance material. If Delegates require further information on access or planning matters they are to call the TAPS line for advice.

The Research Team are unable to ensure that the information listed below provides an accurate & up-to-date snapshot of these matters.

Contents

2 Rituximab ………………………………………………………………………………………………………………………. 1 3 Pharmaceutical Company Compassionate Programs …………………………………………………………… 2 3.1 Roche ………………………………………………………………………………………………………………… 2 3.2 Cellitron ………………………………………………………………………………………………………………… 3 3.3 Sandoz ………………………………………………………………………………………………………………… 3 4 Lions Club ………………………………………………………………………………………………………………… 3 5 Rotary Australia………………………………………………………………………………………………………………. 4 6 References ………………………………………………………………………………………………………………… 5

Rituximab

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Rituximab is a human-murine chimeric anti-CD20-antibody, originally approved for B-cell lymphoma [1]. Upon intravenous administration, it causes rapid and complete depletion of B cells through complement mediated mechanisms in the blood and, to a lesser degree, also in the cerebrospinal fluid [1].

The drug is not approved for use in Devic’s disease, however, has been studied as a disease modifying treatment in multiple sclerosis (MS) with good outcomes [1]. Roche investigated the use of Rituximab in MS which yielded promising signs of efficacy, but development in this indication was discontinued for undisclosed reasons [2]. Regulatory approval for the treatment of MS currently does not exist.

In Australia, Rituximab is marketed as:

•  Mabthera –Roche
•   Riximyo - Sandoz
•  Truxima – Cellitron Healthcare Australia

All are approved for use in Non-Hodgkin’s Lymphoma (NHL), Chronic Lymphocytic Leukemia (CLL), Rheumatoid Arthritis (RA), Granulomatosis with Polyangiitis (GPA), Microscopic Polyangiitis (MPA).

Pharmaceutical Company Compassionate Programs

Drugs that are unapproved for the indication for which they are being requested on compassionate grounds are more challenging to acquire and contribute a significant proportion of likely requests [3].

3.1 Roche

Roche has a positon statement relating to access to medicines outside of public funding. In summary [4]:

•   Roche’s aim is for every person who needs our medicines to be able to benefit from them
•  The Federal and State Governments have primary responsibility for Australia’s universal healthcare system, which provides subsidised access to many medicines via the Pharmaceutical Benefits Scheme (PBS) and public hospitals.
•  Roche acknowledges that the absence of public funding for a prescribed medicine may represent a significant financial burden and difficult choice for patients.

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• In situations where there is no public funding, Roche may be able to assist patients with alternative mechanisms of access to medicines, such as providing subsidies. o All requests for access in these circumstances must come to Roche via a patient’s treating healthcare professional.

In relation to investigational medicinal products, new medicines may be made available to individual patients in special circumstances [5]. For example:

•  Compassionate Use (CU): is a mechanism to provide a new medicine to an individual patient or set of individual patients who have a serious or life threatening disease or condition for which no satisfactory alternative therapy exists or who cannot enter a clinical trial.

3.2 Cellitron

Unable to locate a compassionate use program for this company.

3.3 Sandoz

Novartis (Sandoz is a division of Novartis) delivers what is called a ‘managed access program’. This program allows patients with serious or life threatening diseases or conditions access to certain investigational or unapproved treatments [6].

The Novartis “Managed Access” terminology covers all locally defined pre-approval access mechanisms and programs such as “Compassionate Use”, “Expanded Access”, “Named Patient Supply”, “Special Access Schemes/Programs” etc.

The treating physician can request an investigational or pre-approval product prior to regulatory approval, provided it is allowed by the applicable local laws.

Lions Club

The Lions Club International Foundation (LCIF) supports large-scale projects that address unmet humanitarian needs for entire communities. These projects are led by local Lions who identify the need, develop the plan of action and carry out the project. LCIF does not have a grant program that supports individual assistance. These requests are more appropriately

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directed to the local Lions who often have programs in place to support these types of requests within their communities.

The participant’s local club is Rochedale Springwood – District 201Q1

Rotary Australia

In January 2017, Rotary Australia announced its Compassionate Grants program to help Australians in need.

Grants are assessed by the Rotary Australia Benevolent Society (RABS) and funds distributed to disadvantaged Australians identified by local Rotary Clubs or Rotary Districts as being in need within their local or wider community.

Projects granted funding must meet RABS criteria for registration. They must provide direct relief to people in need. If the intended recipients are disadvantaged, the relief should target that disadvantage.

The concept of disadvantage is unlimited and could have arisen from sickness, suffering, distress, misfortune, disability, destitution, helplessness or poverty, any aspect of the negative side of the human condition. The criteria are not prescriptive but are to be used as a guide to determine the disadvantage.

Sample projects where person or group was potentially disadvantaged under the above criteria:

 Provision of a modified family motor vehicle for a 6 year old with cerebral palsy  Modifications to a home to assist access and functionality for a quadriplegic  Financial assistance for a seriously injured sportsman’s family  Ongoing support for non PBS medicines for a sufferer of Lymes disease  Provision of a specialised bed for a person with Parkinson’s Disease  Supply insulin pumps to three children with juvenile diabetes  Provision of improved prosthetics for an amputee  Assistance to a family who lost everything in a fire  Provision of financial assistance to a young family whose mother drowned

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References

  1.  Ineichen BV, Moridi T, Granberg T, Piehl F. Rituximab treatment for multiple sclerosis. Multiple Sclerosis Journal [Internet]. 2019 2020/02/01; 26(2):[137-52 pp.]. Available from: https://doi.org/10.1177/1352458519858604.
  2. Mullard A. Roche hits multiple sclerosis landmarks. Nature Reviews Drug Discovery [Internet]. 2015 2015/11/01; 14(11):[739- pp.]. Available from: https://doi.org/10.1038/nrd4780.
  3. Medicines Australia. Navigating (Compassionate) Access to Medicines For Trials 2020. Available from: https://www.medicinesaustralia.com.au/wp-content/uploads/2020/11/Whitepaper-Navigating-compassionate-access-to-medicines-for-trials-for-reviewspplus-august2020-1.pdf.
  4. Roche. Access to medicines outside of public funding Roche Australia (Pharmaceuticals) Policy Position 2016 [Available from: https://www.roche-australia.com/content/dam/rochexx/roche-au/roche australia/en en/media/publications/2016-10%20Roche%20Position%20-%20Access%20to%20Medicines%20Outside%20of%20Public%20Funding.pdf.
  5. Roche. RochePosition on Pre-Approval Access to Investigational Medicinal Products 2020 [Available from: https://www.roche.com/dam/jcr:035f3847-505e-484c-b5f6-f666790791de/en/24 Position Pre Approval Access Investigational Medicinal Products reviewed April 2020.pdf.
  6.  Novartis. Novartis Position on Pre-Approval Access to Novartis Products through Novartis Managed Access Programs (MAPs) 2019 [Available from: https://www.novartis.com/sites/www.novartis.com/files/novartis-position-pre-approval-access.pdf.

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