Your details
What is the name of your company and/or organisation? (If applicable)
Company/organisation
Haemophilia Foundation Australia
Terms of Reference 1
A. Are these proposed principles appropriate? With regard to the proposed principles, is anything missing or needing to change?
Please provide your response in the text box below
These proposed principles are important and valuable. We propose some amendments and additions:
Innovation:
Adding a principle that is a commitment to supporting innovation in health technologies. To accommodate current and future trends, the policy around healthcare and treatment will need to include a broad range of activities, including currently conventional medicines, devices, testing, vaccines, biologics, blood and blood products, gene and cell therapies, as well as other new and emerging health care developments that might fall under the umbrella of advanced therapies.
The principle should recognise the benefits of integrating new technologies, genomics, and digital advances into healthcare delivery and that such that innovations in therapies can contribute to cost-effectiveness. This includes accepting that an initial high cost to government for a medicine or technology may mitigate higher future healthcare costs.
Consumer centred approach:
Altering the current wording of the principle to ‘Consumers should be informed, engaged and enabled to participate in medicines policy, recognising that they are the most affected by the health technologies and have a key role in supporting the achievement of the policy’s objectives’.
Consumers are the individuals who experience the benefits and the limitations of the health technology. The first objective of the NMP is to provide ‘timely access to the medicines that Australians need’. Consumers are crucial to defining what this ‘need’ involves – it may for example, be not only clinical needs but relating to quality of life.
Empowering a consumer to participate leaves the responsibility of participating with the consumer. However, enabling the consumer voice to be represented carries with it responsibilities from all stakeholders in the process:
• Including the consumer voice from the beginning of the HTA process to define what outcomes are relevant
• Ensuring that the consumer voice at the table represents the health condition in question
• Valuing the type of data a consumer or consumer organisation can provide.
• Providing guidance to support consumers to contribute to health technology development, the HTA process and safety and quality bodies.
Accountability and transparency:
This is an important principle that will have great value when it is enacted in relation to the objectives.
For example, the first NMP objective is for ‘timely access to the medicines that Australians need…’. If the Health Technology Assessment (HTA) bodies are held accountable for timely access, it will promote addressing the issues in the process that impede timeliness quickly when they are identified. The bodies would be rewarded for implementing agreed solutions, such as bringing relevant expert clinicians to the table early in the HTA process to identify any issues or misconceptions and clarifying sponsor requirements, and this would prevent further inefficiencies such as the need to resubmit or provide new evidence to substantiate funding for a medicine.
Effective accountability and transparency not only requires a flow through from the principles to health policies and program, but it needs agreement and compliance at all jurisdictional regardless of which jurisdiction pays for a health care intervention or technology.
Innovation:
Adding a principle that is a commitment to supporting innovation in health technologies. To accommodate current and future trends, the policy around healthcare and treatment will need to include a broad range of activities, including currently conventional medicines, devices, testing, vaccines, biologics, blood and blood products, gene and cell therapies, as well as other new and emerging health care developments that might fall under the umbrella of advanced therapies.
The principle should recognise the benefits of integrating new technologies, genomics, and digital advances into healthcare delivery and that such that innovations in therapies can contribute to cost-effectiveness. This includes accepting that an initial high cost to government for a medicine or technology may mitigate higher future healthcare costs.
Consumer centred approach:
Altering the current wording of the principle to ‘Consumers should be informed, engaged and enabled to participate in medicines policy, recognising that they are the most affected by the health technologies and have a key role in supporting the achievement of the policy’s objectives’.
Consumers are the individuals who experience the benefits and the limitations of the health technology. The first objective of the NMP is to provide ‘timely access to the medicines that Australians need’. Consumers are crucial to defining what this ‘need’ involves – it may for example, be not only clinical needs but relating to quality of life.
Empowering a consumer to participate leaves the responsibility of participating with the consumer. However, enabling the consumer voice to be represented carries with it responsibilities from all stakeholders in the process:
• Including the consumer voice from the beginning of the HTA process to define what outcomes are relevant
• Ensuring that the consumer voice at the table represents the health condition in question
• Valuing the type of data a consumer or consumer organisation can provide.
• Providing guidance to support consumers to contribute to health technology development, the HTA process and safety and quality bodies.
Accountability and transparency:
This is an important principle that will have great value when it is enacted in relation to the objectives.
For example, the first NMP objective is for ‘timely access to the medicines that Australians need…’. If the Health Technology Assessment (HTA) bodies are held accountable for timely access, it will promote addressing the issues in the process that impede timeliness quickly when they are identified. The bodies would be rewarded for implementing agreed solutions, such as bringing relevant expert clinicians to the table early in the HTA process to identify any issues or misconceptions and clarifying sponsor requirements, and this would prevent further inefficiencies such as the need to resubmit or provide new evidence to substantiate funding for a medicine.
Effective accountability and transparency not only requires a flow through from the principles to health policies and program, but it needs agreement and compliance at all jurisdictional regardless of which jurisdiction pays for a health care intervention or technology.
B. Are these four Objectives still relevant? Should any be modified, or any additional objectives be considered? If so, how and why?
Please provide your response in the text box below
The term ‘medicines’ needs to be changed to keep up with modern medicine and the rapidly advancing environment.
The World Health Organization definition of health technology, ‘The definition of health technology is the application of organized knowledge and skills in the form of medicines, medical devices, vaccines, procedures and systems developed to solve a health problem and improve quality of life.’ (1) reflects an appropriate concept and could be broadened to include advanced therapies so it retains currency.
(1) World Health Organization. Europe Office. Health Technology Assessment. <https://www.euro.who.int/en/health-topics/Health-systems/health-technologies-and-medicines/policy-areas/health-technology-assessment, accessed 4 October 2021>
The World Health Organization definition of health technology, ‘The definition of health technology is the application of organized knowledge and skills in the form of medicines, medical devices, vaccines, procedures and systems developed to solve a health problem and improve quality of life.’ (1) reflects an appropriate concept and could be broadened to include advanced therapies so it retains currency.
(1) World Health Organization. Europe Office. Health Technology Assessment. <https://www.euro.who.int/en/health-topics/Health-systems/health-technologies-and-medicines/policy-areas/health-technology-assessment, accessed 4 October 2021>
Terms of Reference 2
A. Should the current NMP definition of medicines be expanded to include medical devices and vaccines? Why or why not? How would a change in definition of medicines be reflected in the policy’s high-level framework?
Please provide your response in the text box below
It would be valuable to redefine the term ‘medicines’ to encompass ‘health technologies’ that include advanced therapies as well as conventional medicines.
It will be important to have a policy with overarching objectives and principles that encompass the health technologies and other therapies associated with the entire patient journey, from prognosis to diagnosis then treatment, care and sometimes cure.
This would enable a consistent approach across the range of health technologies and therapies associated with each individual health condition – medicines and other advanced therapies, medical devices and diagnostic tools. All of these technologies/therapies have roles to play in the patient journey for a health condition and it is important that development and use of all of them are working to the same goals and principles.
It would also support efficiencies and effectiveness across the range of health technologies and therapies. While the two arms of health technology assessment, PBAC and MSAC, would remain, they would both be working to the same objectives and principles. They would also more easily be able to share ideas on ways to improve processes, for example, with streamlining, transparency, publication of guidelines or timeframes, communication with consumers or industry, rather than each arm of evaluation working to different objectives and imperatives.
One example of this is in bleeding disorders. For people with bleeding disorders, whose treatment is currently funded under the National Blood Agreement via the National Blood Authority Act 2003, a principles-based approach to treatment is critical. There are several heads of authority driving care and treatment, including the involvement of multiple stakeholders across jurisdictions, a national blood management strategy, the Australian Commission on Safety and Quality in Health Care National Safety and Quality Health Service (NSQHS) Standards, and a fast evolving treatment environment where traditional clotting factor replacement therapy manufactured is no longer the only treatment option, and for some patients non-clotting factor products and gene therapy are becoming the most appropriate treatment.
Broadening the title and scope of the policy to encompass a wide range of health technologies and medicines, including advanced therapies, would take into consideration not only medical devices and vaccines, but other treatments that are evolving therapeutic agents or approaches. This may include therapies such as gene therapy and other innovative novel non-factor therapies, including bispecific antibody and RNA interference therapies. It would incorporate the use of precision medicine, where tailoring a formulation or intervention was based on an individual’s genomic profile. This would also allow for further innovations in therapies in the future.
A policy that encompassed the variety of medicines, advanced therapies and other health technologies under the same principles would also support retaining existing health technology assessment bodies that, with the kinds of changes recommended in current government Health Technology Assessment process reviews, will be strengthened and provide Australians with access to the health technologies they need into the future. This is preferable to creating new bodies and losing well-developed processes and structures – throwing out the baby with the bathwater.
It will be important to have a policy with overarching objectives and principles that encompass the health technologies and other therapies associated with the entire patient journey, from prognosis to diagnosis then treatment, care and sometimes cure.
This would enable a consistent approach across the range of health technologies and therapies associated with each individual health condition – medicines and other advanced therapies, medical devices and diagnostic tools. All of these technologies/therapies have roles to play in the patient journey for a health condition and it is important that development and use of all of them are working to the same goals and principles.
It would also support efficiencies and effectiveness across the range of health technologies and therapies. While the two arms of health technology assessment, PBAC and MSAC, would remain, they would both be working to the same objectives and principles. They would also more easily be able to share ideas on ways to improve processes, for example, with streamlining, transparency, publication of guidelines or timeframes, communication with consumers or industry, rather than each arm of evaluation working to different objectives and imperatives.
One example of this is in bleeding disorders. For people with bleeding disorders, whose treatment is currently funded under the National Blood Agreement via the National Blood Authority Act 2003, a principles-based approach to treatment is critical. There are several heads of authority driving care and treatment, including the involvement of multiple stakeholders across jurisdictions, a national blood management strategy, the Australian Commission on Safety and Quality in Health Care National Safety and Quality Health Service (NSQHS) Standards, and a fast evolving treatment environment where traditional clotting factor replacement therapy manufactured is no longer the only treatment option, and for some patients non-clotting factor products and gene therapy are becoming the most appropriate treatment.
Broadening the title and scope of the policy to encompass a wide range of health technologies and medicines, including advanced therapies, would take into consideration not only medical devices and vaccines, but other treatments that are evolving therapeutic agents or approaches. This may include therapies such as gene therapy and other innovative novel non-factor therapies, including bispecific antibody and RNA interference therapies. It would incorporate the use of precision medicine, where tailoring a formulation or intervention was based on an individual’s genomic profile. This would also allow for further innovations in therapies in the future.
A policy that encompassed the variety of medicines, advanced therapies and other health technologies under the same principles would also support retaining existing health technology assessment bodies that, with the kinds of changes recommended in current government Health Technology Assessment process reviews, will be strengthened and provide Australians with access to the health technologies they need into the future. This is preferable to creating new bodies and losing well-developed processes and structures – throwing out the baby with the bathwater.
B. Does the Policy’s title, National Medicines Policy, reflect the breadth of technological developments within the Policy’s scope? If not, how best can these and future developments be appropriately represented in the policy’s title?
Please provide your response in the text box below
If the policy title is to reflect its scope, it would be important to rename it to incorporate the range of health technologies and advanced therapies it covers.
Terms of Reference 4
A. How can the NMP’s focus on consumer centricity and engagement be strengthened? Is anything missing, and what needs to change?
Please provide your response in the text box below
While the existing structural arrangements are important ways of involving consumers, further changes are required to ensure that the relevant consumers are central within the NMP.
HEALTH TECHNOLOGY ASSESSMENT (HTA) PROCESSES
Ensuring the consumer voice is represented in HTA evaluations:
• Ensuring the relevant consumers/patient organisations are embedded in the HTA process from end-to-end, from establishing PICO through to submissions for evaluation
• Requiring evidence in HTA that analyses patients’ experiences
• Including consumers and their representatives in the design of the HTA processes and evaluation of submissions for new therapies
• Including patient-reported measures in evaluation, both their preferences and experiences
• Resourcing patient organisations and other relevant consumer groups to participate in this work
• Provide consumer-focused information and education to support their contribution to the HTA process.
CLINICAL TRIALS
Acknowledging that consumers of health technologies are critical to the development of new therapies, devices and diagnostic tools.
Support consumers to engage in clinical trials by:
• Making information and education available about clinical trials to improving patient engagement in clinical trials
• Support the inclusion of patient-reported evaluation measures in clinical trials
• Resource patient organisations to develop or contribute to the development of patient-reported data that is suitable for clinical trials and health technology assessments.
HEALTH TECHNOLOGY ASSESSMENT (HTA) PROCESSES
Ensuring the consumer voice is represented in HTA evaluations:
• Ensuring the relevant consumers/patient organisations are embedded in the HTA process from end-to-end, from establishing PICO through to submissions for evaluation
• Requiring evidence in HTA that analyses patients’ experiences
• Including consumers and their representatives in the design of the HTA processes and evaluation of submissions for new therapies
• Including patient-reported measures in evaluation, both their preferences and experiences
• Resourcing patient organisations and other relevant consumer groups to participate in this work
• Provide consumer-focused information and education to support their contribution to the HTA process.
CLINICAL TRIALS
Acknowledging that consumers of health technologies are critical to the development of new therapies, devices and diagnostic tools.
Support consumers to engage in clinical trials by:
• Making information and education available about clinical trials to improving patient engagement in clinical trials
• Support the inclusion of patient-reported evaluation measures in clinical trials
• Resource patient organisations to develop or contribute to the development of patient-reported data that is suitable for clinical trials and health technology assessments.