International Alliance of ALS/MND Associations

Equitable Access to Therapies

Equitable Access to Therapies on a Global Scale

Executive Summary

Access to treatments is a fundamental right of all people living with ALS/MND, yet there is an urgent need for equitable access. On a global scale, it takes too long for new treatments to move through traditional pathways from clinical research to market access. Many countries lack the infrastructure to support clinical trials or the resources to fund and deliver therapies. There is also little collaborative engagement among regulators across countries. The ALS/MND community needs greater urgency.

The availability of clinical trials differs from country to country, and there are several barriers to accessing clinical trials that disproportionately affect certain populations, including people who live in developing countries and remote areas with limited access to health centres.

Additionally, Special Access Programs are often available only in high-income countries, resulting in limited access to treatments for people in low- and middle-income countries. Within countries that provide public drug programs, people living with ALS/MND must often wait years through a series of drawn-out, confusing delays caused by sequential reviews and hand-offs before decisions on access are made.

The current lack of global regulatory collaboration creates significant disparities in access to treatment and results in inequitable health outcomes for people living with ALS/MND. Furthermore, it slows down the development and approval of new therapies, leading to limited treatment options for people living with ALS/MND.

Addressing these disparities requires efforts to ensure that people living with ALS/MND have access to safe and effective treatments, regardless of where they live and at minimal cost. This requires collaboration among regulators, healthcare providers, industry, and policymakers to develop strategies to improve access to treatments and ensure that people living with ALS/MND receive the best possible care.

Given the recent scientific and clinical progress in the ALS/MND space, where new potentially efficacious therapies are emerging through successful clinical trials, the International Alliance of ALS/MND Associations supports the development of a cooperative global framework to create an environment for increased clinical trial availability and evaluate the risks and benefits of new therapies, especially given the global nature of drug development and clinical trial conduct.

Rationale for Support

Access to the highest quality treatment available is a fundamental right for people living with ALS/MND. The Fundamental Rights of people with ALS/MND do not stand in isolation from other overarching imperatives, including the United Nations Sustainable Development Goals. The 2030 Agenda for Sustainable Development, adopted by all United Nations Member States in 2015, provides a shared blueprint for peace and prosperity for people and the planet, now and into the future. At its heart are the 17 Sustainable Development Goals (SDGs), which are an urgent call for action by all countries – developed and developing – in a global partnership. They recognize that ending poverty and other deprivations must go hand-in-hand with strategies that improve health and education, reduce inequality, and spur economic growth – all while tackling climate change and working to preserve our oceans and forests.

The Alliance’s Fundamental Rights are further supported by the World Medical Association (WMA) Declaration of Lisbon on the Rights of the Patient, which states that “Every person is entitled without discrimination to appropriate medical care.” (See Appendix 2.) In addition, this fundamental right is embedded in UNESCO’s Universal Declaration on Bioethics and Human Rights. In particular, Article 15 states:

“Sharing of benefits:

Benefits resulting from any scientific research and its applications should be shared with society as a whole and within the international community, in particular with developing countries. In giving effect to this principle, benefits may take any of the following forms:

  • special and sustainable assistance to, and acknowledgement of, the persons and groups that have taken part in the research;
  • access to quality health care;
  • provision of new diagnostic and therapeutic modalities or products stemming from research;
  • support for health services;
  • access to scientific and technological knowledge;
  • capacity-building facilities for research purposes;
  • other forms of benefit consistent with the principles set out in this Declaration.”

Pathways to Access

Not all countries may be able to meet the basic needs of people living with ALS/MND, or there may be limited availability. Within the current access environment, each country or geography has its own processes for investing in, approving, and making treatments available. The following graphic illustrates the Pathway to Access continuum:

Pathway to Access continuum for ALS/MND therapies.

  • Clinical Trials: Clinical trials are an opportunity for people to try new therapies while still within the research phase. Participation in clinical trials is crucial for people living with ALS/MND, as it can provide them with access to cutting-edge therapies that could have therapeutic benefits, the opportunity to contribute to future treatment development, and access to specialized medical care.
  • Expanded Access Programs/Compassionate Use: This is a potential pathway for a person to gain access to an investigational treatment outside of a clinical trial.
  • Open Label Extension Studies: Open label extension studies are an opportunity for people living with ALS/MND to continue to have access to an experimental therapy once their participation in a clinical trial is complete. The programs/studies eliminate gaps in treatment and enable the person to continue treatment under clinical monitoring.
  • Special Access Programs (SAP): Special access programs are designed to provide people with access to therapies that have not yet been approved in their country or are not yet available through a reimbursed pathway. Manufacturers must agree to provide access through this program, often at no cost.
  • Regulatory Approval: Regulatory approval is the process by which the health authority reviews a drug for safety, efficacy, and quality to determine whether it can be approved for sale in the country. A manufacturer must apply directly to the health authority for drug approval.
  • Drug Reimbursement: In some countries, medical care – including the cost of drugs – is covered by public insurance programs. Once a drug has been approved for sale, there may be an additional process that evaluates the clinical and economic evidence (Health Technology Assessment – HTA) of the drug to determine whether state-sponsored (or public) drug programs will cover the cost of the drug.
    Private insurance companies also undertake their own review of a drug to decide whether they will reimburse the cost through their insurance program. These decisions are specific to the company and to the individual drug plans offered.

None of these pathways create an environment where there is equitable access to therapies for anyone who can benefit – nor do they reflect the urgency of living with ALS/MND.

Impact and Influence

Amyotrophic lateral sclerosis (ALS) or motor neuron disease (MND) is a relentless, fatal motor neuron disease that involves the brain and spinal cord, the body’s muscles, and the motor neurons that send signals between the two. The disease moves with startling swiftness, causing progressive paralysis. Over a two-to-five-year period, on average, someone with ALS/MND will lose the ability to walk, talk, eat, move, swallow – and breathe.

Receiving an ALS/MND diagnosis is devastating, and the disease’s physical, emotional, and financial impacts on a person and their family are immense. Yet, the unique and complex care needs of people living with ALS/MND continue to go unmet regardless of where they live – especially as they relate to opportunities for timely access to potentially beneficial therapies.

We recognize the many pathways a drug moves through to reach market availability. As such, we want to ensure that people living with ALS/MND can leverage as many pathways as possible. There are points all along the Pathway to Access Continuum where Patient Advocacy Groups and ALS/MND Organizations can have an effective impact.

  • Clinical Trials: There are several barriers to accessing clinical trials that are inequitable and disproportionately affect certain populations, including individuals who reside in developing countries and remote areas with limited access to trials. Local health authorities must provide support for multidisciplinary clinics within their region capable of supporting clinical trials. Clinical trial sponsors must also work with ALS/MND clinicians on a global scale to ensure clinical trial sites are established in as many countries as possible. Access to clinical trials is a vital and necessary step in ensuring the fundamental right of access to the highest quality treatment. The Alliance’s research strategy will help expand access to clinical trial sites, particularly in the Global South.

    The PALS & CALS Advisory Council of the International Alliance would emphasize that clinical trials should not economically disadvantage participants and that, wherever possible, the cost of each trial must include costs paid by participants to take part. Parking, travel, hotel, and other costs should be included when in-person participation is necessary, and as much telehealth as possible should be embedded to minimize distance from the trial site as an obstacle to participating. Infrastructure should be local, and equipment and materials should be available to minimize disruption.

  • Open Label Extension Studies: The Alliance believes that open label extension studies must be incorporated into all clinical trial designs and protocols to ensure people living with ALS/MND can continue to access experimental therapies once their participation in a clinical trial has ended. Open label extension studies are critical to ensuring there is no disruption to the course of treatment and are vital to collecting real-world evidence to help support clinical care on a global scale.
  • Expanded Access Programs/Compassionate Use: Manufacturers should take into account the opportunity for the global ALS/MND population to access therapies prior to market-access availability.
  • Special Access Programs (SAP): SAPs are often available only in high-income countries, resulting in limited access to treatments for people in low- and middle-income countries, creating significant disparities in access to treatment and resulting in inequitable health outcomes for people living with ALS/MND. Manufacturers must consider utilizing special access programs, especially in low- and middle-income countries or where access is delayed due to lengthy reimbursement processes.
  • Regulatory Approval: Currently, each country or geography has its own regulatory process for approving ALS/MND therapies, and there is little collaborative engagement with other parts of the world. However, as demonstrated through other disease areas, such as oncology, global models for regulatory approval can successfully bring effective therapies to people as early as possible, especially in countries where there may otherwise have been delays with regulatory submission and review. Given the recent scientific and clinical progress in the ALS/MND space, where new potentially efficacious therapies are emerging through successful clinical trials, the time is right for the Alliance to take on the opportunity and responsibility for advancing an alternative, more harmonized global approach.

    The International Alliance of ALS/MND Associations seeks to encourage all international regulatory agencies to develop a framework for global alignment in evaluating potential new therapies and will work to develop a cooperative, harmonized pathway to approval that increases effectiveness and expediency for treatments of ALS/MND.

  • Drug Reimbursement: Streamlined and swift drug reimbursement must be included as part of any global strategy to improve access to innovative therapies. Once an ALS/MND therapy is approved, it must be reviewed for public reimbursement and covered through a single condensed timeframe applicable to all jurisdictions.

A harmonized global approach that allows for timely access to therapies will uphold the rights of individuals with ALS/MND to have equitable access to new treatments and clinical trials in both developing and developed countries. It will benefit not only the ALS/MND community, but all stakeholders involved in therapy development. Furthermore, with greater access to therapies and clinical trials, disease progression could be slowed, and death could be delayed; independence and quality of life could be retained; and perhaps, with this extension of life, new therapies will become available that will cure the disease.

International Alliance of ALS/MND Associations
October 2023

The original language of communication is English, and the accuracy of messaging in any translation cannot be guaranteed.

APPENDICES

Appendix 1: Fundamental Rights for People with ALS/MND and Caregivers

The Alliance’s Fundamental Rights represent the ideal standard for people living with ALS/MND and their caregivers worldwide. They are reviewed and updated over time to reflect the needs and priorities of the global ALS/MND community.

View the current Fundamental Rights for People with ALS/MND and Caregivers

Appendix 2: WMA Declaration of Lisbon on the Rights of the Patient

The World Medical Association’s Declaration of Lisbon outlines key rights of patients, including the right to appropriate medical care without discrimination, freedom of choice, self-determination, information, confidentiality, dignity, and continuity of care.

Read the WMA Declaration of Lisbon on the Rights of the Patient

Appendix 3: UNESCO Universal Declaration on Bioethics and Human Rights

UNESCO’s Universal Declaration on Bioethics and Human Rights establishes principles addressing ethical issues related to medicine, life sciences, and associated technologies. These include equality, justice and equity, access to quality health care, the sharing of benefits from scientific research, and the responsibilities of states in supporting these principles.

Read the UNESCO Universal Declaration on Bioethics and Human Rights