Developments in precision, gene-targeted medicine, as well as other approaches such as immunotherapy and viral oncolytics are showing great promise for treating lethal diseases. Sometimes referred to collectively as personalized medicine, these targeted therapies also highlight a profound gap in the US and Canada. We don’t have a comprehensive policy or strategy for capitalizing on the promise of personalized medicine. From the perspective of people who have incurable diseases, this lack is deadly. Simply put, it takes too long and costs too much to develop and provide access to effective drug treatments for these diseases. Current regulatory and research regimes are cumbersome, expensive and fraught with unnecessary delays.
The regulatory system is clogged and strained (for a summary, please click here).
- The average $800 million it takes to bring a treatment to market means, among other things, that very few drugs actually make it to market. Others that should be tested are not and promising treatments fall by the wayside.
- Tragically, millions of life-years are lost due to approval delays. It can take up to 14 years for a drug to be approved; fast-tracked drugs take about six years.
- Clinical trials are often too complicated and rigid; they must be simpler and easier to access, especially in the case of personalized treatments.
- Reimbursement for the use of approved drugs takes too long, even in the best jurisdictions.
The Life-Saving Therapies Network (LSTN) is a connective hub for patients, researchers, doctors, industry leaders, regulators, healthcare technologists, ethicists and economists who want faster access to better treatments:
- Clinical research reform to permit faster, less expensive access to new therapies for lethal diseases
- Reforming regulatory frameworks for the development and approval of precision treatment for lethal diseases
- Timely and thorough reimbursement for effective lethal disease treatments and companion diagnostics.
- Provide a vehicle for communication and cooperative action among the various key stakeholders and opinion leaders via
- Platforms for debate and discussion
- Posting key articles and news in the field
- Providing links among network members
- Create strategies and a concrete roadmap (action plan) designed to influence research protocols/ regulatory processes and bureaucracies in order to effect faster development of and access to better drugs for lethal diseases in the context of personalized medicine including
- Creating a strong, single voice – to effect the strategies and plan; a voice that marshals resources from the entire LSTN community: patients, oncologists, the pharma industry, medical ethicists, researchers – maybe even payors and regulators
- Providing alternative regulatory frameworks and processes and supporting their active consideration
- Supporting the harmonization of ethics and independent review rules
- Assisting with clinical trial reform
- Creative use of social media
- Supporting publication of books and /or magazine articles
- Producing Ted-talk like pieces focused on the issues
- Participate actively and vigorously in helping to create an international strategy for personalized medicine
LSTN will generate a Roadmap for Change, a three to five year plan which will contain expected outcomes and success measures to create progress-centered regulations that facilitate
- Clinical research reform to permit faster, less expensive access to new therapies for lethal diseases, and
- Reforming regulatory frameworks for the development and approval of treatment for lethal diseases.
As a key first step, LSTN brought together key opinion leaders and stakeholders from the Canadian cancer community – oncologists, industry representatives, researchers, patients and caregivers, regulators – to a Roundtable discussion. The purpose of the Roundtable was to frame the Roadmap and generate commitment to it.
- Preparation for the Roundtable
- Research and targeted interviews with key players to gather information and opinions
- A mapping of key stakeholders, their sensitivities and basic interests, perspectives, areas of convergence and divergence
- Identification of participants in the Roundtable
- Briefing Document
- Circulated to participants prior to the Roundtable
- Thorough, concise understanding of the key issues/challenges, building blocks, roadblocks, and developmental opportunities for moving the conversation forward and affecting regulatory change
- Basis for a few selected interviews to clarify issues, etc.
- Roundtable – Process and Outcomes
- Facilitate discussion in plenary and workshop format
- Create and clarify overall policy issues and goals
- Create overview action plan with recommendations for
- overall milestones
- metrics by which success will be measured
- Generate commitment to public discussion of the need for regulatory and research reform in the context of a personalized medicine strategy including suggestions for
- communication strategy
- forums for promoting public and specialist discussion of the issues among themselves and with policymakers
- vehicles for stakeholders can work together to define actionable solutions
Making the Roadmap a Reality
The Roadmap for Change will be available to all stakeholders and be continuously updated. It will form a living database of evidence, experiences, recommendations and suggestions — the basis for initiatives by the stakeholders working together, in small groups or separately to achieve our goals.