Project Mercury’s work is innovative and often complex. Thus, a little explanation about some of the terms we often use is probably in order!
Access (also referred to as ‘Patient Access’): It refers to the ability for individuals to access the medicines they need at acceptable prices. Unfortunately, in rare diseases like FSHD, medicines are often expensive and getting the government health payers and private health insurers to agree to cover the costs can be seriously delayed or even denied. Click here for an illustration of the access challenge.
Advocacy Group/Organization: Non-profit organizations that focus on a single disease or a broader category of diseases. For example, in Canada, the FSHD Canada Foundation advocates specifically for FSHD and Muscular Dystrophy Canada advocates across all neuromuscular diseases.
Approved Therapies/Medicines: Drugs and medical devices that have received approval from regulatory bodies like the Food and Drug Administration (FDA) in the United States, Health Canada in Canada, European Medicines Agency (EMA) in Europe, etc.
Biopharma Company/Industry: Companies that develop medicines, medical devices and other therapies. There are many companies working in FSHD. Two examples are Fulcrum Therapeutics and Avidity Biosciences. A more complete list of companies can be found here.
Clinical Research: is intended to produce knowledge valuable for understanding FSHD, living with and treating it, and promoting the best possible health outcomes. Examples of clinical research include genetic studies that involves blood tests but no changes in medication; studies of family history that involves gathering insights from family members to learn about people’s medical needs and history; surveys of patients and families to understand the burden and costs of living with FSHD; testing of new FSHD medications or devices (often referred to as clinical trials).
Clinical Trials: There are two types of clinical trials. An interventional clinical trial is a voluntary research study conducted in people and designed to answer specific questions about the safety or effectiveness of drugs, vaccines, medical devices, other therapies, or new ways of using existing treatments. Most interventional clinical trials are sponsored by biopharma companies. An example of an interventional study is the REACH trial sponsored by Fulcrum Therapeutics. Observational studies don’t test potential treatments. Instead, researchers observe participants who have volunteered to participate on their current treatment plan and track health outcomes. An example of an observational study in FSHD is the MOVE study.
Clinician: a person qualified in the clinical practice of medicine, psychiatry, or psychology as distinguished from one specializing in laboratory or research techniques or in theory. Examples of clinicians are primary care doctors, pediatricians, neuromuscular disease specialists, psychiatrists and psychologists, and physical therapists.
Country Working Group (CWG): a formal group of people and organizations in a country working to implement various programs of Project Mercury. Led by a FSHD-specific non-profit advocacy group (most often a member of the World FSHD Alliance), the group includes experts from various fields and volunteer patient engagement leaders. CWG’s work with other CWG’s and the Global Task Force to maximize and share resources to ensure efficiency so work and outputs that only need to be done once are not being duplicated elsewhere. This ‘shared-value’ approach to the network of CWG’s is critical to success in Project Mercury’s vision to speed the delivery of therapies in FSHD.
Global Task Force (GTF): is the oversight body of Project Mercury. The GTF comprises two types of members. Voting Members are the leaders from each of the Country Working Groups. Non-Voting Members are comprised of biopharma company representatives, clinicians, researchers and research network representatives, patient registry managers, patient education experts, experts from patient access and clinical trial readiness and regulatory and payer representatives.
Patient Registry: is an organized system that uses observational study methods to collect data (clinical and other) to evaluate specified outcomes for people affected by FSHD. There are many FSHD registries globally. Registry organizers receive information from multiple sources (patient-reported and clinician-reported chief among them), maintain the information over time, control access to the information and are responsible for keep patient information secure. For Project Mercury, registries are critically important to success in engaging patients and families in clinical research and clinical trial engagement.
Payer: in the health care industry are organizations — such as private health plan providers/insurers, Medicare and Medicaid in the United States, the National Healthcare System in the UK and Canadian Medicare — just to name a few. These organizations set service rates, determine what drugs/therapies for which they cover the costs, collect payments, process claims, and pay healthcare provider claims. For Project Mercury, payers are a critical player in our patient access work.
Regulatory Agency: the drug regulatory authority – are agencies that develop and implement most of the legislation and regulations on pharmaceuticals in their respective country or region. Their main task is to ensure the quality, safety and efficacy of drugs, therapies and medical devices, and the accuracy of product information. Examples of these agencies are the National Institute for Health and Care Excellence (NICE) in the UK, the European Medicines Agency (EMA), the U.S. Food and Drug Administration (FDA), and the Therapeutics Goods Administration (TGA) in Australia – to name a few. Biopharma companies submit their clinical trial data to these agencies when they are seeking approval of their drug, therapy or medical device in that country/region.
Researcher: a health professional who works directly with patients, or uses data from patients (often from patient registries), to do research on health and disease and to develop new treatments. These professionals work in a variety of places, including academic institutions, biopharma companies, government institutions and non-profit advocacy organizations.
Therapy/Therapies/Treatments: is the attempted remediation of a health problem, usually following a medical diagnosis. There are many different types – drugs, medical devices, physical therapy, mental health and behavior therapy, and more. For Project Mercury, unless otherwise described, we use the term in reference to drugs and medical devices.