Facilitating Quality Research

One of the primary reasons that advocacy organizations are formed is to find answers. If all the answers about treatment, management, and life with your condition were readily available, there would be little need for your organization to exist. Sometimes these answers involve treatments, sometimes medical management, but always they are aimed at helping affected individuals live the best life possible. The purpose of this section is to help you to lay out a plan to help your organization define your path to the answers.

The challenge is that the path for every organization and every condition will be different. This chapter will help you through the process by asking you to answer questions. Your answers to the questions will help you define your research plan. The end of the chapter will present three model plans. These plans are not models because they are the best, easiest or recommended. They are just three plans that are working well for their organizations and will serve as examples for you to study.

Identifying Your Needs

Research is an avenue for discovering treatments. First, you need to broaden your idea of research. Research is not just people in white coats and test tubes. Sometimes research is finding answers to simple questions. These questions could be, "How many people are affected with this condition?" "How many have heart involvement?" "What is the genetic component?" Some of these questions do require a laboratory, but many do not. Those that do not can be directly performed by your organization. You may need advice on creating questionnaires, surveys’ or performing the statistical analysis, but these are the questions that advocacy organizations are often best suited to answer.

If you think of the laboratory or clinical components of your research as just bringing in the expertise you need to meet your research goals, then you can allow yourself to take charge of the process. The biggest hurdle is to give yourself permission to go forward. We hope the information in this chapter will help you do just that.

How to Get Started

1. List as many questions as you can think of, including questions from your members. These need not be strictly scientific questions. This list has nothing to do with feasibility, but rather with need and priority. This list needs input from your members and should avoid input from your scientific or medical advisors.
2. Next, give each of the items two rankings. First rank them in order of importance. What is the most important question for your members? Which is the least important? Then rank the same list, but this time put them in order of most easily answered. Which items are the cheapest to answer? The quickest? You may need help from your professional advisory board with this list.
3. Now compare your lists. You should be able too see your priorities emerge. For example, you may decide to focus on one long-term and costly goal and two quick and easy goals.
4. Next look at your long-term and short-term goals. What are the mechanisms that can get these accomplished?

This will depend on many variables.

Are there many researchers already studying your condition, or do you need to drum up converts? If there are already many researchers, then finding new researchers may not be the most efficient approach. You may want to try to get the existing researchers to join your effort. You may wish to create a consortium – and help researchers to share data without threatening their intellectual property.

Are their few researchers working on the condition? Or is no one working on it? Do not despair. Simply figure out the attributes of the condition, look on PubMed and Google Scholar for researchers working on any of these attributes – angiogenesis, apoptosis, immune function – you get the idea. When you find them, contact them (usually the corresponding author’s email address is listed on the abstract or citation. Invite them to think about the condition, offer to find a research cohort, blood and tissue samples, seed funding. Suggest they give a small project related to your condition to a student, fellow, post-doc in their lab. This is the sort of relationship that can grow quickly and eventually the principle investigator or lab director is interested too!

Is funding available from the National Institutes of Health, or another federal agency, to study your condition? Is this funding focused on your particular condition or just the general group of conditions to which your condition belongs?

What percent of your membership would need to participate in order to make progress? If your condition is common, then many narrowly focused projects can be supported by your vast membership. If your condition is rare, then you need to have researchers asking multiple questions, or intial simple ones that can be answered as part of the preliminary work necessary to seek funding.

If you do not yet have a scientific advisory board, call every researcher who has done work on your condition. Even if they are no longer in this line of research, chances are they know who is. Call every name they give you. You will very quickly learn with whom you can work and who will take you seriously.

Internal Links

• Barriers to Rare Disease Research
• Benefits of Collaboration with Advocacy Organization Community
• Blood and Tissue Banks
  • Genetic Alliance BioBank
• Consumers and Researchers
• Educating Membership about Research
• Funding Research by Others
• Getting Needs onto the Research Agenda
• Genetic Privacy
• Orphan Drug Application
• Patient's Bill of Rights
• Promoting Research
• Registries
• Research Models
  • Research Model 1: Recessive Disorder
  • Research Model 2: Chromosomal Disorder
  • Research Model 3: Dominant Disorder