NIH Programs Supporting Collaboration

Learn about special programs offered by the NIH at early stages of therapy development.

• NIH ICs: Determine which NIH ICs are involved in researching the rare disease(s) supported by your group.
  • Contact the specific IC to learn about any special programs they have in place for working with patient groups. For example:
    • The National Cancer Institute has an Office of Advocacy Relations , dedicated to working with patient groups. You can contact them directly by phone: (240) 781-3360 or email: nciadvocacy@nih.gov 
• National Center for Advancing Translational Science (NCATS): NCATS has a number of different programs and funding opportunities focused on the discovery stage of therapy development.
  • General information is available on Patient/Community Engagement & Health Information. 
  • Programs that may be of interest to researchers working with your groups are outlined on Work with NCATS.
• Rare Disease Clinical Research Network (RDCRN) : The RDCRN is a special program supported by the Office of Rare Disease Research (ORDR), NCATS, and collaborating ICs. The program funds research teams, called Consortia, that work to better understand rare diseases and move potential treatments closer to the clinic.
  • Consortia are a network of scientists, clinicians, patients, families, caregivers, and patient groups.
  • There are 20 funded programs per cycle, and a cycle lasts 5 years.
  • Consortia can reapply for another 5 years of funding, but new networks will also compete. 
  • Most Consortia include a group of related conditions. 
  • Each Consortia includes multiple clinical and research sites.
  • The most recent Consortia funding was announced Fall of 2019. 
  • The opportunity to apply to become an RDCRN Consortia is usually announced late spring or early summer the year prior to when the funding cycle will start. Thus, currently a new announcement will occur late spring, early summer 2024: RDCRN Funding Information.
  • In addition, RDCRN Conference on Clinical Research for Rare Diseases (CCRRD) occurs about every 2 to 4 years and focuses on rare disease research methodology.
    • Topics and slide decks from past conferences are available.

Contact Registry

Contact registries typically are focused on including people with a specific disease or condition who may be interested in becoming involved in future clinical trials.

• Type of information stored may include:
  • Contact information of the patient.
  • Demographic information, such as age or sex.
  • Basic health information that may be important to determine clinical trial eligibility.
• Purpose: 
  • Patients in the registry can be notified when a clinical trial or research opportunity becomes available.
  • Patients can learn about opportunities to provide feedback to the FDA and advisory committees.
  • Medical researchers and Industry Partners can seek out patients to be involved in surveys, focus groups, and clinical trials.
• Advantages:
  • Allows researchers in academia and industry to identify people willing to be involved in clinical trials.
  • In most cases, the system sends the information about the trial directly to the patient instead of providing the principal investigator (or PI) with the patient’s contact information.
  • Being part of a contact registry does not obligate a person to join a clinical trial.
  • Minimal effort to set up and maintain.
  • Your group’s member contact list can provide a good base to start a contact registry.
  • If disease pathology and patient population is well characterized, a contact registry may be sufficient. 
• Challenges:
  • Collects only minimal, if any, information about the disease from patient or clinical perspective and therefore will not provide specific information about the disease or patient population.
  • In comparison to a group’s member contact list, a contact registry database:
    • Should separate contact information and other personal identifying information from health information. 
    • Requires clear informed consent so that those providing information know what is being done with information, how it will be stored, and who will have access.

Patient Registries

In the context of therapy development, a patient registry (also called a disease registry) is a database that collects and stores information about patients diagnosed with a specific disease, genetic disorder, or medical condition.

• Registry types: There are 2 general categories of registries.
  • A Contact registry can help connect patients to clinical trials and researchers to patients interested in furthering research.
  • A Natural History Study registry collects information about a disease from a patient and clinical perspective that can inform every stage of the therapy development process. 
• Registry vs. clinical trial: A patient registry can be listed in ClinicalTrials.gov , but these registries differ from treatment-based clinical trials  in several important ways:
  • A registry does not investigate a new therapy.
    • Registries are based on the patient’s actual experience and their current medical care.
    • A treatment clinical trial has certain requirements, for example it will require a volunteer to take a certain treatment or use a specific medical device and follow a specific protocol to track the safety and effectiveness of the therapy.
    • Registries are observational.
    • Clinical trials are experimental.
  • In almost every case, there is no cost to join a registry and medical insurance providers are not involved. 
    • Depending on the clinical trial, a participant’s medical insurance and, ultimately, the participant may be responsible for certain portions or all of the expenses related to the treatment clinical trial protocol. 
• Founders of a registry: A patient or disease registry can be set up by different stakeholders, however, forming a collaboration among several of the key stakeholders can increase the value of the data collected in the registry. Collaborators can include:
  • Patient groups (including their Medical or Scientific Advisors)
  • Clinicians
  • Academic researchers
  • Pharmaceutical companies
  • Biotechnology companies
• Rare and genetic disease registries: A registry is important to a rare disease community and is especially valuable in certain situations. For example, when:
  • Small patient populations make it difficult to attract enough patients to run a clinical trial.
  • The disease is not well characterized, leading to delayed and missed diagnosis.
  • Poor understanding of the natural history of the disease and its progression without treatment makes it difficult to evaluate the effectiveness of a new therapy.
  • No biomarkers have been validated. 
  • Clinical endpoints are unclear.
  • Clinical trial designs do not include the patient perspective, making retention in a clinical trial a challenge. 
• International registries: If a disease is relatively common, registries may only include people from a specific country . However, rare diseases benefit greatly from a global approach for a number of reasons. For example, an international registry:
  • Unifies efforts and data collection methods.
  • Increases statistical significance of data.
• Importance of registry: Registries benefit a number of populations, including patients and their caregivers, clinicians, researchers, industry partners, and regulatory agencies.
  • For patients and their caregivers, registries can:
    • Allow their voice to be heard. 
    • Capture the knowledge gained from living with the disease.
    • Increase the probability that a treatment or cure may be developed.
    • Provide valuable information to support accommodations and services that may  be needed at school, places of employment, or home.
    •  Empower and unite the community.
  • Registries can allow clinicians to: 
    • Better understand how the disease progresses over time.
    • Discover the signs and symptoms that are most responsible for decreasing a  patient’s quality of life. 
    • Explore the heterogeneity of signs, symptoms, and progression within the patient population.
    • Develop diagnostic criteria to decrease time to diagnosis.
    • Publish treatment and management guidelines.
  • For medical researchers, registries:
    • Validate animal models.
    • Establish patient-reported outcome and other clinical outcome measures.
    • Identify biomarkers.
    • Determine disease incidence.
    • Uncover common traits, behaviors, or symptoms not previously realized that may provide a better understanding of the pathology of the disease or new targets for treatment.  
    • Identify unmet medical needs to guide focus of treatment research.
  • Industry partners can gain insight on:
    • The established course of disease, biomarkers, and patient-reported outcome and other clinical outcome measures to increase the ability of a treatment to be evaluated for safety and effectiveness in clinical trials.
    • Improved clinical trials design based on knowledge from the patient perspective which can increase enrollment and retention. 
  • For regulators: 
    • An active, engaged patient community is one of the key components to  a successful therapy development and regulatory review process, especially for rare and genetic diseases.
    • Safety and efficacy of a newly approved treatment or medical device can be tracked accurately in an established natural history registry.
• Learn more:
  • Section 5: Encourage Sharing of Data and Section 6: Providing Data in From Molecules to Medicine: How Patients Can Their Voices Throughout The Drug Development Process (2018) provide a brief overview of the benefits and importance of sharing patient data and examples from different patient groups. This resource was developed by Global Genes .
  • Rare Disease Registries: Advancing Disease Understanding, Treatments, and Cures is a Global Genes document that discusses the importance of registries throughout the therapy development process as well as some factors that are important to building a good registry.
  • List of Registries includes answers to common questions about registries and links to multiple national registries.  This resource is updated periodically and is part of NIH Clinical Research Trials and You.

PFDD Initiative

The PFDD Initiative grew out of the realization that patients, their families, caregivers, and associated patient groups are experts in living with their condition and are uniquely positioned to inform the understanding of the therapeutic context for therapy development and evaluation. 

• FDA-led PFDD public meetings: The FDA wanted to include information in their marketing review process from the patient and caregiver perspective, but needed the collection of this information to be systematic and representative of the whole patient population. FDA-led PFDD public meetings were piloted as a method to collect this data.
  • Patient perspectives were sought concerning 2 main areas:
    • Most bothersome signs/symptoms of their condition and the impact of the condition on daily life, including:
      • Burden of managing the disease.
      • Affected daily activities.
      • Unmet medical needs.
      • Barriers to participating in clinical studies.
    • Current and potential approaches to treatment, such as:
      • Impact on patient’s ability to perform daily activities.
      • Minimum expectations of benefits.
      • Tolerance for harms or risks.
      • Acceptable trade-offs of benefits and risks (i.e., patient preference).
      • Attitudes towards uncertainty.
  • The FDA hosted 24, mostly disease-specific, public PFDD Meetings between 2012 and 2017 to determine the feasibility of such meetings to achieve their goals.
    • Since 2018, the FDA continues to host periodic PFDD meetings, but the focus is on much broader public health issues, such as opioid use disorder, chronic pain, and stimulant use disorder.
  • Sample questions asked included: 
    • Which symptoms have the most significant impact on your daily life?
    • Which symptoms have the most significant impact on your ability to do specific activities?
    • How well does your current treatment regimen treat the most significant symptoms of your disease?
    • What specific things would you look for in an ideal treatment for your condition?
    • What factors do you take into account when making decisions about using treatments?
    • What factors do you take into account when deciding whether to participate in a clinical trial?
  • Additional information gained through the meetings included:
    • Outreach is the key to success.
    • Specific experiences that mattered most to patients.
    • Patient perspectives on meaningful treatment benefits.
    • Patient-focused methods for engaging them in the therapy development process.
  • Following each PFDD meeting, FDA summarizes the input shared by patients and patient representatives in a Voice of the Patient report.
  • You can learn more about these initial meetings and future meetings on the FDA-led Patient-Focused Drug Development (PFDD) Public Meetings webpage. For each meeting there are links to the:
    • Agenda
    • Discussion questions
    • Voice of the Patient report
  • During the period of the first PFDD meetings, the National Health Council developed a tool to help patient groups collect patient information. Although the actual tool is no longer available, the accompanying manual is. The manual describes how your group can gather, translate, and share information. It also includes the efforts of Unite Narcolepsy, one first disease communities to have an FDA-led PFDD, to prepare their community for the public meeting.
    • Implementation Manual: How to Operationalize the National Health Council’s Patient Information Tool (2012)
• Externally-led PFDD: Since the number of public meetings the FDA can host is limited, the FDA encourages patient groups, in collaboration with other stakeholders, to host externally-led PFDD meetings.
  • When appropriately modeled after FDA-led PFDD meetings, externally-led PFDD meetings can inform the FDA and therapy development through the patient voice. 
  • Because the meetings take resources and effort, you may wish to consider whether there is an identified need for a better understanding of the patient perspective for purposes of therapy development for your disease.
  • Meetings may be most helpful for disease areas:  
    • That are chronic, symptomatic, or affect the functioning and activities of daily living.
    • For aspects of the disease that are not formally captured in clinical trials.
    • For which there are currently no therapies or very few therapies, or the available therapies do not directly affect how a patient feels, functions, or survives.
    • That have a severe impact on identifiable subpopulations, such as children or the elderly. 
  • Externally-led PFDD meetings are appropriate for most rare diseases.
  • FasterCures offers a self-guided PFDD Readiness Assessment tool as one means to determine whether your group is ready to plan an externally-led PFDD.
• General elements of PFDD meeting model:
  • Key participants: Patients, patient families and caregivers, patient advocates.
  • Target audience: FDA and other federal agencies, therapy developers, researchers, healthcare professionals.
  • Meetings or sessions are facilitator led discussions.
  • Interactive webcasts increase the diversity of patient voice.
  • Polling tools are used to gather answers to questions from both those in attendance and those attending via webcast.
  • Main discussion topics should include:
    • Symptoms and impacts that matter most to patients, families, and caregivers.
    • Challenges of current approaches to treatment.
  • Web recording of meeting and transcript may be posted on the group website.
  • Summary report (Voice of the Patient) can be submitted to be posted on External Resources or Information Related to Patients’ Experience  (updated periodically).

Planning an Externally-Led PFDD

The FDA has multiple resources to help your group plan a successful externally-led PFDD. FDA’s Center for Drug Evaluation and Research (CDER ) PFDD Program Staff leads the externally-led PFDD program.

• Submit a Letter of Intent (LOI) at least 1 year in advance to the planned meeting date.
  • Refer to Guidelines for Developing a Letter of Intent (LOI) for Externally Led Patient Focused Drug Development Meetings
    • These guidelines state the LOI can be approximately 5 pages long, but current information on the externally-led PFDD webpage states approximately 3 pages long.
  • LOIs are reviewed 4 times per year by the CDER PFDD Program Staff.
• After an LOI is accepted by the FDA, CDER PFDD Program Staff will reach out to you and upon request provide specific recommendations on the planning of the meeting including:
  • Development of agenda.
  • Discussion/polling questions.
• FDA cannot provide a meeting space, facilitator/moderator, or financial assistance. 
• Previous FDA-led PFDD meetings provide examples of agendas, slides, recordings, transcripts, and reports that can help guide the planning process.   
• Meeting should not require hiring event planners, consultants, scientific writers, or other external resources on your team, especially when resources may be limited.
• If resources are limited, consider web-based/virtual meetings or including as a session or workshop during an already planned event or conference.
• Successful meetings include other patient groups and stakeholders in your disease community when possible.
• Seeking outside funding, for example, from therapy developers or large umbrella groups, is encouraged.
  • Disclose any known funding resources in LOI. 
  • If established after LOI submission, include in the planning materials and deliverables.
  • Therapy developers, whether academic or pharmaceutical, should not be involved in the development of the meeting agenda, discussion/polling questions, or selection of patient panelists. 
• If you have questions, contact FDA’s CDER Patient-Focused Drug Development Program Staff at patientfocused@fda.hhs.gov  
• Externally led Patient-Focused Drug Development Meetings , an FDA webpage, provides more information and links to resources in easy to read format and language. 
• For even more detail, you may consider referring to Patient-Focused Drug Development: Collecting Comprehensive and Representative Input Guidance for Industry, Food and Drug Administration Staff, and Other Stakeholders (2018). However, this resource is written in a more formal style and contains high levels of technical language.
  • This draft guidance (Guidance 1) is the first of a series of four PFDD FDA guidance documents. 
  • Guidance 1 answers the questions:
    • Whom do you get input from, and why? 
    • How do you collect the information?

Natural History Study

Natural history study databases contain more detailed clinical information over time, such as age at diagnosis, symptoms, medical images, and test results. Data may be entered by patients, their caregivers, or healthcare professionals.

• Type of information stored may include: 
  • Patient contact information.
  • Clinical information as it becomes available, such as:
    • Diagnosis
    • Signs and symptoms
    • Medical images
    • Test results.
  • Patient experiences, such as:
    • Effect on quality of life.
    • Positive and negatives of current treatments.
  • Family and caregiver perspectives.
  • Clinicians experience.
• Purpose: Data from natural history studies is more complex than that found in contact registries, and can inform every part of the therapy development process, as well as benefit your disease community in other ways. A natural history study registry can:
  • Notify patients within the registry when a clinical trial or research opportunity becomes available.
  • Provide information to help researchers and clinicians understand the disease course over time (natural history), as well as validate animal models.
  • Better characterize the disease. This can include:
    • Range in types and severity of signs and symptoms among patients.
    • Changes in signs and symptoms over time.
    • If genetic, determine whether there is a correlation between genotype and phenotype.
    • Environmental factors.
  • Characterize patient population.
    • Identify subgroups within the population.
  • Understand the day-to-day effects of the disease on the lives of patients and their families from their perspective.
    • Highlight areas of unmet medical need.
    • Uncover environmental and lifestyle factors.
  • Inform research priorities from patient and clinical perspectives.
  • Establish diagnostic criteria.
  • Develop clinical care guidelines.
  • Ensure patients are receiving established clinical guidelines.
  • Identify biomarkers.
  • Collect patient-reported outcomes and other clinical outcomes, which can be used to identify outcome measures to be used in clinical trials. 
  • Increase patient participation and retention in clinical trials.
  • Compile data from the patient perspective that can be used by the FDA as part of their marketing review process.
  • Amass data to advocate for expanded insurance coverage for therapies and services.
  • Track safety and effectiveness of new therapies after FDA marketing approval. 
• Advantages:
  • If the disease and the patient population are not well characterized, the data from a natural history study can affect every stage of the therapy development process.
  • Rewards the entire disease community quickly through development of clear diagnostic criteria or clinical management guidelines.
  • Raises awareness of disease in medical, academic, and pharmaceutical communities.
  • Provides your patient community with a sense of momentum and increase interest in participation in research.
  • Long term rewards include development of more effective, safe treatments.
  • Ultimate reward is a cure for the disease.
• Challenges:
  • Can be costly and time consuming to establish and maintain.
  • May have to overcome patient population concerns about sharing health records or personal experiences. 
  • May be difficult to determine the most important information to collect, especially when disease causes symptoms in multiple organ systems. 
  • Requires outreach beyond your group’s members to attain a representative population.
  • Collecting too much data can make analysis more difficult.
  • Requires Informed Consent, Institutional  Review Board (IRB) approval, and Health Insurance Portability and Accountability Act (HIPAA) certification.
• Grants and more:
  • The FDA Orphan Products Natural History Grants Program is a program funding natural history studies for rare diseases in order to guide therapy development. This funding can also support studies targeted at the validation of biomarkers or the development of clinical outcome measures. 
    • Learn more about the program by reading FAQs – Natural History Grants .
    • Check Find Funding Opportunities periodically for the next application deadline or sign up for email notification. Other grants opportunities offered through the FDA Office of Orphan Product Development (OOPD) are also listed on this  page. 
    • Grant Application Overview also provides information about the Natural History Grant Program through OOPD.
  • Natural History Clinical Trials (2016) is a video from a session at the Global Genes Rare Patient Advocacy Summit regarding the importance of natural history studies and ways in which they can contribute to rare disease research. The presentation also provides information about resources that can help with engagement in natural history studies.

Building a Registry

It is important that the information in your registry database will be helpful to the stakeholders in the therapy development process. You also want to collect the information using the correct methods so that it can be used by medical researchers, industry partners, and the FDA. Because this process is crucial to laying the foundation for the therapy development process, especially for rare and genetic diseases, the National Center for Advancing Translational Sciences (NCATS) established a website devoted to building your registry. 

NCATS Rare Diseases Registry Program (RaDaR) includes step by step guidance to help you develop your registry. It is not a registry platform, but rather a collection of best practices, templates, and resources to help you achieve your goal. So if you want to learn more about building a registry or you are ready to begin, we encourage you to explore RaDaR:

• Rare Diseases Registry Program (RaDaR)

Tips for Success

Tips for patient registries and natural history studies:

• Anticipate popular misconceptions and be prepared to alleviate concerns or unrealistic expectations. Being in a registry:
  • Does not obligate a person to join a clinical trial if they are contacted.
  • Does not guarantee that a person will be asked or able to join a clinical trial.
  • Does not ensure a new treatment or cure will be developed.
  • Does not promise any benefit from the registry in the patient’s lifetime.
• Address your disease community’s concerns about privacy and security early in the process.
• Educate your disease community about short- and long-term goals of the registry.
• Highlight the added benefits, for example, finding others who have the same challenges you are experiencing but that may not be published in the medical literature.
• Empower your patient community to help direct the therapy development process for their disease. 
• Keep those in the registry engaged in the process so that the data continues to grow. 
• Celebrate achievements.

Tips for externally-led PFDD Meetings:

• Because the information produced by externally led PFDD meetings has the potential to affect all stages of the therapy development process, many groups hold PFDD meetings long before there is a possible therapy heading to clinical trials. 
• PFDD-style sessions can be held during annual conferences, scientific workshops, etc.
• Begin outreach early and go beyond your own group’s membership, potentially reaching out to social media groups, clinics, and medical professionals serving the patient population, and larger umbrella organizations.
• Raise funds or find Sponsors to provide financial travel assistance to patients and their families.
• Organize transportation from key centers if possible.
• Provide tips to help your group members and other patients attending a PFDD meeting share their information effectively and relatively concisely.
  • Possibly offer practice opportunities through webinars. 
• Consider alternative ways for patients and/or their families and caregivers to participate who cannot attend the meeting and may not have access to the internet.
  • Paper copies of the questions (survey) can be mailed at the request of patients, families, and caregivers.
  • Including a self-addressed, stamped envelope to increase return of completed surveys.
• Remember to invite medical researchers and representatives from pharmaceutical groups to be listeners at the event.
• FDA has a plain language PowerPoint presentation reviewing both FDA-led and externally-led PFDD meetings: Patient Focused Drug Development .
  • This presentation may be helpful when discussing hosting a meeting with your Board of Directors, key staff members, Scientific/Medical Advisory Board, and other stakeholders.

Overview

Your group may wish to start a patient registry or support a natural history study for your disease. Patient registries and natural history databases are proving to be invaluable tools for stimulating research. Documenting the experiences of patients and their caregivers provides valuable information for researchers, industry partners, and regulators. The type of data collected will vary depending on the purpose of the registry. These databases are helping engage patients in the therapy development process early, often, and effectively.

In addition, your group may consider organizing an Externally-led Patient-Focused Drug Development Meeting (PFDD). The U.S. Food and Drug Administration (FDA) encourages patient groups to coordinate and run meetings to capture patient perspectives on specific diseases and current treatments using the process established through the FDA’s PFDD Initiative. The format of the meetings was developed through the 24 FDA-led PFDD public meetings. Your patient group can submit a Letter of Intent (LOI) to the FDA and receive assistance to adapt the process for your disease(s).

Discovery of Therapeutic Approach

The search for a therapy, whether a drug, biologic, or medical device, begins when a disease or clinical condition does not have an effective treatment currently available. Finding a therapeutic approach relies on research that provides an understanding of the disease process. Although not as costly as preclinical studies and clinical trials, this stage is considered a high risk financially because it may take many years and capital to find a target and subsequently a candidate therapy that will make it through all the clinical trials and be approved for market. This stage also has the greatest potential for failures and often requires researchers to go back to either identifying a new target or candidate therapy. 

• Identifying a target: Using knowledge about the disease, researchers develop a hypothesis that inhibiting or activating a specific target involved in the disease process will be able to change the course of the disease in a positive way. There is no set way to find a potential target beyond an educated guess. 
  • A target can be a gene, RNA, protein, biological pathway, physiological response, virus, or bacteria. 
  • A target must have the potential to be affected by a therapy in a measurable way. For example, the interaction between a target and a drug or biologic must cause a biological response that can be measured both in a test tube or cell culture (in vitro) and in a living organism (in vivo). A target medical device interaction must also be measurable. 
  • A target goes through multiple levels of validation to make sure that, if it is affected in the hypothesized way, the result will indeed be therapeutic. 
• Determining therapy type: After identifying a target, researchers will need to determine the type of therapy that will be able to affect the target. The term “therapy”, as used in NCATS Toolkit, includes:
  • Drugs and medications: Conventionally, drugs are synthesized pharmaceutical molecules or compounds intended to treat or cure a disease. However since the FDA includes biologics in their definition of drugs, a more accurate term for this type of therapy is small molecule drug.
    • Small molecule drugs are currently still the most common therapy that is developed. 
    • Examples include: acetaminophen, ibuprofen, metformin, gabapentin, and amoxicillin. 
  • Biologics: Also called biopharmaceuticals, biologics are derived from biological sources. 
    • Biologics include a wide range of therapies such as vaccines, blood and blood components, allergenics, somatic cells, gene therapy, tissues, and recombinant therapeutic proteins. 
    • Most biologics developed as a therapy are complex mixtures that are not easily identified or characterized. 
    • Examples include: insulin, Botox (onabotulinumtoxina), Humira (adalimumab), and Rituxan (rituximab).
  • Medical devices: In contrast to drugs and biologics, many complex devices involve a number of components that, together, form a system. 
    • Medical devices may be intended to diagnose, cure, treat, or mitigate disease. 
    • The definition of medical device is wide, and examples can range from adhesive bandages and tongue depressors to implanted cardiac and neurological devices, stair-walking wheelchairs, robotic surgical systems, and magnetic resonance imaging (MRI) devices.
• Finding a potential therapy: In the best-case scenario, when focused on drug or biologic development, several potential therapies are identified that have the desired effect. Further testing and screening then narrows the field to several lead candidates for intensive testing before industry or researchers proceed to preclinical studies. 
  • Typically, researchers discover new drugs that can affect a validated disease target through: 
    • Screening many molecular compounds and biologics to find ones that have the potential produce the desired effect when interacting with the target.
      • Prior to screening, researchers develop biological assays (bioassays) that can measure compound-target interactions.
      • When possible the bioassay is adapted to high throughput screening (HTS). 
        • HTS automates and miniaturizes the bioassay using robotics, data processing, and sensitive detectors. 
        • HTS can quickly perform millions of assays to determine the therapeutic-like properties of large libraries of compounds.
      • NCATS Toolkit provides more information and resources to learn more about bioassays in Discovery: Understand Discovery Research Tools.
    • Designing a therapy to create the desired effect. 
    • Repurposing existing treatments that have unanticipated effects and are found to affect the target “accidentally.”
    • Using new technologies, such as those that provide new ways to target therapies to specific sites within the body or to manipulate genetic material.
  • Lead therapies are tested further to ensure their effect is reproducible in other assays or in vitro systems. Identified therapies are also compared to determine which may work best. Further considerations include:
    • Absorption, distribution, metabolism, and excretion properties. In other words, how a body processes the therapy (pharmacokinetics).
    • Impact on other functions within the body (pharmacodynamics).
    • Potential toxicity or side effects.
    • Dose-response curves.
    • Method of administration, for example, whether it is taken in pill form, injected, or inhaled and whether this administration will work for the patient population.
    • Interactions with other drugs and treatments, especially those used by the patient population.
    • Differing effects on different groups of people, such as by age, sex, race, or ethnicity.
    • Ability to be produced and manufactured.
• NCATS Toolkit provides more information on the Discovery stage of therapy development including key areas your group can support the process. Key areas include:
  • Collecting patient data.
  • Facilitating scientific collaboration. 
  • Funding research.