PatientView | USA insights from Patient Groups: A seat at the pricing table

Patient group goals

The percentages below highlight how the 91 patient groups interact with regulators and advocate for patient interests. As respondents may be represented in more than one area, percentages may not total 100%.

interactions

How patient groups interact with drug regulators

Find out what the goals mean

32 mentions

17 mentions

15 mentions

11 mentions

5 mentions

1 mention

actions & advocacy

How patient groups get access to medicines

Find out what the goals mean

27 mentions

22 mentions

3 mentions

2 mentions

English text	Therapy area	Category	Patients reached	Geo reach	Regulatory engagement	Respondent
Our community members participate in this at an individual level, and as an organisation, we meet with industry policy holders.	Cancer > Cancer: women’s	Regulatory partnership > Working with regulatory bodies	10,000	International	Rarely	119028011659
We collaborated on an externally-led Patient-Focused Drug Development meeting with the FDA [Food and Drug Administration] in 2025.	Neurological > Multiple sclerosis	Regulatory partnership > Working with regulatory bodies	100,000	National	Rarely	119039458452
We engage in education, guidance, and institutional dialogue initiatives to promote informed, equitable decisions that place community well-being at their centre.	Patient advocacy > Training	Regulatory partnership > Working with regulatory bodies	100,000	International	Regularly	119063146174
Through written comments, sign-on letters, and participating in roundtable meetings where drugs are being discussed.	Cancer > Cancer: sarcoma	Regulatory partnership > Working with regulatory bodies	10,000	International	Sometimes	119064713792
Just recently hosted an EL-PFDD [Externally-Led Patient-Focused Drug Development] meeting and attended FDA [Food and Drug Administration] meetings with industry partners.	Rare > Rare disorders: genetic	Regulatory partnership > Working with regulatory bodies	5,000	International	Regularly	119065526277
... We work closely with the FDA and other drug agencies, like EMA [European Medicines Agency] and PDMA [Pharmaceuticals and Medical Devices Agency], to look at endpoints for drug trials, or how to improve drug trials to benefit sponsors and patients.	Kidney > Kidney diseases	Regulatory partnership > Working with regulatory bodies	100,000	National	Regularly	119070696943
Participation in activities like PDUFA [Prescription Drug User Fee Act] and MDUFA [Medical Device User Fee and Modernization Act] discussions.	Rare > Rare diseases: most types	Regulatory partnership > Working with regulatory bodies	100,000	National	Rarely	119078556821
We have a policy committee and weigh in on public policy issues through education and advocacy.	Chronic > Palliative	Regulatory partnership > Working with regulatory bodies	100	National	Regularly	119078649119
Discussions about review and approvals of new screening technologies.	Cancer > Cancer: most types	Regulatory partnership > Participate in Health Technology Assessment (HTA) and reimbursement reviews	100,000	National	Rarely	119045117950
Our organisation actively advocates for the interests of the individuals and communities we serve before regulatory authorities and decision-making bodies on matters relating to medicine access, affordability, and coverage recommendations.	Patient advocacy > Training	Regulatory partnership > Participate in Health Technology Assessment (HTA) and reimbursement reviews	100,000	International	Regularly	119063146174
... Our government relations team responds to comment letters and regularly talks with CMS [Centers for Medicare & Medicaid Services] to ensure best outcomes.	Kidney > Kidney diseases	Regulatory partnership > Participate in Health Technology Assessment (HTA) and reimbursement reviews	100,000	National	Regularly	119070696943
Our organization has participated in responses to ICER [Institute for Clinical and Economic Review].	Respiratory > Lung diseases	Regulatory partnership > Participate in Health Technology Assessment (HTA) and reimbursement reviews	100,000	International	Rarely	119078556838
We are typically advisors on ICER [Institute for Clinical and Economic Review] reviews.	Rheumatic > Lupus	Regulatory partnership > Participate in Health Technology Assessment (HTA) and reimbursement reviews	100,000	National	Regularly	119082751826
Testifying on behalf of pharma & device companies in front of the FDA [Food and Drug Administration].	Transplants > Transplants: heart and lung	Regulatory partnership > Attend/facilitate meetings with policymakers/regulators	5,000	Local	Regularly	119014633092
Discussions with FDA [Food and Drug Administration].	General health > Diverse communities	Regulatory partnership > Attend/facilitate meetings with policymakers/regulators	10,000	International	Rarely	119026771169
I personally have testified before the FDA [Food and Drug Administration] and CMS [Centers for Medicare & Medicaid Services].	Transplants > Transplants: heart and lung	Regulatory partnership > Attend/facilitate meetings with policymakers/regulators	1,000	Regional	Sometimes	119037216727
We collaborated on an externally-led Patient-Focused Drug Development meeting with the FDA [Food and Drug Administration] in 2025.	Neurological > Multiple sclerosis	Regulatory partnership > Attend/facilitate meetings with policymakers/regulators	100,000	National	Rarely	119039458452
Meeting with people at CMS [Centers for Medicare & Medicaid Services] to weigh in on drug pricing negotiation potential consequences.	Rare > Rare diseases: most types	Regulatory partnership > Attend/facilitate meetings with policymakers/regulators	5,000	National	Sometimes	119046591776
Elevating the patient voice and sharing insights from our patient experience registry at meetings such as ODAC [Oncologic Drugs Advisory Committee] and MDPNP [Medical Device Patient Network Program] listening sessions.	Cancer > Cancer: other	Regulatory partnership > Attend/facilitate meetings with policymakers/regulators	100,000	International	Regularly	119047535935
Communications with FDA [Food and Drug Administration], participating in public comment sessions.	Blood > Anaemia	Regulatory partnership > Attend/facilitate meetings with policymakers/regulators	5,000	National	Regularly	119050770645
We work with FDA [Food and Drug Administration], Congress, and the White House on federal policies that affect liver patients.	Liver > Liver diseases: most types	Regulatory partnership > Attend/facilitate meetings with policymakers/regulators	100,000	International	Regularly	119054948119
Testifying at ODAC [Oncologic Drugs Advisory Committee].	Cancer > Cancer: other	Regulatory partnership > Attend/facilitate meetings with policymakers/regulators	100,000	International	Sometimes	119059387919
Just recently hosted an EL-PFDD [Externally-Led Patient-Focused Drug Development] meeting and attended FDA [Food and Drug Administration] meetings with industry partners.	Rare > Rare disorders: genetic	Regulatory partnership > Attend/facilitate meetings with policymakers/regulators	5,000	International	Regularly	119065526277
EL-PFDD [Externally-Led Patient-Focused Drug Development] FDA [Food and Drug Administration] Patient Listening Sessions.	Patient advocacy > Health activist	Regulatory partnership > Attend/facilitate meetings with policymakers/regulators	10,000	National	Regularly	119066130575
We have testified at FDA [Food and Drug Administration] Adcomms [Advisory Committees] and have helped organise Patient-Focused Drug Development meetings.	Neuromuscular > Neuromuscular diseases: most types	Regulatory partnership > Attend/facilitate meetings with policymakers/regulators	100,000	National	Sometimes	119068005400
Actively participate in Patient Focused Drug Development with FDA [Food and Drug Administration].	Rheumatic > Lupus	Regulatory partnership > Attend/facilitate meetings with policymakers/regulators	100,000	National	Regularly	119070039917
We have held multiple externally led patient-focused drug development (EL-PFDD [Externally Led Patient-Focused Drug Development]) meetings with the FDA [Food and Drug Administration]. We invite regulators to all our scientific workshops. We educate patients and facilitate meetings with regulators on an ongoing basis.	Kidney > Kidney diseases	Regulatory partnership > Attend/facilitate meetings with policymakers/regulators	100,000	National	Regularly	119070696943
We provide public comment on new drugs to the FDA [Food and Drug Administration].	Blood > Sickle cell disease	Regulatory partnership > Attend/facilitate meetings with policymakers/regulators	10,000	Regional	Sometimes	119074242460
Participation in activities like PDUFA [Prescription Drug User Fee Act] and MDUFA [Medical Device User Fee and Modernization Act] discussions.	Rare > Rare diseases: most types	Regulatory partnership > Attend/facilitate meetings with policymakers/regulators	100,000	National	Rarely	119078556821
Coalition work.	Rare > Rare diseases: patient rights	Regulatory partnership > With other healthcare advocates	100,000	National	Sometimes	119050874740
We lead the national coalition EACH [Ensuring Access through Collaborative Health]/PIC [Patient Inclusion Council] coalition, working on government drug affordability reviews.	Autoimmune > Autoimmune: other	Regulatory partnership > With other healthcare advocates	100,000	International	Regularly	119071535440
In Arizona and, on occasion, more broadly across the USA, we collaborate with other cancer groups, organisations, and legislators.	Cancer > Cancer: blood	Regulatory partnership > With other healthcare advocates	Do not know	Regional	Sometimes	119078394447
Lobbying on the state and federal level on rules/laws that impact our patients.	General health > Healthcare delivery	Impacting policy and legislation > Influence healthcare policy and legislation	100	National	Sometimes	119011450240
Our Cancer Policy Institute advocates for equitable access to medicines to government organizations such as CMS [Centers for Medicare & Medicaid Services].	Cancer > Cancer: other	Impacting policy and legislation > Influence healthcare policy and legislation	100,000	International	Regularly	119020437168
We include a discussion of medication pricing in our conversations with the state and federal government employees. We are also pushing for lifetime coverage of physical therapy for people with PD [Parkinson's Disease], as maintaining balance as the disease progresses saves both providers and insurers money in the long run.	Neurodegenerative > Parkinson’s	Impacting policy and legislation > Influence healthcare policy and legislation	100,000	Regional	Rarely	119020536956
Advocacy with PBM [Pharmacy Benefit Manager], Medicare and state legislatures.	Rheumatic > Lupus	Impacting policy and legislation > Influence healthcare policy and legislation	5,000	Regional	Sometimes	119037794627
Advocacy with PBM [Pharmacy Benefit Manager], Medicare and state legislatures.	Rheumatic > Lupus	Impacting policy and legislation > Influence healthcare policy and legislation	5,000	Regional	Sometimes	119037794627
...The organization's flagship event - the annual Indo US Bridging RARE Summit brings together rare disease stakeholders, including scientists, patient advocates, government officials, biopharma executives, and policy experts who share a common goal: transforming the future of care for the 400 million people worldwide living with rare diseases.	Rare > Rare diseases: most types	Impacting policy and legislation > Influence healthcare policy and legislation	100	International	Regularly	119038170136
Invitation to the White House in 2024 to discuss cancer research funding with the First Lady's office.	Cancer > Cancer: medical research	Impacting policy and legislation > Influence healthcare policy and legislation	1,000	National	Rarely	119044810653
We work with FDA [Food and Drug Administration], Congress, and the White House on federal policies that affect liver patients.	Liver > Liver diseases: most types	Impacting policy and legislation > Influence healthcare policy and legislation	100,000	International	Regularly	119054948119
We frequently engage on state and federal issues related to patient access to care.	Respiratory > Lung diseases	Impacting policy and legislation > Influence healthcare policy and legislation	100,000	National	Rarely	119055217525
We regularly advocate through sign-on letters, policymaker discussions, and policy related programming.	Cancer > Cancer: diverse communities	Impacting policy and legislation > Influence healthcare policy and legislation	5,000	National	Sometimes	119055664199
We both authored and signed on to letters to state and federal (private insurance and CMS [Centers for Medicare & Medicaid Services]) on a variety of issues.	Cardiovascular > Cardiovascular & heart conditions	Impacting policy and legislation > Influence healthcare policy and legislation	Over 100,000	International	Sometimes	119058234393
Campaigning with KidneyCAN for increased federal research funding.	Cancer > Cancer: kidney	Impacting policy and legislation > Influence healthcare policy and legislation	1,000	Regional	Sometimes	119059282158
The Community Liver Alliance represents patient interests to regulators and policymakers responsible for advising on and determining drug pricing. We engage at the federal and state levels to ensure that the lived experiences of people affected by liver disease inform policy decisions related to drug access, affordability, and reimbursement. Our advocacy includes engagement with CMS [Centers for Medicare & Medicaid Services], participation in public comment opportunities (including those related to Medicare Drug Price Negotiation), collaboration with policymakers on PBM [Pharmacy Benefit Manager] reform, and education of regulatory stakeholders about the real-world impact of pricing decisions on patients with chronic and rare liver diseases. We elevate patient and caregiver voices to ensure pricing policies balance innovation with equitable and timely access to life-saving therapies.	Liver > Liver diseases: most types	Impacting policy and legislation > Influence healthcare policy and legislation	100,000	National	Regularly	119059491202
We communicate with federal representatives about the importance of funding.	Cancer > Cancer: brain	Impacting policy and legislation > Influence healthcare policy and legislation	We do not communicate with patients directly	National	Sometimes	119061001133
We bring patient perspectives opposing use of QALYs [Quality-Adjusted Life Years].	Patient advocacy > Health activist	Impacting policy and legislation > Influence healthcare policy and legislation	Do not know	National	Regularly	119065430409
Just one example: we continually advocate (both through regulatory comments and now legislation) for adequate reimbursement for new and innovative drugs in the ESRD [End-Stage Renal Disease] space through TDAPA [Transitional Drug Add-on Payment Adjustment] and a sustainable post-TDAPA payment pathway.	Kidney > Kidney diseases	Impacting policy and legislation > Influence healthcare policy and legislation	Over 100,000	National	Regularly	119068073151
We work with state, federal policymakers and health insurers.	General health > Obesity	Impacting policy and legislation > Influence healthcare policy and legislation	Over 100,000	National	Sometimes	119068989651
As a transgender-led healthcare organization, ETSI [Ending Transmission of Sexual Infections] also brings the perspective of historically marginalized communities into healthcare discussions. The clinic advocates for policies that protect medication affordability, expand HIV prevention services, and strengthen programs that reduce the cost of prescription drugs for safety-net providers. Through patient care, community engagement, and participation in public health programs, ETSI Health Clinic ensures that the voices and needs of vulnerable patients are represented in conversations about medication access and healthcare equity.	HIV > HIV and sexual health	Impacting policy and legislation > Influence healthcare policy and legislation	100	Regional	Regularly	119073874628
Supported efforts to have an ICD-10 (International Classification of Diseases, 10th Revision) code for VEXAS syndrome.	Rare > Autoinflammatory diseases	Impacting policy and legislation > Influence healthcare policy and legislation	1,000	International	Sometimes	119074154009
We provide public comment on new drugs to the FDA. We regularly organize delegations to Capitol Hill to advocate for Medicaid and Medicare support for sickle cell treatment. We advocate with New Jersey legislators for support for sickle cell treatment and services.	Blood > Sickle cell disease	Impacting policy and legislation > Influence healthcare policy and legislation	10,000	Regional	Sometimes	119074242460
Testifying at PDAB meetings [submitting comments to a Patient Drug Affordability Board during a public meeting, hearing, or review process] writing letters to decision-makers at state and federal level [USA].	Rare > Cystic fibrosis	Impacting policy and legislation > Influence healthcare policy and legislation	10,000	National	Sometimes	119078388026
In Arizona and occasionally, the U.S., we collaborate with other cancer groups, organizations, and legislators who support legislation that will help cancer research, the medical/oncology groups, and cancer patients and families.	Cancer > Cancer: blood	Impacting policy and legislation > Influence healthcare policy and legislation	Do not know	Regional	Sometimes	119078394447
We have a policy committee and weigh in on public policy issues through education and advocacy.	Chronic > Palliative	Impacting policy and legislation > Influence healthcare policy and legislation	100	National	Regularly	119078649119
We include a discussion of medication pricing in our discussions with state and federal government employees.	Neurodegenerative > Parkinson’s	Impacting policy and legislation > Participate in public hearings and consultations	100,000	Regional	Rarely	119020536956
We submitted comments to CMS [Centers for Medicare & Medicaid Services] regarding the price negotiation of mental health and neurological conditions.	Mental health > Mental health: stigma	Impacting policy and legislation > Participate in public hearings and consultations	We do not communicate with patients directly	National	Sometimes	119024698241
IndoUSrare participates in RDLA's [Rare Disease Legislative Advocates] Rare Disease Week on Capitol Hill and advocates to legislators on behalf of our patient alliance members and the rare disease community.	Rare > Rare diseases: most types	Impacting policy and legislation > Participate in public hearings and consultations	100	International	Regularly	119038170136
Testify before prescription drug affordability boards.	Bleeding > Bleeding disorders: most types	Impacting policy and legislation > Participate in public hearings and consultations	1,000	Local	Regularly	119044404340
Communications with FDA [Food and Drug Administration], participating in public comment sessions.	Blood > Anaemia	Impacting policy and legislation > Participate in public hearings and consultations	5,000	National	Regularly	119050770645
We regularly advocate through sign-on letters, policymaker discussions, and policy related programming.	Cancer > Cancer: diverse communities	Impacting policy and legislation > Participate in public hearings and consultations	5,000	National	Sometimes	119055664199
We both authored and signed on to letters to state and federal (private insurance and CMS [Centers for Medicare & Medicaid Services]) on a variety of issues.	Cardiovascular > Cardiovascular & heart conditions	Impacting policy and legislation > Participate in public hearings and consultations	Over 100,000	International	Sometimes	119058234393
... Testifying at IRA [Inflation Reduction Act] listening sessions to elevate the patient experience.	Cancer > Cancer: other	Impacting policy and legislation > Participate in public hearings and consultations	100,000	International	Sometimes	119059387919
The Community Liver Alliance engages at the federal and state levels to ensure that the lived experiences of people affected by liver disease inform policy decisions related to drug access, affordability, and reimbursement. The Community Liver Alliance's advocacy includes engagement with CMS [Centers for Medicare & Medicaid Services], participation in public comment opportunities (including those related to Medicare Drug Price Negotiation), collaboration with policymakers on PBM [Pharmacy Benefit Manager] reform, and education of regulatory stakeholders about the real-world impact of pricing decisions on patients with chronic and rare liver diseases.	Liver > Liver diseases: most types	Impacting policy and legislation > Participate in public hearings and consultations	100,000	National	Regularly	119059491202
Through written comments, sign-on letters, and participating in roundtable meetings where drugs are being discussed.	Cancer > Cancer: sarcoma	Impacting policy and legislation > Participate in public hearings and consultations	10,000	International	Sometimes	119064713792
We work with the state agencies that oversee our Medicaid program, our exchange and private insurance. We hold quarterly meetings with them to discuss patient access issues. We also provide letters and testimony to the board that oversees health care costs in our state.	Bleeding > Bleeding disorders: most types	Impacting policy and legislation > Participate in public hearings and consultations	5,000	Regional	Regularly	119069774462
... Our government relations team responds to comment letters and regularly talks with CMS [Centers for Medicare & Medicaid Services] to ensure best outcomes.	Kidney > Kidney diseases	Impacting policy and legislation > Participate in public hearings and consultations	100,000	National	Regularly	119070696943
We provided comments to federal and state regulators around drug pricing in the 340B program [in the US, these reforms seek to overhaul the 1992 federal program that mandates pharmaceutical manufacturers provide steep drug discounts to safety-net hospitals and clinics] when asking for community input to proposed policy changes.	Patient education > Patient information	Impacting policy and legislation > Participate in public hearings and consultations	5,000	National	Sometimes	119073380443
We are not a lobbying organization, we are a 501 c-3 non-profit public charity. We sign on to letters with partner societies who are lobbying for better access to care. We host roundtables and advocacy awareness days with legislators where we raise awareness of current problems and offer solutions. One recent event was with the Governor of Virginia. We are planning a Spine Health Fair at the US Capitol in September.	General health > Lifestyle	Impacting policy and legislation > Participate in public hearings and consultations	100,000	National	Sometimes	119074217249
We provide public comment on new drugs to the FDA [Food and Drug Administration].	Blood > Sickle cell disease	Impacting policy and legislation > Participate in public hearings and consultations	10,000	Regional	Sometimes	119074242460
Work with NYSTAG [New York State Technical Advisory Group-generally refers to ad-hoc or specialised panels of industry experts, academics, and stakeholders assembled by NY state agencies to advise on specific policies, healthcare, or emerging technologies.].	General health > Healthcare delivery	Impacting policy and legislation > Participate in public hearings and consultations	5,000	Regional	Sometimes	119078326039
Testifying at PDAB meetings [submitting comments to a Patient Drug Affordability Board during a public meeting, hearing, or review process] writing letters to decision-makers at state and federal level [USA].	Rare > Cystic fibrosis	Impacting policy and legislation > Participate in public hearings and consultations	10,000	National	Sometimes	119078388026
IndoUSrare participates in RDLA's [Rare Disease Legislative Advocates] Rare Disease Week on Capitol Hill and advocates to legislators on behalf of our patient alliance members and the rare disease community.	Rare > Rare diseases: most types	Impacting policy and legislation > Mobilise parliamentarians and political leaders	100	International	Regularly	119038170136
Invitation to the White House in 2024 to discuss cancer research funding with the First Lady's office.	Cancer > Cancer: medical research	Impacting policy and legislation > Mobilise parliamentarians and political leaders	1,000	National	Rarely	119044810653
We communicate with federal representatives about the importance of funding.	Cancer > Cancer: brain	Impacting policy and legislation > Mobilise parliamentarians and political leaders	We do not communicate with patients directly	National	Sometimes	119061001133
We provide public comment on new drugs to the FDA. We regularly organize delegations to Capitol Hill to advocate for Medicaid and Medicare support for sickle cell treatment. We advocate with New Jersey legislators for support for sickle cell treatment and services.	Blood > Sickle cell disease	Impacting policy and legislation > Mobilise parliamentarians and political leaders	10,000	Regional	Sometimes	119074242460
... We are active in Lupus Caucus [Congressional Lupus Caucus, a bipartisan group of lawmakers focused on raising awareness about lupus and securing federal funding for research and education]	Rheumatic > Lupus	Impacting policy and legislation > Mobilise parliamentarians and political leaders	100,000	National	Regularly	119082751826
Government Relations/Advocacy team signs letters in support of lower drug prices.	Cancer > Cancer: prostate	Pricing and affordability advocacy > Advocate for fair medicine pricing	100,000	National	Sometimes	119011723588
We include a discussion of medication pricing in our discussions with state and federal government employees.	Neurodegenerative > Parkinson’s	Pricing and affordability advocacy > Advocate for fair medicine pricing	100,000	Regional	Rarely	119020536956
Testify before prescription drug affordability boards.	Bleeding > Bleeding disorders: most types	Pricing and affordability advocacy > Advocate for fair medicine pricing	1,000	Local	Regularly	119044404340
Drug assistance programs and co-pay assistance foundation.	Cancer > Cancer: most types	Pricing and affordability advocacy > Advocate for fair medicine pricing	5,000	International	Sometimes	119045428228
We frequently engage on state and federal issues related to patient access to care, occasionally weighing in on issues adjacent to drug pricing, though it is not our primary focus.	Respiratory > Lung diseases	Pricing and affordability advocacy > Advocate for fair medicine pricing	100,000	National	Rarely	119055217525
The Community Liver Alliance represents patient interests to regulators and policymakers responsible for advising on and determining drug pricing. We engage at the federal and state levels to ensure that the lived experiences of people affected by liver disease inform policy decisions related to drug access, affordability, and reimbursement. Our advocacy includes engagement with CMS [Centers for Medicare & Medicaid Services], participation in public comment opportunities (including those related to Medicare Drug Price Negotiation), collaboration with policymakers on PBM [Pharmacy Benefit Manager] reform, and education of regulatory stakeholders about the real-world impact of pricing decisions on patients with chronic and rare liver diseases. We elevate patient and caregiver voices to ensure pricing policies balance innovation with equitable and timely access to life-saving therapies	Liver > Liver diseases: most types	Pricing and affordability advocacy > Advocate for fair medicine pricing	100,000	National	Regularly	119059491202
Promoting 340B reforms [in the US, these reforms seek to overhaul the 1992 federal program that mandates pharmaceutical manufacturers provide steep drug discounts to safety-net hospitals and clinics].	Patient advocacy > Health activist	Pricing and affordability advocacy > Advocate for fair medicine pricing	10,000	Regional	Regularly	119059636045
Have written support letters from the "patient voice" as part of our advocacy efforts related to drug pricing.	Cancer > Cancer: brain	Pricing and affordability advocacy > Advocate for fair medicine pricing	1,000	National	Rarely	119061176158
Our organisation actively advocates for the interests of the individuals and communities we serve before regulatory authorities and decision-making bodies on matters relating to medicine access, affordability, and coverage recommendations.	Patient advocacy > Training	Pricing and affordability advocacy > Advocate for fair medicine pricing	100,000	International	Regularly	119063146174
We bring patient perspectives opposing use of QUALYs [quality-adjusted life years] and opposing importing drug prices from other countries [oustide of the USA], especially those that use QALYs.	Patient advocacy > Health activist	Pricing and affordability advocacy > Advocate for fair medicine pricing	Do not know	National	Regularly	119065430409
Just one example: we continually advocate (both through regulatory comments and now legislation) for adequate reimbursement for new and innovative drugs in the ESRD [End-Stage Renal Disease] space through TDAPA [Transitional Drug Add-on Payment Adjustment] and a sustainable post-TDAPA payment pathway.	Kidney > Kidney diseases	Pricing and affordability advocacy > Advocate for fair medicine pricing	Over 100,000	National	Regularly	119068073151
We work with the state agencies that oversee our Medicaid program, our exchange and private insurance. We hold quarterly meetings with them to discuss patient access issues. We also provide letters and testimony to the board that oversees health care costs in our state.	Bleeding > Bleeding disorders: most types	Pricing and affordability advocacy > Advocate for fair medicine pricing	5,000	Regional	Regularly	119069774462
ETSI [Ending Transmission of Sexual Infections] Health Clinic represents the interests of patients—particularly those impacted by HIV, LGBTQ+ individuals, and underserved communities—by advocating for affordable access to life-saving medications.	HIV > HIV and sexual health	Pricing and affordability advocacy > Advocate for fair medicine pricing	100	Regional	Regularly	119073874628
Pharma pricing.	General health > Obesity	Pricing and affordability advocacy > Advocate for fair medicine pricing	100,000	National	Regularly	119076659022
With our new Public Policy department, this may come up as an opportunity to advocate for drug pricing.	Neurodegenerative > Parkinson’s	Pricing and affordability advocacy > Advocate for fair medicine pricing	100,000	National	Sometimes	119078318937
Cost for SCD [Sickle Cell Disease] treatments as they came to market during the last 7 years.	Blood > Sickle cell disease	Pricing and affordability advocacy > Advocate for fair medicine pricing	5,000	National	Sometimes	119079213376
We have participated in the IRA [Inflation Reduction Act] price negotiation round tables.	Respiratory > Asthma and allergy	Pricing and affordability advocacy > Engage in price-review board negotiations	100,000	National	Regularly	119011501209
We have participated in the IRA [Inflation Reduction Act of 2022] price negotiation round tables [The IRA provides Medicare (the US federal health insurance program for people age 65 and older) powers to negotiate lower prescription costs].	Respiratory > Asthma and allergy	Pricing and affordability advocacy > Engage in price-review board negotiations	100,000	National	Regularly	119011501209
We submitted comments to CMS [Centers for Medicare & Medicaid Services] regarding the price negotiation of mental health and neurological conditions.	Mental health > Mental health: stigma	Pricing and affordability advocacy > Engage in price-review board negotiations	We do not communicate with patients directly	National	Sometimes	119024698241
... meetings with state office of CMS [Centers for Medicare & Medicaid Services].	Bleeding > Bleeding disorders: most types	Pricing and affordability advocacy > Engage in price-review board negotiations	1,000	Local	Regularly	119044404340
Meeting with people at CMS [Centers for Medicare & Medicaid Services] to weigh in on drug pricing negotiation potential consequences.	Rare > Rare diseases: most types	Pricing and affordability advocacy > Engage in price-review board negotiations	5,000	National	Sometimes	119046591776
We educate advocates on outreach to Patient Drug Affordability Boards [Patient Drug Affordability Boards are state government boards that review prescription drug prices and, in some US states, can take actions to help make drugs more affordable.]	Cancer > Cancer: diverse communities	Pricing and affordability advocacy > Engage in price-review board negotiations	100,000	National	Sometimes	119054172981
The Community Liver Alliance's advocacy includes engagement with CMS [Centers for Medicare & Medicaid Services], participation in public comment opportunities (including those related to Medicare Drug Price Negotiation), collaboration with policymakers on PBM [Pharmacy Benefit Manager] reform, and education of regulatory stakeholders about the real-world impact of pricing decisions on patients with chronic and rare liver diseases.	Liver > Liver diseases: most types	Pricing and affordability advocacy > Engage in price-review board negotiations	100,000	National	Regularly	119059491202
PDABs [Prescription Drug Affordability Boards]; IRA [Inflation Reduction Act]; Medicaid P&T [Pharmacy and Therapeutics] committees.	Patient advocacy > Health activist	Pricing and affordability advocacy > Engage in price-review board negotiations	We do not communicate with patients directly	National	Regularly	119064160791
We participated in the Medicare price negotiation roundtables and town hall last year.	Respiratory > Asthma and allergy	Pricing and affordability advocacy > Engage in price-review board negotiations	100,000	National	Regularly	119069847466
Actively participate in patient-focused drug development with the FDA [Food and Drug Administration] and CMS [Centers for Medicare & Medicaid Services]/HHS [U.S. Department of Health and Human Services] Prescription Access discussions.	Rheumatic > Lupus	Pricing and affordability advocacy > Engage in price-review board negotiations	100,000	National	Regularly	119070039917
We lead the national coalition EACH [Ensuring Access through Collaborative Health]/PIC [Patient Inclusion Council] coalition, working on government drug affordability reviews.	Autoimmune > Autoimmune: other	Pricing and affordability advocacy > Engage in price-review board negotiations	100,000	International	Regularly	119071535440
We provided comments to federal and state regulators around drug pricing in the 340B program [in the US, these reforms seek to overhaul the 1992 federal program that mandates pharmaceutical manufacturers provide steep drug discounts to safety-net hospitals and clinics] when asking for community input to proposed policy changes.	Patient education > Patient information	Pricing and affordability advocacy > Engage in price-review board negotiations	5,000	National	Sometimes	119073380443
We communicate to them that many HD [Huntington's disease] families are low income as often the one making the money is the one with HD. So cost of meds can impact whether they can afford the meds.	Neurodegenerative > Huntington’s disease	Pricing and affordability advocacy > Address cost barriers for low-income and vulnerable people	100,000	National	Sometimes	119017688949
Health insurance companies and health insurance reps organize activities for low income communities.	Older people > Older people	Pricing and affordability advocacy > Address cost barriers for low-income and vulnerable people	Over 100,000	International	Sometimes	119035935608
ETSI [Ending Transmission of Sexual Infections] Health Clinic represents the interests of patients—particularly those impacted by HIV, LGBTQ+ individuals, and underserved communities—by advocating for affordable access to life-saving medications and healthcare services. Through its participation in programs such as the 340B Drug Pricing Program [in the US, these reforms seek to overhaul the 1992 federal program that mandates pharmaceutical manufacturers provide steep drug discounts to safety-net hospitals and clinics], ETSI works to ensure that low-income and uninsured patients have access to reduced-cost prescription medications, including HIV treatment and prevention medications like PrEP.	HIV > HIV and sexual health	Pricing and affordability advocacy > Address cost barriers for low-income and vulnerable people	100	Regional	Regularly	119073874628
We regularly provide comments to state P&T [Pharmacy and Therapeutics] committees regarding their formulary inclusions and exclusions.	Respiratory > Asthma and allergy	Pricing and affordability advocacy > Advocating for inclusion of product in reimbursement lists/national formularies	100,000	National	Regularly	119011501209
PDABs [Prescription Drug Affordability Boards]; IRA [Inflation Reduction Act]; Medicaid P&T [Pharmacy and Therapeutics] committees.	Patient advocacy > Health activist	Pricing and affordability advocacy > Advocating for inclusion of product in reimbursement lists/national formularies	We do not communicate with patients directly	National	Regularly	119064160791
Invitation to the White House in 2024 to discuss cancer research funding with First Lady's office. Lastly, I've co-written op-eds and on the local level recorded a video (ACS) targeting NYS lawmakers and the advocacy for increased equity and access to biomarker testing for patients.	Cancer > Cancer: medical research	Pricing and affordability advocacy > Engaging in awareness campaigns	1,000	National	Rarely	119044810653
Advocacy in access to care issues.	Cancer > Cancer: most types	Improving access pathways > Facilitate equitable access to medicines/healthcare	10,000	Regional	Sometimes	119011457943
Our Cancer Policy Institute advocates for equitable access to medicines to government organizations such as CMS [Centers for Medicare & Medicaid Services].	Cancer > Cancer: other	Improving access pathways > Facilitate equitable access to medicines/healthcare	100,000	International	Regularly	119020437168
We include a discussion of medication pricing in our conversations with the state and federal government employees. We are also pushing for lifetime coverage of physical therapy for people with PD [Parkinson's Disease], as maintaining balance as the disease progresses saves both providers and insurers money in the long run.	Neurodegenerative > Parkinson’s	Improving access pathways > Facilitate equitable access to medicines/healthcare	100,000	Regional	Rarely	119020536956
... We sent letters to States to request that they change their step requirements for access to mental health treatments.	Mental health > Mental health: stigma	Improving access pathways > Facilitate equitable access to medicines/healthcare	We do not communicate with patients directly	National	Sometimes	119024698241
Invitation to the White House in 2024 to discuss cancer research funding with First Lady's office. Lastly, I've co-written op-eds and on the local level recorded a video (ACS) targeting NYS lawmakers and the advocacy for increased equity and access to biomarker testing for patients.	Cancer > Cancer: medical research	Improving access pathways > Facilitate equitable access to medicines/healthcare	1,000	National	Rarely	119044810653
Drug assistance programs and co-pay assistance foundation.	Cancer > Cancer: most types	Improving access pathways > Facilitate equitable access to medicines/healthcare	5,000	International	Sometimes	119045428228
We frequently engage on state and federal issues related to patient access to care.	Respiratory > Lung diseases	Improving access pathways > Facilitate equitable access to medicines/healthcare	100,000	National	Rarely	119055217525
The Community Liver Alliance elevates patient and caregiver voices to ensure pricing policies balance innovation with equitable and timely access to life-saving therapies.	Liver > Liver diseases: most types	Improving access pathways > Facilitate equitable access to medicines/healthcare	100,000	National	Regularly	119059491202
We might provide patient stories and testimonials when needed to ensure importance of continued access to a particular drug.	Neurological > Migraine/other headache disorders	Improving access pathways > Facilitate equitable access to medicines/healthcare	100,000	National	Rarely	119060019780
Our organisation actively advocates for the interests of the individuals and communities we serve before regulatory authorities and decision-making bodies on matters relating to medicine access, affordability, and coverage recommendations.	Patient advocacy > Training	Improving access pathways > Facilitate equitable access to medicines/healthcare	100,000	International	Regularly	119063146174
State level Medicaid.	Cancer > Cancer: most types	Improving access pathways > Facilitate equitable access to medicines/healthcare	1,000	National	Rarely	119068136322
We work with the state agencies that oversee our Medicaid program, our exchange and private insurance. We hold quarterly meetings with them to discuss patient access issues. We also provide letters and testimony to the board that oversees health care costs in our state.	Bleeding > Bleeding disorders: most types	Improving access pathways > Facilitate equitable access to medicines/healthcare	5,000	Regional	Regularly	119069774462
We are currently addressing access to biologic medications.	Rare > Rare diseases: immune	Improving access pathways > Facilitate equitable access to medicines/healthcare	10,000	National	Sometimes	119069849386
Actively participate in patient-focused drug development with the FDA [Food and Drug Administration] and CMS [Centers for Medicare & Medicaid Services]/HHS [U.S. Department of Health and Human Services] Prescription Access discussions.	Rheumatic > Lupus	Improving access pathways > Facilitate equitable access to medicines/healthcare	100,000	National	Regularly	119070039917
We are not a lobbying organization, we are a 501 c-3 non-profit public charity. We sign on to letters with partner societies who are lobbying for better access to care. Support with formal cost-approval (reimbursement authorisation) applications and with newly introduced restrictions on treatment options.	General health > Lifestyle	Improving access pathways > Facilitate equitable access to medicines/healthcare	100,000	National	Sometimes	119074217249
Pharma pricing, coverage, indications.	General health > Obesity	Improving access pathways > Facilitate equitable access to medicines/healthcare	100,000	National	Regularly	119076659022
We have on only a few occasions advocated for better access to newer drugs.	Mental health > Mental health: stigma	Improving access pathways > Facilitate equitable access to medicines/healthcare	Over 100,000	International	Rarely	119078209025
Cost for SCD [Sickle Cell Disease] treatments as they came to market during the last 7 years.	Blood > Sickle cell disease	Improving access pathways > Facilitate equitable access to medicines/healthcare	5,000	National	Sometimes	119079213376
... convene MAPRx [Medicare Access for Patients Rx] for Medicare Part D, etc.[MAPRx is guided by the idea that every Medicare Part D beneficiary should have timely, affordable access to the medications they need to live a healthy life.]	Rheumatic > Lupus	Improving access pathways > Facilitate equitable access to medicines/healthcare	100,000	National	Regularly	119082751826
... We sent letters to States to request that they change their step requirements for access to mental health treatments.	Mental health > Mental health: stigma	Improving access pathways > Address disparities in access and management	We do not communicate with patients directly	National	Sometimes	119024698241
We work with the state agencies that oversee our Medicaid program, our exchange and private insurance. We hold quarterly meetings with them to discuss patient access issues. We also provide letters and testimony to the board that oversees health care costs in our state.	Bleeding > Bleeding disorders: most types	Improving access pathways > Address disparities in access and management	5,000	Regional	Regularly	119069774462
IndoUSrare participates in RDLA's [Rare Disease Legislative Advocates] Rare Disease Week on Capitol Hill and advocates to legislators on behalf of our patient alliance members and the rare disease community.	Rare > Rare diseases: most types	Improving access pathways > Improving access to rare/ultra-rare therapies	100	International	Regularly	119038170136
Elevating the patient voice and sharing insights from our patient experience registry at meetings such as ODAC [Oncologic Drugs Advisory Committee] and MDPNP [Medical Device Patient Network Program] listening sessions.	Cancer > Cancer: other	Evidence-based patient input > Use patient insights to inform decisions	100,000	International	Regularly	119047535935
Elevating the patient voice and sharing insights from our patient experience registry at meetings such as ODAC [Oncologic Drugs Advisory Committee] and MDPNP [Medical Device Patient Network Program] listening sessions.	Cancer > Cancer: other	Evidence-based patient input > Use patient insights to inform decisions	100,000	International	Regularly	119047535935
Held an EL-PFDD [Externally-Led Patient-Focused Drug Development] meeting.	Neurodegenerative > Dementia	Evidence-based patient input > Use patient insights to inform decisions	Do not know	National	Sometimes	119052219350
Serve on the Alliance for Breast Cancer Policy [The Alliance for Breast Cancer Policy is a coalition of over 25 national patient advocacy organizations founded by Susan G. Komen. The network unites advocates, community members, and industry partners to drive federal policy changes that improve access to care, eliminate out-of-pocket costs, and improve outcomes]. We were just part of a manuscript written for the launch of the global Breast Cancer Care Quality Index: https://www.mdpi.com/1660-4601/23/2/207	Cancer > Cancer: breast	Evidence-based patient input > Use patient insights to inform decisions	100,000	International	Sometimes	119052475951
We bring patient perspectives opposing use of QALYs [Quality-Adjusted Life Years].	Patient advocacy > Health activist	Evidence-based patient input > Use patient insights to inform decisions	Do not know	National	Regularly	119065430409
Actively participate in Patient Focused Drug Development with FDA [Food and Drug Administration].	Rheumatic > Lupus	Evidence-based patient input > Use patient insights to inform decisions	100,000	National	Regularly	119070039917
We have held multiple externally led patient-focused drug development (EL-PFDD [Externally Led Patient-Focused Drug Development]) meetings with the FDA [Food and Drug Administration].	Kidney > Kidney diseases	Evidence-based patient input > Use patient insights to inform decisions	100,000	National	Regularly	119070696943
As a transgender-led healthcare organization, ETSI [Ending Transmission of Sexual Infections] also brings the perspective of historically marginalized communities into healthcare discussions. The clinic advocates for policies that protect medication affordability, expand HIV prevention services, and strengthen programs that reduce the cost of prescription drugs for safety-net providers. Through patient care, community engagement, and participation in public health programs, ETSI Health Clinic ensures that the voices and needs of vulnerable patients are represented in conversations about medication access and healthcare equity.	HIV > HIV and sexual health	Evidence-based patient input > Use patient insights to inform decisions	100	Regional	Regularly	119073874628
We provide data and real-world knowledge.	Liver > Liver diseases: most types	Evidence-based patient input > Provide Real-World Evidence (RWE) to regulatory agencies	100,000	National	Sometimes	119045306046
The Community Liver Alliance represents patient interests to regulators and policymakers responsible for advising on and determining drug pricing. We engage at the federal and state levels to ensure that the lived experiences of people affected by liver disease inform policy decisions related to drug access, affordability, and reimbursement. Our advocacy includes engagement with CMS [Centers for Medicare & Medicaid Services], participation in public comment opportunities (including those related to Medicare Drug Price Negotiation), collaboration with policymakers on PBM [Pharmacy Benefit Manager] reform, and education of regulatory stakeholders about the real-world impact of pricing decisions on patients with chronic and rare liver diseases. We elevate patient and caregiver voices to ensure pricing policies balance innovation with equitable and timely access to life-saving therapies	Liver > Liver diseases: most types	Evidence-based patient input > Provide Real-World Evidence (RWE) to regulatory agencies	100,000	National	Regularly	119059491202
ETSI [Ending Transmission of Sexual Infections] Health Clinic collaborates with pharmacy partners and public health systems to highlight the real-world impact that medication costs have on patients living with HIV and those at risk. By documenting patient barriers to medication access, the clinic helps inform healthcare partners and policymakers about the financial and systemic challenges patients face.	HIV > HIV and sexual health	Evidence-based patient input > Provide Real-World Evidence (RWE) to regulatory agencies	100	Regional	Regularly	119073874628
We communicate to them that many HD [Huntington's Disease] families are low income, as often the one making the money is the one with HD.	Neurodegenerative > Huntington’s disease	Evidence-based patient input > Leverage patient stories and lived-experience narratives	100,000	National	Sometimes	119017688949
The Community Liver Alliance engages at the federal and state levels to ensure that the lived experiences of people affected by liver disease inform policy decisions related to drug access, affordability, and reimbursement. We elevate patient and caregiver voices to ensure pricing policies balance innovation with equitable and timely access to life-saving therapies.	Liver > Liver diseases: most types	Evidence-based patient input > Leverage patient stories and lived-experience narratives	100,000	National	Regularly	119059491202
We might provide patient stories and testimonials when needed to ensure importance of continued access to a particular drug.	Neurological > Migraine/other headache disorders	Evidence-based patient input > Leverage patient stories and lived-experience narratives	100,000	National	Rarely	119060019780
Have written support letters from the "patient voice" as part of our advocacy efforts related to drug pricing.	Cancer > Cancer: brain	Evidence-based patient input > Leverage patient stories and lived-experience narratives	1,000	National	Rarely	119061176158
Held an EL-PFDD [Externally-Led Patient-Focused Drug Development] meeting.	Neurodegenerative > Dementia	Evidence-based patient input > Conduct surveys/research to generate data	Do not know	National	Sometimes	119052219350
Serve on the Alliance for Breast Cancer Policy [The Alliance for Breast Cancer Policy is a coalition of over 25 national patient advocacy organizations founded by Susan G. Komen. The network unites advocates, community members, and industry partners to drive federal policy changes that improve access to care, eliminate out-of-pocket costs, and improve outcomes]. We were just part of a manuscript written for the launch of the global Breast Cancer Care Quality Index: https://www.mdpi.com/1660-4601/23/2/207	Cancer > Cancer: breast	Evidence-based patient input > Conduct surveys/research to generate data	100,000	International	Sometimes	119052475951
As a transgender-led healthcare organization, ETSI [Ending Transmission of Sexual Infections] also brings the perspective of historically marginalized communities into healthcare discussions. The clinic advocates for policies that protect medication affordability, expand HIV prevention services, and strengthen programs that reduce the cost of prescription drugs for safety-net providers. Through patient care, community engagement, and participation in public health programs, ETSI Health Clinic ensures that the voices and needs of vulnerable patients are represented in conversations about medication access and healthcare equity.	HIV > HIV and sexual health	Evidence-based patient input > Translate evidence and insights into policy input	100	Regional	Regularly	119073874628
... We work closely with the FDA and other drug agencies, like EMA [European Medicines Agency] and PDMA [Pharmaceuticals and Medical Devices Agency], to look at endpoints for drug trials, or how to improve drug trials to benefit sponsors and patients.	Kidney > Kidney diseases	Diagnosis and medicines delivery > Collaborate on clinical trial design	100,000	National	Regularly	119070696943
Supporting legislation for multiple cancer early detection, including TV appearances and social media.	Cancer > Cancer: carers, family, friends	Diagnosis and medicines delivery > Support screening and early detection	1,000	Regional	Regularly	119036085844
Discussions about review and approvals of new screening technologies.	Cancer > Cancer: most types	Diagnosis and medicines delivery > Support screening and early detection	100,000	National	Rarely	119045117950
USPTF [U.S. Preventive Services Task Force] input.	Cancer > Cancer: men's	Diagnosis and medicines delivery > Support screening and early detection	100,000	National	Sometimes	119050776275
Sit on several panels.	Cancer > Cancer: breast	Institutional governance > Ensure representation within governmental advisory bodies	100	Regional	Regularly	119037236943
... The organization's flagship event - the annual Indo US Bridging RARE Summit brings together rare disease stakeholders, including scientists, patient advocates, government officials, biopharma executives, and policy experts who share a common goal: transforming the future of care for the 400 million people worldwide living with rare diseases.	Rare > Rare diseases: most types	Institutional governance > Working effectively within multi-stakeholder networks	100	International	Regularly	119038170136
Serve on the Alliance for Breast Cancer Policy [The Alliance for Breast Cancer Policy is a coalition of over 25 national patient advocacy organizations founded by Susan G. Komen. The network unites advocates, community members, and industry partners to drive federal policy changes that improve access to care, eliminate out-of-pocket costs, and improve outcomes]. We were just part of a manuscript written for the launch of the global Breast Cancer Care Quality Index: https://www.mdpi.com/1660-4601/23/2/207	Cancer > Cancer: breast	Institutional governance > Working effectively within multi-stakeholder networks	100,000	International	Sometimes	119052475951
We are not a lobbying organization, we are a 501 c-3 non-profit public charity. We sign on to letters with partner societies who are lobbying for better access to care. We host roundtables and advocacy awareness days with legislators where we raise awareness of current problems and offer solutions. One recent event was with the Governor of Virginia. We are planning a Spine Health Fair at the US Capitol in September.	General health > Lifestyle	Institutional governance > Working effectively within multi-stakeholder networks	100,000	National	Sometimes	119074217249
We are typically advisors on ICER [Institute for Clinical and Economic Review] reviews, active in Lupus Caucus, convene MAPRx [Medicare Access for Patients Rx] for Medicare Part D, etc.[MAPRx is guided by the idea that every Medicare Part D beneficiary should have timely, affordable access to the medications they need to live a healthy life.]	Rheumatic > Lupus	Institutional governance > Working effectively within multi-stakeholder networks	100,000	National	Regularly	119082751826

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