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    What To Consider Before Repainting Your Classic Car

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    What Your Car’s Paint Color Says About You

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    Top Ways To Upgrade Your Electric Vehicle

    Tips for Crossing Water in Your Jeep Wrangler

    Tips for Crossing Water in Your Jeep Wrangler

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    4 Helpful Tips for Giving Your Car a Glossy Finish

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    4 Common Mistakes To Avoid With Car Restorations

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    Signs That It Might Be Time To Get a New Car

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    Mercedes-Benz to Launch Global Branded High-Power Charging Network in North America

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    Mistakes To Avoid When Starting Your Online Store

    Mistakes To Avoid When Starting Your Online Store

    Ways To Reduce Safety Hazards in the Office

    Ways To Reduce Safety Hazards in the Office

    How To Realistically Invest in Apartment Complexes

    How To Realistically Invest in Apartment Complexes

    Fighting housing shortage: Airbnb & Co should share more data with authorities to prevent fraud 

    Fighting housing shortage: Airbnb & Co should share more data with authorities to prevent fraud 

    How To Transition to Mass-Producing Your Product

    How To Transition to Mass-Producing Your Product

    How To Choose the Right Outdoor Lighting for Your Business

    How To Choose the Right Outdoor Lighting for Your Business

    Tips for Designing an Industrial-Style Restaurant

    Tips for Designing an Industrial-Style Restaurant

    Los Angeles implemented a new tax on luxurious real estate to finance affordable housing and combat homelessness

    Los Angeles implemented a new tax on luxurious real estate to finance affordable housing and combat homelessness

    Working shorter hours: 38 companies in the USA are testing the 4-day week

    Working shorter hours: 38 companies in the USA are testing the 4-day week

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    • Fashion
      Essential Tips for Planning a Great Fashion Show

      Essential Tips for Planning a Great Fashion Show

      Signs That Your Tuxedo Doesn’t Fit Right

      Signs That Your Tuxedo Doesn’t Fit Right

      Make a Bold Style Statement With These Wide-Leg Pants

      Make a Bold Style Statement With These Wide-Leg Pants

      Power Your Winter Workouts With The Best Zip Hoodies for Men

      Power Your Winter Workouts With The Best Zip Hoodies for Men

      The Ultimate Watch Buying Guide

      The Ultimate Watch Buying Guide

      Outdoor Enthusiasts, Take Note: These Watches Are as Tough as You

      Outdoor Enthusiasts, Take Note: These Watches Are as Tough as You

      Our 7 Favorite Field Jackets for Tackling Fall Weather

      Our 7 Favorite Field Jackets for Tackling Fall Weather

      The Best Leather Jackets for Guys – No Matter Your Budget

      The Best Leather Jackets for Guys – No Matter Your Budget

      Campus-Ready Backpacks for Storing *All* the Books

      Campus-Ready Backpacks for Storing *All* the Books

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      • Food & Drink
        Energizing Foods and Drinks To Consume Before a Workout

        Energizing Foods and Drinks To Consume Before a Workout

        Classic Beer Styles You Should Know How to Brew

        Classic Beer Styles You Should Know How to Brew

        The Best Flour Alternatives That Work in Any Recipe

        The Best Flour Alternatives That Work in Any Recipe

        Cheese Tortellini in Creamy Marinara

        Cheese Tortellini in Creamy Marinara

        Unusual Products That Come From Animals and Their Uses

        Unusual Products That Come From Animals and Their Uses

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        Tips for Creating a Homemade Wine Cellar

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        How the Wine You Choose Can Help You have an Eco-Friendly Home

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          As Younger Children Increasingly Die by Suicide, Better Tracking and Prevention Is Sought

          Florida Gov. Ron DeSantis Injects Presidential Politics Into the Covid Vaccine Debate

          Florida Gov. Ron DeSantis Injects Presidential Politics Into the Covid Vaccine Debate

          Tips for Fitting a Doctor’s Visit Into Your Busy Schedule

          Tips for Fitting a Doctor’s Visit Into Your Busy Schedule

          The Shrinking Number of Primary Care Physicians Is Reaching a Tipping Point

          The Shrinking Number of Primary Care Physicians Is Reaching a Tipping Point

          Activist Misuses Federal Data to Make False Claim That Covid Vaccines Killed 676,000

          Activist Misuses Federal Data to Make False Claim That Covid Vaccines Killed 676,000

          Illustrated Report: How Gun Violence Goes Viral

          Illustrated Report: How Gun Violence Goes Viral

          A New Medicare Proposal Would Cover Training for Family Caregivers

          A New Medicare Proposal Would Cover Training for Family Caregivers

          New Alzheimer’s Drug Raises Hopes — Along With Questions

          New Alzheimer’s Drug Raises Hopes — Along With Questions

          Blood Test That Screens for Cancer Raises Hopes, and Worries

          Blood Test That Screens for Cancer Raises Hopes, and Worries

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          • Lifestyle
            Common Dog Owner Mistakes and How To Avoid Them

            Common Dog Owner Mistakes and How To Avoid Them

            3 Reasons To Give an Antique or Vintage Engagement Ring

            3 Reasons To Give an Antique or Vintage Engagement Ring

            Tim Ferris

            Tim Ferriss’ Lifestyle Design: Unleashing Human Potential and Redefining Success

            Outdoor Luxuries To Add to Your Backyard

            Outdoor Luxuries To Add to Your Backyard

            Strategy and business planning ideas. Chess gold on the board.

            Why the Friend of My Enemy is My Enemy

            Huge Money Mistakes New Parents Fall For

            Huge Money Mistakes New Parents Fall For

            Energizing Foods and Drinks To Consume Before a Workout

            Energizing Foods and Drinks To Consume Before a Workout

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            Hobbies That Stimulate Cognitive Function

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            How To Prevent Mold from Growing in a Sauna

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              Primaries underway for America’s third-largest party with grassroots and self-funded candidates

              Primaries underway for America’s third-largest party with grassroots and self-funded candidates

              Carbon capture draws millions in lobbying, billions in federal subsidies

              Carbon capture draws millions in lobbying, billions in federal subsidies

              United leads soaring airline industry lobbying spending

              United leads soaring airline industry lobbying spending

              Deep-pocketed dairy industry continues war on plant-based milk as FDA hears comments on new draft guidance

              Deep-pocketed dairy industry continues war on plant-based milk as FDA hears comments on new draft guidance

              Shein’s lobbying spending soars amid supply chain probes

              Shein’s lobbying spending soars amid supply chain probes

              Virginia legislative candidates in Richmond and Hampton Roads raked in millions in final primary sprint

              Virginia legislative candidates in Richmond and Hampton Roads raked in millions in final primary sprint

              Manchin outraises GOP challengers but has yet to announce 2024 plans

              Manchin outraises GOP challengers but has yet to announce 2024 plans

              Gallego outraises Sinema but falls far short of incumbent’s war chest in Arizona Senate race

              Gallego outraises Sinema but falls far short of incumbent’s war chest in Arizona Senate race

              Elon Musk outmuscled by Mark Zuckerberg’s money in politics

              Elon Musk outmuscled by Mark Zuckerberg’s money in politics

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                Ways To Invest in Real Estate With No Experience

                Ways To Invest in Real Estate With No Experience

                How To Make Your Rental Property More Eco-Friendly

                How To Make Your Rental Property More Eco-Friendly

                Things To Consider Before Investing in Real Estate Overseas

                Things To Consider Before Investing in Real Estate Overseas

                Incredible Areas in Florida To Buy a Vacation Home

                Incredible Areas in Florida To Buy a Vacation Home

                Helpful Home Staging Tips for Virtual Tours

                Helpful Home Staging Tips for Virtual Tours

                Ways To Decorate and Design a Lake House

                Ways To Decorate and Design a Lake House

                Challenges To Managing a Rental Property

                Challenges To Managing a Rental Property

                The Best Landlord: How To Show Tenants You Care

                The Best Landlord: How To Show Tenants You Care

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                The Best Locations To Invest in a Second Home

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                  Reasons Gas Stations Charge for Compressed Air

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                  What To Consider Before Repainting Your Classic Car

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                  What Your Car’s Paint Color Says About You

                  Top Ways To Upgrade Your Electric Vehicle

                  Top Ways To Upgrade Your Electric Vehicle

                  Tips for Crossing Water in Your Jeep Wrangler

                  Tips for Crossing Water in Your Jeep Wrangler

                  4 Helpful Tips for Giving Your Car a Glossy Finish

                  4 Helpful Tips for Giving Your Car a Glossy Finish

                  4 Common Mistakes To Avoid With Car Restorations

                  4 Common Mistakes To Avoid With Car Restorations

                  Signs That It Might Be Time To Get a New Car

                  Signs That It Might Be Time To Get a New Car

                  Mercedes-Benz Charging Network

                  Mercedes-Benz to Launch Global Branded High-Power Charging Network in North America

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                • Business
                  Mistakes To Avoid When Starting Your Online Store

                  Mistakes To Avoid When Starting Your Online Store

                  Ways To Reduce Safety Hazards in the Office

                  Ways To Reduce Safety Hazards in the Office

                  How To Realistically Invest in Apartment Complexes

                  How To Realistically Invest in Apartment Complexes

                  Fighting housing shortage: Airbnb & Co should share more data with authorities to prevent fraud 

                  Fighting housing shortage: Airbnb & Co should share more data with authorities to prevent fraud 

                  How To Transition to Mass-Producing Your Product

                  How To Transition to Mass-Producing Your Product

                  How To Choose the Right Outdoor Lighting for Your Business

                  How To Choose the Right Outdoor Lighting for Your Business

                  Tips for Designing an Industrial-Style Restaurant

                  Tips for Designing an Industrial-Style Restaurant

                  Los Angeles implemented a new tax on luxurious real estate to finance affordable housing and combat homelessness

                  Los Angeles implemented a new tax on luxurious real estate to finance affordable housing and combat homelessness

                  Working shorter hours: 38 companies in the USA are testing the 4-day week

                  Working shorter hours: 38 companies in the USA are testing the 4-day week

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                  • Fashion
                    Essential Tips for Planning a Great Fashion Show

                    Essential Tips for Planning a Great Fashion Show

                    Signs That Your Tuxedo Doesn’t Fit Right

                    Signs That Your Tuxedo Doesn’t Fit Right

                    Make a Bold Style Statement With These Wide-Leg Pants

                    Make a Bold Style Statement With These Wide-Leg Pants

                    Power Your Winter Workouts With The Best Zip Hoodies for Men

                    Power Your Winter Workouts With The Best Zip Hoodies for Men

                    The Ultimate Watch Buying Guide

                    The Ultimate Watch Buying Guide

                    Outdoor Enthusiasts, Take Note: These Watches Are as Tough as You

                    Outdoor Enthusiasts, Take Note: These Watches Are as Tough as You

                    Our 7 Favorite Field Jackets for Tackling Fall Weather

                    Our 7 Favorite Field Jackets for Tackling Fall Weather

                    The Best Leather Jackets for Guys – No Matter Your Budget

                    The Best Leather Jackets for Guys – No Matter Your Budget

                    Campus-Ready Backpacks for Storing *All* the Books

                    Campus-Ready Backpacks for Storing *All* the Books

                    Trending Tags

                    • Food & Drink
                      Energizing Foods and Drinks To Consume Before a Workout

                      Energizing Foods and Drinks To Consume Before a Workout

                      Classic Beer Styles You Should Know How to Brew

                      Classic Beer Styles You Should Know How to Brew

                      The Best Flour Alternatives That Work in Any Recipe

                      The Best Flour Alternatives That Work in Any Recipe

                      Cheese Tortellini in Creamy Marinara

                      Cheese Tortellini in Creamy Marinara

                      Unusual Products That Come From Animals and Their Uses

                      Unusual Products That Come From Animals and Their Uses

                      Tips for Creating a Homemade Wine Cellar

                      Tips for Creating a Homemade Wine Cellar

                      How the wine you choose can help you have an eco-friendly home

                      How the Wine You Choose Can Help You have an Eco-Friendly Home

                      Rum and Cola Holiday Ham

                      Special Centerpieces for Holiday Celebrations

                      ChefsGiving 2020

                      This Holiday Season, How about Cooking for a Cause?

                      Trending Tags

                      • Health

                        As Younger Children Increasingly Die by Suicide, Better Tracking and Prevention Is Sought

                        Florida Gov. Ron DeSantis Injects Presidential Politics Into the Covid Vaccine Debate

                        Florida Gov. Ron DeSantis Injects Presidential Politics Into the Covid Vaccine Debate

                        Tips for Fitting a Doctor’s Visit Into Your Busy Schedule

                        Tips for Fitting a Doctor’s Visit Into Your Busy Schedule

                        The Shrinking Number of Primary Care Physicians Is Reaching a Tipping Point

                        The Shrinking Number of Primary Care Physicians Is Reaching a Tipping Point

                        Activist Misuses Federal Data to Make False Claim That Covid Vaccines Killed 676,000

                        Activist Misuses Federal Data to Make False Claim That Covid Vaccines Killed 676,000

                        Illustrated Report: How Gun Violence Goes Viral

                        Illustrated Report: How Gun Violence Goes Viral

                        A New Medicare Proposal Would Cover Training for Family Caregivers

                        A New Medicare Proposal Would Cover Training for Family Caregivers

                        New Alzheimer’s Drug Raises Hopes — Along With Questions

                        New Alzheimer’s Drug Raises Hopes — Along With Questions

                        Blood Test That Screens for Cancer Raises Hopes, and Worries

                        Blood Test That Screens for Cancer Raises Hopes, and Worries

                        Trending Tags

                        • Lifestyle
                          Common Dog Owner Mistakes and How To Avoid Them

                          Common Dog Owner Mistakes and How To Avoid Them

                          3 Reasons To Give an Antique or Vintage Engagement Ring

                          3 Reasons To Give an Antique or Vintage Engagement Ring

                          Tim Ferris

                          Tim Ferriss’ Lifestyle Design: Unleashing Human Potential and Redefining Success

                          Outdoor Luxuries To Add to Your Backyard

                          Outdoor Luxuries To Add to Your Backyard

                          Strategy and business planning ideas. Chess gold on the board.

                          Why the Friend of My Enemy is My Enemy

                          Huge Money Mistakes New Parents Fall For

                          Huge Money Mistakes New Parents Fall For

                          Energizing Foods and Drinks To Consume Before a Workout

                          Energizing Foods and Drinks To Consume Before a Workout

                          Hobbies That Stimulate Cognitive Function

                          Hobbies That Stimulate Cognitive Function

                          How To Prevent Mold from Growing in a Sauna

                          How To Prevent Mold from Growing in a Sauna

                          Trending Tags

                          • Politics
                            Primaries underway for America’s third-largest party with grassroots and self-funded candidates

                            Primaries underway for America’s third-largest party with grassroots and self-funded candidates

                            Carbon capture draws millions in lobbying, billions in federal subsidies

                            Carbon capture draws millions in lobbying, billions in federal subsidies

                            United leads soaring airline industry lobbying spending

                            United leads soaring airline industry lobbying spending

                            Deep-pocketed dairy industry continues war on plant-based milk as FDA hears comments on new draft guidance

                            Deep-pocketed dairy industry continues war on plant-based milk as FDA hears comments on new draft guidance

                            Shein’s lobbying spending soars amid supply chain probes

                            Shein’s lobbying spending soars amid supply chain probes

                            Virginia legislative candidates in Richmond and Hampton Roads raked in millions in final primary sprint

                            Virginia legislative candidates in Richmond and Hampton Roads raked in millions in final primary sprint

                            Manchin outraises GOP challengers but has yet to announce 2024 plans

                            Manchin outraises GOP challengers but has yet to announce 2024 plans

                            Gallego outraises Sinema but falls far short of incumbent’s war chest in Arizona Senate race

                            Gallego outraises Sinema but falls far short of incumbent’s war chest in Arizona Senate race

                            Elon Musk outmuscled by Mark Zuckerberg’s money in politics

                            Elon Musk outmuscled by Mark Zuckerberg’s money in politics

                            Trending Tags

                            • Real Estate
                              Ways To Invest in Real Estate With No Experience

                              Ways To Invest in Real Estate With No Experience

                              How To Make Your Rental Property More Eco-Friendly

                              How To Make Your Rental Property More Eco-Friendly

                              Things To Consider Before Investing in Real Estate Overseas

                              Things To Consider Before Investing in Real Estate Overseas

                              Incredible Areas in Florida To Buy a Vacation Home

                              Incredible Areas in Florida To Buy a Vacation Home

                              Helpful Home Staging Tips for Virtual Tours

                              Helpful Home Staging Tips for Virtual Tours

                              Ways To Decorate and Design a Lake House

                              Ways To Decorate and Design a Lake House

                              Challenges To Managing a Rental Property

                              Challenges To Managing a Rental Property

                              The Best Landlord: How To Show Tenants You Care

                              The Best Landlord: How To Show Tenants You Care

                              The Best Locations To Invest in a Second Home

                              The Best Locations To Invest in a Second Home

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                              A Bitter Battle Over the ‘Orphan Drug’ Program Leaves Patients’ Pocketbooks at Risk

                              by Staff Writer
                              February 22, 2023
                              in Health
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                              By Sarah Jane Tribble

                              A prescription drug that helps Lore Wilkinson walk and talk despite a rare muscle disease cost her so little for more than a decade that she didn’t even use her insurance to pay for it. But now, her Medicare insurance is shelling out about $40,000 for a one-month supply of the drug, and she fears she’ll be slammed with a $9,000 copayment.

                              “Who can afford that?” said the 91-year-old, who lives in Rochester, Minnesota. (Her first name is pronounced LOR-ee.)

                              Wilkinson, like millions of other people with rare diseases nationwide, is caught up in an ongoing legal and political debate about how the U.S. supports pharmaceutical companies and their research. The FDA made its latest move in the tug of war in late January by saying it would largely ignore a U.S. court ruling involving Firdapse, the drug Wilkinson needs.

                              Firdapse was approved in 2018 by the FDA as an “orphan drug,” a designation that rewards drug companies for developing treatments for rare diseases. When a drugmaker wins approval for an orphan drug, the company is entitled to seven years of exclusive rights to the marketplace, which means the FDA won’t approve another company’s application for a competitive drug for the same use during that period.

                              But after the 11th U.S. Circuit Court of Appeals denied a motion in early 2022, the FDA stopped reviewing applications for certain drugs or handing out exclusivity, agency spokesperson April Grant said. The delay left drugmakers in limbo.

                              Often, drugs granted exclusivity are among the highest priced in the U.S. market. For example, Zolgensma, a one-time treatment for spinal muscular atrophy, carries a $2.25 million price tag. Mary Carmichael, a spokesperson for its manufacturer, Novartis, said Zolgensma has treated more than 3,000 patients globally and nearly all U.S. patients taking the drug as approved by the FDA are covered by commercial or government insurance.

                              The company also continues to invest in research and development as well as clinical studies for the drug to reach more patients, Carmichael said. Most drugs enter the U.S. market armed with a variety of patents and intellectual property protections that stave off competition and allow drugmakers to set prices as they see fit. For drugs that treat rare diseases, the seven years of market exclusivity is part of that armor.

                              A year’s supply of Catalyst Pharmaceuticals’ Firdapse, which Wilkinson takes to treat her Lambert-Eaton myasthenic syndrome, or LEMS, sells for about $375,000 after discounts, said Catalyst spokesperson David Schull. He said the company has financial assistance programs and donates to charitable foundations to help those in need. The goal, Schull said, “is that no LEMS patient is ever denied access to medication for financial reasons.”

                              Catalyst was granted exclusive market rights for Firdapse in 2018, which meant that Wilkinson and other LEMS patients could no longer get a similar drug from another company free of charge.

                              In 2019, amid a patient uproar about the cost, which Sen. Bernie Sanders weighed in on, the FDA granted another company, Jacobus Pharmaceutical, the right to market a competitive product for a subset of pediatric patients.

                              Then Catalyst filed suit against the federal government, contending it had rights to be the exclusive provider for all LEMS patients, regardless of age. The case, Catalyst Pharmaceuticals Inc. v. Becerra, had potentially “far-reaching implications,” wrote Grant, the FDA spokesperson, in an email to KHN. The court’s decision also “raised several novel questions,” she said.

                              The 11th Circuit sided with Catalyst in September 2021. But the FDA’s recent move to effectively disregard the court’s decision is “in the best interest of public health, rare disease patients and rare disease product development,” Grant wrote.

                              Still, the multiyear saga highlights lingering questions about orphan drug exclusivity and how the FDA’s policies may influence drug prices. At issue is the Orphan Drug Act, a 1980s-era law that incentivizes drug companies to research and develop rare-disease drugs. And it’s not the first time the orphan drug program has raised concerns.

                              For decades, the FDA has overseen a two-step process: A drug is first granted an orphan designation because it shows promise to treat a rare disease or condition. Then, once the pharmaceutical company studies and develops the rare-disease drug, the FDA approves its use and awards seven-year market exclusivity, preventing competition.

                              That final step, granting exclusivity, was in the spotlight in Catalyst’s lawsuit against the FDA. Since the Orphan Drug Act was created, the FDA’s staff routinely handed out exclusivity to companies for orphan drugs that treat a subset of patients, such as pediatrics. The goal was to make sure pharmaceutical companies didn’t get total market control for a drug after doing studies on only the “smallest, easiest-to-study populations,” the agency wrote on its website.

                              The Catalyst court decision could hurt children, agency officials wrote.

                              George O’Brien, a partner at Mayer Brown who represents companies regarding the FDA and regulatory practices, said he agreed with the FDA’s decision and its long-term strategy of parceling out exclusivity because a drug’s sales “should be limited to what you studied and got approved.”

                              “Most people think the way the FDA has done it for years is a very sensible way to do it,” O’Brien said. “Good for patients, good for pharma, and good for the FDA.”

                              The FDA said that it will comply with the court’s decision regarding Catalyst but that it doesn’t apply to other companies or drugs. In response to the FDA’s January announcement, Catalyst said it would not be affected. In July 2022, Catalyst bought the rights to Ruzurgi, the Jacobus drug.

                              Now, there is no competitive drug on the market that treats Wilkinson’s disease.

                              Jacobus had provided Wilkinson with the active ingredient of its drug free of charge from 2004 to 2018: “The only thing I paid was shipping.”

                              The FDA’s move to largely rebuke the Catalyst case will likely mean another company will sue the agency again, O’Brien said: “They are in a really tough spot.”

                              “My worry is there is just another lawsuit coming. And its uncertainty. Uncertainty is ultimately bad for patients,” O’Brien said.

                              Drugmakers have taken the FDA to court before over how the agency administers the Orphan Drug Act. In 2014, Depomed won a suit against the agency demanding an exclusivity label on its drug Gralise, which treated nerve pain.

                              The FDA had given Gralise an orphan designation and approval but declined to give it exclusivity because it said it was not clinically superior to another drug already on the market. Then-federal district court judge Kentaji Brown Jackson, who was appointed to the U.S. Supreme Court last year, required the FDA to grant exclusivity, blocking a generic.

                              That case was focused on the clinical superiority of a drug, rather than the scope of exclusivity. After the Gralise decision, the FDA eventually persuaded Congress to amend the law, which may be needed now, O’Brien said. Rachel Sher, a former director of policy at the National Organization for Rare Disorders who is now at Manatt, Phelps, & Phillips, said companies that would benefit from a broader award of exclusivity will sue to force the agency for the same reading of the Orphan Drug Act.

                              “Congress will need to act at some point,” said Sher, who also spent a decade on Capitol Hill as the FDA counsel for the House Energy and Commerce Committee.

                              Congress almost passed an amendment last year when it reauthorized the user fees that help fund the FDA. Then-Sen. Richard Burr (R-N.C.) argued to take the committee-added amendment out of the package, saying drugmakers would otherwise lack the incentives needed to develop drugs for rare diseases, according to Bloomberg Law.

                              Wilkinson, the patient advocate, has her own advice for Congress. The Orphan Drug Act itself — not just the exclusivity provision — needs to be fixed, she said.

                              “They have to change the law,” she said. Pharmaceutical companies should only win orphan drug status and be given exclusivity when they develop “a really new medication, not just by changing one molecule.”

                              Until then, Wilkinson said, she and others are still waiting: “I’m an old lady, and I don’t know if it is going to get fixed.”

                              This story was produced by KHN (Kaiser Health News), a national newsroom that produces in-depth journalism about health issues. Together with Policy Analysis and Polling, KHN is one of the three major operating programs at KFF (Kaiser Family Foundation). KFF is an endowed nonprofit organization providing information on health issues to the nation.

                              KHN (Kaiser Health News) is a national newsroom that produces in-depth journalism about health issues. Together with Policy Analysis and Polling, KHN is one of the three major operating programs at KFF (Kaiser Family Foundation). KFF is an endowed nonprofit organization providing information on health issues to the nation.

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