Watch this video to learn more about the unique challenges in rare disease treatment development, and why “one-size-fits-all” approaches to healthcare policy reform are harmful to continued investment and innovation for rare disease treatments.
Coalition Welcomes Amryt Pharma, Fulcrum Therapeutics and Signature Biologics, LLC
WASHINGTON – October 12, 2021 – The Rare Disease Company Coalition, a unified voice of life science companies committed to discovering, developing and delivering rare disease treatments, today announced the addition of three new companies to the Coalition. Launching in May 2021, the Coalition now boasts a total membership roster of 18 companies and provides a unified voice for innovator companies dedicated to rare disease treatment development.
New members that join the Rare Disease Company Coalition include:
- Amryt Pharma
- Fulcrum Therapeutics
- Signature Biologics, LLC
As Congress considers policy reforms that could impact the ability for companies to develop treatments for the one in ten Americans living with rare disease and have extraordinarily high unmet needs, the Coalition continues to help inform policymakers on the unique circumstances impacting therapy development for the 93% of rare diseases that have no FDA-approved treatments.
“We welcome the three new members to our Coalition, and applaud their commitment to developing rare disease treatments through ongoing research and development while often taking on significant risk and uncertainty,” said Betsy Ricketts, Chair of the Rare Disease Company Coalition. “The sustained growth of the Coalition is reflective of the need for ongoing education and engagement with policymakers on the importance of well-informed policy that can support continued progress for developing treatments for people with rare diseases.”
Life science companies that develop treatments for rare disease patients face numerous challenges from clinical trial enrollment through proving meaningful endpoints given the smaller population sizes and the more limited knowledge known about the disease progression. As a result, a comparatively higher percentage of operating expenses is dedicated to research and development at these life science companies. In 2020, Rare Disease Company Coalition members invested more than $4.1 billion in research and development, representing on average more than half of their annual operating budgets.
This necessary commitment to research and development underscores the importance of supportive policy and regulatory frameworks that can foster a greater understanding of rare diseases and recognizes the distinct considerations associated with the development of and access to rare disease treatments.
To learn more about the Coalition and the need for informed policy and legislative discourse and actions related to rare disease treatment development and commercialization, please visit rarecoalition.com.
About
The Rare Disease Company Coalition represents life science companies committed to discovering, developing and delivering rare disease treatments for the patients we serve. As an education and advocacy-focused coalition of companies, our goal is to inform policymakers of the unique challenges and promises of rare disease drug discovery, development and manufacturing for small population sizes in order for critical innovation to continue. To achieve this goal, we will use our unified voice to advocate for long-term, consistent, equitable and sustainable government policies that enable life science companies to continue to bring hope and provide access to approved treatments to people living with rare diseases. For more information, please visit rarecoalition.com
Media Contact:
Anna Stallmann
media@rarecoalition.com
708-476-1258
By: Leslie Meltzer, Ph.D., Chief Medical Officer at Orchard Therapeutics, a company member of the Rare Disease Company Coalition
In the first 24 to 48 hours of a baby’s life, a small blood sample is taken to detect serious genetic conditions, some of them deadly, that can be treated if diagnosed early. Thanks to newborn screening (NBS), a critical public health program that screens about 4 million babies a year in the U.S., parents have the opportunity for timely diagnosis, medical guidance, and disease intervention for their babies.
This NBS Awareness Month, advocates in the field, while praising the system for its many accomplishments and lives saved, are also raising red flags about its capacity to handle the coming wave of innovative gene and cell therapies approaching regulatory approval in the U.S. These therapies can bring hope to families facing rare genetic conditions, but for many of these disorders diagnosis as early as possible is often critical to successful intervention – making newborn screening crucial.
The NBS system needs modernization.
In the last 15 years, only six conditions were added to the Federal Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) Recommended Uniform Screening Panel (RUSP). On average, it takes approximately six years from the time a condition is nominated until it is added to the RUSP, if it is included at all. Even if added to the RUSP, screening in all 50 states can take up to a decade. For example, SCID was added to the RUSP in 2010; however, the condition was included on the first state panel in 2008 but was not added to all state panels until 2018. That means a baby born in one state can have the advantage of diagnosis, medical guidance, and treatment for an addressable genetic condition, while a baby born over the state line may face disability or early death for lack of testing.
More than 60 new gene therapy treatments – many for serious and rare conditions in children – are expected by 2030. This is therefore the right moment to look carefully at NBS capacities and screening implementation timelines with an eye on what is coming in terms of new therapies. An innovation in rare disease therapy almost always requires timely diagnosis and medical guidance in order for patients to benefit from therapy.
Continue reading here.
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WASHINGTON – September 14, 2021 – The Rare Disease Company Coalition, a unified voice of life science companies committed to discovering, developing and delivering rare disease treatments, today issued the following statement criticizing the draft legislation to amend the Orphan Drug Tax Credit included in the Build Back Better Act which will be considered in the U.S. House of Representatives Committee on Ways and Means this week.
“As one of America’s most underserved medical populations with extraordinarily high unmet medical needs, people living with rare diseases simply cannot shoulder the burden of American economic recovery and infrastructure development, and it is disappointing that Congress continues to advance policies that would limit their access to life-changing therapies,” said Taylor Mason, Executive Director of the Rare Disease Company Coalition. “The proposed changes to the Orphan Drug Tax Credit would directly limit the ability for innovator companies to invest in research and development for the nearly 30 million people battling debilitating and potentially fatal rare diseases, of which 93 percent do not have available treatments. The Rare Disease Company Coalition strongly urges Congress to consider the devastating impact of this legislation.”
The 1983 Orphan Drug Act was passed and built upon to help incentivize investment in rare disease research and therapeutic development where there was little research being done and nearly no treatment options for those diagnosed with a rare disease. Over the last several decades, the Act has proven to be a universal success, enabling life science companies to address a growing number of unmet needs for people living with rare diseases that once were untreatable. Since implementation of the Orphan Drug Act in 1983, there have been more than 1,000 FDA approvals for rare disease treatments, with over 25 percent of those approvals occurring in the last three years. The Orphan Drug Tax Credit, already diminished in 2017 under the Tax Cut and Jobs Act from 50 percent to 25 percent, remains a critical program for sustained innovation and investment for innovator companies that exclusively focus on life-changing development programs for treatments for rare diseases.
The Rare Disease Company Coalition was established in 2021 to help advance policies and regulations that enable the cost-effective and timely development and commercialization of rare disease treatments by educating policymakers on the distinct considerations of life science companies operating in the rare disease space. To learn more about the Coalition and its commitment to rare disease treatment development and commercialization, please visit rarecoalition.com.
About
The Rare Disease Company Coalition represents life science companies committed to discovering, developing and delivering rare disease treatments for the patients we serve. As an education and advocacy-focused coalition of companies, our goal is to inform policymakers of the unique challenges and promises of rare disease drug discovery, development and manufacturing for small population sizes in order for critical innovation to continue. To achieve this goal, we will use our unified voice to advocate for long-term, consistent, equitable and sustainable government policies that enable life science companies to continue to bring hope and provide access to approved treatments to people living with rare diseases. For more information, please visit rarecoalition.com
Media Contact:
Anna Stallmann
media@rarecoalition.com
708-476-1258
WASHINGTON – August 12, 2021 – The Rare Disease Company Coalition, a unified voice of life science companies committed to discovering, developing and delivering rare disease treatments, today issued the following statement in response to President Joe Biden’s remarks outlining a policy proposal that would allow Medicare to negotiate the price of certain prescription drugs.
“Ninety-three percent of rare diseases have no viable, FDA-approved treatment option. President Biden’s remarks calling on Congress to adopt price controls for drugs that have no competition in the market create the wrong incentives for further innovation to treat these devastating diseases,” said Taylor Mason, Executive Director of the Rare Disease Company Coalition. “Research and development for rare disease treatments present unique challenges and require substantially different business models than therapies for larger patient populations. We, as rare disease companies, are committed to working with Congress to address patient affordability, but a policy that seemingly targets innovators who are bringing medicines to patients with high unmet need seems unworkable and, worse yet, could mean fewer innovative medicines for the patients who need them most.”
The Rare Disease Company Coalition was established in 2021 to help advance policies and regulations that enable the cost-effective and timely development and commercialization of rare disease treatments by educating policymakers on the distinct considerations of life science companies operating in the rare disease space. To learn more about the Coalition and its commitment to rare disease treatment development and commercialization, please visit rarecoalition.com.
About
The Rare Disease Company Coalition represents life science companies committed to discovering, developing and delivering rare disease treatments for the patients we serve. As an education and advocacy-focused coalition of companies, our goal is to inform policymakers of the unique challenges and promises of rare disease drug discovery, development and manufacturing for small population sizes in order for critical innovation to continue. To achieve this goal, we will use our unified voice to advocate for long-term, consistent, equitable and sustainable government policies that enable life science companies to continue to bring hope and provide access to approved treatments to people living with rare diseases. For more information, please visit rarecoalition.com
Media Contact:
Anna Stallmann
media@rarecoalition.com
708-476-1258
By: Habib Dable, CEO of Acceleron Pharma, a company member of the Rare Disease Company Coalition
Originally published on MassBio.org (July 20, 2021)
In the biopharmaceutical industry, this refrain is as familiar as it is true. For those of us at companies committed to developing potentially transformative medicines to treat any of the approximately 7,000 recognized rare diseases—93% of which have no FDA-approved treatment—this call to action has never felt more attainable, yet at the same time, more at risk.
As legislators contemplate a range of access and pricing reforms in this era of rising costs and constrained resources, there is a considerable temptation among legislators to paint our industry with a single broad brush. Such one-size-fits-all policy approaches are a very real threat to the innovation that fuels the hopes of the small but needful patient populations we endeavor to serve. Further, such policies fail to recognize or reward the level of risk that smaller, rare-disease-focused companies—historically, the companies best suited to producing true advances in their chosen spaces—incur through much of their existence.
Before becoming CEO of Acceleron, I spent more than 20 years in classic “big pharma” at a multinational corporation with a vast portfolio of marketed drugs to treat prevalent diseases across a breadth of therapeutic areas. Revenues supported nearly 100,000 employees worldwide and funded a robust research pipeline. This is most certainly not the business model my rare disease counterparts and I execute on. Many of us are in the pre-clinical, clinical or early commercial stage, relying almost entirely on investors for funding as we seek meaningful therapeutic advances. Many of us may only have one or two seemingly viable assets, a precarious state in an industry in which failure is far more often the rule rather than the exception.
Continue reading at MassBio.org
By: Zachary Brennan / August 3, 2021
The controversy over the FDA’s accelerated approval pathway is heating up.
Last week, the FDA’s top oncology official Rick Pazdur said the pathway is “under attack,” largely due to the agency’s recent accelerated approval of Biogen’s controversial Alzheimer’s drug and the surrogate endpoint used in that decision. In the meantime, three accelerated approval indications have been pulled since July 1 (two from Bristol Myers Squibb and one from Merck in recent weeks), even as Pazdur called on critics of the pathway to not miss the more positive, big picture, with some cancer drugs proving to be enormously helpful and approved years before their confirmatory trials were completed.
Now, a coalition of rare disease drugmakers is seeking to defend the use of the pathway, raising concerns with a recent call from the nonpartisan MACPAC to increase rebates around drugs approved under the FDA’s accelerated pathway. The rare disease firms claim such a move on rebates would disincentivize drug developers from pursuing therapies in otherwise intractable disease areas, the coalition said in a recent letter to top congressional health committees.
Source: Endpoints News
By: Gabrielle Wanneh / August 2, 2021
Drug makers researching rare disease treatments are urging congressional health committees to reject calls by Medicaid payment advisers to update reimbursement policies for drugs granted accelerated approval by FDA. Patient advocates also are pressing lawmakers to reject the proposals, saying they would curtail access to innovative products and stifle development of rare disease treatments.
At issue are proposals by the Medicaid and CHIP Payment and Access Commission (MACPAC) and some state Medicaid programs to limit the amount public and private insurers pay for costly accelerated approval drugs until drug makers can prove their efficacy.
The Rare Disease Company Coalition, like the patient advocates, argues the proposals would decrease access to new innovative medications, particularly those meant to treat rare and life-threatening diseases that might be too difficult to study using the traditional pathway for approval.
Source: InsideHealthPolicy.com
WASHINGTON – July 29, 2021 – The Rare Disease Company Coalition, a unified voice of life science companies committed to discovering, developing and delivering rare disease treatments, today sent a letter to the leadership of the U.S. Senate Health, Education, Labor & Pensions (HELP) Committee and the U.S. House of Representatives Energy & Commerce Committee. In this letter, the Coalition expresses support for the Food and Drug Administration’s (FDA) Accelerated Approval Program while citing concerns with recent policy recommendations put forth by the Medicaid and CHIP Payment and Access Commission (MACPAC).
“The accelerated approval pathway is a critical mechanism that enables expedited availability of innovative treatments for patients with serious and often life-threatening diseases, including rare diseases that may be too challenging to study using a traditional pathway. In these cases, accelerated approval provides a promising roadmap to make possible the delivery of safe and effective rare disease therapies without compromising FDA’s stringent, science-based approval standards,” said Taylor Mason, Executive Director of the Rare Disease Company Coalition. “As a coalition of companies dedicated to rare diseases, we support policies that preserve the intent of the accelerated approval pathway and give all patients who have debilitating diseases a chance to be treated with cutting edge science. The Coalition urges members of Congress to recognize the harmful consequences of undercutting reimbursement, coverage and patient access to therapies developed under the Accelerated Approval Program.”
Throughout the letter, the Rare Disease Company Coalition highlights how the accelerated approval pathway has enabled the timely delivery of innovative treatments, particularly oncology drugs, and provides a proven regulatory framework that can appropriately enable access to therapies for challenging rare disorders, many of which might not be developed otherwise. The Coalition cites ill-informed policy proposals, including the recent recommendations put forth by MACPAC that selectively target accelerated approval drugs as a threat to patient access and the development of rare disease treatments.
About
The Rare Disease Company Coalition represents life science companies committed to discovering, developing and delivering rare disease treatments for the patients we serve. As an education and advocacy-focused coalition of companies, our goal is to inform policymakers of the unique challenges and promises of rare disease drug discovery, development and manufacturing for small population sizes in order for critical innovation to continue. To achieve this goal, we will use our unified voice to advocate for long-term, consistent, equitable and sustainable government policies that enable life science companies to continue to bring hope and provide access to approved treatments to people living with rare diseases. For more information, please visit rarecoalition.com
Media Contact:
Anna Stallmann
media@rarecoalition.com
708-476-1258
By: Emil D. Kakkis is the CEO, president, and founder of Ultragenyx Pharmaceutical, a company member of the Rare Disease Company Coalition
The FDA’s decision to grant accelerated approval to Biogen’s aducanumab (Aduhelm) for the treatment of Alzheimer’s disease was a difficult and bold one that people with the disease, their families, and other drug developers should be applauding.
When it comes to making new therapies for complex, difficult-to-treat diseases, history has shown that progress can’t be made without taking a first — often controversial — step. Without the FDA’s accelerated approval program and novel first treatments based on new and imperfect biomarker endpoints, HIV would not be a controllable disease today, and we might not have such a flourishing clinical research ecosystem in oncology.
Continue reading at STAT News.
Coalition Welcomes Atara Biotherapeutics, Chiesi Global Rare Diseases, Horizon Therapeutics, and Stealth BioTherapeutics
WASHINGTON – June 30, 2021 – The Rare Disease Company Coalition, a unified voice of life science companies committed to discovering, developing and delivering rare disease treatments, today announced the addition of four new companies to the Coalition. With a total membership roster of 15 companies, the Coalition continues to articulate its voice on the importance of well-informed policies and regulations to enable the cost-effective and timely development and commercialization of rare disease treatments for patients.
New members that join the Rare Disease Company Coalition include:
- Atara Biotherapeutics
- Chiesi Global Rare Diseases
- Horizon Therapeutics
- Stealth BioTherapeutics
“We are pleased to welcome four new companies to our growing Coalition,” said Taylor Mason, Executive Director of the Rare Disease Company Coalition. “The Coalition is committed to fostering greater understanding of the unique circumstances associated with the development of rare disease treatments so that legislation and policies can better support continued development of these therapies as well as patient access and reimbursement to address the incredible unmet need of rare disease patients.”
Life science companies that develop treatments for rare disease patients face numerous challenges from clinical trial enrollment through proving meaningful endpoints given the smaller population sizes and the more limited knowledge known about the disease progression. As a result, a comparatively higher percentage of operating expenses is dedicated to research and development at these life science companies. In 2020, Rare Disease Company Coalition members invested more than $4.6 billion in research and development, representing on average more than half of their annual operating budgets.
This necessary commitment to research and development underscores the importance of supportive policy and regulatory frameworks that can foster a greater understanding of rare diseases and recognizes the distinct considerations associated with the development of and access to rare disease treatments.
About
The Rare Disease Company Coalition represents life science companies committed to discovering, developing and delivering rare disease treatments for the patients we serve. As an education and advocacy-focused coalition of companies, our goal is to inform policymakers of the unique challenges and promises of rare disease drug discovery, development and manufacturing for small population sizes in order for critical innovation to continue. To achieve this goal, we will use our unified voice to advocate for long-term, consistent, equitable and sustainable government policies that enable life science companies to continue to bring hope and provide access to approved treatments to people living with rare diseases. For more information, please visit rarecoalition.com
Media Contact:
Anna Stallmann
media@rarecoalition.com
708-476-1258
WASHINGTON – June 22, 2021 – The Rare Disease Company Coalition, a unified voice of life science companies committed to discovering, developing and delivering rare disease treatments, commented on the release of “Cures 2.0” draft bipartisan legislation by U.S. Representatives Diana DeGette (D-CO) and Fred Upton (R-MI) and welcomes the opportunity to further engage and provide feedback as the legislation is considered.
“The Rare Disease Company Coalition applauds Congresswoman DeGette and Congressman Upton on recognizing the importance of building upon their landmark, bipartisan 21st Century Cures Act and continuing to advance legislation that aims to better deliver modern treatments and cures to the countless Americans seeking options,” said Betsy Ricketts, Chair of the Rare Disease Company Coalition. “As Congress now considers ‘Cures 2.0’ and other legislative and regulatory proposals, we look forward to further engaging and providing feedback on the unique challenges in the development of rare disease treatments so that forthcoming legislation and policies facilitate and do not inadvertently harm the ability to successfully provide treatments for these medically underserved patient populations, now and in the future.”
The Rare Disease Company Coalition was established in 2021 to help advance policies and regulations that enable the cost-effective and timely development and commercialization of rare disease treatments by educating policymakers on the distinct considerations of life science companies operating in the rare disease space. To learn more about the Coalition and its commitment to rare disease treatment development and commercialization, please visit rarecoalition.com.
About
The Rare Disease Company Coalition represents life science companies committed to discovering, developing and delivering rare disease treatments for the patients we serve. As an education and advocacy-focused coalition of companies, our goal is to inform policymakers of the unique challenges and promises of rare disease drug discovery, development and manufacturing for small population sizes in order for critical innovation to continue. To achieve this goal, we will use our unified voice to advocate for long-term, consistent, equitable and sustainable government policies that enable life science companies to continue to bring hope and provide access to approved treatments to people living with rare diseases. For more information, please visit rarecoalition.com
Media Contact:
Anna Stallmann
media@rarecoalition.com
708-476-1258
For Immediate Release
WASHINGTON – June 8, 2021 – The Rare Disease Company Coalition, a unified voice of life science companies committed to discovering, developing and delivering rare disease treatments, today announced appointments to its Executive Committee for 2021, following the Coalition’s launch on May 13, 2021.
The Rare Disease Company Coalition was established to educate policymakers on the distinct considerations of life science companies operating in the rare disease space. The Coalition believes that constructive dialogue with well-informed policymakers will lead to policies and regulations that enable continued innovation and the cost-effective and timely development and commercialization of resulting treatments for the one in 10 Americans living with rare diseases.
The Coalition is governed by a Board of Directors and supported by an Executive Committee that provides oversight of the day-to-day operations of the Coalition. In addition to named elected positions, the Executive Committee has representation from each of the membership classes of the Coalition.
The following company representatives were elected to serve as Executive Committee members for the inaugural year of the Coalition:
- Chair: Betsy Ricketts, Ultragenyx Pharmaceutical
- Vice Chair: Greg Chesmore, Acceleron Pharma
- Secretary: Tim Kanter, Orphazyme US
- Member-at-Large: Christine Harrison, Orchard Therapeutics
- Member-at-Large: Deirdre Parsons, Alnylam Pharmaceuticals
- Member-at-Large: Geoff Werth, Harmony Biosciences
Supporting the work of the Coalition, Taylor Mason was appointed to serve as Executive Director of the Coalition. In addition, the following individuals have been appointed Committee Chairs for 2021:
- Policy Committee Chair: Diane Berry, Sarepta Therapeutics
- Operations Committee Chair: John Jackimiec, Aeglea BioTherapeutics
To learn more about the Coalition and its commitment to rare disease treatment development and commercialization, please visit rarecoalition.com.
About
The Rare Disease Company Coalition represents life science companies committed to discovering, developing and delivering rare disease treatments for the patients we serve. As an education and advocacy-focused coalition of companies, our goal is to inform policymakers of the unique challenges and promises of rare disease drug discovery, development and manufacturing for small population sizes in order for critical innovation to continue. To achieve this goal, we will use our unified voice to advocate for long-term, consistent, equitable and sustainable government policies that enable life science companies to continue to bring hope and provide access to approved treatments to people living with rare diseases. For more information, please visit rarecoalition.com.
Media Contact:
Anna Stallmann
media@rarecoalition.com
708-476-1258
For Immediate Release
With one in 10 Americans living with an often devastating and debilitating rare disease and limited treatment options, a coalition of life science companies cites a “one-size-fits-all” approach to healthcare policy and regulations as detrimental to continued innovation that has potential to systematically improve quality of life for millions
WASHINGTON, DC – May 13, 2021 – The Rare Disease Company Coalition, a unified voice of life science companies committed to discovering, developing and delivering rare disease treatments, today announced their organizational launch. This first-of-its-kind alignment of organizations will inform and help educate policymakers on the unique circumstances facing life science companies when developing and bringing to market therapies for very small and differentiated patient populations. The Coalition will also advocate for policies and regulations that enable cost-effective and more timely delivery of treatments by recognizing these differences.
Collectively, the Rare Disease Company Coalition represents life science companies that have brought to market 22 treatments and are presently working on more than 160 rare disease development programs, many of which would be first-to-market therapies if approved. These diseases are devastating and often life-threatening: 80 percent of rare diseases are genetic in origin, 50 percent impact children, and 30 percent of those children won’t live to see their 5th birthday. The Coalition is composed of companies continuing to change those statistics by discovering, developing and bringing valuable treatments – and even potential cures – to market for these patients awaiting a treatment approved by the Food and Drug Administration (FDA).
The pharmaceutical industry continues to lead the nation in research and development expenditures by dedicating on average between fifteen to twenty percent of sales to fostering innovation. Companies focused on rare diseases take on an outsized commitment to and risk in research and development. In 2020, Rare Disease Company Coalition members invested more than $4.1 billion in research and development, representing approximately 65 percent of their annual operating budgets. Moreover, the majority of Coalition members spend more annually in research and development than revenues generated, including the over one-third of members that are in the preclinical stage and currently do not generate revenue.
Building on the results of these capital investments, smaller companies are now driving the groundbreaking innovation happening across multiple disease areas. As reported by the Congressional Budget Office, 70 percent of the nearly 3,000 therapies in Phase III clinical trials are from small drug companies or those with annual revenues of less than $500 million. Moreover, about one-third of new treatments approved by the FDA have been developed by companies with annual revenues of less than $100 million.
Expanding on years of advocacy work through successful industry associations and rare disease patient-focused organizations, the Rare Disease Company Coalition will specifically work to:
- Inform and help educate policymakers on the unique circumstances of the rare disease company business model, specifically focusing on the different issues created by working with small and differentiated patient population sizes;
- Advocate for public policy and regulatory frameworks that account for and recognize the distinct considerations of life science companies operating in the rare disease space;
- Work with policymakers to establish long-term, consistent, equitable and sustainable research incentives for rare diseases and access to the resulting innovation;
- Increase awareness of the innovation happening within rare disease companies.
Founding members of the Coalition include:
- Acceleron Pharma
- Aeglea BioTherapeutics
- Agios Pharmaceuticals
- Alnylam Pharmaceuticals
- Harmony Biosciences
- Orchard Therapeutics
- Orphazyme US, Inc.
- Sarepta Therapeutics
- Taysha Gene Therapies
- Ultragenyx Pharmaceutical
In the coming month, the Coalition will engage with policy stakeholders on potentially impactful drug and healthcare policies and regulations currently under discussion, including prescription drug pricing, to highlight the consequences that blanket legislation can have on continued innovation for rare disease treatments.
About
The Rare Disease Company Coalition represents life science companies committed to discovering, developing and delivering rare disease treatments for the patients we serve. As an education and advocacy-focused coalition of companies, our goal is to inform policymakers of the unique challenges and promises of rare disease drug discovery, development and manufacturing for small population sizes in order for critical innovation to continue. To achieve this goal, we will use our unified voice to advocate for long-term, consistent, equitable and sustainable government policies that enable life science companies to continue to bring hope and provide access to approved treatments to people living with rare diseases. For more information, please visit rarecoalition.com
Media Contact:
Anna Stallmann
media@rarecoalition.com
708-476-1258
By: Kate Goodwin / May 13, 2021
There are more than 7,000 rare diseases identified in the world today. While “rare” means that less than 200,000 people in the US are affected, to suffer from a rare disease is not actually rare at all.
Around 10% of the American population suffers from a rare disease. A shocking 90% of rare diseases do not have an approved treatment on the market.
Source: BioSpace