On this page you can find important Policy Reports from the nation’s leading thought leaders in the Rare Disease Community.

Kids v Cancer Creates Pediatric Cancer Legislative Yearbook for 2016

Kids v Cancer compiled a yearbook summarizing the achievements of pediatric cancer advocacy organizations in 2016 and outlining steps that pediatric cancer organizations plan to take in 2017. The Pediatric Cancer Legislative Yearbook 2016 is available here.




From FDA Law Blog: Priority Review Voucher Updates- Valuation, Eligibility, Reauthorization

From FDA Law Blog by Alexander J. Varond

Hardly a week goes by without news of FDA’s priority review voucher (“PRV”) programs. The last several weeks are no exception. In this blog post, we focus on five key updates related to FDA’s tropical disease and rare pediatric disease (“Pediatric”) PRV programs:

  1. 1. United Therapeutics Corporation (“United Therapeutics”) sold its Pediatric PRV for $350 million;
  2. 2. DA added Chagas disease and neurocysticercosis to its list of eligible tropical diseases;
  3. 3. Sanofi-Adventis (“Sanofi”) successfully redeemed a Pediatric PRV to beat Amgen to market and became the first PCSK9 cholesterol therapy approved in the U.S.;
  4. 4. The Senate released its own version of the Advancing Hope Act; and
  5. 5. Three Duchenne muscular dystrophy sponsors received rare pediatric disease designation.

PRV Prices Continue to Climb

On August 19, United Therapeutics announced the sale of its rare Pediatric PRV for an incredible $350 million to AbbVie Inc. We discussed FDA’s issuance of this Pediatric Voucher to United Therapeutics for Unituxin here. As the graph below shows, the value of PRVs continues to climb dramatically.











FDA Adds Chagas Diseases and Neurocysticercosis to the List of Qualifying Tropical Diseases

On August 20, 2015, FDA published a final order to add two new diseases, Chagas disease and neurocysticercosis, to the list of qualifying tropical diseases. Recall that, in order for a drug product to be eligible for a PRV, four requirements must be met:

  1. 1. The application must be for a listed tropical disease;
  2. 2. The application must be submitted either as a 505(b)(1) NDA or a 505(b)(2) application;
  3. 3. The drug that is the subject of the application must not contain a previously-approved active moiety; and
  4. 4. The application must qualify for a 6-month priority review under FDA’s policies.

Thus, a sponsor for a drug that treats Chagas disease or neurocysticercosis that meets the requirements above would be eligible for a tropical disease PRV.

Section 524(a)(3)(R) of FD&C Act permits FDA to include “[a]ny other infectious disease for which there is no significant market in developed nations and that disproportionately affects poor and marginalized populations.” (Emphasis added.) In its final order, the Agency also affirmed its commitment to using “a flexible approach” to tropical disease designations based on “scientifically informed, qualitative assessment of disease candidates.” FDA also announced the establishment of a public docket that will remain open to receive future suggestions for tropical disease designations. That docket is here.

The final order explains FDA’s interpretation of the key elements of section 524(a)(3)(R) of FD&C Act:

“Developed nations” – According to the order, “FDA will use a country’s presence on the World Bank’s list of ‘low income economies’ . . . as evidence that the country should not be considered a ‘developed nation’ for purposes of ‘tropical disease’ determination.”

“No significant market” – FDA notes the difficulty in providing a rigid definition of “no significant market” and instead proposes the following factors be considered in determining whether a “significant market” exists in developed countries: (1) occurrence of the disease in developed nations; and (2) the existence of a sizeable indirect market for the tropical disease drug (e.g., government, including the military) that would constitute a financial incentive for drug development.

Disproportionately affects poor and marginalized populations – As with the term “no significant market,” FDA rejected a single definition in favor of four factors that must be weighed. These factors are: (1) the proportion of global disability-adjusted life years for the disease that is attributable to developing countries; (2) the relative burden of the disease in the most impoverished populations within the countries in which it is found; (3) the relative burden of the disease in infants, children, and other marginalized segments of the population; and (4) whether WHO has designated it as a neglected tropical disease.

Applying these standards, FDA exercised the authority granted to it by the Adding Ebola to the FDA Priority Review Voucher Program Act in December 2014 and added Chagas disease and neurocysticercosis to its list of diseases that qualify for the tropical disease voucher program.

Bonus:  On August 24, 2015, Mid-Atlantic BioTherapeutics, Inc. submitted a citizen petition requesting that FDA add the rabies virus to the list of qualifying tropical diseases.  Notably, rabies is on WHO’s list of neglected tropical diseases, as is cysticercosis/taeniasis.

Sanofi Successfully Uses its PRV

On August 19, 2015, FDA announced that Sanofi-Adventis had redeemed a Pediatric PRV for its Praluent (alirocumab) NDA.  With its approval on July 24, 2015, Praluent became the first approved PCSK9 cholesterol therapy in the United States.  The PCSK9 therapeutic class is expected to a blockbuster, with sales projected to be in the billions of dollars per year.

The interesting part of this story is that Amgen’s Repatha beat Sanofi’s Praluent to market in Europe; but, ostensibly, through its redemption of a rare pediatric disease priority review voucher, Sanofi was able to reach the U.S. market before Amgen.  The Praluent approval came a month ahead of Repatha’s August 27, 2015 PDUFA date and approval.

Drugs that are first-to-market often enjoy considerable benefits over second-comers.  U.S. payers, for example, are quick to cover new drugs, and products that are first-to-market often have significant marketing advantages and sales momentum.  Sanofi’s $67.5 million move to accelerate its entry into the U.S. market for its PCSK9 therapy will be closely watched.  After all, it will be a bellwether of whether industry can justify the high prices paid for priority review vouchers.  If things go well for Sanofi, the demand for and price of subsequent PRVs should be high.

An Update to the Advancing Hope Act

In March 2014, Representative G.K. Butterfield (D-NC) introduced the Advancing Hope Act (H.R. 1537) in the House of Representatives.  In our post on the bill, we noted the effort to make the Pediatric Voucher program permanent.  We also noted the bill’s proposal to change the tropical disease voucher program.  At the time, we noted that such changes not only seemed out of place, but they could be counterproductive.

On July 28, 2015, the Advancing Hope Act (S. 1878) was introduced in the Senate.  The Senate version of the bill scrapped the amendments to the tropical disease priority review voucher program.  Instead, the Senate version proposes to make the Pediatric Voucher program permanent and to designate pediatric cancers and sickle cell anemia as rare pediatric diseases.

Rare Pediatric Disease Designations Granted for DMD

On August 19, FDA awarded rare pediatric disease designation to BioMarin’s,Sarepta’s, and Santhera’s investigational products for Duchenne Muscular Dystrophy.  With the rare pediatric disease designation, each company will likely be eligible for a Pediatric Voucher upon approval of its drug product.

Bipartisan Policy Center Releases Bold Recommendations to Advance Medical Innovation, Help Cure Life-Threatening Diseases and Improve U.S. Competitiveness in the Global Marketplace

From an article by Joann Donnellan – Today there are 10,000 known diseases in the United States, but only 500 have established treatments. Life-threatening diseases such as Alzheimer’s, cancer, heart disease, and many others lack effective treatments or cures. The urgent need to find cures is impeded by the process of bringing promising drugs and devices to market. A new report by the Bipartisan Policy Center (BPC) recommends a series of policy actions that Congress can take to help reduce the time and cost of developing and delivering safe and effective medical products to patients. Such actions could considerably increase the competitiveness of U.S. companies in the global marketplace.

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Fact sheets on healthcare exchanges for every State

Fact sheets on every state were compiled by Avalere Health, a leading advisory company focused on healthcare business strategy and public policy.

The key facts for each state includes information on:  deductibles, cost sharing and formulary analysis.

Take a look at the state map below to view key facts about 2014 health plans and prescription drug coverage in your state’s health insurance exchange.

State oversight can help ensure that health insurance exchange coverage offers the access to needed medicines the Affordable Care Act promised. States can continue to protect patients in their state through the following actions:

  • Enforcing non-discrimination requirements, which apply to benefit design and provider networks;
  • Establishing stronger rules regarding exceptions and appeals processes, which help enrollees get the medicines and care their doctors recommend; and
  • Enhancing the state’s marketplace website or advocating for a Healthcare.gov page that allows for easier plan comparisons, including searchable formularies and estimates of total out-of-pocket costs.



CHI – Combating Rare Disease in the 21st Century

Read the full report here: CHI Rare Disease Policy Report


Although called rare or orphan diseases – defined as conditions affecting fewer than 200,000 people in the United States – there are more than 7,000 such conditions, affecting 30 million Americans. Collectively, rare diseases represent a significant public health challenge.

Quite often these are genetic, childhood conditions, in some cases caused by a single gene mutation that may or may not be inherited from a parent’s genes. Because they are so rare, and many families may not have prior experience with the condition, simply getting a diagnosis can be an agonizing, years-long process. In addition, because these diseases are so uncommon, many doctors have no experience treating them and there may only be a handful of physicians in the world who have any experience with a given condition.

Even after diagnosis, patients still face barriers to care: 95 percent of rare diseases have not one single U.S. Food and Drug Administration (FDA) approved drug treatment. The absence of treatments for rare diseases actually used to be much worse. While many products and therapies were developed to treat cancer, heart disease and other common conditions, from 1973-1983, fewer than 10 rare disease therapies were developed.

FDA Law Blog: FDA Issues Final Orphan Drug Regulations

By Michelle L. Butler 

On June 12th, the final rule to amend FDA’s 1992 orphan drug regulations was published in the Federal Register.  78 Fed. Reg. 35117 (June 12, 2013).  This final rule, which largely finalizes the revisions as proposed in October 2011, will be effective August 12, 2013.  We previously posted on the details of the proposed rule, as well as litigation that is still ongoing concerning some of the issues discussed by FDA in the proposed and final rules.

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Shire Launches Report that Quantifies the Health, Psycho-social and Economic Impact of Rare Diseases

Lexington, Massachusetts, US – April 9, 2013 – Shire plc (LSE: SHP, NASDAQ: SHPG) today launched a Rare Disease Impact Report, which uncovers the health, psycho-social, and economic impact of rare diseases on patient and medical communities in the United States (US) and United Kingdom (UK). The report, developed in collaboration with an external advisory board of thought leaders in the medical, advocacy, health policy and health economics fields, will be distributed at the World Orphan Drug Congress in Washington, DC (April 9-11) and is available for immediate download at www.rarediseaseimpact.com.

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PhRMA Report: Innovation in the Biopharmaceutical Pipeline: A Multi-Dimensional View

For Immediate Release:  January 17, 2013

Today, the Pharmaceutical Research and Manufacturers of America (PhRMA) released a report entitled “Innovation in the Biopharmaceutical Pipeline: A Multi-Dimensional View.”  The report was conducted by researchers at the Analysis Group, and examines the drug pipeline to illustrate the type of research conducted by biopharmaceutical companies.

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Which Orphans Will Find a Home? The Rule of Rescue in Resource Allocation for Rare Diseases


Hastings Center Report, Volume 42, Issue 1, pages 27–34, January-February 2012


The rule of rescue describes the moral impulse to save identifiable lives in immediate danger at any expense. Think of the extremes taken to rescue a small child who has fallen down a well, a woman pinned beneath the rubble of an earthquake, or a submarine crew trapped on the ocean floor. No effort is deemed too great. Yet should this same moral instinct to rescue, regardless of cost, be applied in the emergency room, the hospital, or the community clinic?

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Report to the President on Propelling Innovation in Drug Discovery, Development, and Evaluation

On September 25, 2012, the President’s Council of Advisors on Science and Technology (“PCAST”) released a report entitled “Report to the President on Propelling Innovation in Drug Discovery, Development, and Evaluation.”

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