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Rare Diseases Lead to Unique Legislation


Pharmaceutical Executive

In 1983-with the help of Abbey Meyers and the drive she had to help her son (more on that here)-the Orphan Drug Act was passed to incentivize pharma to step up and play an active role in drug development for the 30 million children and adults in the U.S.


Since 1983, pharma and patent organizations have been working together to influence policy aimed at improving the lives of those with rare diseases.

In 1983-with the help of Abbey Meyers and the drive she had to help her son (more on that here)-the Orphan Drug Act was passed to incentivize pharma to step up and play an active role in drug development for the 30 million children and adults in the U.S. who suffer from rare diseases. The Orphan Drug Act recognized that because there are so few patients affected by each of these rare diseases, the likelihood of pharma reaping a strong ROI from bringing such drug treatments to market is slim. Thus, the Act aimed to sweeten the deal for pharma by providing seven years of market exclusivity for the designated orphan indication of the product; federal grants to fund clinical trials of designated orphan products and protocol assistance by the FDA; and tax credits for such clinical trials.

Despite such efforts, it could arguably be said that orphan diseases have still historically not gotten adequate attention from Big Pharma, as the industry rode the blockbuster wave of the 1990s. In the past few years, however, with the reality of patent cliffs finally hitting home, industry is again starting to pay attention to the untapped market of those 30 million Americans. “Pharma companies are naturally looking for new places to grow-and if there aren’t any more big markets to grow in, you have to grow in a bunch of smaller markets,” says Craig Kephart, president of Centric Health Resources, a direct-distribution company focused on specialty pharma and orphan diseases. “We need to raise awareness around rare diseases, and around the fact that as a society, we’ve already made the choice that these are people that need our help the most, when we passed the Orphan Drug Act back in the ‘80s.”

In that vein, 2011 saw the introduction of the Creating Hope Act, a bipartisan bill introduced by Senator Bob Casey (D-PA) last March that would encourage the development of new treatments for rare and neglected diseases that disproportionately affect children. The law tells pharma companies that if they focus on a rare or neglected childhood disease and try to bring a drug to market there, they’ll get a voucher for one of their other non-rare drugs to qualify for expedited review through the FDA-thus cutting up to five months off the review process. Organizations such as Kids v Cancer are applauding the Act, pointing out on its website that “research and development for new drugs from pharmaceutical companies comprises 60 percent of funding for adult cancer drugs and close to zero for childhood cancers.” Similarly, the Congressional Childhood Cancer Caucus, which also supports the proposed law, notes on its website that “the cancers in children are different from cancers in adults, such that drugs developed to treat an adult malignancy, such as colon cancer, may not be useful for a cancer that occurs in children, such as neuroblastoma,” and that “developing products for children is particularly challenging because of the difficulties associated with conducting clinical trials on this patient population.” As of December, more than 100 cosponsors had signed on in support of the bill, including the National Organization for Rare Disorders (NORD), the Biotechnology Industry Organization (BIO), Shire, Novartis, and GlaxoSmithKline. The bill has not yet passed.

Most recently, Senator Kay Hagan (D-NC) proposed yet another bill intended to accelerate the development of medical treatments for people with unmet medical needs. Introduced on February 16 of this year, the Transforming the Regulatory Environment to Accelerate Access to Treatments (TREAT) Act focuses on regulatory innovation in areas such as the accelerated approval and fast track processes to speed the development of therapies for patients with serious or life-threatening diseases. The bill will be referred to the Senate Health, Education, Labor and Pensions (HELP) Committee that oversees healthcare issues, of which Senator Hagan is a member. “For patients suffering today from rare diseases for which there are no current treatments, medical advances can’t come fast enough. This legislation creates a consistent process and a clear and effective pathway at FDA that will encourage the development of innovative treatments for patients otherwise dependent on the uncertainty of medical breakthroughs,” said Hagan in a recent press release on the Senator’s website. According to the release, the bill “allows the FDA Commissioner to better utilize waivers when potential conflicts of interest are outweighed by the need to have input from leading medical and scientific experts. It also recommends that patients and disease research organizations have more representation on FDA advisory committees … [and] ensures that drug sponsors are provided explanations when their drugs are turned down so that they might address concerns, improve the treatment, and get approved medications to patients more quickly.”

A common debate about such proposed bills, and about fast track and expanded access programs in general, is whether or not moving drugs more quickly through the pipeline compromises safety. But according to Kephart, “When you talk to the [rare disease] patient community, they’re way more willing to accept risk associated with a product, because they have nothing. When you’re trying to find needles in a haystack to start with in forming clinical trials, trying to find a group of patients that meets study criteria for such a rare disease is tough-and it’s a big recruiting cost. These are therefore small groups of people who have no treatment in many cases, so they’re willing to take an ‘experimental’ drug, so to speak, if it’s been shown to be reasonably safe.”

The announcement of the newly proposed bill and the general swell of interest in orphan diseases comes perfectly timed to correlate with Rare Disease Day (RDD), hosted by NORD, today, February 29.

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